Medical Arm of the Interagency Registry for Mechanically Assisted Circulatory Support
Conditions: Heart Failure, Systolic Heart Failure
Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

 
 
Date:  November 16, 2012 
 
 
Re:  
Administrative Change for Protocol, Version Date 11/08/2011 
 
To: 
Sites participating in the Medical Arm of the Interagency Registry for Mechanically 
Assisted Circulatory Support (MedaMACS) 
 
NCT#: NCT01932294 
 
From:  Mary Lynne Clark 
 
Regulatory Director 
 
INTERMACS 
            mlclark@uab.edu 
 
The following administrative change impacts the MedaMACS Protocol, Version Date 11/8/2011. 
This letter must be submitted with your initial IRB submission. In the event you have already 
submitted the initial IRB application, please forward this letter to your Institutional Review Board 
(IRB) as soon as possible. IRB acknowledgment must be forwarded to the MedaMACS Data 
Coordinating Center.   
 
Please file this letter with all copies of the protocol.  Please file this letter and any  
IRB correspondence in your regulatory file and other pertinent files.   
 
List of Changes to the MedaMACS Protocol, Version Date 11/8/2011: 
 
 The following text, Section 2, Specific Aims, Aim 4 has been removed from 
the protocol: 
 
“To develop and advance a team of emerging clinical investigators who will increase 
awareness of mechanical circulatory support and serve as role models for other young 
investigators.    
 
Hypothesis: Focused mentorship of early career investigators will foster the next 
generation of leaders in the clinical care of patients with advanced heart disease. 
 
4.1.1 Junior investigators will take the lead in designing and analyzing research questions 
of their own with the mentorship of a more senior investigator at each clinical site.  
Presentation of MedaMACS data at national meetings and manuscript development 
will be the responsibility of junior investigators.  A centralized publications 
committee with ensure equitable distribution of topics of interest so there is synergy 
across centers and appropriate extramural mentorship is available.   
 
4.1.2 Survey will be made among the young investigators for VAD-related publications, 
progression to independent research status, and affiliation to clinical VAD programs 
at 5 years after initiation of MedaMACS enrollment.” 
 

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MedaMACS continues to develop and advance a team of emerging clinical investigators to increase 
awareness of mechanical circulatory support and serve as role models for young investigators.  
However, we feel that this can best be addressed in a separate format and should not be included in 
the study protocol.   
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Medical Arm of the Interagency 
Registry for Mechanically  
Assisted Circulatory Support 
(Medamacs) Study 
 
 
 
 
 
 
 
 
 
Version 11/8/2011 

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1. 
INTRODUCTION  
 
Despite widespread use of evidence-based medical therapies, including neurohormonal 
blockers and biventricular pacing, mortality and morbidity from systolic heart failure remain high.1  
Breakthroughs in mechanical circulatory support technology have extended survival and improved 
quality in advanced heart failure patients awaiting cardiac transplantation and in inotrope-dependent 
patients who are ineligible for transplant.2-4  The INTERMACS Registry (Interagency Registry of 
Mechanical Assist Circulatory Support) has facilitated the refinement of patient selection for this 
therapy by analysis of those patients who have received devices5, 6   
Integral to the original intent of INTERMACS was comparison to ambulatory patients living 
with advanced heart failure who were not currently receiving mechanical circulatory support 
devices. As the first years of INTERMACS were dominated by INTERMACS profiles 1-2 with 
medical survival of hours to weeks, comparison to outcomes for ambulatory patients without 
devices was initially of limited relevance. However, the lack of information on outcomes with 
continued medical therapy has limited the ability to define and advance indications in the “less 
sick” despite INTERMACS-documented progress that should attract ambulatory patients, who 
currently make up fewer than 20% of all patients receiving mechanical support.6  (Figure 1) 
The development of a contemporary parallel population of ambulatory patients with heart 
failure is necessary in order to meet the commitment defined for INTERMACS at its inception in 
2005 and re-emphasized for the renewal beginning 2010, for INTERMACS to 1). Facilitate the 
refinement of patient selection to maximize outcomes with current devices within approved 
indications, and to 2). Guide and expedite clinical trials for new indications and/or new devices.  
This is true both for survival outcomes and for outcomes beyond survival, measured in terms of 
quality of life, functional capacity, and satisfaction with the chosen strategy of care.  
The future impact of mechanical circulatory support on public health will be experienced by 
ambulatory heart failure patients in whom support can be employed electively for long-term benefit 
with diminishing operative risk.  At present, the size and clinical characteristics of the population 
with advanced ambulatory heart failure despite optimal contemporary medical therapy remain 
unknown since few such patients have been enrolled in clinical trials.  Patients with less severe 
heart failure can clearly identify thresholds for consideration of a ventricular assist device, or 
VAD.7 A better understanding of this population and their unique priorities is vital in order to 
anticipate the optimal expansion of mechanical circulatory support into the less critically ill 
population. 
These needs will be addressed in the Medical Arm for Mechanically Assisted Circulatory 
Support (MedaMACS) Pilot Study.  MedaMACS will characterize the types of patients who are not 
receiving an LVAD currently due to the intersection of relative contra-indications, to their own 
preferences, or to their characterization as “less sick” either by perception or by absolute criteria as 
currently defined for lifetime VAD support.  The nature of contemporary “optimal” medical therapy 
will be reported, and the major risk scores for heart failure will be calculated and challenged.  
MedaMACS will incorporate specific consideration of frailty as measured by gait speed, the 
proposed new “geriatric vital sign”.  In relation to the characteristics at baseline and over time, 
MedaMACS will provide information on medical outcomes in terms of timed endpoints of VAD, 
transplant, or death.  There will also be determination of adverse events of stroke and 
hospitalizations for comparison to those experienced after VAD.  Of equal weight will be the 
determination of functional capacity, quality of life, and satisfaction with therapy.  For many 
ambulatory patients with chronic heart failure, the magnitude and predictability of expected 
improvement in functional status with a VAD will likely influence their VAD decisions more than 
the margin of survival benefit. These are pivotal components of decision-making for advanced heart 

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failure in order to progress toward the 21st century goal of patient-centered care.  The MedaMACS 
program will provide landmark data from which to refine selection for mechanical circulatory 
support from the ambulatory heart failure population within which the greatest benefit of 
mechanical support is anticipated.    
Since INTERMACS was originally conceived, penetration of this technology has advanced 
steadily but increasingly requires expanded expertise and commitment beyond the original 
investigators.  The presence and influence of a vital young academic cardiology cadre will be 
crucial to maintain skill and focus on clinical application during rapid evolution of mechanical 
support technology.  MedaMACS has been designed to meet the scientific goals required for 
progress while fostering the development of a core group of emerging heart failure cardiology 
investigators at key sites.  This group will provide leadership in the identification and follow-up of 
the cascading sequence of changing populations for mechanical support.   
 
 
2.  
SPECIFIC AIMS 
  
Aim 1: To identify prospectively a population of ambulatory patients followed on optimal 
medical therapy for whom chronic heart failure limits both function and survival to a range 
where elective implantation of left ventricular assist devices should offer meaningful benefit.   
Hypothesis:  It will be possible to define baseline characteristics that predict a one year mortality 
of ≥35±5% for patients on medical therapy and ≤25±5% in patients who have had VAD.   
 
1.1 Characterize ambulatory patients with advanced heart failure by baseline variables and 
interval change, including functional capacity, frailty, and interest in technical 
intervention. There will be two baseline measurements one month a part (used to predict 
early stability) and a repeated set at 1 year (used to predict outcome between 1 and 2 
years). These outcomes will include: a). All clinical parameters required for current risk 
score models and prediction, b). Follow-up values and changes from baseline to 1 month 
and baseline to 1 year as additional variables from which to predict outcomes on medical 
therapy, c). Functional capacity, quality of life, and patient preferences at baseline and 
after time. 
 
1.2 Distinguish between risk factors that predict mortality whether therapy is medical or 
mechanical, and those risk factors that predict outcomes with different impact depending 
on whether therapy is medical or VAD. This will be stratified by whether patient is 
eligible for transplant or not.  
 
1.3  Record outcomes for ambulatory patients with advanced heart failure for comparison to 
VAD, to include a). Hospitalizations, VAD, transplant, survival without VAD or 
transplant, b). Major adverse events leading to hospitalization, including infection, 
neurologic or bleeding, c). Functional capacity and quality of life, d). Eligibility for 
transplant, e). Changes in resuscitation status and enrollment in hospice. 
 
Aim 2:  To design an integrated endpoint of survival and objective functional assessment that 
provides more discrimination between chronic ambulatory heart failure and current device 
outcomes than survival alone.  
Hypothesis: It will be possible to identify a composite endpoint of survival and function that will 
show a 30% difference between outcomes on medical therapy and outcome with initial VAD.  

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2.1 Compare survival and functional outcomes for MedaMACS patients and recipients of 
single LVAD.   Within each Profile 4-7, comparisons will be made for a). Clinical 
characteristics, b). Outcomes, and c). Outcomes for populations from INTERMACS and 
MedaMACS defined by other baseline variables and risk scores.  
 
2.2 Define changes in 6 minute walk between baselines and 12 and 24 months after 
enrollment, to determine timing, frequency and degree of improvement for Profiles 4-7 
after entry for medical therapy, and compare to INTERMACS results.  
 
2.3 Define changes in quality of life as assessed by Euroqol-5D and Kansas City 
Cardiomyopathy Questionnaire.  
 
2.4 Analyze distribution of integrated functional survival endpoints to a). Inform individual 
patient decisions between VAD and medical therapy, b). Provide basis of evidence Level 
B for VAD guidelines, and c). Estimate sample sizes for pivotal randomized trials.  
 
Aim 3:  To evaluate patient perceptions about their cardiac condition, ventricular assist 
device technology, preferences for their care, and thresholds for considering device implant.  
Hypothesis: The likelihood of patients expressing preference for a VAD will increase with their 
functional limitation and with their predicted risk of 1 year mortality. 
 
3.1 Patient functional capacity by questionnaire and 6 minute walk will be assessed twice at 
baseline and again at 1 year and 2 years. These will be compared to their expressed 
willingness to consider VAD at their own level of function and from a series of 
hypothetically worse states. 
 
3.2 Available relevant mortality prediction models will be used to estimate 1 year survival 
from baseline and from the one-year re-evaluation.  These will be compared to patients’ 
expressed willingness to consider VAD at their own anticipated level of survival and from 
a series of hypothetically worse states.  
 
Aim 4:  To develop and advance a team of emerging clinical investigators who will increase 
awareness of mechanical circulatory support and serve as role models for other young 
investigators.    
Hypothesis: Focused mentorship of early career investigators will foster the next generation of 
leaders in the clinical care of patients with advanced heart disease. 
 
4.2 Junior investigators will take the lead in designing and analyzing research questions of 
their own with the mentorship of a more senior investigator at each clinical site.  
Presentation of MedaMACS data at national meetings and manuscript development will be 
the responsibility of junior investigators.  A centralized publications committee with 
ensure equitable distribution of topics of interest so there is synergy across centers and 
appropriate extramural mentorship is available.   
 
4.3 Survey will be made among the young investigators for VAD-related publications, 
progression to independent research status, and affiliation to clinical VAD programs at 5 
years after initiation of MedaMACS enrollment.  

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3.  
BACKGROUND - Gaps in Current Medical Trial Data 
 
Survival Prediction 
The available information from the randomized trials of REMATCH, INTREPID, and the 
combined experiences of other inotrope-dependent patients provides a boundary of approximately 
75% one-year mortality on medical therapies.2,8  On the other side are the experiences in 
ambulatory outpatients with advanced heart failure, in which one-year mortality is no more than 
20% in trial of beta blockers, aldosterone antagonists, and cardiac resynchronization. These medical 
trial populations thus cannot be aligned with experiences with approved VADs, in whom the one-
year mortality was in the range of 30% as reported specifically in the HeartMate II device trial.4  
The one-year mortality in the broader experience of approved devices for destination therapy is 
declining toward 20% in INTERMACS when outcomes are isolated for LVAD without right 
ventricular support.6  These sets of medical data thus leave unaddressed the wide chasm of one year 
medical mortality between 25 and 75%, within which falls the largest anticipated target population 
of ambulatory patients for current implantable VADs.  
This chasm cannot be crossed by community registry populations which describe overall 
one-year mortality of 30-50%, but an average age is 75.9,10  with prevalence of co-morbidities such 
as chronic pulmonary disease and peripheral vascular disease in the range of 30%, with dementia, 
stroke, cancer each in the range of 10-20%.10  Multiple risk scores exist from these populations for 
the identification of patients at high risk for death from heart failure,11-13 but they do not fully 
address the challenge of identifying ambulatory patients both sick enough and well enough to 
benefit from VADs.  On the other end, scores developed from low-ejection fraction HF outpatient 
trials depict a young population with one-year survival rates in the range of 80-90%. In the pyramid 
of risk for death during heart failure, the largest level is the bed of risk at the base, in which the 
patient remains regardless of therapy received, with such risks as intrinsic liver and lung disease, 
and the intermediate levels where malnutrition and renal dysfunction compromise peri-operative 
outcomes and offer uncertain potential for improvement. (Figure 2)  Potential for benefit is reflected 
best at the top of the pyramid with risk factors such as hemodynamics and biomarkers that are 
addressed directly by VAD (or transplant).  The robust Seattle Heart Failure Score versions, derived 
from disparate trial populations, have been used to identify very low risk patients and patients with 
heart failure severity unlikely to benefit from ICD therapy.11  This score will provide the central 
screen for the ongoing REVIVE-IT trial in ambulatory heart failure.  The MedaMACS population is 
designed to surround the 50 patients on medical therapy in the REVIVE it trial, by including 
patients deemed sicker by recent hospitalization, but also less sick patients who have a Seattle score 
below the 1.5 threshold.  The accuracy of current criteria to select this spectrum of patients has been 
shown in analysis of the first140 patients of the MedaMACS screening pilot. (Figure 3) 
 
Functional Capacity Prediction 
 
As we look toward the “less sick” populations, it has been shown that patient interest in 
VAD is heavily influenced by the degree of functional limitation. The increasing focus on shared 
decision-making in advanced heart failure requires that patients be given adequate information to 
choose among available options.  This requires better understanding of the expected functional 
outcome on medical therapy as well as with VADs.  Current reports of those patients providing 
serial information on quality of life through Kansas City questionnaires indicate improvement in the 
range of 40 points after VAD,  which exceeds that demonstrated with any other therapy thus far. 
However, these are from baseline levels substantially worse than anticipated for most ambulatory 

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heart failure.14  Similarly, 6 minute walk distances show major improvement but the majority of 
patients were unable to perform them at baseline.14  Contemporary information about the trajectory 
of quality of life and functional capacity over 12-24 months for patients ambulatory at baseline will 
be crucial to understanding and communicating expected benefits from VAD.  
 
Benefit Emphasized Over Risk 
MedaMACS outcomes will accelerate the progress of mechanical circulatory support into 
the ambulatory population.  Outcomes will include the rate of progression to transplant or VAD 
after enrollment for 2 years.  For those patients remaining alive on medical therapy, information 
will include key adverse events and functional capacity in detail for one year and general life 
satisfaction at one and 2 years.  Taken in conjunction with INTERMACS data, the MedaMACS 
baseline and outcome data will facilitate the important evolution from risk scores to “Benefit 
Scores”, which reflect both the anticipated risk without MCS and the likelihood of good outcomes 
with MCS. This will be used both for overall survival, survival without major events, and adjusted 
scores that include quality of life and functional capacity in survivors. 
   
 
4.  
PRELIMINARY DATA 
 
INTERMACS Provides the Framework  
 
INTERMACS has established the key questions in the potential VAD populations along 
with the data field framework upon which to answer them for ambulatory patients on medical 
therapy.  INTERMACS has validated clinical patient profiles and has established new branch points 
for decisions regarding transplant.15  Outcome fields for specific adverse events, re-hospitalization, 
cardiac function, systemic markers of disease, and functional capacity are designed to capture not 
only endpoints but the crucial midpoints that allow an integration of function and survival over 
time.  The architecture for INTERMACS has been designed from its inception to favor entry of 
parallel information for patients receiving contemporary medical therapies.   The experience from 
INTERMACS will illuminate the establishment of MedaMACS. 
 
MedaMACS Screening Pilot Confirms Feasibility 
 
A MedaMACS Screening Pilot study has completed enrollment at 10 JCAHO-certified 
mechanical circulatory support programs across the United States.  Using similar entry criteria as 
outlined below, 168 patients with advanced ambulatory systolic heart failure and recurrent 
hospitalizations were enrolled over 7 months.  Comprehensive baseline data of usual care practices 
was recorded along with a 14 item patient questionnaire and the Euroqol-5D instrument.  Rapid 
enrollment confirmed that a population of advanced ambulatory heart failure patients could be 
readily identified and enrolled in an observational study.  Data from the screening pilot suggests 
that there may be a cohort of patients who may benefit from LVAD therapy who are not yet being 
considered for mechanical support.16 (Figure 4)  In addition, the patient survey instrument 
successfully provided information on patient perception of VAD technology, thresholds for implant, 
and goals of therapy.17 The screening pilot is also being used to field test telephone administered 
outcome measures at 6 and 12 months after enrollment.  Baseline entry criteria, data elements and 
survey items have been refined based on collaborative feedback from the screening pilot effort.  The 
successful design and implementation of the screening pilot over a short time-line demonstrates that 
such an effort can be led by junior investigators committed to clinical research in advanced heart 
disease.   
 

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5.  
STUDY DESIGN AND POPULATION 
 
Screening and Determination of Eligibility 
 
The MedaMACS Pilot Study is a prospective, observational study of ambulatory patients 
with advanced heart failure.  The study will enroll patients who have not yet received an LVAD but 
who receive their care at a hospital with a JCAHO-certified mechanical circulatory support 
program.  
 
Participants will be screened for this study if they are between 18 and 80 years old with low 
ejection fraction history and hospitalization for heart failure within the past year.   All subjects who 
have been admitted to the heart failure service will be screened and recruited for study participation, 
either as an inpatient or during their first outpatient follow-up appointment by the investigator and 
research staff caring for patients in the advanced heart failure program of the enrolling center.  
Eligibility for enrollment will be based only upon information that is clinically available at the time 
of screening.  The history of prior hospitalizations will be available from clinical records.  Routine 
evaluation and triage of ambulatory patients with advanced heart disease includes echocardiography 
and functional assessment with peak oxygen consumption and frequently 6 minute walk distance, 
The elements of information required for estimation of the Seattle Heart Failure Risk score will be 
gleaned from laboratory data, or imputed as described in the Seattle score literature. The research 
coordinator will enter this data onto a currently available website for calculation of the Seattle 
score.  
 
Eligibility will be determined by the inclusion and exclusion criteria listed below. The 
inclusion criteria will be checked individually for “all that apply, with numeric values where 
indicated.  The exclusion criteria will be verified by individual checkboxes. After determination of 
eligibility, a member of the study research team will approach individual subjects who are potential 
candidates for participation and for whom the subject’s primary physician has given permission to 
approach.  No group of persons will be excluded without a good scientific or ethical reason to do so.  
Incarcerated prisoners have been excluded by this protocol.  For patients who meet inclusion 
criteria but who have an exclusion criteria related to non-cardiac conditions (Exclusion Criterion #2, 
#3, or #9) and for eligible patients not providing informed consent, a screening log will be kept that 
includes their age, ejection fraction, race and gender.  This basic information is necessary to assess 
completeness of patient capture and possible bias in the screening process and in the process to 
obtain informed consent.  No further information will be collected on patients who do not meet the 
inclusion criteria.  This screening log will help determine the total number of advanced ambulatory 
heart failure patients eligible for MedaMACS participation at each study center. 
 
We anticipate a total of 350 patients enrolled from 12 centers in the United States over a 12 
month period.  The screening pilot enrolled 168 patients at 10 sites during a 7 month period.  Once 
all sites are activated with enrollment of approximately 3 patients per month per site, enrollment 
should be complete by 12 months.  
 
Inclusion Criteria 
1. 
Age 18-80 years 
2. 
NYHA class III-IV heart failure for 45 of the last 60 days 
3. 
Left ventricular ejection fraction ≤ 35% 
4. 
Heart failure diagnosis or typical symptoms for 12 months 
5. 
Use of evidence based oral medications (beta-blockers, ACE-inhibitors/ARBs, 
aldosterone antagonist) for at least 3 months prior to enrollment or documented 
medication contraindication or intolerance. 

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6. 
Hospitalization for heart failure within the previous 12 months (other than for elective 
procedure) 
7. 
Informed consent given 
 
In addition, they must have at least one of the following:  
 
A. An additional unplanned hospitalization during the previous 12 months for a total of at 
least 2 inpatient hospitalizations lasting >24 hours with heart failure as the primary or 
secondary diagnosis within the previous 12 months  
 
OR  
 
B.  
1) Peak oxygen uptake (VO2) <55% of age- and sex-predicted (using Wasserman equation) 
OR a peak VO2 ≤16 ml/kg/min for men and ≤14 ml/kg/min for women in a test with an 
RER >1.08 on cardiopulmonary exercise testing. 
2) 6-minute walk distance <300 meters without non-cardiac limitation.  
3) Serum BNP > 1000  (NT-proBNP > 4000 pg/ml) as outpatient or at hospital discharge.  
 
OR 
 
C. Seattle Heart Failure Model Score > 1.5.   
 
Available information will be obtained on all of these inclusion criteria for comparison 
regardless of which meets eligibility.  
 
Exclusion Criteria 
1. Age >80 years or <18 years 
2. Non-cardiac diagnosis anticipated to limit 2-year survival (≥30-50% mortality within 2 years 
from non-cardiac diagnosis)   
3.  Primary functional limitation from non-cardiac diagnosis even if not likely to limit survival 
4. QRS > 120msec and planned biventricular pacemaker implant or biventricular pacemaker 
implantation within past 90 days 
5. Current home intravenous inotrope therapy 
6. Chronic hemodialysis or peritoneal dialysis 
7. Scheduled for non-ventricular assist device cardiac surgery on current hospital admission 
8. Obvious anatomical or other major contra-indication to any cardiac surgery in the future (e.g. 
previous pneumonectomy, advanced connective tissue disease) 
9. Actively listed for heart transplant as UNOS Status 1 or 2 
10. History of cardiac amyloidosis 
11. Dominant lesion of at least moderate aortic or mitral stenosis or congenital structural heart 
defect.  
 
Informed Consent 
Informed consent is mandatory and will be obtained from all subjects prior to participation 
in this clinical study. Informed consent will be obtained in accordance with IRB policies and 
procedures.  Prior to inclusion in the study, it will be the responsibility of the Investigator to give 
each subject full and adequate verbal and written information about the objectives and the 

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procedures of the study and the possible risks involved. Each subject will be informed of their right 
to withdraw from the study at any time and for any reason without sanction, penalty, or loss of 
benefits to which they are otherwise entitled and that withdrawal from the study will not jeopardize 
their future medical care.   When a potential subject is the investigator’s own patient, it will be 
made clear to the potential subject that participation in the study is entirely voluntary and that their 
decision will not affect their care now or in the future. 
Furthermore, it will be the responsibility of the Investigator to obtain a signed Informed 
Consent form from each subject prior to administering any questionnaires, recording data into the 
Registry, or performing a 6 minute walk at a time other than when customarily performed in their 
routine clinical care.  Informed consent documents will be written in language understandable to the 
subject or subject’s legally authorized representative.  One copy of the signed Informed Consent 
form will be given to the subject and another retained by the Investigator.  
 
 
6.   
PROCEDURES AND ASSESSMENTS 
 
Clinical, Demographic and Laboratory Data  
 
It has been verified that the requested data is normally acquired in the routine care of these 
types of patients at the referral centers for the primary investigators.  Laboratory data will be 
obtained from routine blood draws as clinically indicated.  Peak VO2 and echocardiographic 
parameters will be measured once during the baseline period and once at the one-year visit as 
clinically indicated.  In those centers selected for MedaMACS, as in most centers with high volume 
advanced heart disease evaluation, peak VO2 and echocardiographic assessment are routine 
components of regular and serial evaluation.  Medications, cardiac imaging, and exercise testing 
will occur at the discretion of the physicians caring for each study participant.  Patient therapies and 
testing will conform to standards of care and published clinical guidelines for heart failure 
management.18, 19  Subjects will have additional functional assessment of both endurance (6 minute 
walk) and frailty (gait speed) one month after consent to measure early stability.  Registry data will 
be obtained by chart review from both the electronic medical record and paper charts.  Note all 
dates for baseline data will be by intervals rather than calendar dates. There will be a total of six 
data collection time points, four with face to face clinical encounters and another two by telephone: 
(Figure 5).   
 Baseline A – at consent (face to face encounter) 
 Baseline B – 3-6 weeks after consent (face to face encounter) 
 6 month telephone call by study site personnel 
 12 month follow-up – 10-14 months after consent (face to face encounter) 
 18 month telephone call by study site personnel 
 24 month follow-up – 22-26 months after consent (face to face encounter; study close) 
 
Patient Survey 
The patient survey instrument patient will assess quality of life, health-related stress, along 
with impression of and thresholds for consideration of VAD therapy .  All survey responses are for 
research only and as such will be kept confidential and not be shared with members of the medical 
team.  This written survey will be administered at Baseline A, Baseline B, the 12 month visit and 
again at the 24 month study close-out visit.  Patients will receive the Kansas City Cardiomyopathy 
Questionnaire to determine disease specific quality of life and with the Euroqol-5D questionnaire 
including the thermometer.  There will also be a brief instrument on health-related stress identical to 

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the tool being used in INTERMACS.  The quality of life instruments will be completed 
independently by the patients in a single session.   
After quality of life instruments have been completed, study coordinators will read a 
description of a VAD and provide a simple picture.  The description of a VAD will include 
information on implantation, anticipated benefits and potential risks.  The surveys will only be 
administered at face-to-face visits to allow a study nurse to assist with the VAD portion of the 
instrument.  Patients will complete the VAD portion of the survey after receiving this standard 
information and a study nurse will be present to answer any queries about the VAD items.  Similar 
VAD survey items were administered in the screening pilot with excellent response rates.17 Any 
patient who fails to complete a survey will have a missing data form filled outlining why the self-
reported instruments were not completed.  In all, there are 40 questions for the patients to answer in 
a written format, which should take approximately 35-40 minutes for most subjects.  Original 
copies of the written survey instruments will be kept on site by study investigators and the data will 
be entered electronically by the coordinators or their data assistants on site (Appendix A).  
  
Follow-up Telephone Contact at 6 and 18 Months 
 
A telephone call will be made by local site investigators or their study staff to patients at 6 
and 18 months after study enrollment, but can be replaced by face-to-face visits if occurring as part 
of their routine care.  The major purpose of this telephone call is to assess patient vital status and 
important interval events such as transplantation, placement of VAD, or other major procedures.  
The second purpose is to estimate the number of hospitalizations after study enrollment and the 
frequency of other major adverse events as recalled by patients.  Any major endpoint or adverse 
event will be confirmed or amended by review of the interim medical record by site investigators. 
At the time of each study follow-up visit, the records will be reviewed for evidence of any major 
events leading to hospitalization that might have been missed in the intervening phone interviews.  
Patients will be asked the questions determining functional class and profile.  They will be 
asked a small subset of questions from the baseline questionnaire related to preferences for 
resuscitation and for VAD.  They will be asked to bring their medications to the telephone to list 
them with doses.   
 
Clinical Assessment at 12 months   
 
It is anticipated that patients will be seen at some time between 10 and 14 months after the 
baseline study.  Clinical information including routine laboratory values, physical examination, and 
symptoms will be reported.  An echocardiogram and peak oxygen consumption will be recorded if 
obtained for clinical indications, as is routine practice for patients with this severity of illness 
followed at major centers. The six minute walk functional test will be repeated along with 5 meter 
gait speed to assess frailty.  Patients will be re-administered the questionnaires regarding functional 
status, quality of life, and preferences for VAD as at baseline.  
 
End of study at 24 months 
A final face to face encounter at 24 months (between 22-26 months) will be completed and 
the study will end at that point.  Routine clinical assessment components and other study data 
elements will be recorded as available from the clinical record, including results of imaging and 
exercise testing for measurement of peak oxygen consumption, if performed at or after the 18 
month time point. The six minute walk functional test will be repeated along with 5 meter gait 
speed to assess frailty.  Patients will be re-administered the questionnaires regarding functional 
status, quality of life, and preferences for VAD as at baseline. Finally, at 24 months each patient 

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will receive the VAD survey and questionnaire to assess quality of life and perception of VAD 
technology.  After the two year face to face visit, study participation will end. 
 
Data Elements 
 
A full list of data elements to be collected along with collection times may be found in 
Appendix B.  All data will be recorded in a web based data entry system.   
 
 
7. 
DATA SAFETY AND MONITORING 
 
Data Safety and Coordination 
Data and safety monitoring, including guarding the rights, safety and welfare of subjects, 
will be the responsibility of the individual site investigators.  The data coordinating center for the 
study will be the University of Alabama Birmingham (UAB). All information collected in this 
registry will be held confidential to the extent permitted by law. No published or unpublished report 
or visual or speaking presentation about this study will include any material that will identify the 
subject as a participant in this study.  No specimens will be collected.  Subjects may withdraw 
consent from the study at any time.  Data will not be used by collaborators for any purposes other 
than those described in this protocol. 
 
Data will be transferred to the coordinating center electronically for all data.  Datasets 
containing de-identified individual data and summary data will be available to the INTERMACS 
and MedaMACS investigators and industry partners. All of the data systems feature multiple levels 
of security, which protect patient data by the most stringent requirements. All are fully compliant 
with the Health Insurance Portability and Accountability Act (HIPPA) and are certified by the 
Health Resources and Services Administration (HRSA).  Any plans for data to be shared with 
outside entities as part of contractual arrangements will be within the conditions specified in the 
patient consent form and detailed in contracts to be reviewed separately.  
 
Study Monitoring 
The Principal Investigator and UAB study staff will regularly monitor the data from the 
registry, review and assess the performance of its operation, and make recommendations, as 
appropriate, to participating institutions with respect to: 
 
 Enrollment and data timeliness and completeness from individual enrolling sites  
 Issues related to participant safety and informed consents  
 Adequacy of study progress in terms of recruitment, quality control, and 
          
data analysis 
 Issues pertaining to participant burden 
 Achievement on the main study goals 
 Possible modifications in the study protocol  
 Overall scientific direction of the registry 
 
 
8. 
ANALYSIS PLAN 
 
Planned Analysis 
 
The prospective data analysis is planned to be descriptive and correlative, with primary 
hypothesis outlined in the specific aims. Data collected are designed to generate future hypotheses.  

MEDAMACS Pilot Protocol 
11/08/2011 
 
  
 
 
 
 
13
Actuarial survival curves will be generated and compared to known risk models, such as the Seattle 
Heart Failure Score and the Heart Failure Survival Score, for both heart failure populations and 
VAD populations from INTERMACS.19, 20  In addition, new models will be developed to allow 
comparison of anticipated outcomes after VADs using INTERMACS data.  It is fully recognized 
that these comparisons will be inevitably limited through confounding by indication.  The intent is 
to identify a range of baseline profiles for which anticipated outcome with medical survival can be 
projected.  From this information, hypotheses can be generated for target populations in whom 
VAD therapy might be expected to yield better outcomes.  
 
 “Primary” Endpoint and Power 
As this is a registry designed to determine outcomes for particular groups of patients, there 
will be more than one “primary” endpoint depending on the level of severity of illness.  Knowing 
that survival in isolated VAD patients dominated by Profile I and II has reached 70% at one year,  it 
is reasonable to assume that survival will be at least as good or better with VAD in less sick 
patients.  We hypothesize that it will be possible to define baseline characteristics that predict a one 
year mortality of > 35 + 5% for patients on medical therapy and < 25 + 5% in patients who have 
had VAD.  Seeking a confidence interval of approximately + 5%, this could be accomplished 
initially with approximately 350 patients if they have similar risk.  We anticipate that some patients 
will have better survival and some may have worse survival than this, such that there will be 
overlapping groups, and a small proportion of patients will have other events ending their medical 
history, such as transplant and deterioration to worse INTERMACS profiles for which they receive 
VADs.   
For groups within which survival is > 60% at 1 year on medical therapy, a composite of 
survival and function will be the most important for comparison to VAD therapy.  We hypothesize 
that it will be possible to identify a composite endpoint of survival and function that will show a 
30% difference between outcomes on medical therapy and outcome with initial VAD.  The nature 
of these survival-function endpoints will be explored during the first year of MEDAMACS data.  
The two simplest will be done with the endpoints of days alive out of hospital, and quality-adjusted 
life years according to the Euroqol.  However, more complex endpoints will be tested for survival 
and functional scores at specific intervals.  (Figure 5) 
Information will be collected regarding the major adverse events that lead to hospitalization 
both with and without mechanical circulatory support, as listed in the data appendix: neurologic 
event, non-CNS bleeding, sepsis, ventricular tachyarrhythmia or ICD firing. The hospitalizations, 
adverse events and deaths will be attributed on site by the investigators in drop-down menus.  There 
will be no independent adjudication of causes of hospitalizations or deaths.  The general categories 
of causality are broad and not the focus of the primary hypotheses. Thus, the lack of formal 
adjudication should not compromise the data for the endpoints of death, transplant, and VAD, 
which are objectively determined.  The secondary endpoints that relate to functional capacity and to 
quality of life will also be specifically determined.  
 
Statistical Methods 
Baseline data between different patient groups will be compared using student’s t-test for 
normally distributed continuous variables and Wilcoxon Rank Sum for non-normally continuous 
variables.  Kaplan-Meier curves will be constructed for time to death, VAD or transplant for the 
two-year study period.  Event rates including cause-specific hospitalizations will be generated for 
different groups of patients and analyzed.  Analysis plans for specific research questions will be 
developed under guidance of the DCC after approval by the Research and Publication Committee. 

MEDAMACS Pilot Protocol 
11/08/2011 
 
  
 
 
 
 
14
All summary statistics and subsequent analysis will be performed using SAS statistical software 
(Cary, NC).  
 
Publications 
 
Publications will be authored by the primary investigators at the participating sites.  The senior site 
investigators will be offered inclusion as authors for the manuscripts on which they supervise the 
efforts of the investigator from their own site as first author.  Options may become available for 
research coordinators to author manuscripts on secondary analyses of topics of their special interest.  
 
 
9. 
MATURATION OF MEDAMACS 
 
 
The launching of MedaMACS will focus on the ambulatory population closely proximal to 
the common threshold for implementation of mechanical circulatory support.  The foundation of the 
10 sites entering patients for the MedaMACS screening pilot will allow rapid initiation of the new 
protocol, with the additional 2 sites.  The fastidious definition of entry criteria offers a fine-tuning 
knob to dial up or down disease acuity of patients enrolled, as this becomes necessary.   However, 
the general approach to baseline data collection, risk profiling, assessment of adverse events and 
outcomes, is planned for cascading applications to progressively “more well” populations to keep 
pace with those enrolling into INTERMACS.   The next promising target population will be the 
population of patients waiting at home after listing for transplant.  As the improving outcomes of 
implantable VADs are demonstrated by INTERMACS to advance beyond a bridge to transplant, 
past even the current focus on the transplant-ineligible population, MedaMACS will prepare the 
field on which mechanical circulatory support will finally compete with cardiac transplantation as 
primary therapy to improve quality life for a substantial proportion of the patients currently 
suffering from advanced heart failure.  
 
 
10. 
PARTICIPATING CENTERS 
 
 
Study protocol and informed consent forms will be approved by local institutional review 
board prior to study enrollment.  Participating centers will include: 
 
 Brigham and Women’s Hospital (Boston, MA) 
 Cleveland Clinic (Cleveland, OH) 
 Cedars Sinai Hospital (Los Angeles, CA) 
 Duke University Hospital (Durham, NC) 
 Hospital of the University of Pennsylvania (Philadelphia, PA) 
 University of Alabama, Birmingham (Birmingham, AL) 
 University of Colorado (Denver, CO) 
 University of Iowa Hospitals (Iowa City, IA) 
 University of Michigan Hospital (Ann Arbor, MI)  
 University of Pittsburgh Medical Center (Pittsburgh, PA) 
 University of South Florida,  (Tampa FL) 
 University of Texas Southwestern Medical Center (Dallas, TX) 
 

MEDAMACS Pilot Protocol 
11/08/2011 
 
  
 
 
 
 
15
It is anticipated that sites will begin to be activated early in 2012 such that several will have 
some initial experience prior to the first investigators’ meeting.  (Figure 6)  This meeting is planned 
for March 2012 to include both investigators and a research nurse, with one plenary session for 
general trial structure and goals, followed by split sessions for the investigators and the research 
nurses.  The focus of the investigator sessions will be review of the hypotheses, primary and 
secondary endpoints, initial timetable for publications of baseline data, and primary endpoint data 
after completion of one year of follow-up for all patients enrolled within year 1.  The research 
nurses will receive orientation and instruction regarding the informed consent procedures and 
documentation, conduct of the study, and electronic data entry.   
 
 
REFERENCES 
 
1. Lloyd-Jones D, Adams RJ, Brown TM, et al. Heart disease and stroke statistics—2010 update: a 
report from the American Heart Association.  Circulation  2010;121(7):e46-e215. 
2. Rose EA, Gelijns AC, Moskowitz AJ, et al. Long-term mechanical left ventricular assistance for 
end-stage heart failure. The New England journal of medicine. 2001;345(20):1435-1443. 
3. Miller LW, Pagani FD, Russell SD, et al. Use of a continuous-flow device in patients awaiting 
heart transplantation. The New England journal of medicine. 2007;357(9):885-896. 
4. Slaughter MS, Rogers JG, Milano CA, et al. Advanced heart failure treated with continuous-flow 
left ventricular assist device. The New England journal of medicine. 2009;361(23):2241-
2251. 
5. Kirklin JK, Naftel DC. Mechanical circulatory support: registering a therapy in evolution. Circ 
Heart Fail. 2008;1(3):200-205. 
6. Kirklin JK, Naftel DC, Kormos RL, et al. Third INTERMACS Annual Report: the evolution of 
destination therapy in the United States. J Heart Lung Transplant. 2011;30:115-123. 
7. Stewart GC, Brooks K, Pratibhu PP, et al. Thresholds of physical activity and life expectancy for 
patients considering destination ventricular assist devices. J Heart Lung Transplant. 
2009;28(9):863-869. 
8. Rogers, J.G., et al., Chronic mechanical circulatory support for inotrope-dependent heart failure 
patients who are not transplant candidates: results of the INTrEPID Trial. J Am Coll 
Cardiol, 2007. 50(8): p. 741-7. 
9. Yancy, C.W., et al., Clinical presentation, management, and in-hospital outcomes of patients 
admitted with acute decompensated heart failure with preserved systolic function: a report 
from the Acute Decompensated Heart Failure National Registry (ADHERE) Database. J Am 
Coll Cardiol, 2006. 47(1): p. 76-84. 
10. Setoguchi S, Stevenson LW, Schneeweiss S. Repeated hospitalizations predict mortality in the 
community population with heart failure. American heart journal. 2007;154(2):260-266. 
11.  Levy, W.C., et al., The Seattle Heart Failure Model: prediction of survival in heart failure. 
Circulation, 2006. 113(11): p. 1424-33. 
12.  O'Connor, C.M., et al., The ESCAPE Discharge Risk Score: Don't  Go Home Without It 
(abstract). J  Cardiac Failure, 2006 12(6): p. S117. 
13.  Aaronson, K.D., et al., Development and prospective validation of a clinical index to predict 
survival in ambulatory patients referred for cardiac transplant evaluation. Circulation, 1997. 
95(12): p. 2660-7. 
14.  Rogers JG, Aaronson KD, Boyle AJ, Russell SD, Milano CA, Pagani FD, Edwards BS, Park S, 
John R, Conte JV, Farrar DJ, Slaughter MS. Continuous flow left ventricular assist device 

MEDAMACS Pilot Protocol 
11/08/2011 
 
  
 
 
 
 
16
improves functional capacity and quality of life of advanced heart failure patients. J Am Coll 
Cardiol. 2010;55(17):1826-1834. 
15. Stevenson LW, Pagani FD, Young JB, et al.  INTERMACS profiles of advanced heart failure: 
the current picture. J Heart Lung Transplant. 2009;28(6):535-541. 
16. Cowger JA, Kittleson M, Patel C, et al. End stage heart failure: Are we missing patients who 
may benefit from left ventricular assist device support?  American Heart Associated 
Scientific Sessions 2011 (abstract) 
17. Stewart GC, Kittleson M, Cowger JA, et al. Who wants an LVAD for ambulatory heart failure?  
Heart Failure Society of American Annual Scientific Meeting 2011 (abstract)  
18. Hunt SA, Abraham WT, Chin MH, et al. Focused update incorporated into the ACC/AHA 2005 
guidelines for the diagnosis and management of heart failure in adults.  Circulation  
2009;119(14):e391-e479. 
19. Lindenfeld J, Albert NM, Boehmer JP, et al.  HFSA 2010 Comprehensive Heart Failure Practice 
Guideline.  J Cardiac Failure  2010;16(6):e1-e194. 
20. Lietz K, Long JW, Kfoury AG, et al.  Outcomes of left ventricular assist device implantation as 
destination therapy in the post-REMATCH era: implications for patient selection. 
Circulation. 2007;116(5):497-505. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

MEDAMACS Pilot Protocol 
11/08/2011 
 
  
 
 
 
 
17
 
 
 
 
 
 
 
FIGURE 1: 
 
IIIB
IIIB-IV
IV
IV 
IV
IV
IV
NYHA
Official Shorthand
PROFILE-LEVEL
“Housebound”,
Comfortable at rest, 
symptoms with 
minimum activity ADL
INTERMACS
LEVEL 5
(FF, A)
Resting symptoms 
on oral therapy at 
home
INTERMACS
LEVEL 4
(FF, A)
Stable but Ino-
Dependent
INTERMACS
LEVEL 3
“Sliding fast” on ino
INTERMACS
LEVEL 2
Advanced Class III 
INTERMACS
LEVEL 7
“Walking wounded”-
ADL possible but 
meaningful activity 
limited
INTERMACS
LEVEL 6
(FF, A)
“Crash and burn”
INTERMACS
LEVEL 1
MED and VAD both poor
Need temporary bridge
To successful VAD
MEDAMACS
Targets
VAD better than
MED
Current Status
Of Information
INTREPID
REMATCH
HMII DT
Medical 
HF
Outpt Trials
Only
Entered
In
Hospital
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

MEDAMACS Pilot Protocol 
11/08/2011 
 
  
 
 
 
 
18
 
FIGURE 2: 
      
Pyramid of Risk Factors for HF Death
BNP
Na  
Malnutrition
Inflammation
Risk removed with
Transplant or VAD
Peri-op risk
may diminish 
later
De-conditioning
SBP
Renal function
Age
RV dysfunction 
For VAD
Liver dysfunction
Non-compliance
Limited social support
BED of RISK
Cardiac-related
Functional impairment
6 MW, Peak VO2
Hemodynamic
Compromise
(echo,cath,etc)
?
Figure 3
 
FIGURE 3: 
 
SHFM < 1.5
SHFM 1.5-2.5
SHFM > 2.5
Total N =140
N=90
N=39
N=11
EF
0.18
0.16
0.18
NYHA IV
IIIB
IIIA
6%
43%
51%
18%
41%
41%
55%
36%
9%
INTERMACS
4 (resting symptoms)
5
6
7
17%
33%
34%
16%
31%
23%
39%
8%
46%
46%
9%
Mod-Sev RV Dysfn
34%
56%
64%
Cardiac Index
(72% pts)
2.2
2.0
2.0
No Eval Yet for either 
Tranplant or VAD
57%
44%
27%
Profiles in Ongoing MEDAMACS Screening Pilot
Profiles in Ongoing MEDAMACS Screening Pilot
EF, ejection fraction; SHFM, Seattle Heart Failure Model Score; 
RV, right ventricle; VAD, ventricular assist device
 

MEDAMACS Pilot Protocol 
11/08/2011 
 
  
 
 
 
 
19
 
 
FIGURE 4: 
Level of Illness in MEDAMACS 
Screening Pilot
---
---
13.1 (4.1)
Peak VO2, ml/kg/min
182 (140)
---
253 (134)
6 minute walk test, m
15 (6)
17 (5)
17 (6)
LV ejection fraction, %
MEDAMACS
N=140
REMATCH2
HMII-DT3
Inotrope use
20%
(within 6 mo)
71%
(at implant)
79%
(at implant)
Systolic blood pressure, 
mmHg
112 (18)
101 (15)
104 (14)
RA pressure, mmHg
13 (7)
----
13 (6)
Wedge pressure, mmHg
22 (9)
25 (10)
24 (8)
Cardiac index, L/min*m2
2.1 (0.5)
1.9 (1.0)
2.0 (0.6)
Continuous variables, mean (SD)
Adapted from Cowger et al.16
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

MEDAMACS Pilot Protocol 
11/08/2011 
 
  
 
 
 
 
20
FIGURE 5: 
 
6MW
Gait speed
Euroqol+KCCQ
VAD Survey
Events
Risk Scores
Treatments
Baseline
B
MedaMACS Study Timeline
Inpt or
outpt
1 Month
Re-Look 
Baseline
6 mos
Phone
Interview
1 Yr
Face-to-face
18m        2 Yrs
Phone
Interview
outpt
A
B
C
Time
Zero
D
Telephone Contact
6MW
Gait speed
Euroqol+KCCQ
VAD Survey
Risk Scores
6MW
Gait speed
Euroqol+KCCQ
VAD Survey
Events
Treatments
Baseline
A
6MW
Gait speed
Euroqol+KCCQ
VAD Survey
Events
Risk Scores
Treatments
Study Site Phone Calls 6 and 18 mos
Events (hosp, stroke, transplant, vad, inotropes, death)
Meds, Euroqol, NYHA/INTERMACS profile
One
month
Consent
1mo.
12mos 
24mos
Face-to-face 
encounters
End
1-Yr Visit
C
2-Yr Visit
D
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

MEDAMACS Pilot Protocol 
11/08/2011 
 
  
 
 
 
 
21
FIGURE 6: 
 
MedaMACS Pilot
2012
2013
2014
2015
First Site Activation
(2/1/2012)
Enrollment
N=350 x 12sites
Design MedaMACS II
2016
OSMB Review &
DCC IRB
(11-12/2011)
6M Telephone
18M Telephone
1 Year Visit
2 Year Visit & 
Closeout
WBDE Programming
Double Baselines
Completed
DATABASE
CLOSED
1 Yr Data 
Complete
IRB
STUDY SITE 
STUDY SITE 
DCC + STEERING
DCC + STEERING
MedaMACS Registry Timeline
Pilot Complete
Late-Breaking
Submission
Baseline Data 
ISHLT/ACC
Final Pilot
Analysis
Training
3/11/12
Annual Mtg
@ISHLT
Annual Mtg
@ISHLT
Annual Mtg
@ISHLT
Late-Breaking
Submission
6M Data Complete
Estimated 
# Active Pts
0        90
160        250        270        260         250          235  
210       180      150 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

MEDAMACS Pilot Protocol 
11/08/2011 
 
  
 
 
 
 
22
APPENDIX A:   SURVEY INSTRUMENTS 
 
 
Part I: Physician Questionnaire: 
 
Baseline A only 
 
1.  How long has patient been under care of your heart failure program? 
 
 
A. < 3 months                
 
B.  3-12 months               
 
C.  1-2 years          
 
D.  > 2 years 
 
2. If  <3 months, which best describes the route of presentation: (as a drop-down from A in 
previous) 
 
A.   New onset event or diagnosis within your institution 
B.   Unspecified evaluation of severe heart failure 
 
C.   Cardiac transplant and/or VAD evaluation 
 
3.  If a referral (B or C above), who referred the patient?   (as a drop-down from B/C in previous) 
 
 
A. Local internist 
B. Local cardiologist 
C. Cardiac Surgeon 
D. Self-referral 
E. Unsure 
F. Other 
 
 
Baseline A, B, 12 months and 24 months: (face to face encounters) 
 
4.  What is your best estimate of the likelihood of becoming sick enough to warrant urgent Stage D 
intervention within one year?  This includes home inotropes, hospice, VAD, and urgent transplant. 
A. Highly LIKELY 
B. Moderately LIKELY 
C. Uncertain 
D. Moderately UNLIKELY 
E. Highly UNLIKELY 
 
5.  If it became medically indicated, what is likelihood that this patient would be eligible for 
transplant?  
A. Highly LIKELY 
B. Moderately LIKELY 
C. Uncertain 
D. Moderately UNLIKELY 
E. Highly UNLIKELY 

MEDAMACS Pilot Protocol 
11/08/2011 
 
  
 
 
 
 
23
 
6.  If a VAD became medically indicated, what is the likelihood that they would require 
biventricular mechanical support rather than LVAD alone? 
(answer only if C, D, E in question 5) 
A. Definitely need Biventricular support 
B. Probably need Biventricular support 
C. Uncertain 
D. Probably LVAD only 
E. Definitely LVAD only 
 
7.  If not likely to be a transplant candidate and mechanical support became medically indicated, 
what is likelihood the patient would be eligible for destination LVAD alone as lifetime support? 
 (answer only if C, D, E above) 
A.  Highly LIKELY 
B. Moderately LIKELY 
C. Uncertain 
D. Moderately UNLIKELY 
E. Highly UNLIKELY 
 
 

MEDAMACS Pilot Protocol 
11/08/2011 
 
  
 
 
 
 
24
 Part II: Patient Quality of Life Questionnaires 
 
 
Baseline A,  B,  12 months, and 24 months 
 
1. Which of the following best describes your main activity? 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Actively working 
 
 
Full Time ⁯ Part Time ⁯  
 
 
 
Student 
 
 
 
Full Time ⁯ Part Time ⁯  
 
 
 
Seeking work  
 
 
Full Time ⁯ Part Time ⁯  
 
 
 
Too sick to work (disabled)  
 
     ⁯ 
 
 
 
Retired 
 
 
 
 
     ⁯ 
 
 
 
Keeping house 
 
 
 
     ⁯ 
 
 
 
Other (please specify)  
 
       _________ 
 
2.  How many of your close friends or relatives do you see in person, speak to on the telephone,  
     or contact via the internet at least once a month?  __________  (please count each person 1 time) 
 
3.  How much stress do you feel you’ve been under during the past 1 month, related to your health 
issues?  
 
1             2            3             4             5            6             7            8           9            10  
       No stress                                                                                                   Very much stress   
 
4. How well do you feel you’ve been coping with or handling your stress during the past 1 month, 
related to your health issues? 
 
 
1            2           3           4           5          6          7          8            9           10 
        Coping very poorly   
 
 
 
                        Coping very well 
 
5. How confident are you that you can do the tasks and activities needed to manage your heart 
failure so as to reduce how much having heart failure affects your everyday life? 
                    
1            2           3           4           5      6           7              8              9              10 
       Not at all confident 
 
 
                                                     Totally confident 
 
6. How satisfied are you with the results of your therapy for heart failure during the past 6 months?  
Please circle a number from 1 to 10, with 1 meaning that you’re not satisfied at all, and 10 meaning 
that you’re very satisfied. 
 
 
 
1          2           3          4           5          6             7              8              9              10  
 
Not satisfied  
 
 
 
 
 
          Very satisfied 
 
 
 
 
 
 
 
 
 

MEDAMACS Pilot Protocol 
11/08/2011 
 
  
 
 
 
 
25
 
 
 
EUROQOL – 5D + Thremometer 
 
 
By placing a checkmark in one box in each group below, please indicate which statements best describe your own health state today. 
 
Mobility 
I have no problems in walking about 
 
 
I have some problems in walking about 
 
 
I am confined to bed 
 
 
 
Self-Care 
I have no problems with self-care 
 
 
I have some problems washing or dressing myself 
 
 
I am unable to wash or dress myself 
 
 
 
Usual Activities (e.g. work, study, housework, family or 
leisure activities) 
I have no problems with performing my usual activities 
 
 
I have some problems with performing my usual activities 
 
 
I am unable to perform my usual activities 
 
 
 
Pain/Discomfort 
I have no pain or discomort 
 
 
I have moderate pain or discomfort 
 
 
I have extreme pain or discomfort 
 
 
 
Anxiety/Depression 
I am not anxious or depressed 
 
 
I am moderately anxious or depressed 
 
 
I am extremely anxious or depressed 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

MEDAMACS Pilot Protocol 
11/08/2011 
 
  
 
 
 
 
26
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
To help people say how good or bad a health state is, we have 
drawn a scale (rather like a thermometer) on which the best state 
you can imagine is marked 100 and the worst state you can 
imagine is marked 0.    
 
 
We would like you to indicate on this scale how good or bad your 
own health is today, in your opinion. Please do this by drawing a 
line from the box below to whichever point on the scale indicates 
how good or bad your health state is today. 
 
 
 
  
9 0 
8 0 
7 0 
6 0 
5 0 
4 0 
3 0 
2 0 
1 0 
100 
Worst 
imaginable 
health state 
0 
Best  
imaginable 
health state 
Your own 
health state 
today 

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 27 
 
THE KANSAS CITY CARDIOMYOPATHY QUESTIONNAIRE: 
 
The following questions refer to your heart failure and how it may affect your life.  Please read and complete 
the following questions.  There are no right or wrong answers. Please mark the answer that best applies to you.  
Heart Failure affects different people in different ways.  Some feel shortness of breath while others feel 
    fatigue.  Please indicate how much you are limited by heart failure (shortness of breath or fatigue) in 
    your ability to do the following activities over the past 2 weeks. 
 
 
 
 
           Place an X in one box on each line 
Activity  
    Extremely 
Quite a bit     Moderately   Slightly          Not at all     Limited for other reasons 
 
 
     limited 
 limited           limited 
   limited             limited 
 or did not do the activity 
Dressing yourself          ⁯  
      ⁯  
⁯      
    ⁯                       ⁯                                    ⁯ 
Showering/Bathing             ⁯ 
      ⁯  
⁯     
    ⁯                       ⁯                                    ⁯ 
Walking 1 block on  
    level ground 
         ⁯                    ⁯                 ⁯      
    ⁯                       ⁯                                    ⁯ 
Doing yard work, 
    housework or 
    carrying groceries           ⁯ 
      ⁯                 ⁯   
    ⁯                       ⁯                                    ⁯ 
Climbing a flight of 
     stairs without 
     stopping 
⁯                   ⁯                 ⁯   
    ⁯                       ⁯                                    ⁯ 
Hurrying or jogging 
     (as if to catch a bus)       ⁯                   ⁯                 ⁯      
    ⁯                       ⁯                                    ⁯ 
 
1. Compared with 2 weeks ago, have your symptoms of heart failure (shortness of breath, fatigue or ankle 
swelling)   
      changed?  My symptoms of heart failure have become… 
 
     Much worse 
 Slightly worse 
 Not changed 
 Slightly better 
  Much better           I’ve had no symptoms 
  
 
 
 
 
 
 
 
 
 
   over the last 2 weeks 
      ⁯  
       ⁯  
      ⁯                        ⁯                          ⁯  
 
⁯ 
 
2. 
Over the past 2 weeks, how many times did you have swelling in your feet, ankles or legs when you woke 
up in the morning? 
 
      Every          3 or more times       1-2 times a           Less than once         Never over the 
       morning        a week, but not            week                     a week                   past 2 weeks 
                                every day 
 
         ⁯  
       ⁯  
        ⁯                        ⁯                         ⁯ 
 
 
 
3. 
Over the past 2 weeks, how much has swelling in your feet, ankles or legs bothered you? 
     It has been…  
      
    Extremely 
Quite a bit 
Moderately 
 Slightly 
Not at all 
I’ve had no swelling 
     bothersome 
bothersome 
bothersome 
bothersome          bothersome 
  
         ⁯  
       ⁯  
      ⁯                        ⁯                           ⁯ 
          ⁯ 
 
4. 
Over the past 2 weeks, on average, how many times has fatigue limited your ability to do what you want? 
  

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 28 
All of the time       Several times          At least once a         3 or more times 
1-2 times per 
Less than once a 
Never over 
                                  per day                         day                  per week but not            week                          week                 the past 2                        
                                                                                                    every day                                                                                 weeks  
      ⁯                     ⁯  
      ⁯                        ⁯                          ⁯  
          ⁯                  ⁯  
 
5. 
Over the past 2 weeks, how much has your fatigue bothered you? 
     It has been… 
 
 Extremely 
Quite a bit 
Moderately 
Slightly  
Not at all 
I’ve had no fatigue 
    bothersome 
bothersome 
bothersome 
bothersome 
bothersome 
           ⁯  
       ⁯  
      ⁯                        ⁯                        ⁯                        ⁯ 
 
6. Over the past 2 weeks, on average, how many times has shortness of breath limited your ability to do what you wanted? 
 
All of the time       Several times          At least once a         3 or more times 
1-2 times per 
Less than once a      Never over 
                                  per day                         day                  per week but not            week                          week                 the past 2                        
                                                                                                    every day                                                                                 weeks  
       ⁯                     ⁯  
      ⁯                        ⁯                        ⁯  
          ⁯                  ⁯  
 
7. Over the past 2 weeks, how much has your shortness of breath bothered you? 
 
  Extremely 
Quite a bit 
Moderately 
 Slightly  
Not at all               I’ve had no shortness 
     bothersome 
bothersome 
bothersome 
bothersome          bothersome                     of breath 
  
          ⁯  
       ⁯  
      ⁯                        ⁯                        ⁯                        ⁯ 
 
8. Over the past 2 weeks, on average, how many times have you been forced to sleep sitting up in a chair or with at 
least 3 pillows to prop you up because of shortness of breath? 
 
     Every night 
3 or more times 
1-2 times a 
Less than once 
Never over the  
                               a week, but not 
    week  
     a week 
  past 2 weeks 
  
 
every day 
 
          ⁯  
       ⁯  
        ⁯                       ⁯                      ⁯ 
 
9. Heart Failure symptoms can worsen for a number of reasons.  How sure are you that you know what to do, or 
whom to call, if your heart failure gets worse? 
 
      Not at all  
Not very sure 
Somewhat sure 
Mostly sure 
Completely sure 
          sure 
          ⁯  
       ⁯  
        ⁯                       ⁯                     ⁯ 
 
10. How well do you understand what things you are able to do to keep your heart failure symptoms from getting 
worse? (for example, weighing  yourself, eating a low salt diet, etc.) 
 
        Do not 
   Do not  
Somewhat 
   Mostly  
Completely 
      understand 
understand            understand 
understand 
understand 
      at all 
very well 
         ⁯  
       ⁯  
        ⁯                     ⁯                      ⁯ 
 
 
11. 
Over the past 2 weeks, how much has your heart failure limited your enjoyment of life? 
 
 It has extremely It has limited 
    It has  
It has slightly 
It has not limited my 
    limited my 
my enjoyment 
moderately 
  limited my 
enjoyment of life 

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 29 
  enjoyment of 
  of life quite 
 limited my 
enjoyment of 
        at all 
       life                      a bit  
enjoyment of life         life 
 
       ⁯  
       ⁯  
        ⁯                     ⁯                      ⁯ 
 
 
 
12. If you had to spend the rest of your life with your heart failure the way it is right now, how would you feel about 
this? 
 
     Not at all 
    Mostly 
Somewhat          Mostly satisfied 
Completely 
      satisfied 
dissatisfied 
  satisfied 
   
 
   satisfied         
         ⁯  
       ⁯  
        ⁯                     ⁯                      ⁯ 
 
13. Over the past 2 weeks, how often have you felt discouraged or down in the dumps because of your heart failure? 
 
     I felt that way I felt that  way 
I occasionally 
I rarely felt that 
I never felt that  
     all of  the 
most of the 
felt that way 
         way 
        way   
         time 
    time  
        
 
          ⁯  
       ⁯  
        ⁯                     ⁯                      ⁯ 
 
14. 
How much does your heart failure affect your lifestyle?  Please indicate how your heart failure may have 
limited your participation in the following activities over the past 2 weeks? 
 
  
 
 
 
Please place an X in one box on each line 
 
Activity  
    Severely 
Limited         Moderately   Slightly               Did not              Does not apply 
or did 
 
 
     limited             quite a bit         limited 
   limited              limit at all            not do for other 
reasons 
Hobbies, recreational 
       activities 
         ⁯  
      ⁯  
⁯      
    ⁯                       ⁯                               ⁯ 
Working or doing  
    household chores         ⁯                    ⁯                 ⁯      
    ⁯                       ⁯                               ⁯ 
Visiting family or friends 
    out of your home            ⁯  
      ⁯                 ⁯   
    ⁯                       ⁯                               ⁯ 
Intimate relationships 
   with loved ones 
⁯                   ⁯                 ⁯   
    ⁯                       ⁯                               ⁯ 
 
 
Developed by John Spertus et al.,  Mid America Heart Institute, Saint Luke’s Hospital, Kansas City, MO. 
 
 
 
 
 
 
 
 
 
 

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 30 
 
 
 
Part III: MedaMACS VAD Survey 
 
Thank you for taking the time to fill out this short survey.  We will be asking you several 
questions about your heart failure and a new therapy for heart failure.  This survey 
should take no more than 15 minutes of your time.  Your responses will remain 
confidential.   
1. Based on how you feel today and what you know about your heart failure, what is your 
best estimate of how much longer you have to live?  (choose one) 
A. Less than 6 months 
B. Between 6 months to 2 year  
C. Between 2 to 5 years 
D. More than 5 years 
 
Ventricular Assist Device 
 
There are many effective medical therapies available to treat congestive heart 
failure.  Sometimes the heart can become too weak to pump enough blood to the body.  
At that stage, drugs may not be enough to treat heart failure.   
Mechanical heart pumps called ventricular assist devices, or VADs, are a way to 
improve the circulation of blood throughout the body.  These pumps do not replace the 
heart.  They only assist the heart in pumping blood to the body.  Once blood flow is 
improved, many patients have more energy and breathe easier.  Clinical studies show that 
select patients with severe heart failure live longer with an assist device than with drug 
treatments alone.  
Placement of a VAD requires major open heart surgery.  The pump is placed 
inside the chest and abdomen and is connected to the heart.  The VAD also has a power 
line that leaves the body through the skin in the front of the abdomen and is attached to a 
power supply outside the body.  On average patients will remain in the hospital for about 
one month after surgery.  Once discharged from the hospital, most patients are able to 
return home and live independently.   
 
2. Based on how you feel right now and knowing only the above information above, 
which statement best describes how you would feel about having an assist device placed? 
 
A. I would DEFINITELY want it 
B. I would PROBABLY want it 
C. I don’t know if I would want it or not 

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 31 
D. I would PROBABLY NOT want it. 
E. I would DEFINITELY NOT want it 
 
3.  Suppose that your doctor told you with certainty that you only had a limited about of 
time to live.  The next series of questions will ask you to imagine different scenarios 
where you only have a certain amount of time left to live.      
(Check one box) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Would you want a ventricular assist device 
if you had: 
 
Probably 
YES 
Unsure 
Probably 
NOT 
A. Less than 1 month to live? 
 
 
O 
 
O 
 
O 
B.  Less than 6 months to live? 
 
 
O 
 
O 
 
O 
C.  Less than 2 years to live? 
 
O 
 
O 
 
 
O 
D.  Less than 5 years to live? 
 
O 
 
O 
 
 
O 

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 32 
 
 
 
 
 
 
4.  The next series of questions asks to imagine that you are experiencing various degrees 
of illness related to your heart failure.  Please consider each scenario in terms of how 
limited you might feel and whether or not you would want an assist device to feel better.  
(Check one box) 
 
 
 
 
 
 
Would you want an assist device if: 
 
Probably 
YES 
Unsure 
Probably 
NOT 
1.  In the intensive care unit with hours or days to 
live. 
 
 
O 
 
O 
 
O 
2.  In the hospital on intravenous medicines to 
keep you alive, with only days or weeks to live. 
 
 
O 
 
O 
 
O 
3.  At home requiring continuous medicine 
through an intravenous 24 hours a day with 
weeks to months to live. 
 
O 
 
O 
 
O 
4.  At home and always breathless at rest and with 
light activities such as dressing or bathing. 
 
O 
 
O 
 
O 
5.  At home, comfortable at rest but breathless 
when walking around the house. 
 
O 
 
O 
 
O 
6.   At home but breathless after walking more 
than one block or more than one flight of stairs 
 
O 
 
O 
 
O 
7.   Not breathless during daily activities at home 
or after walking several blocks, but breathless 
with all other activities  
 
O 
 
O 
 
O 

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 33 
 
 
 
 
 
 
 
 
 
5. Prior to this survey, have you heard about a ventricular assist device (VAD)? 
 
A. Yes 
 
B. No 
 
C. Don’t know 
 
 
6.  If you have heard about an assist device before, how did you first hear about a VAD?  
 
A.  Television/Radio 
 
B.  Newspaper/Magazine 
 
C.  Your health care provider 
 
D.  Family members or friends 
 
E.  The Internet 
 
7. Knowing what you know now about different treatments for severe heart failure, which 
of these therapies would you rather have? 
 
A. Ventricular Assist Device 
 
B. Heart Transplant 
 
C. Don’t know 
 
8. Do you have a designated health care proxy or durable power of attorney for health 
care? 
 
A. Don’t know 
B. Yes 
 
C. No 

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 34 
 
The next series of questions focuses on treatment choices near the end of life.  Once 
again, all responses are confidential and will not be shared with your medical team.  
Please let us know if you wish to discuss any of these matters with your physicians. 
 
9. Many life-sustaining therapies are available near the end of the life.  These include 
dialysis, breathing machines, tubes for feeing, and whether or not you would wish to be 
resuscitated if your breathing or heart stops beating.  Has your physician talked about 
your wishes regarding such life-sustaining therapies? 
A. Yes 
B.  No 
C. Don’t know 
 
10. At this time, if there are any life-sustaining therapies you do not want, please circle 
them below: 
  
 
A. Chest compressions 
 
B. Being placed on a breathing machine 
 
C. Kidney dialysis 
 
D. Transfer to the Intensive Care Unit (ICU) 
 
E.  Feeding tube if unable to eat 
 
 
 
 
 
 
 
 
 
 
 
 
 

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 35 
 
 
 
 
 
 
 
Part IV: Missing Data Form 
 
 
 
 
 
 
 
 
Check one response regarding WHY “self-report instruments” were not completed by the 
PATIENT: 
 
_____   too sick  
______ too tired  
_____   too stressed, anxious, and / or depressed 
_____   can’t concentrate 
_____   no time / too busy 
_____   too much trouble / don’t want to be bothered / not interested 
_____   unwilling to complete instruments, no reason given 
______ unable to read English and / or illiterate 
______ administrative (check specific reason below) 
_____No time, coordinator too busy to administer self-report instruments 
_____Coordinator forgot to administer self-report instruments 
_____Unable to contact patient face-to-face or per telephone  
_____Patient did not return mailed self-report instruments within the 
window for instrument completion 
 
 
______ other reason   
 
 
(describe)_____________________________________ 
 
 
 
 
 
 
 

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 36 
 
 
 
 
 
 
 
 
Part V: Supplemental Questions: 
 
 
1. In general, how comfortable would you be if your life depended on interacting with 
technology every day?  Please draw an “X” on the line below that best describes how you 
feel. 
 
  
 
 
Highly Uncomfortable 
 
 
 
             
      Very Comfortable  
 
 
 
 
 
 
2. Treatments for heart disease can range from pills to pacemakers and even major heart 
surgeries.  Some patients are willing to undergo more aggressive treatment to survive, 
while others are reluctant to consider more aggressive therapies and wish to focus on 
comfort alone.  Based on how you feel today, please draw an “X” on the line below to 
indicate how you feel about medical treatments for your heart failure.   
 
 
 
Focus on Comfort Only 
 
 
 
                     Do Anything to Survive 
 
 
 
 
 
 
 
 
 
 
 

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 37 
 
 
 
 
 
 
 
 
 
 
APPENDIX B: DATA ELEMENTS 
 
DEMOGRAPHICS 
Baseline 
A 
Baseline 
B 
6m 
12m 
18m 
24m 
Name 
X 
 
 
 
 
  
Social security number 
X 
 
 
 
 
  
Date of birth 
X 
 
 
 
 
  
Gender 
X 
 
 
 
 
  
Ethnicity  
X 
 
 
 
 
  
Race 
X 
 
 
 
 
  
Marital status 
X 
 
 
X 
 
  
Highest education level 
X 
 
 
 
 
  
Working for income 
X 
 
 
X 
 
  
Height 
X 
X 
 
 
 
  
Weight 
X 
X 
X 
X 
X 
X 
Blood type 
X 
 
 
 
 
  
Inpatient/Outpatient status 
X 
X 
 
 
 
  
Context of referral* 
X 
 
 
 
 
  
  
  
 
 
 
 
  
CLINICAL 
Baseline 
A 
Baseline 
B 
6m 
12m 
18m 
24m 
Time since first cardiac diagnosis 
X 
 
 
X 
 
X 
Heart failure hospitalizations in last 
12mos 
X 
 
 
X 
 
X 
Primary cardiac diagnosis 
X 
 
 
 
 
  
  Cancer 
X 
 
 
 
 
  
  Congenital heart disease 
X 
 
 
 
 
  
  Coronary artery disease 
X 
 
 
 
 
  
  Dilated cardiomyopathy 
X 
 
 
 
 
  
    Adriamycin 
X 
 
 
 
 
  
    Toxic/Alcoholic 
X 
 
 
 
 
  
    Familial 
X 
 
 
 
 
  
    Idiopathic 
X 
 
 
 
 
  
    Ischemic 
X 
 
 
 
 
  
    Myocarditis 
X 
 
 
 
 
  
    Other 
X 
 
 
 
 
  
    Post-partum 
X 
 
 
 
 
  
    Viral 
X 
 
 
 
 
  
  Hypertrophic 
X 
 
 
 
 
  

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 38 
  Sarcoidosis 
X 
 
 
 
 
  
  Other 
X 
 
 
 
 
  
Previous cardiac operations 
X 
 
 
X 
 
X 
  None 
X 
 
 
X 
 
X 
  CABG 
X 
 
 
X 
 
X 
  Aortic valve repair/replacement 
X 
 
 
X 
 
X 
  Mitral valve repair/replacement 
X 
 
 
X 
 
X 
  Congenital card surg 
X 
 
 
X 
 
X 
  Aneurysmectomy 
X 
 
 
X 
 
X 
  Other 
X 
 
 
X 
 
X 
INTERMACS patient profile 
X 
X 
X 
X 
X 
X 
  Modifiers: A, FF, none 
X 
X 
X 
X 
X 
X 
Current NYHA class 
X 
X 
X 
X 
X 
X 
Prior heart transplant evaluation 
X 
X 
 
X 
 
X 
  Eval outcome: accept, reject, defer 
X 
X 
 
X 
 
X 
Prior DT VAD evaluation 
X 
X 
 
X 
 
X 
  Eval outcome: accept, reject, defer 
X 
X 
 
X 
 
X 
Diabetes 
X 
X 
 
X 
 
X 
Home oxygen 
X 
X 
 
X 
 
X 
Previous renal replacement 
X 
X 
 
X 
 
X 
  Any dialysis 
X 
X 
 
X 
 
X 
  Any ultrafiltration 
X 
X 
 
X 
 
X 
Recent intubation 
X 
X 
 
X 
 
X 
Recent intraaortic counterpulsation 
(within 6m) 
X 
X 
 
X 
 
X 
Inotrope Use in last 6 months 
X 
X 
 
X 
 
X 
  Timing; which drugs 
X 
X 
 
X 
 
X 
Length of time followed by program 
X 
 
 
 
 
 
Referral source 
X 
 
 
 
 
 
   Reason for referral 
X 
 
 
 
 
  
COMORBID CONCERNS 
Baseline 
A 
Baseline 
B 
6m 
12m 
18m 
24m 
Advanced age 
X 
 
 
X 
 
X 
Patient refuses transplant 
X 
 
 
X 
 
X 
Frailty 
X 
 
 
X 
 
X 
Contraindication to immunotherapy 
X 
 
 
X 
 
X 
Malnutrition/cachexia 
X 
 
 
X 
 
X 
Large body mass index 
X 
 
 
X 
 
X 
Musculoskeletal limitations 
X 
 
 
X 
 
X 
History of solid organ cancer 
X 
 
 
X 
 
X 
History lymphoma, leukemia 
X 
 
 
X 
 
X 
Major stroke 
X 
 
 
X 
 
X 
Other cerebrovascular disease 
X 
 
 
X 
 
X 
Renal dysfunction 
X 
 
 
X 
 
X 
Pulmonary disease 
X 
 
 
X 
 
X 
Severe diabetes 
X 
 
 
X 
 
X 
Peripheral vascular disease 
X 
 
 
X 
 
X 
Risk of recurrent infection 
X 
 
 
X 
 
X 
Pulmonary hypertension 
X 
 
 
X 
 
X 
Recent pulmonary embolus 
X 
 
 
X 
 
X 

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 39 
Allosensitization 
X 
 
 
X 
 
X 
Heparin-induced thrombocytopenia 
X 
 
 
X 
 
X 
Current infection 
X 
 
 
X 
 
X 
Limited cognition/understanding 
X 
 
 
X 
 
X 
Limited social support 
X 
 
 
X 
 
X 
History of illicit drug use 
X 
 
 
X 
 
X 
History of alcohol abuse 
X 
 
 
X 
 
X 
History of smoking 
X 
 
 
X 
 
X 
Currently smoking 
X 
 
 
X 
 
X 
Severe depression 
X 
 
 
X 
 
X 
Other major psychiatric disorder 
X 
 
 
X 
 
X 
Repeated non-compliance 
X 
 
 
X 
 
X 
More than one prior sternotomy 
X 
 
 
X 
 
X 
Mediastinal radiation 
X 
 
 
X 
 
X 
Thoracic aortic disease 
X 
 
 
X 
 
X 
History of hepatitis 
X 
 
 
X 
 
X 
Chronic coagulopathy 
X 
 
 
X 
 
X 
History of human immunodeficiency 
virus or AIDS 
X 
 
 
X 
 
X 
History of GI ulcers 
X 
 
 
X 
 
X 
History of atrial arrhythmias 
X 
 
 
X 
 
X 
History of symptomatic ventricular 
tachycardia or defibrillator shocks 
X 
 
 
X 
 
X 
Other co-morbidity (specify) 
X 
 
 
X 
 
X 
  
  
 
 
 
 
  
PHYSICAL 
Baseline 
A 
Baseline 
B 
6m 
12m 
18m 
24m 
Heart rate 
X 
X 
 
X 
 
X 
Blood pressure 
X 
X 
 
X 
 
X 
Jugular venous pressure 
X 
X 
 
X 
 
X 
S3 gallop 
X 
X 
 
X 
 
X 
S4 gallop 
X 
X 
 
X 
 
X 
Peripheral edema 
X 
X 
 
X 
 
X 
Ascites 
X 
X 
 
X 
 
X 
Hepatomegaly 
X 
X 
 
X 
 
X 
  
  
 
 
 
 
  
EKG 
Baseline 
A 
Baseline 
B 
6m 
12m 
18m 
24m 
Rate 
X 
X 
 
X 
 
X 
Rhythm: atria, ventricles, pacing type 
X 
X 
 
X 
 
X 
QRS duration 
X 
X 
 
X 
 
X 
  
  
 
 
 
 
  
ECHOCARDIOGRAPHY 
Baseline 
A 
Baseline 
B 
6m 
12m 
18m 
24m 
Date 
X 
 
X 
 
(X) 
Left ventricular (LV) ejection fraction 
X 
 
X 
 
(X) 
LV end-diastolic dimension 
X 
 
X 
 
(X) 
Right ventricular (RV) Indices 
X 
 
X 
 
(X) 
  Qualitative RV Function 
X 
 
X 
 
(X) 
  Qualitative RV Size 
X 
 
X 
 
(X) 
  Maximum mid RV dimension 
X 
 
X 
 
(X) 

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 40 
  Tricuspid annular plane excursion 
X 
 
X 
 
(X) 
  Tricuspid regurgitant velocity 
X 
 
X 
 
(X) 
Tricuspid regurgitation (none, mild, mod, 
sev) 
X 
 
X 
 
(X) 
Mitral regurgitation (none, mild, mod, 
sev) 
X 
 
X 
 
(X) 
Aortic stenosis (none, mild, mod, sev) 
X 
 
X 
 
(X) 
Aortic insufficiency  (none, mild, mod, 
sev) 
X 
 
X 
 
(X) 
Inferior vena cava size 
X 
 
X 
 
(X) 
Inferior vena cava respiration variation: 
yes, blunted, none 
X 
 
X 
 
(X) 
Ancillary indices of RV function 
X 
 
X 
 
(X) 
  
  
 
 
 
 
  
RIGHT HEART CATHETERIZATION 
Baseline 
A 
Baseline 
B 
6m 
12m* 
If 
avail 
18m 
24m 
Date 
X 
 
 
X 
 
 
Therapies at cath: no iv, iv, iabp 
X 
 
 
X 
 
 
Systolic blood pressure 
X 
 
 
X 
 
 
Diastolic blood pressure 
X 
 
 
X 
 
 
heart rate 
X 
 
 
X 
 
 
Right atrial pressure 
X 
 
 
X 
 
 
Pulmonary artery systolic pressure 
X 
 
 
X 
 
 
Pulmonary artery diastolic pressure 
X 
 
 
X 
 
 
Pulmonary capillary wedge pressure 
X 
 
 
X 
 
 
PA saturation 
X 
 
 
X 
 
 
Cardiac output 
X 
 
 
X 
 
 
Cardiac index 
X 
 
 
X 
 
 
  
  
 
 
 
 
  
EXERCISE TESTING 
Baseline 
A 
Baseline 
B 
6m 
12m 
18m 
24m 
6 minute walk distance 
X 
X 
 
X 
 
X 
15 ft walk time (gait speed/frailty) 
X 
X 
 
X 
 
X 
Cardiopulmonary exercise testing  
X 
 
X 
 
  
  Resting heart rate 
X 
 
X 
 
  
  Peak heart rate 
X 
 
X 
 
  
  Peak oxygen uptake 
X 
 
X 
 
  
  Peak oxygen uptake % predicted 
X 
 
X 
 
  
  Ventilatory efficiency (Ve/VCO2) 
X 
 
X 
 
  
  Peak respiratory exchange ratio 
X 
 
X 
 
  
  
  
 
 
 
 
  
MEDICAL AND DEVICE THERAPIES 
Baseline 
A 
Baseline 
B 
6m 
12m 
18m 
24m 
Meds: Current/Previous/No/Unknown 
  
 
 
 
 
  
Angiotensin converting enzyme inhibitor 
X 
X 
X 
X 
X 
X 
Angiotensin receptor blocker 
X 
X 
X 
X 
X 
X 
Beta adrenergic blocker 
X 
X 
X 
X 
X 
X 
Aldosterone antagonist 
X 
X 
X 
X 
X 
X 
Potassium supplement 
X 
X 
X 
X 
X 
X 
Long acting nitrate  
X 
X 
X 
X 
X 
X 
Hydralazine 
X 
X 
X 
X 
X 
X 

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 41 
Phosphodiesterase inhibitor 
X 
X 
X 
X 
X 
X 
Digoxin 
X 
X 
X 
X 
X 
X 
Loop diuretic + daily dose 
X 
X 
X 
X 
X 
X 
Metolazone/thiazide and frequency 
X 
X 
X 
X 
X 
X 
Aspirin 
X 
X 
X 
X 
X 
X 
Clopidogrel 
X 
X 
X 
X 
X 
X 
Coumadin 
X 
X 
X 
X 
X 
X 
Other anticoagulant 
X 
X 
X 
X 
X 
X 
Statin 
X 
X 
X 
X 
X 
X 
Allopurinol 
X 
X 
X 
X 
X 
X 
Amiodarone 
X 
X 
X 
X 
X 
X 
Other antiarrhythmic 
X 
X 
X 
X 
X 
X 
ICD 
X 
X 
X 
X 
X 
X 
  If yes, dates of any ICD shocks 
X 
X 
X 
X 
X 
X 
Cardiac resynchronization 
X 
X 
X 
X 
X 
X 
  If < 6 months, implant date 
X 
X 
X 
X 
X 
X 
Home oxygen 
X 
X 
X 
X 
X 
X 
  
  
 
 
 
 
  
BLOOD LABORATORIES 
Baseline 
A 
Baseline 
B 
6m 
12m 
18m 
24m 
Chemistry 
  
 
 
 
 
  
  Sodium 
X 
X 
 
X 
 
X 
  Potassium 
X 
X 
 
X 
 
X 
  Creatinine 
X 
X 
 
X 
 
X 
  Blood urea nitrogen 
X 
X 
 
X 
 
X 
  Total bilirubin 
X 
 
 
X 
 
X 
  Direct bilirubin 
X 
 
 
X 
 
X 
  Aspartate aminotransferase/SGOT 
X 
 
 
X 
 
X 
  Alanine aminotransferase/SGPT 
X 
 
 
X 
 
X 
  B-type natriuretic peptide (BNP) 
X 
X 
 
X 
 
X 
  NT-pro-BNP 
X 
X 
 
X 
 
X 
Metabolism 
  
 
 
 
 
  
  Total cholesterol 
X 
 
 
X 
 
X 
  Low density lipoprotein 
X 
 
 
X 
 
X 
  High density lipoprotein 
X 
 
 
X 
 
X 
  Triglycerides 
X 
 
 
X 
 
X 
  Uric acid 
X 
 
 
X 
 
X 
  C-reactive protein 
X 
 
 
X 
 
X 
  Pre-albumin 
X 
 
 
X 
 
X 
  Albumin 
X 
 
 
X 
 
X 
Hematologic 
X 
 
 
X 
 
X 
  White blood cell 
X 
 
 
X 
 
X 
  % lymphocytes 
X 
 
 
X 
 
X 
  Hemoglobin 
X 
 
 
X 
 
X 
  Hematocrit 
X 
 
 
X 
 
X 
  Platelet 
X 
 
 
X 
 
X 
  International normalized ratio 
X 
 
 
X 
 
X 
  Lupus anticoagulant if available 
X 
 
 
X 
 
X 
  
  
 
 
 
 
  

MedaMACS Pilot Protocol 
 
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Page 42 
QOL/PREFERENCES 
Baseline 
A 
Baseline 
B 
6m 
12m 
18m 
24m 
EuroQoL  
X 
X 
X 
X 
X 
X 
  Thermometer 
X 
X 
X 
X 
X 
X 
  5 functional domains 
X 
X 
X 
X 
X 
X 
Kansas City Cardiomyopathy 
Questionnaire 
X 
X 
 
X 
 
X 
Patient VAD survey instrument 
X 
X 
 
X 
 
X 
Missing data survey 
 X 
X 
 
X 
 
X 
EVENTS/OUTCOMES 
Baseline 
A 
Baseline 
B 
6m 
12m 
18m 
24m 
Death 
  
X 
X 
X 
X 
X 
Primary cause of death (assigned by PI) 
  
X 
X 
X 
X 
X 
  Sudden death 
  
X 
X 
X 
X 
X 
Transplant 
  
X 
X 
X 
X 
X 
Listed for Transplant 
  
X 
X 
X 
X 
X 
  Listing status 
  
X 
X 
X 
X 
X 
VAD, if yes then 
  
X 
X 
X 
X 
X 
  Type of circulatory support 
  
X 
X 
X 
X 
X 
  Device name 
  
X 
X 
X 
X 
X 
  Intended Support Strategy 
  
X 
 
X 
 
X 
Other Cardiac Procedure/Surgery 
(name) 
  
X 
 
X 
 
X 
Chronic inotrope infusion 
  
X 
 
X 
 
X 
Resuscitation (Code) Status 
 
X 
 
X 
 
X 
Hospice  
 
X 
 
X 
 
X 
Recovery of EF to ≥40% 
 
X 
 
X 
 
X 
Current NYHA Functional Class 
  
X 
 
X 
 
X 
Current INTERMACS Profile 
  
X 
 
X 
 
X 
Hospitalization, reason for admission 
 
X 
 
X 
 
X 
  Heart failure with iv diuresis or inotrope 
 
X 
 
X 
 
X 
  Neurologic event 
 
X 
 
X 
 
X 
  Major bleeding (non CNS) 
 
X 
 
X 
 
X 
     Transfusion 
 
X 
 
X 
 
X 
  Psychiatric episode 
 
X 
 
X 
 
X 
  Major infection 
 
X 
 
X 
 
X 
     Localized infection 
 
X 
 
X 
 
X 
     Sepsis 
 
X 
 
X 
 
X 
     Type of Infection 
 
X 
 
X 
 
X 
         Bacterial 
 
X 
 
X 
 
X 
         Fungal 
 
X 
 
X 
 
X 
         Viral 
 
X 
 
X 
 
X 
         Protozoan 
 
X 
 
X 
 
X 
         Unknown 
 
X 
 
X 
 
X 
      Intervention (IV, PO, surgery, other) 
 
X 
 
X 
 
X 
  Cardiac tachyarrhythmia  
 
X 
 
X 
 
X 
  Syncope cause unknown 
 
X 
 
X 
 
X 
  Fever treated with antibiotics cause    
unknown 
 
X 
 
X 
 
X 
  Arterial non-CNS thromboembolism 
 
X 
 
X 
 
X 
  Venous thromboembolic event 
 
X 
 
X 
 
X 

MedaMACS Pilot Protocol 
 
11/08/2011 
 
 
 
 
 
 
Page 43 
  Myocardial infarction 
 
X 
 
X 
 
X 
  Other cardiac cause (specify) 
 
X 
 
X 
 
X 
  Non-cardiac cause (specify) 
 
X 
 
X 
 
X 
  Elective procedure - cardiac 
 
X 
 
X 
 
X 
  Elective procedure – noncardiac 
 
X 
 
X 
 
X 
  Abnormal lab values 
 
X 
 
X 
 
X