Are we Meeting Patient Treatment Goals With Guideline-based Therapy for Psoriatic Arthritis
Conditions: Psoriatic Arthritis
Sponsor: Johns Hopkins University

September 25, 2020 
Principal Investigator: Ana-Maria Orbai 
Application Number: 00184580 
JHMIRB eFormA  01 
Version 3 Dated:   06/2007 
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Are we achieving patient treatment goals with guideline-based 
therapy for Psoriatic Arthritis? 
NCT03620188 
9/25/2020 

September 25, 2020 
Principal Investigator: Ana-Maria Orbai 
Application Number: 00184580 
 
JHMIRB eFormA  01 
Version 3 Dated:   06/2007 
 
Page 2 of 8 
 
 
 
 
 
 
 
JHM IRB - eForm A – Protocol 
*************************************************************************************************** 
 
Are we achieving patient treatment goals with guideline-based 
therapy for Psoriatic Arthritis? 
 
Ana-Maria Orbai MD MHS, Principal Investigator 
 
 
1. 
Abstract 
Psoriatic arthritis (PsA) is a heterogeneous autoimmune disease that occurs in one in three people with 
the skin disease psoriasis. PsA can cause arthritis (joint inflammation), enthesitis (tendon and ligament 
inflammation), sausage digits (swollen entire finger or toe), spondyloarthritis (spinal inflammation). Skin 
involvement by psoriasis is also highly variable in terms of psoriasis type and location.  Through 
combined skin and musculoskeletal involvement, psoriatic disease has a significant life impact with 
decrease quality of life including uncomfortable symptoms, ability to participate in life and functioning.  
Medications used to treat PsA have sometimes an uneven effect on the various PsA manifestations 
where some are more effective for skin while others more effective for the joints.  In this context, clinical 
care and treatment of PsA is a complex process which balances disease activity with medication risks 
and benefits as well as patient priorities.  Professional PsA treatments guidelines state that PsA treatment 
goals are disease remission or low disease activity.  Several studies to date have shown that physicians 
tend to overestimate remission and low disease activity in PsA patients when compared to disease 
activity indices.  Also patients and physicians frequently do not align on perceptions of remission or low 
disease activity.  In the proposed study we aim to identify predictors of successful treatment from a 
patient perspective on a range of disease measures including psoriasis, arthritis, enthesitis, dactylitis, 
patient reported outcomes, and laboratory assessments which are routinely collected in the clinical care 
of PsA.  Secondary endpoints are to quantify longitudinally how stable a state of treatment success is 
from a patient perspective, and to define score ranges for disease measurements, including health-
related quality of life measures, that correspond to treatment success from a patient perspective.  The 
impact of this research is that we will be able to define parameters predictive of achieving 
treatment success from a patient perspective, which will then inform goals of care for psoriatic 
arthritis. 
 
2. 
Objectives 
Primary objective 
To determine predictors of reaching PsA treatment goals/treatment success (primary outcome) from a 
patient perspective in an academic PsA guideline-based treatment cohort.  Conversely, to determine 
adverse predictors to reaching this desirable state.  The primary outcome determination is based on the 
patients’ report of whether they are/are not at goal with their PsA treatment. 
 
Secondary objective(s) 
To estimate the following parameters:  
A. Prevalence of patients reporting they met PsA treatment goals (successful treatment) from their 
perspective. 
 

September 25, 2020 
Principal Investigator: Ana-Maria Orbai 
Application Number: 00184580 
 
JHMIRB eFormA  01 
Version 3 Dated:   06/2007 
 
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B. Frequency and direction of longitudinal transitions between treatment goals/met not met states: 
3 observations/participant. 
C. Score ranges that correspond to a status of patients’ treatment goals met on a set of PsA 
outcome measures used in the study, including composite disease activity measures. 
D. On the same set of PsA outcome measures, meaningful change values in patients who 
transition longitudinally to the better and worse categories. 
 
3. 
Background  
Efficacy of therapeutics in PsA is incomplete, considering at most 60% of patients achieve the primary 
efficacy outcome in clinical trials.  More so, treatment goals from the patient perspective, as well as 
alignment of patient opinion with index-based definitions of treatment success are part of the knowledge 
gap in PsA.  In clinical practice, desirable treatment targets have been defined by experts as Disease 
Activity in Psoriatic Arthritis (DAPSA) low disease activity or remission, or Minimal Disease Activity 
(MDA)2.  The clinical DAPSA score is computed by adding the sum of tender (out of 68) and swollen (out 
of 66) joints with patient global assessment and patient reported pain (each on a scale of 0-10).  DAPSA 
remission state is defined by a score≤ 4, and DAPSA low disease activity as ≤ 14.  MDA is a checklist 
based state defined as achieving five out of the following seven criteria: ≤ 1 swollen joint, ≤ 1 tender joint, 
≤ 1 tender entheseal point, HAQ-DI score ≤ 0.5 (range 0-3), patient reported pain ≤ 1.5, patient global 
assessment ≤ 2, body surface area psoriasis ≤ 3%.  It is of great interest to ascertain if guideline-based 
PsA clinical care achieves individual patient goals as articulated by patients, and to identify predictors of 
achieving individual patient goals from PsA treatment. 
 
4. 
Study Procedures 
All study assessments will be programmed in the electronic medical record (Epic EMR) with the help of the 
epic physician builder who is co-investigator on the study (Dr. Thomas Grader Beck, MD).  Demographics, 
key comorbidities (cardiovascular disease, hypertension, diabetes, obesity, osteoporosis, thromboembolic 
disease, cancer), routine laboratories including inflammatory markers, and disease characteristics will be 
updated at every visit.  Information that does not change such as disease onset for psoriasis and 
psoriatic arthritis will be recorded only at baseline.  Study visits will occur at 3-4 month intervals in 
conjunction with clinical care.  Disease assessments will comprise physician assessments (Table 1 a,b), 
laboratories (Table 2), and patient reported outcomes (Table 3 a,b).  Medication doses and changes in 
medications will be recorded longitudinally. Disease activity measures will be programmed in the medical 
record such that scores are automatically generated from the primary data elements as illustrated in Table 
4. 
Table 1a. Physician assessments 
PsA Phenotype 
smartform  
Elements 
CASPAR 
classification 
criteria (cumulative) 
Psoriasis (active, history, family history), Nail psoriasis, Dactylitis, Negative 
rheumatoid factor, Juxtaarticular new bone (radiographic PsA) 
Extraarticular 
manifestations 
(cumulative) 
Uveitis, Inflammatory bowel disease, Inflammatory back pain, Psoriasis type 
(plaque, pustular, inverse, guttate, erythrodermic) and special locations 
(scalp, palmoplantar, intergluteal cleft/perianal), Elevated CRP 
Comorbidities (from 
problem list) 
Cancer of skin (not melanoma), Melanoma skin cancer, Cancer other than 
skin (fill in), Coronary artery disease, Diabetes, Fatty liver disease, Obesity , 
Osteoporosis, Stroke, Thyroid disease, Vitiligo 

September 25, 2020 
Principal Investigator: Ana-Maria Orbai 
Application Number: 00184580 
 
JHMIRB eFormA  01 
Version 3 Dated:   06/2007 
 
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Table 1b. Disease activity measures 
Psoriatic arthritis 
areas/domains 
Physician assessments  
Musculoskeletal 
Arthritis  
Dactylitis 
Enthesitis 
Spondyloarthritis 
Tender/swollen joint counts 66/68 
Dactylitis index (LDI basic) (0-20) 
Enthesitis index (SPARCC) (0-18) 
Antropometrics (Cervical range of motion anteroposterior and lateral: occiput to 
wall, degrees lateral rotation; Thoracic expansion; Lumbar spine: Schober 10 
and 15 cm, Lateral lumbar flexion)* 
Skin and nails 
Psoriasis body surface area affected (BSA%); Psoriasis location (scalp, upper 
extremities, lower extremities, torso, abdomen); Psoriasis phenotype (plaque, 
pustular, inverse, guttate, erythrodermic) 
Investigator global assessment (0-4); Nail disease (pitting, onycholysis, and 
surface area affected) 
* assessed only at baseline 
 
Table 2. Laboratory assessments 
Psoriatic arthritis areas/domains 
Laboratory test  
Inflammatory markers 
C reactive protein (CRP, mg/dL), ESR 
 
We will also assess complete blood counts and comprehensive metabolic panel at every visit as part of 
usual clinical care. We will retrospectively abstract serologic information collected as part of usual clinical 
care from the medical record (rheumatoid factor, anti-cyclic citrullinated peptide (anti-CCP) status, HLA-
B27, and ANA). 
 
Table 3a. Patient reported treatment success impression 
 
The final questions will be refined with patient research partners to make sure these are formulated such 
that patients understand we are assessing at each visit whether they have achieved their personal 
treatment goals for their psoriatic arthritis. 
 
Patient impression of PsA treatment goals met 
Response options 
1. “Today, considering the level of control of your 
psoriatic arthritis and psoriasis, do you consider 
your treatment has been successful?” 
1. yes 
2. no  
2. “If in your opinion, psoriatic arthritis and 
psoriasis treatments have NOT been successful 
for you, please choose the three most important 
manifestations and life impact aspects that are 
still active for you (from your psoriatic arthritis and 
psoriasis).” 
1. inflamed joints, 2. inflamed tendons, 3. 
back/spine inflammation, 4. skin psoriasis, 5. 
nail psoriasis, 6. scalp psoriasis, 7. Pain, 8. 
Fatigue, 9. Stiffness, 10. Ability to be physically 
active/exercise, 11. Ability to participate with 
family activities, 12. Ability to work, 13. Ability to 
participate in social activities, 14. Emotional 
impact, 15. Ability to be independent in my daily 
activities, 16. Other, please fill in. 
 

September 25, 2020 
Principal Investigator: Ana-Maria Orbai 
Application Number: 00184580 
 
JHMIRB eFormA  01 
Version 3 Dated:   06/2007 
 
Page 5 of 8 
 
 
 
 
Patient impression of PsA treatment goals met 
Response options 
3. Patient acceptable symptom state: “If you were to 
remain for the next few months as you were during 
the past week, would this be acceptable or 
unacceptable for you?” 
 
1. acceptable 
2. unacceptable 
 
Table 3b. Patient reported outcomes for disease status and life impact 
Psoriatic arthritis areas/domains 
Patient reported outcomes 
Musculoskeletal 
Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) 
Health related quality of life 
Psoriatic Arthritis Impact of Disease (PsAID12, PsA specific) 
PROMIS Profile 29 (generic) 
 
Pain 
Pain visual analog scale (VAS, 0-100 mm) 
PROMIS Pain interference (Profile 29) 
Physical function 
PROMIS Physical Function 10 
PsA patient globals 
Patient global Arthritis (GRAPPA) (VAS, 0-100 mm) 
Patient global Psoriasis (GRAPPA) (VAS, 0-100 mm) 
Fatigue 
PROMIS Fatigue 8a 
Participation, Depression, Anxiety 
Sleep quality 
PROMIS Profile 29 
 
Table 4. Disease activity measures (programmed in EMR and calculated automatically from 
elementary data) 
Disease activity measure 
Components and calculation 
Disease Activity Psoriatic 
Arthritis (DAPSA) 
 
Cutoffs 
Remission ≤ 4 
Low 4 ≤ 14 
Moderate 14 ≤ 28 
High ≥ 28 
Tender/swollen joint counts: 68/66 
Patient global (0-10) 
Patient pain (0-10) 
C reactive protein (mg/dL) 
 
DAPSA = 68tender joints + 66swollen joints + patient global + pain 
+ CRP (ranges from 0 to more than 154) 
 
Clinical DAPSA = 68tender joints + 66swollen joints + patient 
global + pain (0-154) 
 
Very low disease activity 
(VLDA) and Minimal disease 
activity (MDA) 
 
VLDA (7/7 criteria met) 
MDA (5/7 criteria met) 
Set of 7 criteria: 
1. Tender joint count 68 ≤ 1 
2. Swollen joint count 66 ≤ 1 
3. Patient global ≤ 2/10 
4. Pain ≤ 1.5/10 
5. Health assessment questionnaire disability index ≤ 0.5 
6. Enthesitis ≤ 1 
7 Body surface area psoriasis ≤ 3% 
 
 

September 25, 2020 
Principal Investigator: Ana-Maria Orbai 
Application Number: 00184580 
 
JHMIRB eFormA  01 
Version 3 Dated:   06/2007 
 
Page 6 of 8 
 
 
 
 
 
Patients will be enrolled in CRMS and the CRMS study flag in Epic will be used to identify patients for 
research visits and data extraction. The unique study identifier will be linked to the patients’ JHH medical 
record number and only study staff will have access to the key table. This will allow study staff to keep 
track of enrollment and study visits and to specify data query parameters to the EPIC team at the stage of 
data extraction. If the patient signs consent and has a study visit on the same day, the CRMS flag will not 
be available to trigger specific Epic content, since the update from CRMS to EPIC takes about 24 hours. 
Because of this, we will institute a patient-level Smartdata element in Epic that will be flagged after the 
patient signs consent. This will also indicate inclusion in the study and can be used to trigger specific Epic 
content for the visit. 
 
Safety assessments: All adverse events (AE) will be recorded and managed per usual clinical care. 
Serious adverse events (SAE) are defined as adverse events that meet any of the following criteria: result 
in death; are life-threatening (i.e., in the opinion of the Investigator(s) the subject is at immediate risk of 
death from the AE); require inpatient hospitalization or prolongation of existing hospitalization; result in  
persistent or significant disability/incapacity (a substantial disruption of the subject’s ability to conduct 
normal life functions); represent a congenital anomaly/birth defect; constitute an important medical event.   
As part of usual clinical care, some patients participating in the study will be taking apremilast.  Any SAE 
due to apremilast must be reported immediately (i.e., within 24 hours of the Investigators’ knowledge of 
the event) to Amgen Inc Safety by facsimile.  A written report (prepared by the Investigator(s) using an 
SAE Report Form or a 3500A Medwatch form  is to be faxed to Safety (see below for contact information). 
 
Amgen Inc Drug Safety Contact Information: 
Amgen Inc Corporation      
Global Drug Safety and Risk Management    
86 Morris Ave.  
Summit, NJ  07901       
Fax: (908) 673-9115 
E-mail: drugsafety@Celgene.com 
 
Pregnancies: Pregnancies and suspected pregnancies (including a positive pregnancy test regardless of 
age or disease state) of a female subject or the female partner of a male subject occurring while the 
subject is on apremilast, or within 30 days of the subject’s last dose of study drug, are considered 
immediately reportable events.  Apremilast is to be discontinued immediately.  The pregnancy, suspected 
pregnancy, or positive pregnancy test must be reported to Amgen Inc Safety immediately facsimile using 
the Pregnancy Report form provided by Amgen Inc. 
 
5. 
Inclusion/Exclusion Criteria 
 
Inclusion criteria: English speaking/reading adults, ages 18 to 95 years who are patients in the Johns 
Hopkins Arthritis Center and/or the Psoriatic Arthritis Clinical Program (PI, Ana-Maria Orbai MD MHS 
directs the PsA program) and are followed every 3-4 months. Participants have to meet CASPAR 
classification criteria for psoriatic arthritis (supplementary material), and be able to interact with touch 
screen computer.   
Exclusion criteria: None 
 
6. 
Drugs/ Substances/ Devices – Not Applicable 
 
 
 

September 25, 2020 
Principal Investigator: Ana-Maria Orbai 
Application Number: 00184580 
 
JHMIRB eFormA  01 
Version 3 Dated:   06/2007 
 
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7. 
Study Statistics 
Grouping of treatment goals met or not met is based on the primary outcome at each study visit (Fig1).  
 
Figure 1. Determining patient status in terms of treatment goals met (treatment has been successful from 
the patient’s perspective) or not. 
 
a. Predictors of successful treatment/reaching PsA treatment goals (primary outcome) from a 
patient perspective: In the longitudinal dataset with up to three visits per participant, we will examine 
predictors of ‘treatment goals met’ status using logistic regression with covariates disease characteristics 
and patient reported variables.  We will identify predictors associated with a transition state from unmet to 
met, a stable status of treatment goals met, as well as predictors of stable status of unmet goals or 
transition in the negative direction (state variable with 4 categories: unmet to met, stable met, stable 
unmet, met to unmet).  Full visits for this study must be in-person so that the physician assessment of the 
patient’s disease is complete. 
b. Prevalence of patients reporting successful treatment/they meet PsA treatment goals from their 
perspective: We will perform exploratory data analysis to estimate the prevalence of ‘patient treatment 
goals met’ status in this longitudinal guideline based therapy cohort.  We will then compare characteristics 
of ‘treatment goals met’ vs ‘not met’ patient groups using proportions for binary/categorical (chi square 
tests) and means for continuous variables (t-tests). 
c. Frequency and direction of longitudinal transitions between treatment goals met(successful 
treatment)/not met states: 3 observations/participant Inperson Visits only: We will determine how 
patient states change over time to define stability of the notion of successful treatment.  Interim 
Telemedicine visit data will be collected to fill gaps between extended in-person intervals.  
d. Score ranges that correspond to a status of patients’ treatment goals are met (succesful 
treatment) on a set of PsA outcome measures used in the study, including composite disease 
activity measures:  We will calculate average score ranges for multiple variables of interest that 
correspond to a status of successful treatment for patients.   
e. Meaningful change values in patients who transition longitudinally to the better and worse 
categories: We will determine change scores in clinical outcomes corresponding to a meaningful change 
from the patient perspective (MCII).  We will determine MCII for various measures using a predictive 
approach (logistic regression with ROC and AUC).  We will perform a similar analysis in patients who go 
the other direction (from treatment goals met to treatment goals not met). 
f. Telemedicine Visits:  Data from telemedicine visits will be collected and used to fill gaps in 
understanding the changes to the patient’s health status over time.  This is important do to the length of 
time between In-Person visits due to COVID-19. 
 
8. 
Risks 

September 25, 2020 
Principal Investigator: Ana-Maria Orbai 
Application Number: 00184580 
 
JHMIRB eFormA  01 
Version 3 Dated:   06/2007 
 
Page 8 of 8 
 
 
This study is a longitudinal observational study of usual guideline based treatment of psoriatic arthritis.  
There are no medical risks associated with participating in this research.  There are no additional risks to 
questionnaire completion. Only the investigators will have access to a list linking personal identifying  
 
 
 
information to study number.  There are no known financial or legal risks associated with participation in 
this study. 
 
9. 
Benefits 
There are no direct benefits to participants from participating in the study.  It is possible that some patients 
will benefit from better communicating their disease experience to their own healthcare providers.  
Benefits for society include a better understanding of how patients define treatment success, experience 
and self-manage their disease, and interact with their healthcare providers regarding their disease. 
 
10. 
Payment and Remuneration 
Participants will not receive monetary compensation for their participation.  Coupons will be provided to 
cover the cost of parking at the time of each study visit.   
 
11. 
Costs 
There are no costs associated with participating in this study.