informed consent to participate in
what if i change my

You can change your mind at any time about allowing us to use your samples and health information for research. We ask that you contact [Principal Investigator] in writing and let him/her know you do not want us to use your research samples or health information for research. His/her mailing address is on the first page of this form. If you withdraw yourself from this protocol, even if you allowed your samples to be used for research, your samples will not be used from that point and they will be discarded. However, samples and information that have already been shared with other researchers cannot be taken back or destroyed.
informed consent to participate in
new information available during the

During this research study, new information about the study drug or the risks and benefits of the study may become known to the study doctors. If this happens, they will tell you about the new information. The new information may mean that you can no longer participate in the study, or that you may not want to continue in the study. If this happens, the study doctor will stop your participation in the study and you will be offered all available care to suit your needs and medical conditions.
informed consent to participate in
privacy confidentiality and use of

Your confidentiality is one of our main concerns. We will do our best to make sure that the personal information in your medical and research records remains confidential. We will not discuss or publish information about your health with any unauthorized person or persons. However, we cannot guarantee total privacy. Your personal information may be given out if required by law. If information from this study is published or presented at scientific meetings, your name and other personal information will not be used. Your study number is not related to your name, social security number or medical record number at [*insert facility name*]. A description of this clinical trial will be available on http://www.ClinicalTrials.gov, as required by U.S. Law. This Web site will not include information that can identify you. At most, the Web site will include a summary of the results. You can search this Web site at any time. Information about your transplant from your original medical records may be seen or sent to national and international transplant registries, including:
- The Center for International Blood and Marrow Transplant Research (CIBMTR)  
- The National Marrow Donor Program (NMDP) - The Food and Drug Administration (FDA)
- The National Institutes of Health (NIH), which include the National Heart, Lung, and Blood Institute (NHLBI) and the National Cancer Institute (NCI)
- Data and Coordinating Center of the Blood and Marrow Transplant Clinical Trials Network
(BMT CTN), and
- Other authorized study organizations
We will not identify you by name in any publications or reports that come from these organizations or groups.
informed consent to participate in
ending your participation

The study doctor or the study sponsor may stop the study at any time, and you may be asked to leave the study. We may ask you to leave the study if you do not follow directions or if you suffer from side effects of the treatment. The study sponsor may decide to end the study at any time. If you are asked to leave the study, the reasons will be discussed with you. Possible reasons to end your participation in this study include:
- You do not meet the study requirements.  
- You need a medical treatment not allowed in this study.  
- The study doctor decides that it would be harmful to you to stay in the study.  
- You are having serious side effects. - You become pregnant. - You cannot keep appointments or take study drugs as directed. - The study is stopped for any reason.
informed consent to participate in
physical injury as a result

It is important that you tell your study doctor or study staff if you feel that you have been hurt or injured because of taking part in this study. You will get medical treatment if you are injured as a result of taking part in this study. You and/or your health plan will be charged for this treatment. The study will not pay for this treatment. In case of injury resulting from this study, you do not lose any of your legal rights to seek payment by signing this form.
informed consent to participate in
compensation or payment

You will not be paid for your participation in the research study. You will not get compensation or reimbursement for any extra expenses (travel, meals, etc.) you may have through your participation on this trial.
informed consent to participate in
costs & reimbursements

Most of the visits for this research study are standard medical care for patients undergoing allogeneic transplants and will be billed to your insurance company. You and/or your health plan/insurance company will need to pay for some or all of the costs of standard treatment in this study. You or your insurance will not be charged for the busulfan blood samples required for the study or the optional blood sample for research on this study.
informed consent to participate in
ethical review

The ethical aspects of this research study have been reviewed and approved by [*name of IRB*].
informed consent to participate in
further information

If you need any information about this study, or if you have any problems while you are participating in this study you can contact the study doctor or his/her staff. They may be contacted at the telephone numbers listed below. [*Insert name and contact details*].
informed consent to participate in
independent contact

If you wish to speak to someone not directly involved in the study, or if you have any complaints about any aspect of the project, the way it is being conducted or any questions about your rights as a research participant, then you may contact [*Insert appropriate contact details*].
informed consent to participate in
health insurance portability and accountability

As a research participant, I authorize the Principal Investigators and the researcher's staff to use and disclose my individual health information for the purpose of conducting the research study:
A Randomized, Multi-Center, Phase III Study of Allogeneic Stem Cell Transplantation Comparing Regimen Intensity in Patients with Myelodysplastic Syndrome or Acute Myeloid Leukemia  
B. **Individual Health Information to be Used or Disclosed**:
My individual health information that may be used or disclosed to do this research includes:
- Demographic information (for example: date of birth, sex, weight).  
- Medical history (for example: diagnosis, complications with prior treatment). - Findings from physical exams.
- Laboratory test results obtained at the time of work up and after transplant (for example:
blood tests, biopsy results).
informed consent to participate in
c. parties who may disclose

The researcher and the researcher's staff may collect my individual health information from: [*List hospitals, clinics or providers from which health care information can be requested*].  
D. **Parties Who May Receive or Use My Individual Health Information**:
The individual health information disclosed by parties listed in item c and information disclosed by me during the course of the research may be received and used by the following parties:
- Principal Investigator and the researcher's staff:
Dr. Bart Scott, Co-Principal Investigator Dr. Mitchell Horwitz, Co-Principal Investigator
- National Heart, Lung, and Blood Institute (NHLBI) and the National Cancer Institute
(NCI), both of the National Institutes of Health (NIH),
- Study sponsors: Blood and Marrow Transplant Clinical Trials Network (BMT CTN),
Data and Coordinating Center
- U.S. government agencies that are responsible for overseeing research such as the Food and Drug Administration (FDA) and the Office of Human Research Protections (OHRP)
- U.S. government agencies that are responsible for overseeing public health concerns such as the Centers for Disease Control (CDC) and federal, state and local health departments.
informed consent to participate in
e. right to refuse to

I do not have to sign this Authorization. If I decide not to sign the Authorization, I will not be allowed to participate in this study or receive any treatment related to research that is provided through the study. My decision not to sign this authorization will not affect any other treatment, payment, or enrollment in health plans or eligibility for benefits.
informed consent to participate in
f. right to revoke

I can change my mind and withdraw this authorization at any time by sending a written notice to the Principal Investigator to inform the researcher of my decision. If I withdraw this authorization, the researcher may only use and disclose the protected health information already collected for this research study. No further health information about me will be collected by or disclosed to the researcher for this study.
informed consent to participate in
g. potential for re-disclosure

My individual health information disclosed under this authorization may be subject to redisclosure outside the research study and no longer protected.  
Examples include potential disclosures for law enforcement purposes, mandated reporting or abuse or neglect, judicial proceedings, health oversight activities and public health measures.
informed consent to participate in
h. this authorization does not

Researchers also want to learn how to better predict possible health problems and how to make transplants more successful. Much of this research is done using human tissue or blood. We would like to store a sample of your blood for use in future research studies. Your blood would be collected at your transplant center before your transplant. We would keep the sample at a central place called the BMT CTN Research Sample Repository (this will be called the "Repository" in the rest of the consent form). A Repository is a place that protects, stores and sends out samples for approved research studies. Some general things you should know about letting us store your blood samples for research are:  
- We will only store samples from people who give us permission. You should feel free to talk over your decision with your family, friends, doctor, and health care team. If you decide to not let us store research samples now or in the future, it will not affect your medical care.  
- Research is meant to gain knowledge that may help people in the future. You will not get any direct benefit from taking part.  
- All testing done on your blood is for research purposes. You or your doctor will not be given results and they will not be added to your medical record.  
- You will not get paid for any samples or for any products that may be developed from current or future research.
If you agree to provide a blood sample, here is what will happen:
1. A single 6 mL sample of your blood (approximately 1 teaspoon) will be collected before your transplant and stored solely for research purposes. The collection will be done at the same time as the routine blood collection done for the study.  
2. The research sample will be given unique bar code designation that cannot be linked to you by the researcher testing your samples.
3. Researchers can apply to study the materials stored in the Repository.  
4. Materials stored in the Repository will be used mainly by clinicians and researchers in the BMT CTN network. In the future, the remaining research samples and clinical data will be made available outside of this network. Researchers from other universities, the
government, and drug or health-related companies can apply to use the samples and information. Only skilled researchers will be allowed to use the samples and information.  
5. The BMT CTN Steering Committee or the BMT CTN Biomarkers Committee must approve each study application before they will share samples or information with researchers. This kind of review is to make sure that the investigators requesting the samples are qualified, and that the research they propose has a high potential of success and for contribution of scientific knowledge.  
6. DNA from your stored blood sample might be used in genome-wide association (GWA)
or pharmacogenomics studies for a future project either done or supported by the National Institutes of Health (NIH). Genome-wide association studies are a way for scientists to identify genes involved in human disease. This method searches the genome for small genetic changes that are more common in people with a particular disease than in people without the disease. Each study can look at hundreds of thousands of genetic changes at the same time. Researchers use data from this type of study to find genes that may add to a person's risk of developing a certain disease. Pharmacogenomics studies are similar genetic tests but look specifically at genes related to how the body breaks down medications. If your coded samples are used in such a study, the researcher is required to add your test results and sample information into a shared, public research database. This public database is called the NIH Genotype and Phenotype Database and it is managed by the National Center for Biotechnology Information (NCBI). The NCBI will never have any information that would identify you, or link you to your information or research samples.  
7. A new federal law (2009), called the Genetic Information Nondiscrimination Act (GINA)
generally makes it illegal for health insurance companies, group health plans, and employers of 15 or more persons to discriminate against you based on your genetic information. Health insurance companies and group health plans may not request your genetic information that we get from this research. This means that they must not use your genetic information when making decisions regarding insurability. Be aware that this new federal law will not protect you against genetic discrimination by companies that sell life insurance, disability insurance, or long-term care insurance.
informed consent to participate in
statement of consent

The purpose of storing blood samples, the procedures involved, and the risks and benefits have been explained to me. I have asked all the questions I have at this time and I have been told whom to contact if I have more questions. I have been told that I will be given a signed copy of this consent form to keep. I understand that I do not have to allow the use of my blood for future research. If I decide to not let you store research samples now or in the future, it will not affect my medical care in any way. I voluntarily agree that my blood and information can be stored indefinitely by the BMT CTN and/or NHLBI Repositories for research to learn about, prevent, or treat health problems. I also understand that my DNA and health information may or may not be used in genome-wide association studies.  
❑ I agree to allow my blood samples to be stored for research.  
❑ I do not agree to allow my blood samples to be stored for research.
Signature Date TITLE: **BMT CTN 0901:** A Randomized, Multi-Center, Phase III Study of Allogeneic Stem Cell Transplantation Comparing Regimen Intensity in Patients with Myelodysplastic Syndrome or Acute Myeloid Leukemia  
CO-INVESTIGATOR: Mitchell Horwitz, MD Duke University 2400 Pratt St. DUMC 3961 Durham, NC 27710 Phone: (919) 668-1045  
| Fred Hutchinson Cancer Research Center 1100 Fairview Avenue North, D1-100 Seattle, WA 98109-1023 Phone: (206) 667-1990   |
|--------------------------------------------------------------------------------------------------------------------------|  
- I have read and understood this Consent Form. The nature and purpose of the research study has been explained to me.  
- I understand that the transplant intensity will be randomly assigned to me. - I have had the chance to ask questions, and understand the answers I have been given. I
understand that I may ask questions at any time during the study.  
- I freely agree to be a participant in the study. - I understand that I may not directly benefit from taking part in the study.  
- I understand that, while information gained during the study may be published, I will not be identified and my personal results will stay confidential.  
- I have had the chance to discuss my participation in this research study with a family member or friend.  
- I understand that I can leave this study at any time, and doing so will not affect my current care or prevent me from receiving future treatment.  
- I understand that I will be given a copy of this signed consent form.
informed consent to participate in
participant signature print name date

I certify that I have provided a verbal explanation of the details of the research study, including the procedures and risks. I believe the participant has understood the information provided. Signature of Counseling Physician Date
appendix c
laboratory procedures 1. busulfan pharmacokinetics

Patients receiving busulfan (Bu) as part of the conditioning regimen regardless of the intensity will participate in this ancillary study if their transplant center agrees to participate in this study. Even though Bu PK is routinely done in some instances to optimize Bu exposure and minimize toxicity with myeloablative Bu doses, this practice is not done in patients who receive reduced intensity Bu doses. This study will analyze Bu PK in all eligible patients at a central laboratory with the objective of correlating PK results with posttransplant outcomes, including rates of relapse, treatment-related mortality, disease-free and overall survival. Patients who receive Bu will be asked to provide up to 7 peripheral blood samples, using a pre-set sampling strategy after the first dose of Bu. Samples will be collected up to 8 hours post administration of Bu. Patients who already have Bu PK performed as part of their routine clinical care will need additional samples to be analyzed at a central laboratory for this study. They can be collected on the same day or another day busulfan is given.
appendix c
samples required

One 3 mL peripheral blood sample will be collected in a 6 mL-fill, sodium heparincontaining Vacutainer® tubes at up to seven scheduled post-Bu dose sample collection time points for a total of 6 or 7 samples (21 mL maximum). Peripheral blood samples will be placed on ice immediately after collection, centrifuged at 1000 g at 4ºC for 5 minutes for plasma separation. The plasma will be removed from each vacutainer and placed in a single polypropylene cryovial corresponding for each time point. The cryovials will be labeled and frozen at -70ºC. The plasma samples must be well frozen prior to shipping to the project laboratory.
appendix c
samples shipment

Samples will collected at 6-7 different time points depending on the route of Bu administration and Bu dosing schedule. The table below outlines the proposed sampling strategy after the first dose of Bu. The final schedule will be provided in the BMT CTN 0901 Laboratory Sample Guide.  
| Dosing                                                                                                                                                                                   | Bu Sample Collection Time Points   |           |          |           |          |                   |         |
|------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------|------------------------------------|-----------|----------|-----------|----------|-------------------|---------|
| Schedule                                                                                                                                                                                 | #1                                 | #2        | #3       | #4        | #5       | #6                | #7      |
| IV                                                                                                                                                                                       | End of Infusion                    | EOI+      | EOI+     | 240 min   | 300 min  | 360 min  (6 hrs)2 | ---     |
| (EOI)1                                                                                                                                                                                   | (4hrs)2                            | (5 hrs)2  |          |           |          |                   |         |
| Q6 hours                                                                                                                                                                                 | 15 min                             | 30 min    |          |           |          |                   |         |
| IV                                                                                                                                                                                       | End of Infusion                    | EOI+      | 240 min  | 300 min   | 360 min  | 480 min  (8hrs)2  | ---     |
| Q24 Hours                                                                                                                                                                                | (EOI) 1                            | 15 min    | (4 hrs)2 | (5 hrs) 2 | (6 hrs)2 |                   |         |
| Oral                                                                                                                                                                                     | 15 min                             | 30 min    | 60 min   | 120 min   | 240 min  | 300 min           | 360 min |
| Busulfan                                                                                                                                                                                 | (1 hrs)                            | (2 hrs)   | (4 hrs)  | (5 hrs)   | (6 hrs)  |                   |         |
| (suspension) Oral                                                                                                                                                                        | 30 min                             | 60 min    | 90 min   | 120 min   | 240 min  | 300 min           | 360 min |
| Busulfan                                                                                                                                                                                 | (1 hr)                             | (1.5 hrs) | (2 hrs)  | (4 hrs)   | (5 hrs)  | (6 hrs)           |         |
| (tablet) 1 For the initial EOI sample, be sure all of the drug has been delivered and the lines have been thoroughly  flushed by Saline before drawing the end of infusion (EOI) sample. |                                    |           |          |           |          |                   |         |
appendix c
samples shipment and test result

Clinical centers will ship the set of six or seven frozen plasma cryovials in 5 kg of dry ice by priority overnight FedEx either on (1) the day of collection (Monday through Thursday) or (2) on the day following collection if sample collection is performed Monday through Wednesday. If the sample collection occurs Friday through Sunday, the samples will need to be stored for Monday shipment. Testing results will be provided by the Project Laboratory to the submitting clinical center, for subsequent entry into the Advantage EDC study data system.
appendix c
2. optional sample for undefined

Patients consenting to provide optional research samples to be submitted to the BMT CTN Research Sample Repository for future testing will have an additional baseline peripheral blood sample collected.
appendix c
samples required

A 6 mL peripheral blood sample will be collected at within 30 days of the initiation of preparative regimen in a 6 mL-fill, EDTA containing Vacutainer® tube (lavender). The peripheral blood sample will be stored upright in a rack at room temperature while preparing to ship the sample on the same day to the BMT CTN Research Sample Repository. Centers should arrange to have these patient samples collected on Monday-Thursday for receipt at the repository on Tuesday-Friday.
appendix c
sample shipment

Clinical centers will ship the peripheral blood tube at ambient temperature by priority overnight FedEx on the day of collection directly to the BMT CTN Research Sample Repository for processing and long-term storage of whole blood aliquots.  
| SAMPLE                                                                                                                                                                                                                           |                                                                            |                                                                                                                                                                                                                                                                                                                                                                                            |
|----------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------|----------------------------------------------------------------------------|--------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------|
| COLLECTION,                                                                                                                                                                                                                      |                                                                            |                                                                                                                                                                                                                                                                                                                                                                                            |
| PROCESSING AND  STORAGE  REQUIREMENTS                                                                                                                                                                                            |                                                                            |                                                                                                                                                                                                                                                                                                                                                                                            |
| RESEARCH SAMPLE PURPOSE                                                                                                                                                                                                          | SAMPLE                                                                     |                                                                                                                                                                                                                                                                                                                                                                                            |
| COLLECTION                                                                                                                                                                                                                       | SHIPPING SPECIFICATIONS                                                    |                                                                                                                                                                                                                                                                                                                                                                                            |
| TIME POINTS                                                                                                                                                                                                                      |                                                                            |                                                                                                                                                                                                                                                                                                                                                                                            |
| RESEARCH TOPIC                                                                                                                                                                                                                   | TYPE OF  SAMPLE                                                            | Cryovials set will be shipped frozen in  5 kg on dry ice directly to the Project  Laboratory by priority overnight Fed  Ex delivery for processing and  busulfan testing. Guidelines for the  final specimen collection schedule,  handling and shipment to the Project  Laboratory is detailed in the BMT  CTN 0901 Laboratory Research  Sample Guide.                                    |
| Gently mix blood with  heparin by inverting the  tube 8-10 times. Centrifuge  at 1000 g at 4ºC to separate  plasma. Freeze the plasma  in polypropylene cryovials.  Ship the complete set of PK  samples to Project  Laboratory. |                                                                            |                                                                                                                                                                                                                                                                                                                                                                                            |
| Up to seven 3 mL  peripheral blood  samples collected  in 6 mL sodium  heparin  containing, greentop vacutainer  tube.                                                                                                                                                                                                                                  | Up to 7 time  points from                                                  |                                                                                                                                                                                                                                                                                                                                                                                            |
| completion of the  first dose of Bu  until 6-8 hours  later.                                                                                                                                                                     |                                                                            |                                                                                                                                                                                                                                                                                                                                                                                            |
| Busulfan                                                                                                                                                                                                                         |                                                                            |                                                                                                                                                                                                                                                                                                                                                                                            |
| Pharmacokine tics Studies1                                                                                                                                                                                                       | Serial  Busulfan Levels                                                    | Peripheral blood tube will be shipped  at ambient temperature on the day of  collection, directly to the BMT CTN  Research Sample Repository by  priority overnight Fed Ex delivery for  processing and long-term storage of  whole blood aliquots. Guidelines for  specimen handling and shipment to  the Repository are detailed in the  BMT CTN 0901 Laboratory Research  Sample Guide. |
| One 6 mL                                                                                                                                                                                                                         |                                                                            |                                                                                                                                                                                                                                                                                                                                                                                            |
| peripheral blood  sample collected  in an EDTA  containing,  lavender-top  vacutainer tube.                                                                                                                                      | Gently mix blood with                                                      |                                                                                                                                                                                                                                                                                                                                                                                            |
| EDTA by inverting the tube  8-10 times. Store upright in  a rack at room temperature  while preparing to ship to  the BMT CTN Research Sample Repository.                                                                        | Pre-transplant Within 30 days of  the initiation of  preparative  regimen. |                                                                                                                                                                                                                                                                                                                                                                                            |
| (Optional)                                                                                                                                                                                                                       |                                                                            |                                                                                                                                                                                                                                                                                                                                                                                            |
| Investigational  Future  Research  Sample2                                                                                                                                                                                       | Undefined  Future  Studies                                                 |                                                                                                                                                                                                                                                                                                                                                                                            |
| 1 To be collected only from patients receiving busulfan at participating transplant centers.                                                                                                                                     |                                                                            |                                                                                                                                                                                                                                                                                                                                                                                            |  
1 To be collected only from patients receiving busulfan at participating transplant centers.  
2 To be collected only from patients consenting to future research sample.
appendix d international prognostic scoring
appendix d international prognostic scoring

| IPSS* for MDS:  Survival and AML Evolution   |             |              |       |       |     |
|----------------------------------------------|-------------|--------------|-------|-------|-----|
| Prognostic                                   | Score Value |              |       |       |     |
| Value                                        | 0           | 0.5          | 1.0   | 1.5   | 2.0 |
| BM blasts (%)                                | <5          | 5-10         | 11-20 | 21-30 |     |
| Karyotype 1                                  | Good        | Intermediate | Poor  |       |     |
| Cytopenias                                   | 0 or 1      | 2 or 3       |       |       |     |  
Scores for risk groups are as follows:  Low = 0; INT-1 = 0.5-1.0; INT-2 = 1.5-2.0; and High = ≥2.5.  
.  
International Prognostic Scoring System [21]
t Good, normal, -Y, del(5q), del(20q); Poor, complex (≥3 abnormalities) or chromosome 7 anomalies; Intermediate, other abnormalities.
appendix e swog cytogenetic classification
appendix d international prognostic scoring

| Risk status                                                             | SWOG coding                                                             |
|-------------------------------------------------------------------------|-------------------------------------------------------------------------|
| inv(16)/t(16;16)/del(16q), t(15;17) with/without secondary aberrations; |                                                                         |
| Favorable                                                               | t(8;21) lacking del(9q) or complex karyotypes                           |
| Normal, +8, +6, −Y, del(12p)                                            |                                                                         |
| Intermediate                                                            |                                                                         |
| Unfavorable                                                             | del(5q)/−5, −7/del(7q), abn 3q, 9q, 11q, 20q, 21q, 17p, t(6;9), t(9;22) |
| and complex karyotypes (≥ 3 unrelated abn)                              |                                                                         |
| Unknown                                                                 | All other abnormalities                                                 |
appendix f karnofsky performance score
appendix d international prognostic scoring

| Index          | Specific Criteria                                                             | General                                                                                                                |
|----------------|-------------------------------------------------------------------------------|------------------------------------------------------------------------------------------------------------------------|
| 100            | Normal, no complaints, no evidence of disease.                                | Able to carry on normal activity; no  special care needed.                                                             |
| 90             | Able to carry on normal activity, minor signs or  symptoms of disease.        |                                                                                                                        |
| 80             | Normal activity with effort, some signs or  symptoms of disease.              |                                                                                                                        |
| 70             | Care for self, unable to carry on normal activity                             | Unable to work, able to live at                                                                                        |
| or to do work. | home and care for most personal  needs, varying amount of assistance          |                                                                                                                        |
| 60             | Requires occasional assistance from others but  able to care for most needs.  | needed.                                                                                                                |
| 50             | Requires considerable assistance from others  and frequent medical care       |                                                                                                                        |
| 40             | Disabled, requires special care and assistance.                               | Unable to care for self, requires  institutional or hospital care or  equivalent, disease may be rapidly  progressing. |
| 30             | Severely disabled, hospitalization indicated, but  death not imminent.        |                                                                                                                        |
| 20             | Very sick, hospitalization necessary, active  supportive treatment necessary. |                                                                                                                        |
| 10             | Moribund                                                                      |                                                                                                                        |
| 0              | Dead                                                                          |                                                                                                                        |
appendix g
appendix d international prognostic scoring

| Comorbidities                                                                   | Definition                                               | Score   |
|---------------------------------------------------------------------------------|----------------------------------------------------------|---------|
| Migraine/headache                                                               | 0                                                        |         |
| Osteoporosis                                                                    | 0                                                        |         |
| Osteoarthritis                                                                  | 0                                                        |         |
| Hypertension                                                                    | 0                                                        |         |
| Gastrointestinal                                                                | Including inflammatory bowel disease                     | 0       |
| Mild pulmonary                                                                  | DLCo and/or FEV1 >80% or                                 | 0       |
| Dyspnea on moderate activity                                                    |                                                          |         |
| Mild renal                                                                      | Serum creatinine 1.2-2 mg/dl                             | 0       |
| Endocrine                                                                       | 0                                                        |         |
| Bleeding                                                                        | 0                                                        |         |
| Coagulopathy                                                                    | Deep venous thrombosis or pulmonary embolism             | 0       |
| Asthma                                                                          | 0                                                        |         |
| Arrhythmia                                                                      | 1                                                        |         |
| Myocardial                                                                      | Coronary artery disease, congestive HF, history of       | 1       |
| medically documented MI, EF50%                                                 |                                                          |         |
| Mild hepatic                                                                    | Chronic hepatitis, Bilirubin >ULN- 1.5 X ULN, or         | 1       |
| AST/ALT >ULN-2.5XULN                                                            |                                                          |         |
| Cerebro-vascular                                                                | History of transient ischemic attack or cerebro-vascular | 1       |
| accident                                                                        | accident                                                 |         |
| Morbid obesity                                                                  | 1                                                        |         |
| Diabetes                                                                        | Requiring treatment                                      | 1       |
| Depression/anxiety                                                              | 1                                                        |         |
| Infection                                                                       | Requiring continuation of treatment after Day 0          | 1       |
| Rheumatologic                                                                   | SLE, RA, polymyositis, mixed CTD, polymyalgia            | 2       |
| rheumatica                                                                      |                                                          |         |
| Moderate pulmonary                                                              | DLCo and/or FEV1 66-80% or                               | 2       |
| Dyspnea on slight activity                                                      |                                                          |         |
| Peptic ulcer                                                                    | Patients who have required treatment                     | 2       |
| Moderate-severe renal                                                           | Serum creatinine >2 mg/dl, on dialysis, or prior renal   | 2       |
| transplantation                                                                 |                                                          |         |
| Valvular heart disease                                                          | Except mitral valve prolapse                             | 3       |
| Prior solid tumor                                                               | Requiring treatment with chemotherapy                    | 3       |
| Moderate-severe hepatic                                                         | Liver cirrhosis, Bilirubin >1.5 X ULN, or AST/ALT        | 3       |
| >2.5XULN                                                                        |                                                          |         |
| Severe pulmonary                                                                | DLCo and/or FEV1 65% or                                 | 3       |
| Dyspnea at rest or requiring oxygen                                             |                                                          |         |
| Total score is the sum of all comorbidities present at time of transplantation. |                                                          |         |
appendix h
human subjects

Subject consent: Candidates for the study will be identified as described in Chapter 4 of the protocol. The Principal Investigator or his/her designee at each transplant center will contact the candidates and enroll them onto the study. The study coordinator at each center will provide the patient with information about the purpose of the study and obtain consent. The Network will provide a template of the consent form to each center. Each center will customize the template according to their local requirements and submit it for review by the local Internal Review Board (IRB). The DCC will verify the adequacy of the consent forms. Each center must provide evidence of IRB approval.  
Confidentiality: Confidentiality will be maintained by individual names being masked and assigned a patient identifier code. The code relaying the patient's identity with the ID code will be kept separately at the center. The ID code will be transmitted to the network. Participation of women, children, minorities and other populations: Women and ethnic minorities will be included in this study. Children will not be included. Accrual will be monitored within each center with the expectation that the enrolled patient population is representative of the transplanted patient population at each center. Representation will be examined by comparing gender, race, ethnicity, and age distributions. Accrual of minority patients will be expected to be in proportion to the number of minority patients transplanted at each center. The DCC and NHLBI will discuss enrollment anomalies with the centers.  
-
appendix i
references

1 SEER Cancer Statistics Review, 1975-2001. Bethesda, MD: National Cancer Institute; 2004.  
2 Bennett JM, Catovsky D, Daniel MT, et al. Proposed revised criteria for the classification of acute myeloid leukemia. A report of the French-American-British Cooperative Group. Ann Intern Med 1985;103(4):620-625.  
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