Clinical trial: NCT01339910
Section: Confidentiality
Subsections: What if I change my mind?|Privacy, Confidentiality and Use of Information|B. Individual Health Information to be Used or Disclosed:|B. Individual Health Information to be Used or Disclosed:|B. Individual Health Information to be Used or Disclosed:|Blood Samples for Future Research (optional)

samples and information that have already been shared with other researchers cannot be taken
back or destroyed.

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  
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.  
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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  
Demographic information (for example: date of birth, sex, weight)  
Medical history (for example: diagnosis, complications with prior treatment)  
Findings from physical exams  
1 HIPAA is the Health Insurance Portability and Accountability Act of 1996, a federal law related to privacy of health
information  
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Laboratory test results obtained at the time of work up and after transplant (for example:
blood tests, biopsy results)
C. Parties Who May Disclose My Individual Health Information:
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.
E. Right to Refuse to Sign this Authorization:
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.
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.
G. Potential for Re-disclosure:
My individual health information disclosed under this authorization may be subject to re-
disclosure outside the research study and no longer protected.  
Laboratory test results obtained at the time of work up and after transplant (for example:
blood tests, biopsy results)  
C. Parties Who May Disclose My Individual Health Information:  
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.  
E. Right to Refuse to Sign this Authorization:  
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.  
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.  
G. Potential for Re-disclosure:
My individual health information disclosed under this authorization may be subject to re-
disclosure outside the research study and no longer protected.  
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Examples include potential disclosures for law enforcement purposes, mandated reporting or
abuse or neglect, judicial proceedings, health oversight activities and public health measures.

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.  
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.  
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Clinical trial: NCT01339910
Section: Duration of Study Involvement
Subsections: Study Participation

You will need to go to clinic at least once before the study begins. Your participation in the
study starts after your sign this consent form. After your transplant you will have weekly
evaluations for the first 3 months of this study. After 3 months, you will have an evaluation at 6,
12 and 18 months after your transplant.  
These evaluations will be done if you are in the hospital ward or clinic, or if your disease
becomes active again after the transplant.
Clinical trial: NCT01339910
Section: Participant's Rights
Subsections: Right to ask Questions and/or Withdraw|Further Information|Independent Contact

You have the right to ask questions about the study at any time. If you have questions about
your rights as a participant or you want to leave the study, please contact [insert contact info].  
Being in this study is voluntary. You can choose to not be in this study, or leave this study at
any time.  
If you choose to not take part or to leave this study, your regular medical care will not be
affected in any way. The conditioning regimen of your transplant will be the standard of care. If
you decide to leave this study after taking the study treatment, or are asked to leave by your
doctor for medical reasons, you will be asked to come back to the doctor's office for tests for
your safety and as part of your routine medical care.  
Even if you withdraw from the study, the information collected from your participation will
be included in the study evaluation, unless you specifically ask that it not be included.
Your study doctor and study staff will be available to answer any questions that you may
have about your participation in, or your withdrawal from this study.  
Even if you withdraw from the study, the information collected from your participation will
be included in the study evaluation, unless you specifically ask that it not be included.  
Your study doctor and study staff will be available to answer any questions that you may
have about your participation in, or your withdrawal from this study.

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].

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].  
Health Insurance Portability and Accountability Act 1 (HIPAA1) Authorization to use and
disclose individual health information for research purposes  
A. Purpose:  
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
Clinical trial: NCT01339910
Section: Possible Risks, Discomforts, and Inconveniences
Subsections: Risks and Discomforts|Risks Related to Medications or Radiation Used in Conditioning Regimens|Risks Related to the Medication Used to Help Prevent Graft-versus-Host Disease|Risks Related to the Medication Used to Help Prevent Graft-versus-Host Disease|Risks Related to the Medication Used to Help Prevent Graft-versus-Host Disease|Risks Related to the Medication Used to Help Prevent Graft-versus-Host Disease|Risks Related to the Medication Used to Help Prevent Graft-versus-Host Disease|Physical Injury as a Result of Participation|Costs & Reimbursements|||||

The risks and discomforts of stem cell transplant are the same if you join this study, or if you do
not join this study. The differences in side effects from medications are because of the different
levels of treatment strength.  
High intensity treatments usually have more side effects early after transplant compared to
reduced intensity treatments. Other problems with transplant, such as graft-versus-host disease
(GVHD) and infections happen equally in patients who have high intensity or reduced intensity
treatments.

All chemotherapy and radiation treatments used as conditioning in this study are commonly used
in allogeneic stem cell transplantation. The side effects can change based on the amount of drug
given. This is true for busulfan, which is used for reduced intensity and high intensity treatments
but in different amounts.  
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Graft-versus-Host Disease (GVHD) is a medical condition that can become serious enough to
cause death. GVHD is a common development after allogeneic stem cell transplant. It happens
when the donor cells attack and damage your organ tissues after transplant. GVHD can cause:  
Skin rashes
Feeling sick to your stomach (nausea)
Throwing up (vomiting)
Abdominal pain
Diarrhea
Liver damage or jaundice (yellowing of the skin or eyes)  
Skin rashes  
Feeling sick to your stomach (nausea)  
Throwing up (vomiting)  
Abdominal pain  
Diarrhea  
Liver damage or jaundice (yellowing of the skin or eyes)  
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Your doctor will prescribe medication to prevent GVHD. You will start GVHD prevention
around the time you get your donor cells, and it can last many months after the transplant. These
medications do not completely prevent GVHD and more drugs might be needed to manage this
complication.  
Your doctor will decide which GVHD prevention treatment is the best choice for you. This
decision is not part of the research study. Your doctor will also decide your medications based
on what is regularly used for transplant in this hospital or clinic. Below is a list of commonly
used drugs used to prevent GVHD. Your doctor may choose to use other medications than what
is listed here.  
Tacrolimus: This drug is used to try to prevent GVHD. Early side effects you may have
include: feeling sick to your stomach (nausea) or throwing up (vomiting) after you
swallow. Other side effects include high blood pressure (hypertension), shaking hands
(tremor), increased hair growth and possibly how clearly you can think or make decisions
(mental function).
If you have these effects, they generally go away if your doctor lowers the amount of
medication you take. A few patients have had a seizure while on this medication.
Your liver or kidneys might not work as well as they did before. If this happens, your
doctor may lower the amount of drug you take or stop giving the drug completely. You
might be more likely to have kidney problems if you need to take other medications at the
same time. This is especially true for drugs that we know might cause kidney problems,
such as antibiotics. Sometimes, the kidney damage caused is serious enough for you to
need an artificial kidney machine (hemodialysis).
Some patients given tacrolimus develop diabetes and must take insulin while taking
tacrolimus.
It is very important that you do not eat grapefruit or drink grapefruit juice.
Grapefruit has an ingredient called bergamottin, which can affect some of the
treatment drugs, including tacrolimus, used in this study. Common soft drinks that
have bergamottin are Fresca, Squirt and Sunny Delight.
Methotrexate: This is also a medication used to try to prevent GVHD. Methotrexate
causes damage to cells and can affect many different parts of your body. It may cause
mouth sores or mouth inflammation. Or if you already have these problems from your
treatments and other medications, they can get worse.  
Tacrolimus: This drug is used to try to prevent GVHD. Early side effects you may have
include: feeling sick to your stomach (nausea) or throwing up (vomiting) after you
swallow. Other side effects include high blood pressure (hypertension), shaking hands
(tremor), increased hair growth and possibly how clearly you can think or make decisions
(mental function).  
If you have these effects, they generally go away if your doctor lowers the amount of
medication you take. A few patients have had a seizure while on this medication.  
Your liver or kidneys might not work as well as they did before. If this happens, your
doctor may lower the amount of drug you take or stop giving the drug completely. You
might be more likely to have kidney problems if you need to take other medications at the
same time. This is especially true for drugs that we know might cause kidney problems,
such as antibiotics. Sometimes, the kidney damage caused is serious enough for you to
need an artificial kidney machine (hemodialysis).  
Some patients given tacrolimus develop diabetes and must take insulin while taking
tacrolimus.  
It is very important that you do not eat grapefruit or drink grapefruit juice.
Grapefruit has an ingredient called bergamottin, which can affect some of the
treatment drugs, including tacrolimus, used in this study. Common soft drinks that
have bergamottin are Fresca, Squirt and Sunny Delight.  
Methotrexate: This is also a medication used to try to prevent GVHD. Methotrexate
causes damage to cells and can affect many different parts of your body. It may cause
mouth sores or mouth inflammation. Or if you already have these problems from your
treatments and other medications, they can get worse.  
Methotrexate may slow down the recovery of blood cells after transplantation.
Methotrexate can also cause kidney damage. If your kidneys are already damaged for  
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other reasons, it can make your kidneys worse. If kidney damage does happen, your
doctor might give you a lower dose of methotrexate, or stop giving it completely.  
Tacrolimus and Methotrexate: These medications can affect your body's immune
system and make it easier for you to get infections. Even simple infections can become
very serious and even life-threatening. As a result, you might have more infections for
several months after transplant, especially viral infections and pneumonia.
Risks Related to the Transplant Procedure: The following risks are part of the
transplant process and not connected to any one medication or the transplanted donor
cells.
-
Bleeding: Platelets help your blood to clot. When you have low amounts of
platelets, you may have bleeding problems. Once your new bone marrow starts to
grow, your platelets will increase and your blood will start to clot normally again.
Bleeding problems can range from minor bleeding, such as nosebleeds or
bruising, to more serious bleeding in your brain and lungs. Serious bleeding can
be very dangerous and can happen if your platelet levels stay low. Usually, we
can prevent major bleeding problems with transfusions of platelets. However, if
your body does not respond well to transfused platelets, you may be at serious
risk for bleeding.
Veno-occlusive Disease (VOD): High dose chemotherapy, radiation therapy and
medications used to prevent GVHD can cause veno-occlusive disease (VOD).
VOD causes severe damage to the liver. Symptoms include jaundice (yellowing
of the skin and eyes), weight gain, and extra fluid build-up in the belly (abdominal
cavity) and other parts of the body. We can usually manage veno-occlusive
disease very well, to the point where it goes away. However, complications can
happen with VOD that may put your life in danger.
Mouth Sores and Diarrhea: The large doses of chemotherapy and radiation
cause irritation in the lining of the mouth and intestines. This can result in painful
mouth sores and diarrhea. If you have severe mouth sores, we will give you
medicine to help control the pain. If your mouth sores are very bad, you may not
be able to eat normally until the sores are healed. Mouth sores get better when
your white blood count starts to rise, and your donor cells start to grow (also
called engraftment).
Capillary Leak Syndrome: This can happen from your chemotherapy and
radiation treatments. The blood vessels may become 'leaky' and fluid enters your
abdomen, lungs, and other tissues. You may gain water weight and not go to the
bathroom as often as you normally do. Capillary leak syndrome can be difficult  
Tacrolimus and Methotrexate: These medications can affect your body's immune
system and make it easier for you to get infections. Even simple infections can become
very serious and even life-threatening. As a result, you might have more infections for
several months after transplant, especially viral infections and pneumonia.  
Risks Related to the Transplant Procedure: The following risks are part of the
transplant process and not connected to any one medication or the transplanted donor
cells.  
-
Bleeding: Platelets help your blood to clot. When you have low amounts of
platelets, you may have bleeding problems. Once your new bone marrow starts to
grow, your platelets will increase and your blood will start to clot normally again.  
Bleeding problems can range from minor bleeding, such as nosebleeds or
bruising, to more serious bleeding in your brain and lungs. Serious bleeding can
be very dangerous and can happen if your platelet levels stay low. Usually, we
can prevent major bleeding problems with transfusions of platelets. However, if
your body does not respond well to transfused platelets, you may be at serious
risk for bleeding.  
Veno-occlusive Disease (VOD): High dose chemotherapy, radiation therapy and
medications used to prevent GVHD can cause veno-occlusive disease (VOD).
VOD causes severe damage to the liver. Symptoms include jaundice (yellowing
of the skin and eyes), weight gain, and extra fluid build-up in the belly (abdominal
cavity) and other parts of the body. We can usually manage veno-occlusive
disease very well, to the point where it goes away. However, complications can
happen with VOD that may put your life in danger.  
Mouth Sores and Diarrhea: The large doses of chemotherapy and radiation
cause irritation in the lining of the mouth and intestines. This can result in painful
mouth sores and diarrhea. If you have severe mouth sores, we will give you
medicine to help control the pain. If your mouth sores are very bad, you may not
be able to eat normally until the sores are healed. Mouth sores get better when
your white blood count starts to rise, and your donor cells start to grow (also
called engraftment).  
Capillary Leak Syndrome: This can happen from your chemotherapy and
radiation treatments. The blood vessels may become 'leaky' and fluid enters your
abdomen, lungs, and other tissues. You may gain water weight and not go to the
bathroom as often as you normally do. Capillary leak syndrome can be difficult  
B-14

to manage if extra fluid enters your lungs and makes it hard to breathe. You may
die if fluid continues to build up in your lungs.  
Unexpected Organ Damage and Other Side Effects: You might have
unexpected, life-threatening heart, lung, kidney, or liver damage as a result of
your transplant. High doses of chemotherapy and radiation can cause very bad
lung damage that may not get better with time or medications. If this happens,
you may need to use oxygen or even a respirator. The lung damage may get
worse and be life-threatening. Rarely, multiple organ failure (such as lung and
kidney failure) can happen, which can lead to death.
Fluid Build-up: We will give you intravenous (IV) fluids during the transplant
process and it can be hard for your body to eliminate this fluid. We will also give
you Furosemide, which is a medication that can help your body get rid of the
extra fluid. One risk of Furosamide is hearing loss. Some side effects may be
loss of body chemicals such as potassium and sodium.
Late Effects: You may have side effects happen a few months to many years
after your transplant.
You may have problems with your thyroid gland that require you to take
thyroid medication.
You may get cataracts earlier in life compared to a person who has not had
a transplant. If you develop cataracts (cloudiness in the eyes) they may
need treatment.
Your kidneys could be affected and cause anemia (low red blood cell
count) or high blood pressure.
You may develop a second cancer as a result of the chemotherapy,
radiation and/or underlying disease. If secondary cancers happen they
generally do not develop until 10 to 15 years after your transplant.
We do not know the long-term effects of transplant on your heart, lungs
and brain.
Unforeseen Risks: New risks might appear at any time during the study that are
different from the risks listed in this Consent Form. We will promptly tell you of
any new information that may affect your decision to participate.  
Unexpected Organ Damage and Other Side Effects: You might have
unexpected, life-threatening heart, lung, kidney, or liver damage as a result of
your transplant. High doses of chemotherapy and radiation can cause very bad
lung damage that may not get better with time or medications. If this happens,
you may need to use oxygen or even a respirator. The lung damage may get
worse and be life-threatening. Rarely, multiple organ failure (such as lung and
kidney failure) can happen, which can lead to death.  
Fluid Build-up: We will give you intravenous (IV) fluids during the transplant
process and it can be hard for your body to eliminate this fluid. We will also give
you Furosemide, which is a medication that can help your body get rid of the
extra fluid. One risk of Furosamide is hearing loss. Some side effects may be
loss of body chemicals such as potassium and sodium.  
Late Effects: You may have side effects happen a few months to many years
after your transplant.  
You may have problems with your thyroid gland that require you to take
thyroid medication.  
You may get cataracts earlier in life compared to a person who has not had
a transplant. If you develop cataracts (cloudiness in the eyes) they may
need treatment.  
Your kidneys could be affected and cause anemia (low red blood cell
count) or high blood pressure.  
You may develop a second cancer as a result of the chemotherapy,
radiation and/or underlying disease. If secondary cancers happen they
generally do not develop until 10 to 15 years after your transplant.  
We do not know the long-term effects of transplant on your heart, lungs
and brain.  
Unforeseen Risks: New risks might appear at any time during the study that are
different from the risks listed in this Consent Form. We will promptly tell you of
any new information that may affect your decision to participate.  
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Risk to the unborn: The treatments in this study have NOT been proven to be safe at
any stage of pregnancy. Therefore, if you are pregnant or nursing, you are not eligible
for this study. Women who have the potential of becoming pregnant must use some form
of effective birth control while receiving chemotherapy, TBI, and GVHD prophylaxis.
Effective birth control is defined as the following:
Refraining from all acts of vaginal intercourse (ABSTINENCE)
Consistent use of birth control pills
Injectable birth control methods (Depro-Provera, Norplant)
Tubal sterilization or male partner who has undergone a vasectomy
Placement of an IUD (intrauterine device)
Use, with every act of intercourse, of a diaphragm with contraceptive jelly and/or
condoms with contraceptive foam.
Sterility and future childbearing potential for men and women. Chemotherapy
and/or irradiation may affect your ability to have children. Male patients may
become sterile (unable to produce sperm) and should discuss with their doctor
regarding sperm banking prior to transplantation. Female patients who have
attained puberty may find that their menstrual cycle becomes irregular or stops
permanently. However, this DOES NOT MEAN THAT YOU CANNOT
BECOME PREGNANT, and you must use some effective method of birth control
during transplant and afterwards until you are off GVHD prophylaxis. Damage to
reproductive tissue may result in infertility (inability to have children). It is not
known if the damage could result in birth defects. You should discuss these risks
and options in detail with your doctor before entering this study.  
Risk to the unborn: The treatments in this study have NOT been proven to be safe at
any stage of pregnancy. Therefore, if you are pregnant or nursing, you are not eligible
for this study. Women who have the potential of becoming pregnant must use some form
of effective birth control while receiving chemotherapy, TBI, and GVHD prophylaxis.
Effective birth control is defined as the following:  
Refraining from all acts of vaginal intercourse (ABSTINENCE)  
Consistent use of birth control pills  
Injectable birth control methods (Depro-Provera, Norplant)  
Tubal sterilization or male partner who has undergone a vasectomy  
Placement of an IUD (intrauterine device)  
Use, with every act of intercourse, of a diaphragm with contraceptive jelly and/or
condoms with contraceptive foam.  
Sterility and future childbearing potential for men and women. Chemotherapy
and/or irradiation may affect your ability to have children. Male patients may
become sterile (unable to produce sperm) and should discuss with their doctor
regarding sperm banking prior to transplantation. Female patients who have
attained puberty may find that their menstrual cycle becomes irregular or stops
permanently. However, this DOES NOT MEAN THAT YOU CANNOT
BECOME PREGNANT, and you must use some effective method of birth control
during transplant and afterwards until you are off GVHD prophylaxis. Damage to
reproductive tissue may result in infertility (inability to have children). It is not
known if the damage could result in birth defects. You should discuss these risks
and options in detail with your doctor before entering this study.

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.

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.  
B-18

| Cyclophosphamide    | Likely Side Effects (May happen in more than 20% of patients)                                                                                                                                              | Less Likely (May happen in less than 20% of patients)                                        | Rare (May happen in less than 2% of patients)       |
|---------------------|------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------|----------------------------------------------------------------------------------------------|-----------------------------------------------------|
|                     | Damage to male (testes) and female (ovaries) sex glands Diarrhea Fluid retention Hair loss Infertility Irregular or no menstrual cycles Loss of appetite Nausea, Vomiting Suppression of the immune system | Bleeding in the bladder Inflammation of the heart muscle (heart failure) Shortness of breath | Allergic reaction Lung fibrosis Serious skin rashes |



| Fludarabine    | Likely Side Effects (May happen in more than 20% of patients)             | Less Likely (May happen in less than 20% of patients)                | Rare (May happen in less than 2% of patients)                        |
|----------------|---------------------------------------------------------------------------|----------------------------------------------------------------------|----------------------------------------------------------------------|
|                | Diarrhea Mouth sores Nausea and vomiting Suppression of the immune system | Fever Numbness in the extremities Sleepiness Visual changes Weakness | Coma Cough Inflammation of the lung Interstitial Pneumonia Skin rash |



| Busulfan    | Likely Side Effects (May happen in more than 20% of patients)                                                                                                                                                           | Less Likely (May happen in less than 20% of patients)                                                                                                                              | Rare (May happen in less than 2% of patients)    |
|-------------|-------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------|------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------|--------------------------------------------------|
|             | Abdominal discomfort Constipation Diarrhea Dizziness Fluid retention Headache Heartburn Insomnia Lack of appetite Mouth sores Nausea and vomiting Running nose Skin rashes Irregular or no menstrual cycles Tachycardia | Cough Hepatic Veno- occlusive disease High blood pressure High magnesium and phosphorus levels in the blood High sugar levels in the blood Infertility Low blood pressure Seizures | Cataracts Lung fibrosis                          |



| Melphalan    | Likely Side Effects (May happen in more than 20% of patients)    | Less Likely (May happen in less than 20% of patients)    | Rare (May happen in less than 2% of patients)                  |
|--------------|------------------------------------------------------------------|----------------------------------------------------------|----------------------------------------------------------------|
|              | Constipation Diarrhea Hair loss Mucositis Nausea and vomiting    | Heart rhythm abnormalities Hepatitis Kidney failure      | Allergic reaction Interstitial Pneumonia Seizure Lung fibrosis |



| Likely ("Likely" refers to a side effect that is expected to occur in more than 20% of patients.)                                                                                                                                                                           | Less Likely ("Less likely" refers to a side effect that is expected to occur in 20% or fewer patients.)    | Rare, but Serious (These possible risks have been reported in rare occurrences, typically less than 2% of patients. They may be serious if they occur.)    |
|-----------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------|------------------------------------------------------------------------------------------------------------|------------------------------------------------------------------------------------------------------------------------------------------------------------|
| Diarrhea Nausea                                                                                                                                                                                                                                                             | Lung inflammation Pneumonia Redness of the skin Serious liver problems                                     | Risk of developing other cancers in the future Difficulty swallowing Back problems Kidney problems Learning problems                                       |
| Stomach cramps                                                                                                                                                                                                                                                              |                                                                                                            |                                                                                                                                                            |
| Vomiting (throwing up) Painful swelling of the salivary glands under the ears for a few days Short-term hair loss Anemia Infection Bleeding Cataracts Sterility (inability to have children) Slow growth Hormone problems (such as thyroid disease or diabetes) Mouth sores |                                                                                                            |                                                                                                                                                            |
Clinical trial: NCT01339910
Section: Procedures
Subsections: Background|Procedures|Before You Start Your Treatment|Randomization|Study Evaluations|Study Evaluations|Health Quality of Life|Blood Samples for Future Research (optional)

This research study is sponsored by The National Institutes of Health (NIH) through the Blood
and Marrow Transplant Clinical Trials Network (BMT CTN).  
Conditioning Regimen  
The conditioning regimen is a combination of chemotherapy and/or radiation given to patients
before the donor cells are infused. This treatment allows donor cells to engraft and start growing
in your bone marrow. The treatment also helps to kill cancer cells that might not be detectable.  
Different chemotherapy drugs can be used as part of the conditioning regimen. Some common
combinations of chemotherapy drugs used for transplant are:  
Busulfan + cyclophosphamide or fludarabine
Fludarabine + melphalan
Radiation + cyclophosphamide  
Busulfan + cyclophosphamide or fludarabine  
Fludarabine + melphalan  
Radiation + cyclophosphamide  
Each combination of chemotherapy drugs or radiation has a different strength. This strength can
also be described as the treatment "intensity."  
Stem cell transplant destroys cancer in two ways.  
The treatment (or conditioning regimen) destroys cancer cells.
The immune cells from the donor can recognize cancer cells and kill them.  
The treatment (or conditioning regimen) destroys cancer cells.  
The immune cells from the donor can recognize cancer cells and kill them.  
High intensity treatments are also known as myeloablative conditioning (MAC) regimens. These
treatments work well to destroy cancer cells because they use very high amounts of
chemotherapy or radiation. High intensity treatments can also have more side effects during and
after transplant.  
Using a lower or "reduced" intensity treatment before transplant can have fewer serious
problems from the chemotherapy drugs. While the cancer killing effects may also be lower,  
B-4

Before you join the study, we will evaluate your general health, medical history, and your current
medications.  
B-5

You will have at least one clinic visit before you begin the study. This visit will collect
information about your:  
Physical health (including history, height, weight and temperature);
Heart, lung and kidney function;
Chest x-ray;
Bone marrow biopsy and aspirate;
Routine blood tests, including cell counts, liver and kidney function;
Routine markers of infectious diseases, including hepatitis, herpes, HIV, syphilis,
varicella zoster (shingles) among others;
Pregnancy test (if it applies to you);
HLA typing for you and your donor; and,
Health quality of life for English speaking patients (see below).  
Physical health (including history, height, weight and temperature);  
Heart, lung and kidney function;  
Chest x-ray;  
Bone marrow biopsy and aspirate;  
Routine blood tests, including cell counts, liver and kidney function;  
Routine markers of infectious diseases, including hepatitis, herpes, HIV, syphilis,
varicella zoster (shingles) among others;  
Pregnancy test (if it applies to you);  
HLA typing for you and your donor; and,  
Health quality of life for English speaking patients (see below).

We selected five different treatment options based on the ones that are most often used by
transplant centers. The treatment options are listed in Table 1.  
Your doctor will choose one reduced intensity treatment (A or B in the table below) and one high
intensity treatment (C, D or E in the table below) to use for this study. These are often the most
commonly used at [insert Institution name]. A computer program will then assign you by chance
to either the reduced intensity or the high intensity treatment option.  
You will have an equal chance of being placed in either group. This means that half of the
people in the study will be in the reduced intensity group and half will be in the high intensity
group.  
B-6

We will measure your health at specific times during your study participation. These tests and
how often they are scheduled are standard for what we do for all patients receiving an allogeneic
transplant. We would do them even if you were not part of this study.  
History, physical exam and weight: weekly for 3 months, 6, 12 and 18 months.
Routine blood tests, including cell counts, liver and kidney function: weekly for 3
months, 6, 12 and 18 months.
Bone marrow biopsy and/or aspirate: at Day 100 and 18 months.
Graft-versus-host disease (GVHD) and infections monitoring: weekly for 3 months, 6,
12 and 18 months.
Side effects or toxicity: monthly for the first 3 months, then at 6, 12 and 18 months.
Blood or bone marrow tests to find out the proportion of donor cells present in the
recipient (chimerism) at 1, 3 and 18 months.
Blood samples to determine the level of busulfan in your blood after the first dose (if you
received busulfan as part of your treatment) only if your transplant center is participating
in the ancillary study.
Health quality of life for English speaking patients (see below) at 3, 12 and 18 months.  
History, physical exam and weight: weekly for 3 months, 6, 12 and 18 months.  
Routine blood tests, including cell counts, liver and kidney function: weekly for 3
months, 6, 12 and 18 months.  
Bone marrow biopsy and/or aspirate: at Day 100 and 18 months.  
Graft-versus-host disease (GVHD) and infections monitoring: weekly for 3 months, 6,
12 and 18 months.  
Side effects or toxicity: monthly for the first 3 months, then at 6, 12 and 18 months.  
Blood or bone marrow tests to find out the proportion of donor cells present in the
recipient (chimerism) at 1, 3 and 18 months.  
Blood samples to determine the level of busulfan in your blood after the first dose (if you
received busulfan as part of your treatment) only if your transplant center is participating
in the ancillary study.  
Health quality of life for English speaking patients (see below) at 3, 12 and 18 months.  
Blood Samples for Busulfan Pharmacokinetics  
Some transplant centers may be participating in this ancillary study.  
Researchers are trying to learn more about how your body breaks down one of the drugs
(busulfan) given as part of the conditioning regimen in this study. Samples for this test will be
collected from you only if you receive this drug and your transplant center is participating in this
ancillary study. These tests measure how much busulfan is concentrated in the blood. Busulfan  
B-7

levels are already done routinely in some settings in order to avoid too high levels. Every patient
can have different levels of this drug after receiving the same dose of busulfan.  
The goal of this study is to see if these levels can be tied to the success of the transplant.
This study will explore the levels of busulfan in these two treatment intensities and compare with
what happens after transplant.  
This study will collect up to seven blood samples within 6 to 8 hours after a dose of busulfan was
given to you. Each blood sample volume is 3 mL (1/2 teaspoon). Once all seven blood samples
are collected from you, they will be sent to a laboratory for testing. None of your personal
information will be shared with the laboratory.  
The busulfan blood tests are part of this clinical trial at select centers, but your center may repeat
these tests as part of the routine transplant procedure. If this happens, your doctor will either
collect 6 mL (1 teaspoon) each time as described above, or collect 3 mL for the research tests on
another day that busulfan is given.

We will ask you about your general health and how well you feel while you participate in this
study. Even though different treatments may treat a disease equally well, there might be a
difference in how patients feel or the side effects they have after their treatment. This is
important information for when we evaluate the treatments in this study.  
We will collect information by using surveys. The surveys will ask about:  
How you feel
What symptoms you might have and how they affect you
How well can you do regular daily activities  
How you feel  
What symptoms you might have and how they affect you  
How well can you do regular daily activities  
You will need to fill out the surveys and each survey should take about 30 minutes to finish.
Your answers will help us understand how your transplant treatment affects how you feel, what
you can do, and your general quality of life.

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.  
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  
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  
B-21
Clinical trial: NCT01339910
Section: Purpose of Research
Subsections: Introduction|Background|Purpose|Possible Benefits|Statement of Consent

We are inviting you to join a research study. The main goals of the study are to:  
Compare two kinds of treatments used to destroy diseased cells and prepare your body
for transplant. This process is also called a conditioning regimen.
Measure how well your disease (acute myeloid leukemia or myelodysplastic syndrome)
responds to the treatment.  
Compare two kinds of treatments used to destroy diseased cells and prepare your body
for transplant. This process is also called a conditioning regimen.  
Measure how well your disease (acute myeloid leukemia or myelodysplastic syndrome)
responds to the treatment.  
Combinations of chemotherapy and sometimes radiation are used as a treatment to destroy
cancer cells and help donor cells start to grow in your bone marrow. Depending on the
combination used, each treatment (or conditioning regimen) can have a different intensity or
strength.  
High intensity treatment uses high doses of chemotherapy or radiation.
Reduced intensity treatment uses lower doses of chemotherapy or radiation.  
High intensity treatment uses high doses of chemotherapy or radiation.  
Reduced intensity treatment uses lower doses of chemotherapy or radiation.  
Both kinds of treatments are used by stem cell transplant doctors around the world and are not
experimental. Our goal is to see if one kind of treatment is better than the other for people who
have a stem cell transplant to treat either their acute myeloid leukemia (AML) or
myelodysplastic syndromes (MDS).  
If you volunteer to join this study, we will randomly assign you to receive either a high intensity
or a reduced intensity treatment before you receive the stem cells from your donor.  
We believe this study will last about 18 months for most patients who decide to join. About 356
patients will take part in the study at transplant centers around the United States. We will
explain the two different treatments in this consent form. Every participating clinic will report
their results, SO we can compare and share the results at the end of the study.  
This consent form tells you about the study, its possible risks and benefits, other options
available to you, and your rights as a participant in the study. Please take your time to make
your decision.  
Everyone who takes part in research at [insert facility name] should know that:  
Being in any research study is voluntary.
You may or may not benefit from being in the study. Knowledge we gain from this study
may benefit others.
If you join the study, you can quit the study at any time.
If you decide to quit the study, it will not affect your care at [insert name of. facility or
institution].  
Being in any research study is voluntary.  
You may or may not benefit from being in the study. Knowledge we gain from this study
may benefit others.  
If you join the study, you can quit the study at any time.  
If you decide to quit the study, it will not affect your care at [insert name of. facility or
institution].  
B-3

studies show that immune cells given during the transplant can help destroy remaining cancer
cells. Transplants with reduced intensity conditioning (RIC) regimens are often used for people
who cannot have high doses of chemotherapy drugs or radiation because of their age or other
medical problems.  
This study will compare high intensity and reduced intensity treatments used to destroy cancer
cells and prepare bone marrow for transplant. Our goal is to see if one kind of treatment is better
than the other for people who have a stem cell transplant to treat either their AML or MDS.

You are invited to join this research study because you have AML or MDS and are currently
being evaluated for an allogeneic transplant. The main goal of this study is to see if patients with
AML or MDS have better results with transplants using reduced intensity treatment compared to
high intensity treatment.

Taking part in this study may or may not make your health better compared to receiving the
transplant through your routine medical care. We do know that the information from this study
will help doctors learn more about selection of conditioning regimen intensities. This
information could help patients in the future who are in need of an allogeneic transplant.

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.  
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  
B-23
Clinical trial: NCT01339910
Section: Voluntary Participation
Subsections: Introduction|Other Treatment Choices

Please ask the study staff questions about anything that you do not understand, or if you
would like to have more information.
You can ask questions now or any time during the study.
Please take the time you need to talk about the study with your doctor, study staff, and
your family and friends. It is your decision to be in the study. If you decide to take part,
please sign and date the end of the Consent Form.  
Please ask the study staff questions about anything that you do not understand, or if you
would like to have more information.  
You can ask questions now or any time during the study.  
Please take the time you need to talk about the study with your doctor, study staff, and
your family and friends. It is your decision to be in the study. If you decide to take part,
please sign and date the end of the Consent Form.  
You and your doctor will discuss other treatment choices if you do not want to participate
in this study.

It is your choice to join this study. If you decide you do not want to participate, you may still
receive a transplant for treatment of your disease. It is possible that you may have a treatment
and evaluations that are very similar to what would be if you joined this study.  
Your study doctor will discuss these choices with you. If you decide you do not want to join this
research study, your medical care will not be affected in any way.  
B-8
Clinical trial: NCT01339910
Section: Withdrawal from Study
Subsections: What if I change my mind?|New Information Available During the Study|Ending Your Participation

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,  
B-16

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.

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.  
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.