About blood and marrow transplant research

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Learn about research and clinical trials related to your child's blood and marrow transplant.

Key points

  • Research studies and clinical trials are conducted to improve on and find better ways of preventing, diagnosing and treating specific medical conditions.
  • If your child is asked to participate in a trial, make sure you understand what will be involved before giving consent.
  • Choosing not to participate or withdrawing from the study will not impact the care your child receives.

You may be asked to allow your child to participate in a research project that is being run on children undergoing a blood and marrow transplant (BMT).

If you are asked to take part in a study, you may listen to the project description and decide if you want to participate. When making a decision about whether or not to participate, make sure that the researcher:

  • answers all of your questions to your satisfaction
  • explains the reasons for the study and what they hope to accomplish
  • explains what is expected of your child and your family

If you feel all your questions are answered and you consent to having your child participate in the research project, you will be asked to sign a consent form. A copy of this form will be handed to you for your records and will be part of your child's hospital chart. You may withdraw your child from the project at any time. If you withdraw or choose not to participate, it will not affect the care your child receives.

What are the different kinds of research?

A lot of research is conducted to learn more about diseases that only happen in children or that have a different course in children than in adults. Doctors, nurses, and other health professionals also study children’s mental and physical development in research that can ultimately benefit all children.

Different types of research studies include:

  • “chart reviews,” which look at children’s existing hospital records to study the frequency or course of a disease, to look at diagnostic methods, or to assist in planning a research study of future patients
  • studies that involve collecting additional samples (for example, blood, bone marrow cells, or CSF fluid). These samples are usually only collected if they are part of procedures during normal treatment
  • studies that involve talking to patients or their parents and using questionnaires, interviews, or information collected to study disease outcomes, behaviour, opinions, quality of life, or patient satisfaction
  • studies that involve direct contact with study participants. These studies may include physical examinations, imaging, and specimen collection to study diseases or diagnostic methods
  • intervention studies, which look at a therapy such as a drug, natural health product, medical device, surgery, or behavioural therapy. Intervention studies include clinical trials.

What is a clinical trial?

A clinical trial is a research study or scientific experiment that examines better ways to use drugs or other therapies in treatment. It asks a specific question to try and find better ways of preventing, diagnosing, or treating different medical conditions. For example, clinical trials could study new drugs or new combinations of known drugs. Usually, there are four different phases of clinical trials. The most “experimental” stage is Phase I, which is used to determine the safety of a drug or medical procedure. By the time a treatment reaches a phase III clinical trial, it has been studied well. When a phase III trial is conducted under an organizing group of experts, it may also be called a protocol.


Randomization is used in studies that involve two or more groups. Each group gets a different treatment and patients have to be placed in one of the groups. Randomization means that the patients are placed in a group by chance. This makes sure the results are not biased. A bias may be possible if a person decides who goes in which group. Usually a computer will assign a patient to a group. Most research protocols need randomization at diagnosis or shortly after diagnosis

Why is research important?

Research is necessary to improve the success of treatments. As an example of the dramatic impact of research, consider the case of childhood leukemia. In the early 1960s, when children were diagnosed with leukemia, there was hardly any chance that they would live. Today, for some types of leukemia, the vast majority of children will survive the disease. This success has been achieved as the result of research. This research took place in the form of clinical trials. Each time a trial showed an improvement with a new type of treatment, this new treatment could benefit more children.

After a clinical trial ends

After each clinical trial, the researchers will write about their findings. They will describe the procedure, the participants, treatment results, side effects, and their understanding of the results. The researchers will send their report to a scientific journal. Other experts who are not connected to the study will read and analyze this report. This is called peer review. If these experts agree that the trial was of high quality, the report will be published.

After reading the report, other researchers in different cities and countries should be able to repeat similar trials to see if they get the same results. Larger and larger numbers of patients will participate.

When a number of trials show that a drug or treatment is effective, it is approved by a national regulatory body, such as Health Canada, the U.S. Food and Drug Administration, or the Medicines Control Agency in the United Kingdom. Usually, it is first approved for a very particular disease and situation. Later trials help pave the way for the drug to be approved for use with different diseases or age groups.

Research and treatment protocols

A formal protocol is a written plan that describes in detail how to treat a particular illness. It describes the drugs, dosages, and tests that are needed. It tells the treatment team how often and when each drug should be given based on a schedule. Each protocol is based on research that shows which treatments are expected to be effective.

Experts from many hospitals work together to develop protocols. The results from each hospital are gathered and studied. Researchers have developed international groups to oversee and coordinate these efforts. Protocols are closely monitored during their use and can change if new research shows there are other drugs or combinations of drugs that are more effective.

Last updated: March 5th 2010