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NIH clinical trials on MDS

Clinical research—to advance diagnostics and to develop better treatments—requires patients, but one big challenge in a rare disease such as is patient recruitment. If patients don’t agree to be “on protocol”, or if only a few enroll, clinical trials fail or take years to reach full accrual. Patients are often surprised to learn that unless they are treated on a research trial, data concerning their outcome is not utilized for research purposes. Also often not appreciated is that even experts with huge clinics rely on carefully conducted and interpreted trials to draw conclusions that are applicable to their own patients. Theoretical assumptions, animal experiments, and anecdotal clinical experiences can help in formulating research and designing a trial, but they do not replace actual clinical results. One recent example in aplastic anemia was our NIH trial, in which Phillip Scheinberg was the principal investigator, that surprisingly showed a major difference in favor of horse anti-thymocyte globulin (ATG) compared to rabbit ATG.

Patients on protocols often do better than those who receive standard treatments or worse, combinations of treatments devised by an inexperienced doctor. Research usually is carried out in hospitals with large experience in , which is very valuable in severe disease. Research protocols also provide an opportunity to receive a novel, potentially beneficial therapy. Clinical trials are intended to “advance the field”, and even when not successful they are highly informative to future patients and their doctors as to the choices and their risks and benefits. Participating in research empowers patients and can give them satisfaction in contributing to new developments that may help them but surely will help others.

Recently, our institution completed a pilot study using a novel agent for aplastic anemia called eltrombopag (also known as Promacta®). Eltrombopag is a drug designed to mimic a special protein in the body called thrombopoietin (TPO). Like naturally occurring TPO, eltrombopag causes the body to make more platelets. Eltrombopag has been approved by the U.S. Food and Drug Administration (FDA) to treat low blood counts in adults with chronic ITP (immune thrombocytopenic purpura). Eltrombopag may also cause the body to make more red and white blood cells.

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Popular Q&A

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Lymphoma and Clinical Trials - What You Need to Know.?

Cancer research is developing rapidly. New questions need to be answered, and new treatments need to be tested. A clinical trial is a controlled way of finding out whether a new treatment works better than old ones, or whether a paticular way of treating is of any good.

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