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clinical trial

Platelet drug shows clinical benefits for severe, unresponsive aplastic anemia


What is Eltrombopag?

Eltrombopag is a chemical designed to bind to and activate the receptor for thrombopoietin, a cytokine produced by the body essential for production of platelets and it now appears also hematopoietic stem cells. Eltrombopag can be given orally and has been approved by the FDA for treating chronic immune thrombocytopenia (ITP)

How does it work?

It appears to stimulate hematopoietic stem cells to multiply, as well as stimulate increased production of platelets from platelet precursor cells.

How is it given and for how long?

It is given orally as a pill once or twice a day. Some patients with ITP have been treated with the drug for years. In our aplastic anemia trial several patients have been on the drug for over two years.

Is Eltrombopag being used for both children and adults?

In ITP, trials have been carried out in children, but it is not yet FDA-approved for children. The aplastic anemia trial reported in New England Journal of Medicine included only adults. However, we are designing a new trial for patients with refractory aplastic anemia that includes children. We also currently have a trial open for patients with new onset aplastic anemia combining eltrombopag with standard ATG and cyclosporin treatment, and this trial enrolls children.

How long before patients see response?

Most patients did not begin to respond until after 12 weeks of treatment, and did not reach their maximum response for a year or more.

What if there is no response? Are patients still able to try other therapies?

As far as we know there are no reasons that patients would not be able to try other therapies. Some patients that failed eltrombopag treatment on our trial went on successfully to have allogeneic stem cell transplantation.

Are there any side effects?

Very few. At very high doses some patients can become jaundiced and in some patients there is liver inflammation that necessitates lowering the dosage.

Are you still enrolling patients for any trials with Eltrombopag?

Yes, we are carrying out several trials at present. We have one for previously untreated moderate aplastic anemia in adults, another for refractory aplastic anemia in adults (an extension of the published study), and a trial for combining eltrombopag with ATG and cyclosporine in children and adults with previously-untreated aplastic anemia.

One of the trials is also for patients with low- or intermediate-risk MDS.

Do you have a timeline for the Phase 3 clinical trial period and how soon Eltrombopag could be possibly be approved for treatment of aplastic anemia?

It is difficult to predict. We hope that given the long-term safety data from the ITP trials, and the rarity of aplastic anemia, approval may be hastened.

The complete citation for the article is:
Eltrombopag and Improved Hematopoiesis in Refractory Aplastic Anemia

Matthew J. Olnes, M.D., Ph.D., Phillip Scheinberg, M.D., Katherine R. Calvo, M.D., Ronan Desmond, M.D., Yong Tang, M.D., Ph.D., Bogdan Dumitriu, M.D., Ankur R. Parikh, M.D., Susan Soto, B.S.N., Angelique Biancotto, Ph.D., Xingmin Feng, M.D., Ph.D., Jay Lozier, M.D., Ph.D., Colin O. Wu, Ph.D., Neal S. Young, M.D., and Cynthia E. Dunbar, M.D.

N Engl J Med 2012; 367:11-19 July 5, 2012

Learn more about the 20 active studies recruiting for eltrombopag at

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Clinical Trials In Depth

There is a lot you should know if you are considering a clinical trial. There are many types of trials. And there are three phases of a clinical trial. There are certain rights you have as a clinical trial participant, along with many safeguards to protect you and your health. This section gives you information to help you decide whether a clinical trial is right for you.

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