Platelet drug shows clinical benefits for severe, unresponsive aplastic anemia

July 4, 2012

Eltrombopag, a drug that was designed to stimulate production of platelets from the bone marrow and thereby improve blood clotting, can raise blood cell levels in some people with severe aplastic anemia who have failed all standard therapies.

About one-third of aplastic anemia cases do not respond to standard therapy, a combination of immune-suppressing drugs. Although is an option for some, patients without a matched donor have few treatment options. The findings of this new clinical study, carried out by the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health, suggest eltrombopag could be a second-line for them.

"Eltrombopag and Improved Hematopoiesis in Refractory Aplastic Anemia," will be published online July 5 in the .

Aplastic anemia is a rare blood disorder, with about 600 new cases in the U.S. each year. Aplastic anemia results from the destruction of bone marrow stem cells, which mature into that carry oxygen, that fight infection, and platelets that prevent excess bleeding. Symptoms of the disorder include fatigue, frequent infections, and hemorrhaging. In severe cases unresponsive to treatment, death can occur.

Eleven of 25 participants enrolled in this phase 2 study showed improved production of at least one type of blood cell (red blood cell, white blood cell, or platelet) after 12 weeks of oral eltrombopag therapy. Among the seven volunteers who continued taking the pills long-term (8-32 months), six eventually showed an improvement in all three types of blood cells, and were able to maintain safe blood counts without needing red blood cell or platelet transfusions. Overall the drug was well tolerated, with few side effects.

The research team in the NHLBI Hematology Branch tested eltrombopag because this drug had previously been shown to boost platelet levels in both healthy people and people with reduced platelets due to hepatitis C infection or immune thrombocytopenia, blood disorders that like aplastic anemia result in low platelet counts and increased risk of bleeding.

The encouraging finding in this study was improvement in red blood cell and white cell counts in some aplastic anemia patients, suggesting that the drug can stimulate bone marrow stem cells and perhaps have wider utility than initially predicted.

Explore further: Standard aplastic anemia therapy improves patient outcomes better than newer version

More information: Learn more about this study at:
Learn more about aplastic anemia at:

Related Stories

Recommended for you

Monkeys in Asia harbor virus from humans, other species

November 19, 2015

When it comes to spreading viruses, bats are thought to be among the worst. Now a new study of nearly 900 nonhuman primates in Bangladesh and Cambodia shows that macaques harbor more diverse astroviruses, which can cause ...

One-step test for hepatitis C virus infection developed

November 14, 2015

UC Irvine Health researchers have developed a cost-effective one-step test that screens, detects and confirms hepatitis C virus (HCV) infections. Dr. Ke-Qin Hu, director of hepatology services, will present findings at the ...

Computer model reveals deadly route of Ebola outbreak

November 10, 2015

Using a novel statistical model, a research team led by Columbia University's Mailman School of Public Health mapped the spread of the 2014-2015 Ebola outbreak in Sierra Leone, providing the most detailed picture to date ...


Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.