Drug May Aid Patients With Rare Anemia

Eltrombopag shows benefit in patients with aplastic anemia

/ Author:  / Reviewed by: Joseph V. Madia, MD

(RxWiki News) Patients with a severe form of aplastic anemia may not see a benefit with traditional therapies. However, a platelet-enhancing drug could be an option for individuals with the rare blood disorder.

Promacta (eltrombopag) appears to improve blood clotting and increase blood cell levels in aplastic anemia patients, according to a recent small clinical trial suggesting the therapy could act as a second-line treatment.

"Discuss treatment options for severe anemia with a hematologist."

Aplastic anemia affects about 600 new patientsin the U.S. each year and occurs when the body's bone marrow does not produce enough new blood cells.This includes white blood cells to fight infection, red blood cells to carry oxygen and platelets which aid in the process of clotting blood.

Matthew J. Olnes, MD, PhD, from the National Institutes of Health, found that a significant portion of participants in the phase 2 study no longer needed the platelet transfusions that they had previously required.

During the study, researchers followed 25 patients with severe aplastic anemia who took 50 milligrams of eltrombopag daily, though the amount could be increased to a maximum of 150 milligrams if needed, for a period of 12 weeks.

The study sample was small because the blood disorder is uncommon, providing a smaller pool of potential participants. Results were tracked through bone marrow biopsies and other lab work.

Of the patients, 11 demonstrated improved production of at least one blood cell (red, white or platelet) after 12 weeks of taking the oral medication. Hemoglobin levels improved in six participants, while nine patients experienced increased neutrophil counts, the most abundant type of white blood cells in the body. Also, nine patients previously dependent on red blood cell transfusions no longer needed them following the therapy.

Following the initial 12 weeks, seven patients continued the treatment between 8 and 32 months. Six of those participants showed improvement in production of all three types of blood cells and no longer needed transfusions.

The study, funded by the National Institutes of Health, was recently published in the New England Journal of Medicine.

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Review Date: 
July 9, 2012
Last Updated:
July 10, 2012