Eltrombopag Linked to Clinical Response in Aplastic Anemia

Clinically significant multilineage response in some patients with severe refractory aplastic anemia

THURSDAY, July 5 (HealthDay News) -- For some patients with severe refractory aplastic anemia, treatment with eltrombopag is associated with clinically significant improvements in platelet, erythroid, and neutrophil lineages at 12 weeks, according to a study published in the July 5 issue of the New England Journal of Medicine.

Matthew J. Olnes, M.D., Ph.D., from the National Institutes of Health in Bethesda, Md., and colleagues conducted a phase 2 study involving 25 patients with immunosuppression-refractory aplastic anemia. Patients received 50 mg daily eltrombopag and, if necessary, the dose was increased up to a maximum of 150 mg daily, for 12 weeks.

At 12 weeks, the researchers found that 11 of the patients (44 percent) had a hematologic response in at least one lineage, with minimal toxic effects. Platelet transfusions were no longer needed in nine patients. Hemoglobin levels improved (median increase, 4.4 g per deciliter) in six patients, including three who were previously dependent on red-cell transfusions and no longer needed transfusions. Neutrophil counts increased in nine patients (median increase, 1,350 per mm³). Normalization of trilineage hematopoiesis was seen in serial bone marrow biopsies in patients who had a response, without increased fibrosis. There were no consistent changes in immune function.

"Treatment with eltrombopag was associated with multilineage clinical responses in some patients with refractory severe aplastic anemia," the authors write.

GlaxoSmithKline supplied the eltrombopag for the trial.

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