(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, MD, PhD, 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 50mg daily eltrombopag and, if necessary, the dose was increased up to a maximum of 150mg daily, for 12 weeks.

At 12 weeks, the researchers found that 11 of the patients (44%) had a hematologic response in at least 1 lineage, with minimal toxic effects. Platelet transfusions were no longer needed in 9 patients. Hemoglobin levels improved (median increase, 4.4g/dl) in 6 patients, including 3 who were previously dependent on red-cell transfusions and no longer needed transfusions. Neutrophil counts increased in 9 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.

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“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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