Aplastic anemia is a difficult to treat diseae. Those under the age of 50, have an option of a stem cell translantation, but for older patients, the only effective current treatment to prevent the complications of underproducing blood cells, is immunosupressive therapy. A recently approved drug for Idiopathic Throbocytopenic purpura(ITP) can potentially raise platelet counts in aplastic anemia patients.
A recent phase II trial included 25 patients, who receive Promacta with a slowly raised dose. Nine patients acieved a platelet rise of 20,000 cells/mm3 or more above baseline or independence from platelet transfusions for at least 8 weeks if previously transfusion-dependent. Four patients had a neutrophil response. Eight maintained a response without relapse for a median of 10 months. and six had a response in all three lineages.
These are promising results but the exchange of letters that followed the publication of this study drew attention to the potential risk of inducing progression to myelodysplastic syndromes or acute myeloid leukemia needs to be conisdered in patients with aplastic anemia. There are reports of progression to acute myeloid leukemia in patients with myelodysplastic syndromes who are treated with a similar drug and the researchers who published this study cautioned that treatment should be limited to clinical trials at this time.
There is an active clinical trial: Eltrombopag for Moderate Aplastic Anemia. NCT01328587
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