A NEW STANDARD IMMUNOSUPPRESSION REGIMEN IN SEVERE APLASTIC ANEMIA
Hypocellular marrow and pancytopenia are the defining characteristics of the bone marrow failure syndromes, of which aplastic anemia is the prototype. Severe aplastic anemia is fatal if left untreated.
The results of a trial supported by the National Institutes of Health (NIH) and published in the Journal in 2012 showed that eltrombopag, a thrombopoietin-receptor agonist developed for the treatment of patients with chronic immune thrombocytopenia, was successful in treating 44% of patients with severe aplastic anemia who had not responded to the initial immunosuppression.
Eltrombopag was initiated on day 14 at a dose of 150 mg per day, and treatment was continued through 6 months, or through 3 months in patients who had a complete response at 3 months. The experimental therapy was superior to the standard therapy with respect to the primary end point.
Horse ATG-cyclosporine plus eltrombopag was superior to standard therapy at all significant time points and resulted in a higher and more rapid complete and overall hematologic response, which led to earlier transfusion independence, fewer pancytopenia-related complications, and improved quality of life.