Among patients with previously untreated severe aplastic anemia, adding eltrombopag to standard immunosuppression therapy appears to be both safe and effective for inducing a hematologic response, according to research published in The New England Journal of Medicine.

Acquired severe aplastic anemia is characterized by primary bone marrow failure and pancytopenia. Therapies introduced over the past 50 years, including antithymocyte globulin (ATG) and cyclosporine, which now constitute standard immunosuppression treatment, improved both hematopoietic recovery and survival.

Some patients, however, are refractory to immunosuppressive therapy, and there are limited treatment options in this population. For this open-label, randomized phase 3 trial, researchers evaluated whether adding eltrombopag, an oral thrombopoietin-receptor agonist, to standard first-line therapy improved outcomes among patients with severe or very severe aplastic anemia.

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Overall, 197 patients were enrolled and randomly assigned to group A (horse ATG and cyclosporine; 101 patients) or group B (horse ATG and cyclosporine plus eltrombopag; 96 patients). In the overall study population, the median age was 53 years, 55% of patients were male sex, and 34% of patients had very severe anemia.

The overall median follow-up was 24 months. In groups A and B, the 3-month complete response rate was 10% and 22%, respectively (odds ratio, 3.2; P =.01). The 6-month overall response rate was 41% vs 68% in group A vs B, respectively, and the median time to first response was 8.8 vs 3 months.

The event-free survival rate after the follow-up period was 34% in group A vs 46% in group B. Although the percentage of patients with somatic mutations increased in both groups over the study period, no effect on the hematologic response was noted at end of follow-up. The authors noted that adverse events occurred at a similar rate in the 2 groups.

“This prospective randomized trial showed that the addition of eltrombopag to horse ATG plus cyclosporine, as compared with horse ATG plus cyclosporine alone, was beneficial in patients with severe aplastic anemia,” the authors wrote. “The addition of eltrombopag induced a response that was of higher quality and occurred faster without increasing toxic effects.”

Disclosure: The study author(s) declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of authors’ disclosures. 


Peffault de Latour R, Kulasekararaj A, Iacobelli S, et al. Eltrombopag added to immunosuppression in severe aplastic anemia. N Engl J Med. 2022;386(1):11-23. doi:10.1056/NEJMoa2109965