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Adding Eltrombopag to Standard Care in Severe or Very Severe Aplastic Anemia
Standard treatment for severe or very severe aplastic anemia includes horse antithymocyte globulin (ATG) with cyclosporine. Unfortunately, over the last several decades there have not been any improvements to this standard of care, despite efforts with other agents. Eltrombopag, an oral thrombopoietin-receptor agonist, was found to be effective as a single agent in refractory aplastic anemia in a phase 2 trial (NEJM JW Oncol Hematol Sep 2012 and N Engl J Med 2012; 367:11), and when added to ATG/cyclosporine in previously untreated severe disease in a nonrandomized phase 1/2 trial (NEJM JW Oncol Hematol Jun 2017 and N Engl J Med 2017; 376:1540).
Now, investigators report a randomized, phase 3, multicenter study comparing horse ATG/cyclosporine with or without eltrombopag in 197 previously untreated patients with a confirmed diagnosis of severe or very severe aplastic anemia. The primary endpoint was a complete hematological response at 3 months (hemoglobin >10g/dL, absolute neutrophil count >1.0 x 109/L, platelet count >100 x 109/L).
At 3 months, the rate of complete hematological response was 22% in patients treated with eltrombopag, compared to 10% in those treated without eltrombopag (P=0.01); the overall response (complete or partial) rate was 59% versus 31%, respectively. Median time to first response was 3.0 months with eltrombopag versus 8.8 months without it. Older age (≥40 years) and very severe disease were negative predictors of response.
Adverse event rates were similar in the two groups. At 6 months, new or additional somatic mutations were acquired in 39% and 53% of patients treated with and without eltrombopag, respectively.
This phase 3 study demonstrates improved hematological responses in patients with severe or very severe aplastic anemia treated with eltrombopag plus horse ATG/cyclosporine. Moreover, the time to response was shorter with the addition of eltrombopag. Despite the improved response, overall survival was statistically similar in the two groups. Of note, a sizeable proportion of patients in both groups acquired new somatic mutations, yet response rates were not negatively impacted by this finding.
Peffault de Latour R et al.
Title: Eltrombopag added to immunosuppression in severe aplastic anemia.
Source: N Engl J Med 2022 Jan 6; [e-pub]. (Abstract/FREE Full Text)