Adding Eltrombopag to Standard Care in Severe or Very Severe Aplastic Anemia

The addition of eltrombopag resulted in higher rates of complete hematological responses at 3 months.

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/cyclosporin…

Reviewing Author

Brady L. Stein, MD, MHS

Disclosures

Leadership Positions in Professional SocietiesAmerican Society of Hematology (Senior Editor, ASH Self-Assessment Program; guideline development)

Brady L. Stein, MD, MHS

Disclosures

Leadership Positions in Professional SocietiesAmerican Society of Hematology (Senior Editor, ASH Self-Assessment Program; guideline development)

Adding Eltrombopag to Standard Care in Severe or Very Severe Aplastic Anemia

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Reviewing Author