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Eltrombopag Added to Standard Immunosuppressive Therapy Improves Response in Severe Aplastic Anemia

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The addition of eltrombopag to standard immunosuppressive therapy led to significant increases in blood counts in almost 50% of treatment-naïve patients with severe aplastic anemia enrolled in a phase 1-2 study. The study included 92 consecutive patients who were consecutively enrolled in one of three cohorts that differed with regard to the timing and duration of eltrombopag therapy: cohort 1,  eltrombopag administered from day 14 to 6 months; cohort 2, eltrombopag administered from day 14 to 3 months; and cohort 3, eltrombopag administered from day 1 to 6 months.

At 6 months, the complete response rate and overall response rate were as follows: cohort 1, 33% and 80%; cohort 2, 26% and 87%; and cohort 3, 58% and 94%, respectively. At a median follow-up of 2 years, the survival rate was 97%, with 1 patient dying from a nonhematologic cause. Marked increases in bone marrow cellularity, CD34+ cell number, and frequency of early hematopoietic progenitors were observed. Two patients had severe rashes, which resulted in early discontinuation of eltrombopag.

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In May 2018, the FDA granted eltrombopag (Promacta) priority review for severe aplastic anemia based on a 52% complete response rate and 85% overall response rate when the agent was added to standard immunosuppressive therapy,

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Townsley DM, Scheinberg P, Winkler T, et al. Eltrombopag added to standard immunosuppression for aplastic anemia. N Engl J Med. 2017;376(16):1540-1550. doi: 10.1056/NEJMoa1613878.
Novartis. FDA expedites review of Novartis drug Promacta® for first-line severe aplastic anemia (SAA) [press release]. Released May 30, 2018. Accessed September 24, 2018.

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