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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.

Read more here.

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,

Read more here.

References

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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