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Long-term eltrombopag demonstrated efficacy in restoring platelet counts and decreasing risk of bleeding in patients with chronic/persistent immune thrombocytopenia (ITP) lasting more than 6 months, according to a study published in Blood.1

Previous studies showed that eltrombopag treatment for 6 months or less effectively increased platelet counts and reduced bleeding among patients with chronic ITP. The purpose of this analysis was to evaluate the safety and efficacy of eltrombopag long term.

For the phase 3 EXTEND trial ( Identifier: NCT00351468), an extension study of 4 clinical trials for eltrombopag, researchers enrolled 302 patients with chronic ITP with a starting dose of eltrombopag 50 mg daily and titrated doses accordingly. Eligible patients must have had ITP lasting for 6 months or longer and received prior treatment for the disease. 

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The median duration of treatment was 2.37 years. Forty-five percent (135) of patients completed the study, and 25% (75) of patients continued treatment for 4 or more years.

Study patients experienced an increase in median platelet count to 50×109/L or greater by week 2. Approximately 86% of patients reached platelet counts of 50×109/L at least once in the absence of rescue treatment, and 52% of patients had a continuous response lasting 25 weeks or longer.

Lower rates of response were observed in patients with platelet levels lower than 15×109/L, patients who had received more previous therapies, and/or patients who underwent splenectomy.

After 1 year of treatment, bleeding symptoms decreased from 57% at baseline to 16%.

Reported adverse events that led to treatment discontinuation for 41 patients included hepatobiliary effects, cataracts, deep vein thrombosis, cerebral infarction, headache, and myelofibrosis.


  1. Wong RSM, Saleh MN, Khelif A, et al. Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood. 2017 October 17. doi: 10.1182/blood-2017-04-748707 [Epub ahead of print]