Eltrombopag safety and efficacy for primary chronic immune thrombocytopenia in clinical practice

Abstract

Background

Eltrombopag is effective and safe in chronic immune thrombocytopenia (ITP). However, clinical trials may not accurately reflect what happens in clinical practice. We evaluated the efficacy and safety of eltrombopag in primary chronic ITP in a real-world setting.

Methods

164 primary chronic ITP patients from 40 Spanish centers, who had been treated with eltrombopag, were retrospectively evaluated.

Results

The median age of our cohort (72% women) was 63 years (interquartile range, IQR, 45–75 years). The median time with ITP diagnosis was 81 months (IQR, 30–192 months). The median number of therapies prior to eltrombopag was 3 (IQR, 2–4). At the time of eltrombopag start, 45 patients (30%) were receiving concomitant treatment for ITP. Forty-six patients (30%) had bleeding signs/symptoms the month before treatment start. The median platelet count at eltrombopag initiation was 22 x 109/L (IQR, 8-39 x 109/L). 135 patients (88.8%) achieved a platelet response. The median time to platelet response was 12 days (95% CI, 9-13 days). Maintained platelet response rate during the 15-month period under examination was 75.2%. 28 patients (18.4%) experienced adverse events, mainly grades 1–2.

Conclusion

Eltrombopag is highly effective and well tolerated in unselected patients with primary chronic ITP.

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