Repeated short-term use of eltrombopag in patients with chronic immune thrombocytopenia (ITP)

James B. Bussel, Mansoor N. Saleh, Sandra Y. Vasey, Bhabita Mayer, Michael Arning, Nicole L. Stone

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59 Citations (Scopus)


Eltrombopag is a thrombopoietin-receptor agonist that stimulates platelet production and increases platelet counts in patients with chronic immune thrombocytopenia (ITP). This open-label, single-arm study evaluated consistency of response and safety following repeated intermittent dosing of eltrombopag 50 mg daily over 3 cycles (1 cycle = up to 6 weeks on therapy followed by up to 4 weeks off therapy). The primary endpoint was proportion of patients with a response (platelet count ≥50 × 109/l and ≥2× baseline) in Cycle 1 who subsequently responded in Cycle 2 or 3. Fifty-two of 65 evaluable patients (80%) responded in Cycle 1; these responding patients comprised the primary analysis population. Of these, 45/52 (87%) responded in Cycle 2 or 3 [95% confidence interval (CI), 74-94%] and 34/48 (71%; 95% CI, 56-83%) responded in both Cycles 2 and 3. Time to response was consistent, with >50% of responders responding by Day 8 in each cycle. Bleeding rates relative to baseline decreased by approximately 50% during each treatment cycle. The frequency or severity of adverse events, most commonly headache, did not increase over successive cycles. If a chronic ITP patient not requiring consistent therapy responds to short-term eltrombopag, then subsequent courses of eltrombopag, as needed, are likely to be safe and effective.

Original languageEnglish
Pages (from-to)538-546
Number of pages9
JournalBritish Journal of Haematology
Issue number4
Publication statusPublished - Feb 2013


  • Autoimmunity
  • Bleeding
  • Intermittent dosing
  • Platelet
  • Safety


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