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Repeated short‐term use of eltrombopag in patients with chronic immune thrombocytopenia (ITP)
Author(s) -
Bussel James B.,
Saleh Mansoor N.,
Vasey Sandra Y.,
Mayer Bhabita,
Arning Michael,
Stone Nicole L.
Publication year - 2013
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.12169
Subject(s) - eltrombopag , medicine , clinical endpoint , adverse effect , gastroenterology , platelet , thrombopoietin , dosing , immune thrombocytopenia , thrombopoietin receptor , randomized controlled trial , genetics , stem cell , haematopoiesis , biology
Summary 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 × 10 9 /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.