Prolonged remission from eltrombopag in chronic refractory idiopathic thrombocytopenic purpura

Abstract

The therapy of ITP has recently been revolutionized with the introduction of thrombopoeitin stimulating agents. However, these medications are known to increase the platelet count only while the medication is being administered, with a rapid fall of the platelet count to baseline pre-therapy levels on discontinuation. We report the case of a patient with chronic refractory ITP who has attained a prolonged remission after a short course of eltrombopag, with normalization of the platelet count, which is sustained 8 months after discontinuation of the medication.

Tapering thrombopoietin receptor agonists in primary immune thrombocytopenia: Expert consensus based on the RAND/UCLA modified Delphi panel method

“…While it was previously thought that patients with ITP would have to remain on TPO-RAs indefinitely to maintain adequate platelet counts, recent case reports and cohort studies have shown that selected patients with ITP can safely discontinue treatment. [7][8][9][10][11][12][13][14][15][16][17][26][27][28][29][30][31][32][33][34][35][36][37][38][39] However, to date, no formal guidelines identify which patients can successfully discontinue TPO-RAs. 4,20 In response to this need, we developed consensus statements on when it is appropriate to consider tapering TPO-RAs; broad guidance on how to taper patients off therapy, how to monitor patients after discontinuation, and how to restart therapy in the event of relapse is also provided.…”

“…No human subjects were involved in this research; thus, ethics committee approval was not required. [26][27][28][29][30][31][32][33][34][35][36][37] 11 cohort studies, [7][8][9][10][11][12][13][14][15][16][17] and two analyses of pooled clinical trial data 38,39 on sustained remission in patients with ITP after discontinuation of TPO-RAs. We did not formally appraise the quality of evidence using a standard tool.…”

TPO‐RAs in pITP: description of a case series and analysis of predictive factors for response

“…In the last few years, some reports have suggested the possibility of maintaining the response after discontinuation of either romiplostim or eltrombopag, but the suspension criteria adopted by each single center have not been homogeneous (11,18,(26)(27)(28)(29)(30)(31)(32)(33)(34)(35)(36)(37)(38)(39)(40)(41)(42). Seven retrospective series of persisting response have been published by different authors (35,(37)(38)(39)(40)(41)(42).…”

“…In the last few years, some reports have suggested the possibility of maintaining the response, without other concomitant or rescue therapies, after discontinuation of either romiplostim or eltrombopag in 3-39% and 4-33% of patients, respectively (11,18,(26)(27)(28)(29)(30)(31)(32)(33)(34)(35)(36)(37)(38)(39)(40)(41)(42). These observations generated hypotheses about the mechanisms of action of the TPO-RAs.…”

Efficacy and safety of eltrombopag in the treatment of severe chronic immune thrombocytopenia in children of China: A single-center observational study

“…3,4 Continuous long-term treatment with eltrombopag for up to 3 years was reported to be safe, and the drug maintained its efficacy when used at a stable dose. 5,6 Two international, randomized, double-blind, placebo-controlled trials of eltrombopag in pediatric patients with CITP demonstrated that the platelet response rate was 36%–44%. 7,8 However, the safety of eltrombopag in children of China with severe ITP is largely unknown.…”