Efficacy and safety of eltrombopag in treatment-refractory primary immune thrombocytopenia

Abstract

Eltrombopag was used in patients with chronic primary immune thrombocytopenia (ITP) who did not tolerate or were refractory to two or more previous treatments. The primary aims of the study were to determine the efficacy and safety of long-term eltrombopag treatment. Data were extracted from medical chart records retrospectively. Platelet count of at least 50 000/μl at any time point during the treatment was defined as the 'response'. Median duration of eltrombopag treatment was 29 weeks (11-74). The number of patients who had a platelet count of at least 50 000/μl at any time point was 26 (83.9%). The response was achieved by the second week in most of the patients. Concomitant ITP medications were withdrawn in nine out of the 11 patients. Eltrombopag was discontinued in one patient due to sustained response despite discontinuation of the treatment. Age, sex, concomitant ITP treatments, and previous ITP treatment failures had no impact on the treatment response. The treatment was discontinued due to thrombosis in only four patients. Four patients experienced a minor bleeding event. Hepatotoxicity and all other adverse events were mild and manageable. Eltrombopag is effective, safe, and well tolerated in the long-term treatment of chronic ITP patients.

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

“…Articles that included patients with ITP, treatment with TPO‐RAs, and cessation of TPO‐RAs were reviewed and summarized. The summary included evidence from 12 case reports, 26‐37 11 cohort studies, 7‐17 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.…”

“…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.…”

“…5,6 However, some retrospective and prospective cohort studies have shown that 3%-33% of patients with ITP, including those treated early in their ITP course, may go into remission and maintain hemostatic platelet counts after tapering and discontinuing TPO-RAs (although studies differed in their definition of remission and follow-up duration). [7][8][9][10][11][12][13][14][15][16][17] Most of these cohort studies were not designed to assess the feasibility of TPO-RA discontinuation. The true prevalence of patients able to maintain a hemostatic platelet count off therapy is unknown, and while a gradual tapering to discontinuation has been advised to avoid rebound thrombocytopenia, 18,19 factors associated with successful discontinuation have not been identified.…”

Eltrombopag in immune thrombocytopenia: efficacy review and update on drug safety

“…Lastly, a Turkish group reported their experience with 31 chronic ITP patients who had been refractory to, at least, two previous treatments, in a retrospective study on their response to eltrombopag. 57 The median duration of treatment was 29 weeks. The response (i.e.…”

Thrombopoietin and its receptor expression in pediatric patients with chronic immune thrombocytopenia

“…Romiplostim generated sustained remission in cITP and pITP patients. In 2015, eltrombopag was also approved by the FDA for use in children >6 years of age with ITP who were not responsive to first line therapies [27][28][29][30][31][32]. The success of these therapies confirmed the role of TPO and its receptors in the pathogenesis of ITP.…”