Use of eltrombopag in aplastic anemia in Europe

Abstract

Eltrombopag (ELT), an oral thrombopoietin receptor agonist, has recently emerged as a promising new drug for the treatment of aplastic anemia (AA). How ELT is used outside of clinical trials in the real-world setting and results of this treatment are not known. We conducted therefore a retrospective survey on the use of ELT in AA among EBMT member centers. We analyzed the 134 patients reported in our survey together with 46 patients recently published by Lengline et al. The median follow-up from start of ELT treatment was 15.3 months, with 85.6% patients alive at last follow-up. Importantly, only 28.9% of our patients received ELT according to the FDA/EMA label as monotherapy in the relapsed/refractory setting, whereas 16.7% received ELT upfront. The overall response rate in our cohort was 62%, very similar to the results of the pivotal ELT trial. In multivariate analysis, combination therapy with ELT/cyclosporine/ATG and response to previous therapy were associated with response. Overall survival was favorable with a 1-year survival from ELT start of 87.4%. We identified age, AA severity before ELT start and response to ELT as variables significantly associated with OS. Two patients transformed to MDS; other adverse events were mostly benign. In sum, ELT is used widely in Europe to treat AA patients, mostly in the relapsed/refractory setting. Response to

Eltrombopag is effective in patients with relapse/refractory aplastic anemia—report from a single center in China

“…Since then, clinical trials from different countries have confirmed the promising value of eltrombopag in AA of various severities [8][9][10][11]. Recently, real-world experience of eltrombopag on patients with AA has been described in Europe with an overall survival rate of 62% [12]. It has been shown that patients from Asian countries have different pharmacokinetics of eltrombopag; however, very few data have been published 13 .…”

Correlation of the Plasma Concentration of Eltrombopag With Efficacy in the Treatment of Refractory Aplastic Anemia: A Single-Centre Study in China

“…were excluded. Some patients refused to sign the consent form for blood testing, especially those with a poor response, which may partly explain the relatively high response rate (70.5%) in our cohort compared to that in the literature (Olnes et al, 2012;Desmond et al, 2014;Lengline et al, 2018;Ecsedi et al, 2019). Although the response rate was calculated at the time when blood was obtained to determine the ELT concentration, which may have been too short for an efficacy evaluation, most of the patients had already been exposed to ELT for more than 3 months in our cohort.…”

“…Elevated transaminase is the most common dose-dependent toxic effect of ELT (Ecsedi et al, 2019), according to published data. Other side effects mentioned in the literature include skin rash, elevated bilirubin, skin pigmentation, nausea, fatigue, cough, diarrhea, headache, and possible clone evolution (Lum and Grainger, 2016;Scheinberg, 2019).…”

Effective treatment with tacrolimus for patients with non-severe aplastic anemia refractory/ intolerant to cyclosporine A: a retrospective study

“…Although intensive immunosuppressive therapy with ATG, or even HSCT can further improve the response rate, these treatments are relatively expensive, risky, sometimes unavailable, and not suitable to old and weak patients [15]. Eltrombopag, an oral thrombopoietin receptor agonist [16], launched in China recently, has emerged as a promising new drug for the treatment of aplastic anemia [17,18]. But the price is too high for the regular use for most of the patients.…”