Use of eltrombopag in aplastic anemia in Europe

• DOI: 10.1007/s00277-019-03652-8
• Publication Date: 2019-03-26T16:24:29Z
• Authors: Matyas Ecsedi; Étienne Lengline; Cora Knol-Bout; Paul Bosman; Dirk-Jan Eikema; Boris Afanasyev; Alexei Maschan; Peter Dreger; Constantijn J. M....; Beatrice Drexler; Agostino Cortelezzi; Bernard Drénou; Andrea Patriarca; Benedetto Bruno; Daniela Onofrillo; Edoardo Lanino; Drazen Pulanic; Ranka Serventi-Se...; Alice Garnier; Per Ljungman; Francesca Bonifazi; Sabrina Giammarco; Olivier Tournilhac; Pietro Pioltelli; Alicia Rovó; Antonio M. Risitano; Régis Peffault de...; Carlo Dufour; Jakob Passweg
• Keywords: Adult; Male; 610 Medicine & health; Kaplan-Meier Estimate; Benzoates; Young Adult; 03 medical and health sciences; 0302 clinical medicine; Receptors; Humans; Aged; Proportional Hazards Models; Retrospective Studies; Aplastic; Anemia, Aplastic; [SDV.MHEP.HEM]Life Sciences [q-bio]/Human health and pathology/Hematology; Anemia; Middle Aged; Drug Utilization; Aplastic anemia; Eltrombopag; Growth factors; Immunosuppression; Adult; Aged; Anemia, Aplastic; Benzoates; Drug Evaluation; Drug Utilization; Europe; Female; Follow-Up Studies; Humans; Hydrazines; Kaplan-Meier Estimate; Male; Middle Aged; Myelodysplastic Syndromes; Proportional Hazards Models; Pyrazoles; Receptors, Thrombopoietin; Retrospective Studies; Young Adult; 3. Good health; Europe; Hydrazines; Thrombopoietin; Myelodysplastic Syndromes; Eltrombopag; Drug Evaluation; Pyrazoles; Female; Aplastic anemia; Growth factors; Receptors, Thrombopoietin; Immunosuppression; Follow-Up Studies

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 ELT is similar to the clinical trial data across different age groups, treatment lines, and treatment combinations and results in favorable survival.