Romiplostim is effective for eltrombopag-refractory aplastic anemia: results of a retrospective study
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ORIGINAL ARTICLE
Romiplostim is effective for eltrombopag‑refractory aplastic anemia: results of a retrospective study Masataka Ise1 · Hiromitsu Iizuka1 · Yoshimasa Kamoda1 · Masako Hirao1 · Michiko Kida1 · Kensuke Usuki1 Received: 11 May 2020 / Revised: 29 July 2020 / Accepted: 7 August 2020 © Japanese Society of Hematology 2020
Abstract Eltrombopag (EPAG) and romiplostim (ROM), thrombopoietin receptor-agonists with demonstrated efficacy against aplastic anemia (AA) in prospective controlled studies, were authorized in Japan for use in adults with aplastic anemia in 2017 and 2019, respectively. So far, no data are available on the potential contribution of switching from ROM to EPAG or vice versa in terms of efficacy or tolerance. Efficacies and tolerance profiles of ten patients, who failed to respond to the maximum dose of EPAG and then switched to ROM, were evaluated. All ten patients received a maximum dose of ROM (20 μg/kg/week). At a median follow-up of twelve months, seven of ten patients (70%) had achieved either neutrophil, erythroid, or platelet response, including one complete response. No patients showed platelet count fluctuations that were reported during ROM treatment for immune thrombocytopenia. In univariate analysis of the relationship between efficacy and demographics, the response had a correlation with neither factors. None of the patients stopped the ROM treatment because of adverse events. Although a larger number of patients and a longer follow-up period are needed to confirm our findings, our results show the efficacy of ROM in patients with EPAG-refractory AA. Keywords Aplastic anemia · Romiplostim · Eltrombopag · Thrombopoietin receptor-agonists
Introduction Aplastic anemia (AA) is an acquired bone marrow disease characterized by trilineage marrow hypoplasia and a paucity of hematopoietic stem and progenitor cells due to an autoimmune attack on the bone marrow [1]. It is estimated that the incidence of AA is 0.7–4.1 cases per million people worldwide with the prevalence between men and women being approximately equal. The incidence rate of AA in Asia is 2–3 times higher than in the west, and the number of patients with AA was estimated to be 9455, with an annual incidence of 8.2 per 1,000,000 people [2]. There is a biphasic distribution with peaks at 10–25 and over 60 years of age. Immunosuppressive therapy with horse anti-thymocyte globulin (ATG) and cyclosporine (CSA) with or without eltrombopag (EPAG) is the standard of care for patients with severe AA, who are not eligible for transplantation [3–5]. * Kensuke Usuki [email protected] 1
Department of Hematology, NTT Medical Center Tokyo, 5‑9‑22 Higashigotanda, Shinagawa‑ku, Tokyo 141‑8625, Japan
While hematological responses correlate strongly with long-term survival, approximately 30% of the patients fail to respond, and therapeutic options for those with refractory/ relapse AA are limited. Recovery in AA may be limited due to not only the ongoing immune attack but also the profound depletion of tissue stem cells. Thes
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