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PubMed · 42472670

Eltrombopag Added to Standard Immunosuppressive Treatment as Front-Line Therapy for Severe Aplastic Anemia: Long-Term Outcomes of the Phase-3 Randomized Superiority EBMT-SAAWP RACE Study.

Antonio M Risitano·Simona Iacobelli·Austin Kulasekararaj·Marleen van Os·Sofie R Terwel·Joe Tuffnell·Brian Piepenbroek·Morag Griffin·Constantijn J M Halkes·Christian Recher·Fiorenza Barraco·Edouard Forcade·Juan Carlos Vallejo·Beatrice Drexler·Jean-Baptiste Mear·Roochi Trikha·Shreyans Gandhi·Anna Maria Raiola·L G M Daenen·Marco R de Groot·Etienne Daguindau·Erfan Nur·Wilma Barcellini·Nigel H Russell·Louis Terriou·Anna Paola Iori·Walter Barberi·Anna Sureda·Isabel Sánchez-Ortega·Blanca Xicoy·Isidro Jarque·James Cavenagh·Flore Sicre de Fontbrune·Camilla Frieri·Talha Munir·Jennifer M L Tjon·Suzanne Tavitian·Aline Praire·Laurence Clement·Florence Rabian·Luana Marano·Anita Hill·Elena Palmisani·Petra Muus·Serena Marotta·Fabiana Cacace·Marica Laurino·Jakob R Passweg·Gérard Socié·Ghulam J Mufti·Carlo Dufour·Régis Peffault de Latour·Severe Aplastic Anaemia Working Party of the EBMT
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Abstract

The RACE study (NCT02009747) compared horse antithymocyte globulin (hATG) plus cyclosporine A (CsA)&#x2009;&#xb1;&#x2009;eltrombopag as initial immunosuppressive treatment (IST) for severe aplastic anemia. Here we report the final 2-year analysis of this prospective randomized phase III study. One hundred ninety-seven treatment-naive patients were randomized to standard IST (hATG 40&#x2009;mg/kg&#x2009;&#xd7;&#x2009;4&#x2009;days and CsA 5&#x2009;mg/kg/day; arm A; n&#x2009;=&#x2009;101) or standard IST&#x2009;+&#x2009;eltrombopag at the dose of 150&#x2009;mg/day (arm B; n&#x2009;=&#x2009;96) from day +14 until 6&#x2009;months (or 3&#x2009;months, in case of complete response). The median follow-up was 23.2&#x2009;months. The 2-year cumulative incidence of complete response was significantly superior in arm B (62.4% vs. 35.3%; p&#x2009;<&#x2009;0.001). The 2-year overall survival (OS) was 86% in arm A and 91% in arm B (p&#x2009;=&#x2009;0.081), with hazard ratio (HR), adjusted for age and disease severity, of 0.54 (p&#x2009;=&#x2009;0.064). The 2-year disease-free survival (DFS) was 56% vs. 37% (adjusted HR&#x2009;=&#x2009;0.49; p&#x2009;<&#x2009;0.001), while event-free survival (EFS) was 48% vs. 32% (p&#x2009;<&#x2009;0.001), with adjusted HR&#x2009;=&#x2009;0.54 (p&#x2009;<&#x2009;0.001), both significantly superior for arm B. The cumulative incidence of relapse was comparable in the two arms, while evolution to clinical paroxysmal nocturnal hemoglobinuria was 8% in arm A and 1% in arm B (p&#x2009;=&#x2009;0.041). The risk of clonal evolution remained negligible, with one patient in arm A and two in arm B developing karyotypic abnormalities. The initial hematological response benefit of eltrombopag added to IST as front-line treatment of AA is associated with better 2-year OS, DFS, and EFS without increased risk of secondary myeloid malignancies.

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HumansBenzoatesAnemia, AplasticHydrazinesFemalePyrazolesMaleImmunosuppressive AgentsAntilymphocyte SerumCyclosporineAdultMiddle AgedTreatment OutcomeDrug Therapy, CombinationAdolescentYoung AdultFollow-Up StudiesProspective StudiesJournal ArticleRandomized Controlled TrialClinical Trial, Phase IIIMulticenter StudyEquivalence Trial

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Antonio M Risitano, Simona Iacobelli, Austin Kulasekararaj, Marleen van Os, Sofie R Terwel, Joe Tuffnell, Brian Piepenbroek, Morag Griffin, Constantijn J M Halkes, Christian Recher, Fiorenza Barraco, Edouard Forcade, Juan Carlos Vallejo, Beatrice Drexler, Jean-Baptiste Mear, Roochi Trikha, Shreyans Gandhi, Anna Maria Raiola, L G M Daenen, Marco R de Groot, Etienne Daguindau, Erfan Nur, Wilma Barcellini, Nigel H Russell, Louis Terriou, Anna Paola Iori, Walter Barberi, Anna Sureda, Isabel S&#xe1;nchez-Ortega, Blanca Xicoy, Isidro Jarque, James Cavenagh, Flore Sicre de Fontbrune, Camilla Frieri, Talha Munir, Jennifer M L Tjon, Suzanne Tavitian, Aline Praire, Laurence Clement, Florence Rabian, Luana Marano, Anita Hill, Elena Palmisani, Petra Muus, Serena Marotta, Fabiana Cacace, Marica Laurino, Jakob R Passweg, G&#xe9;rard Soci&#xe9;, Ghulam J Mufti, Carlo Dufour, R&#xe9;gis Peffault de Latour, Severe Aplastic Anaemia Working Party of the EBMT. 2026-07-19. Eltrombopag Added to Standard Immunosuppressive Treatment as Front-Line Therapy for Severe Aplastic Anemia: Long-Term Outcomes of the Phase-3 Randomized Superiority EBMT-SAAWP RACE Study.. https://doi.org/10.1002/ajh.70445

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Adolescent subjectAdult subjectClinical Trial, Phase III subjectDrug Therapy, Combination subjectEquivalence Trial subjectFemale subjectFollow-Up Studies subjectHumans subjectJournal Article subjectMale subjectMiddle Aged subjectMulticenter Study subjectRandomized controlled trials study

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