{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,3]],"date-time":"2026-09-03T14:12:09Z","timestamp":1788444729206,"version":"build-2803163510"},"reference-count":33,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2026,7,19]],"date-time":"2026-07-19T00:00:00Z","timestamp":1784419200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2026,7,19]],"date-time":"2026-07-19T00:00:00Z","timestamp":1784419200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100008272","name":"Novartis Pharmaceuticals Corporation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008272","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004319","name":"Pfizer","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004319","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100006396","name":"Alexion Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006396","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000289","name":"Cancer Research UK","doi-asserted-by":"publisher","award":["A22324"],"award-info":[{"award-number":["A22324"]}],"id":[{"id":"10.13039\/501100000289","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2026,10]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>\n                    The RACE study (NCT02009747) compared horse antithymocyte globulin (hATG) plus cyclosporine A (CsA)\u2009\u00b1\u2009eltrombopag as initial immunosuppressive treatment (IST) for severe aplastic anemia. Here we report the final 2\u2010year analysis of this prospective randomized phase III study. One hundred ninety\u2010seven treatment\u2010naive patients were randomized to standard IST (hATG 40\u2009mg\/kg\u2009\u00d7\u20094\u2009days and CsA 5\u2009mg\/kg\/day; arm A;\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u2009101) or standard IST\u2009+\u2009eltrombopag at the dose of 150\u2009mg\/day (arm B;\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u200996) from day +14 until 6\u2009months (or 3\u2009months, in case of complete response). The median follow\u2010up was 23.2\u2009months. The 2\u2010year cumulative incidence of complete response was significantly superior in arm B (62.4% vs. 35.3%;\n                    <jats:italic>p<\/jats:italic>\n                    \u2009&lt;\u20090.001). The 2\u2010year overall survival (OS) was 86% in arm A and 91% in arm B (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.081), with hazard ratio (HR), adjusted for age and disease severity, of 0.54 (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.064). The 2\u2010year disease\u2010free survival (DFS) was 56% vs. 37% (adjusted HR\u2009=\u20090.49;\n                    <jats:italic>p<\/jats:italic>\n                    \u2009&lt;\u20090.001), while event\u2010free survival (EFS) was 48% vs. 32% (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009&lt;\u20090.001), with adjusted HR\u2009=\u20090.54 (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009&lt;\u20090.001), 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 (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.041). 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\u2010line treatment of AA is associated with better 2\u2010year OS, DFS, and EFS without increased risk of secondary myeloid malignancies.\n                  <\/jats:p>","DOI":"10.1002\/ajh.70445","type":"journal-article","created":{"date-parts":[[2026,7,20]],"date-time":"2026-07-20T01:30:29Z","timestamp":1784511029000},"page":"2520-2532","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["<scp>Eltrombopag<\/scp>\n                    Added to Standard Immunosuppressive Treatment as Front\u2010Line Therapy for Severe Aplastic Anemia: Long\u2010Term Outcomes of the Phase\u20103 Randomized Superiority\n                    <scp>EBMT<\/scp>\n                    \u2010\n                    <scp>SAAWP RACE<\/scp>\n                    Study"],"prefix":"10.1002","volume":"101","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4744-5109","authenticated-orcid":false,"given":"Antonio M.","family":"Risitano","sequence":"first","affiliation":[{"name":"Clinical Study Unit EBMT  Leiden the Netherlands"},{"name":"Department of Clinical Medicine and Surgery Federico II University  Naples Italy"},{"name":"Hematology and Hematopoietic Transplant Unit AORN S. Giuseppe Moscati  Avellino Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3743-756X","authenticated-orcid":false,"given":"Simona","family":"Iacobelli","sequence":"additional","affiliation":[{"name":"Clinical Study Unit EBMT  Leiden the Netherlands"},{"name":"Department of Public Health and Infectious Diseases University of Rome Sapienza  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3180-3570","authenticated-orcid":false,"given":"Austin","family":"Kulasekararaj","sequence":"additional","affiliation":[{"name":"Clinical Study Unit EBMT  Leiden the Netherlands"},{"name":"Haematological Medicine, King's College Hospital NHS Foundation Trust  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marleen","family":"van Os","sequence":"additional","affiliation":[{"name":"Clinical Study Unit EBMT  Leiden the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sofie R.","family":"Terwel","sequence":"additional","affiliation":[{"name":"Clinical Study Unit EBMT  Leiden the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joe","family":"Tuffnell","sequence":"additional","affiliation":[{"name":"Leiden Study Unit EBMT  Leiden the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0006-5990-2211","authenticated-orcid":false,"given":"Brian","family":"Piepenbroek","sequence":"additional","affiliation":[{"name":"Leiden Study Unit EBMT  Leiden the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4287-0595","authenticated-orcid":false,"given":"Morag","family":"Griffin","sequence":"additional","affiliation":[{"name":"Department of Haematology St James's University Hospital  Leeds UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6573-0045","authenticated-orcid":false,"given":"Constantijn J. M.","family":"Halkes","sequence":"additional","affiliation":[{"name":"Department of Hematology Leiden University Medical Center  Leiden the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3332-4525","authenticated-orcid":false,"given":"Christian","family":"Recher","sequence":"additional","affiliation":[{"name":"Hematology Department Centre Hospitalier Universitaire de Toulouse  Toulouse France"},{"name":"Institut Universitaire du Cancer de Toulouse Oncopole  Toulouse France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fiorenza","family":"Barraco","sequence":"additional","affiliation":[{"name":"Centre Hospitalier Universitaire Lyon Sud  Lyon France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8873-2868","authenticated-orcid":false,"given":"Edouard","family":"Forcade","sequence":"additional","affiliation":[{"name":"CHU Bordeaux, H\u00f4pital Haut\u2010Leveque  Pessac France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8484-8097","authenticated-orcid":false,"given":"Juan Carlos","family":"Vallejo","sequence":"additional","affiliation":[{"name":"University Hospital of Salamanca  Salamanca Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Beatrice","family":"Drexler","sequence":"additional","affiliation":[{"name":"Hematology University Hospital Basel  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean\u2010Baptiste","family":"Mear","sequence":"additional","affiliation":[{"name":"Clinical Hematology, University Hospital of Rennes  Rennes France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-7273-1463","authenticated-orcid":false,"given":"Roochi","family":"Trikha","sequence":"additional","affiliation":[{"name":"Haematological Medicine, King's College Hospital NHS Foundation Trust  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3532-231X","authenticated-orcid":false,"given":"Shreyans","family":"Gandhi","sequence":"additional","affiliation":[{"name":"Haematological Medicine, King's College Hospital NHS Foundation Trust  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5118-4167","authenticated-orcid":false,"given":"Anna Maria","family":"Raiola","sequence":"additional","affiliation":[{"name":"Haematology and Cellular Therapies, IRCCS Ospedale Policlinico San Martino  Genova Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9835-3223","authenticated-orcid":false,"given":"L. 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