{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,31]],"date-time":"2026-10-31T01:20:50Z","timestamp":1793409650886,"version":"4.5.0"},"reference-count":250,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2024,4,5]],"date-time":"2024-04-05T00:00:00Z","timestamp":1712275200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Immunol."],"abstract":"<jats:p>Acquired aplastic anemia (AA) is an immune-mediated bone marrow (BM) failure where marrow disruption is driven by a cytotoxic T-cell\u2013mediated autoimmune attack against hematopoietic stem cells. The key diagnostic challenge in children, but also in adults, is to exclude the possible underlying congenital condition and myelodysplasia. The choice of treatment options, either allogeneic hematopoietic cell transplantation (alloHCT) or immunosuppressive therapy (IST), depends on the patient\u2019s age, comorbidities, and access to a suitable donor and effective therapeutic agents. Since 2022, horse antithymocyte globulin (hATG) has been available again in Europe and is recommended for IST as a more effective option than rabbit ATG. Therefore, an update on immunosuppressive strategies is warranted. Despite an improved response to the new immunosuppression protocols with hATG and eltrombopag, some patients are not cured or remain at risk of aplasia relapse or clonal evolution and require postponed alloHCT. The transplantation field has evolved, becoming safer and more accessible. Upfront alloHCT from unrelated donors is becoming a tempting option. With the use of posttransplant cyclophosphamide, haploidentical HCT offers promising outcomes also in AA. In this paper, we present the state of the art in the management of severe AA for pediatric and adult patients based on the available guidelines and recently published studies.<\/jats:p>","DOI":"10.3389\/fimmu.2024.1378432","type":"journal-article","created":{"date-parts":[[2024,4,5]],"date-time":"2024-04-05T00:19:23Z","timestamp":1712276363000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":37,"title":["The state of the art in the treatment of severe aplastic anemia: immunotherapy and hematopoietic cell transplantation in children and adults"],"prefix":"10.3389","volume":"15","author":[{"given":"Agnieszka","family":"Piekarska","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katarzyna","family":"Pawelec","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Szmigielska-Kap\u0142on","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marek","family":"Ussowicz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2024,4,5]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra1413485","article-title":"Aplastic anemia","volume":"379","author":"Young","year":"2018","journal-title":"N Engl J Med"},{"key":"B2","doi-asserted-by":"publisher","first-page":"70","DOI":"10.1007\/s11864-017-0511-z","article-title":"Diagnosis and treatment of aplastic anemia","volume":"18","author":"Peslak","year":"2017","journal-title":"Curr Treat Options Oncol"},{"key":"B3","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V45.3.355.bloodjournal453355","article-title":"Selection of patients for bone marrow transplantation in severe aplastic anemia","volume":"45","author":"Camitta","year":"1975","journal-title":"Blood"},{"key":"B4","doi-asserted-by":"publisher","first-page":"13","DOI":"10.1053\/j.seminhematol.2021.12.002","article-title":"Aplastic anemia: pathophysiology","volume":"59","author":"Giudice","year":"2022","journal-title":"Semin Hematol"},{"key":"B5","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2010.08194.x","article-title":"Hepatitis-associated aplastic anaemia: epidemiology and treatment results obtained in Europe. 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