{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,20]],"date-time":"2026-10-20T01:02:40Z","timestamp":1792458160978,"version":"4.5.0"},"reference-count":36,"publisher":"American Society of Hematology","issue":"1","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2023,1,5]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>Allogeneic hematopoietic stem cell transplantation (alloSCT) is curative for severe inborn errors of immunity (IEIs), with recent data suggesting alloSCT in adulthood is safe and effective in selected patients. However, questions remain regarding the indications for and optimal timing of transplant. We retrospectively compared outcomes of transplanted vs matched nontransplanted adults with severe IEIs. Seventy-nine patients (aged \u2265 15 years) underwent alloSCT between 2008 and 2018 for IEIs such as chronic granulomatous disease (n\u00a0= 20) and various combined immune deficiencies (n\u00a0= 59). A cohort of nontransplanted patients from the French Centre de R\u00e9f\u00e9rence D\u00e9ficits Immunitaires H\u00e9r\u00e9ditaires registry was identified blindly for case-control analysis, with \u22643 matched controls per index patient, without replacement. The nontransplanted patients were matched for birth decade, age at last review greater than index patient age at alloSCT, chronic granulomatous disease or combined immune deficiencies, and autoimmune\/lymphoproliferative complications. A total of 281 patients were included (79 transplanted, 202 nontransplanted). Median age at transplant was 21 years. Transplant indications were mainly lymphoproliferative disease (n\u00a0= 23) or colitis (n\u00a0= 15). Median follow-up was 4.8 years (interquartile range, 2.5-7.2). One-year transplant-related mortality rate was 13%. Estimated disease-free survival at 5 years was higher in transplanted patients (58% vs 33%; P\u00a0= .007). Nontransplanted patients had an ongoing risk of severe events, with an increased mean cumulative number of recurrent events compared with transplanted patients. Sensitivity analyses removing patients with common variable immune deficiency and their matched transplanted patients confirm these results. AlloSCT prevents progressive morbidity associated with IEIs in adults, which may outweigh the negative impact of transplant-related mortality.<\/jats:p>","DOI":"10.1182\/blood.2022015482","type":"journal-article","created":{"date-parts":[[2022,9,27]],"date-time":"2022-09-27T22:09:32Z","timestamp":1664316572000},"page":"60-71","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":21,"title":["Allogeneic stem cell transplantation compared to conservative management in adults with inborn errors of immunity"],"prefix":"10.1182","volume":"141","author":[{"given":"Morgane","family":"Cheminant","sequence":"first","affiliation":[{"name":"1Clinical Haematology, Necker-Enfants Malades University Hospital, Assistance Publique-H\u00f4pitaux de Paris, Paris, France"},{"name":"2Laboratory of Hematological Disorders, Universit\u00e9 Paris Cit\u00e9, Institut Imagine, Institut National de la Sant\u00e9 et de la Recherche M\u00e9dicale, Unit\u00e9 Mixte de Recherche 1163, Paris, France"},{"name":"3Centre de R\u00e9f\u00e9rence D\u00e9ficits Immunitaires H\u00e9r\u00e9ditaires, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1163-2295","authenticated-orcid":false,"given":"Thomas A.","family":"Fox","sequence":"additional","affiliation":[{"name":"4University College London Institute of Immunity and Transplantation, University College London, London, United Kingdom"},{"name":"5Department of Immunology, Royal Free London National Hospital Service Foundation Trust, London, United Kingdom"},{"name":"6Department of Haematology, University College London Hospitals National Hospital Service Foundation Trust, London, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mickael","family":"Alligon","sequence":"additional","affiliation":[{"name":"3Centre de R\u00e9f\u00e9rence D\u00e9ficits Immunitaires H\u00e9r\u00e9ditaires, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olivier","family":"Bouaziz","sequence":"additional","affiliation":[{"name":"7Math\u00e9matiques Appliqu\u00e9es \u00e0 Paris 5, Unit\u00e9 Mixte de Recherche Centre National de la Recherche Scientifique 8145, Universit\u00e9 Paris Cit\u00e9, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"B\u00e9n\u00e9dicte","family":"Neven","sequence":"additional","affiliation":[{"name":"3Centre de R\u00e9f\u00e9rence D\u00e9ficits Immunitaires H\u00e9r\u00e9ditaires, Paris, France"},{"name":"8Service d\u2019H\u00e9matologie-Immunologie et Rhumatologie P\u00e9diatrique, Necker-Enfants Malades University Hospital, Assistance Publique-H\u00f4pitaux de Paris, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6719-3693","authenticated-orcid":false,"given":"Despina","family":"Moshous","sequence":"additional","affiliation":[{"name":"3Centre de 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