{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,20]],"date-time":"2026-10-20T09:39:38Z","timestamp":1792489178784,"version":"4.5.0"},"reference-count":37,"publisher":"American Society of Hematology","issue":"21","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2025,5,22]]},"abstract":"<jats:sec>\n                  <jats:title>Abstract<\/jats:title>\n                  <jats:p>Chronic granulomatous disease (CGD) is an inborn error of immunity characterized by defective NAD phosphate oxidase function, leading to impaired microbial killing, recurrent infections, and granulomatous inflammation. Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for CGD, particularly effective when a fully HLA-matched donor is available. However, the place of HLA-haploidentical HSCT remains less established. This retrospective multicenter study analyzed outcomes of 64 patients with CGD (53 males; 46 with X-linked CGD) who underwent a first HSCT with HLA-haploidentical family donors, with either in vitro T-cell receptor (TCR)\u03b1\u03b2\/CD19 depletion or in vivo depletion using posttransplant cyclophosphamide (PTCY). The mean age at transplant was 5.8 years (range, 0-33). Patients exhibited a high disease burden before HSCT, with 45% experiencing infections in the 6 months before HSCT and 67% exhibiting inflammation. Outcomes in the entire cohort showed a 3-year overall survival, event-free survival (EFS), and grade 3 to 4 graft-versus-host disease (GVHD)\u2013free EFS of 75.9%, 70.2%, and 56.1%, respectively, and were not affected by the type of depletion or age. The cumulative incidence (CI) of primary graft failure (PGF) was 20.6%. The CI of grade 2 to 4 acute GVHD was higher in the PTCY group (P = .04), whereas the CI of grade 3 to 4 GVHD was not. These results indicate that HLA-haploidentical HSCT is a feasible transplant option for patients with CGD lacking HLA-matched donors. Further refinement of transplant protocols is necessary to mitigate graft failure and acute GVHD, ultimately improving access and outcomes for this life-saving therapy.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1182\/blood.2024026915","type":"journal-article","created":{"date-parts":[[2025,3,16]],"date-time":"2025-03-16T21:05:56Z","timestamp":1742159156000},"page":"2518-2527","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":13,"title":["HLA-haploidentical stem cell transplantation for chronic granulomatous disease: an EBMT-IEWP retrospective study"],"prefix":"10.1182","volume":"145","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3323-0235","authenticated-orcid":false,"given":"Quentin","family":"Riller","sequence":"first","affiliation":[{"name":"1Pediatric Unit of Immunology, Hematology, and Rheumatology, Necker Hospital, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mathias","family":"Hauri","sequence":"additional","affiliation":[{"name":"2Department of Stem Cell Transplantation, University Children's Hospital, Z\u00fcrich, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Su Han","family":"Lum","sequence":"additional","affiliation":[{"name":"3Translational and Clinical Research Institute, Newcastle University, Great North Children\u2019s Hospital, Newcastle upon Tyne, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeroen","family":"Knippenberg","sequence":"additional","affiliation":[{"name":"4European Bone Marrow Transplantation, Leiden Study Unit, Leiden, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tiarlan","family":"Sirait","sequence":"additional","affiliation":[{"name":"4European Bone Marrow Transplantation, Leiden Study Unit, Leiden, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2354-2588","authenticated-orcid":false,"given":"Alexandra","family":"Laberko","sequence":"additional","affiliation":[{"name":"5Department of Pediatric Hematology, Oncology, and Immunology, Dmitry Rogachev National Medical Research Center, Moscow, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1161-9138","authenticated-orcid":false,"given":"Khalid","family":"Halahleh","sequence":"additional","affiliation":[{"name":"6Adult Bone Marrow Transplantation Program, King Hussein Cancer Centre, Amman, Jordan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4681-4726","authenticated-orcid":false,"given":"Hasan","family":"Hashem","sequence":"additional","affiliation":[{"name":"7Division of Pediatric Blood and Marrow Transplantation, Department of Pediatrics, King Hussein Cancer Center, Amman, Jordan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2015-3541","authenticated-orcid":false,"given":"Musa","family":"Karakukcu","sequence":"additional","affiliation":[{"name":"8Department of Pediatric Hematology and Oncology, Faculty of Medicine, Erciyes University, Kayseri, Turkey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5991-2958","authenticated-orcid":false,"given":"Henrik","family":"Sengeloev","sequence":"additional","affiliation":[{"name":"9Department of Pediatrics and Adolescent Medicine, Rigshospitalet, Copenhagen, Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fulvio","family":"Porta","sequence":"additional","affiliation":[{"name":"10Department of Pediatric Hematology, Universit\u00e1 degli Studi di Brescia, Brescia, 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University Hospital, Oslo, Norway"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5715-501X","authenticated-orcid":false,"given":"Maura","family":"Faraci","sequence":"additional","affiliation":[{"name":"18Department of Hematology, Oncology, and Stem Cell Transplantation, IRCCS Istituto Giannina Gaslini, Genoa, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5037-6175","authenticated-orcid":false,"given":"Claudia","family":"Wehr","sequence":"additional","affiliation":[{"name":"19Department of Hematology, Oncology, and Stem Cell Transplantation, Faculty of Medicine and Medical Center, University of Freiburg, Freiburg im Breisgau, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wolfgang","family":"Holter","sequence":"additional","affiliation":[{"name":"20Department of Pediatric Haematology and Oncology, St. Anna Children's Hospital, Department of Pediatrics and 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