{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,21]],"date-time":"2026-10-21T00:12:46Z","timestamp":1792541566255,"version":"4.5.0"},"reference-count":41,"publisher":"American Society of Hematology","issue":"25","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2024,6,20]]},"abstract":"<jats:sec>\n                  <jats:title>Abstract<\/jats:title>\n                  <jats:p>In the setting of a learning collaborative, we conducted an international multicenter phase 2 clinical trial testing the hypothesis that nonmyeloablative\u2013related haploidentical bone marrow transplant (BMT) with thiotepa and posttransplant cyclophosphamide (PTCy) will result in 2-year event-free survival (no graft failure or death) of at least 80%. A total of 70 participants were evaluable based on the conditioning protocol. Graft failure occurred in 8 of 70 (11.4%) and only in participants aged &amp;lt;18 years; all had autologous reconstitution. After a median follow-up of 2.4 years, the 2-year Kaplan-Meier\u2013based probability of event-free survival was 82.6%. The 2-year overall survival was 94.1%, with no difference between children and adult participants. After excluding participants with graft failure (n\u00a0= 8), participants with engraftment had median whole blood donor chimerism values at days\u00a0+180 and\u00a0+365 after transplant of 100% (n\u00a0= 58), respectively, and 96.6% (57\/59) were off immunosuppression 1 year after transplant. The 1-year grade 3 to 4 acute graft-versus-host disease (GVHD) rate was 10%, and the 2-year moderate\u2013severe chronic GVHD rate was 10%. Five participants (7.1%) died from infectious complications. We demonstrate that nonmyeloablative haploidentical BMT with thiotepa and PTCy is a readily available curative therapy for most adults, even those with organ damage, compared to the more expensive myeloablative gene therapy and gene editing. Additional strategies are required for children to decrease graft failure rates. The trial was registered at www.clinicaltrials.gov as #NCT01850108.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1182\/blood.2023023301","type":"journal-article","created":{"date-parts":[[2024,3,17]],"date-time":"2024-03-17T18:00:57Z","timestamp":1710698457000},"page":"2654-2665","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":53,"title":["An international learning collaborative phase 2 trial for haploidentical bone marrow transplant in sickle cell disease"],"prefix":"10.1182","volume":"143","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0484-5903","authenticated-orcid":false,"given":"Adetola A.","family":"Kassim","sequence":"first","affiliation":[{"name":"1Division of Hematology\/Oncology, Department of Medicine, Vanderbilt University School of Medicine, Nashville, TN"},{"name":"2Vanderbilt-Meharry Sickle Cell Disease Center of Excellence, Vanderbilt University School of Medicine, Nashville, 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School of Medicine, Nashville, TN"},{"name":"2Vanderbilt-Meharry Sickle Cell Disease Center of Excellence, Vanderbilt University School of Medicine, Nashville, TN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7577-0152","authenticated-orcid":false,"given":"Ali D.","family":"Alahmari","sequence":"additional","affiliation":[{"name":"6Adult Hematology and Stem Cell Therapy and Cellular Therapy Program, King Faisal Specialist Hospital, Riyadh, Saudi Arabia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4578-9423","authenticated-orcid":false,"given":"Adriana","family":"Seber","sequence":"additional","affiliation":[{"name":"7Pediatric Hematopoietic Stem Cell Transplantation Program, Pediatric Hematopoietic Cell Transplantation, Hospital Samaritano Higienopolis-Americas, S\u00e3o Paulo, Brazil"},{"name":"8Pediatric Hematopoietic Stem Cell Transplantation Program, Pediatric Hematopoietic Cell Transplantation, Instituto de Oncologia Pediatrica\u2013Graacc\/Unifesp, S\u00e3o Paulo, Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0343-2610","authenticated-orcid":false,"given":"Carmem","family":"Bonfim","sequence":"additional","affiliation":[{"name":"9Pediatric Bone Marrow Transplantation Program, Hospital Pequeno Pr\u00edncipe\/Instituto de Pesquisa Pele Pequeno Principe, Curitiba, Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4508-8934","authenticated-orcid":false,"given":"Belinda Pinto","family":"Sim\u00f5es","sequence":"additional","affiliation":[{"name":"10Department of Medicine, Ribeir\u00e3o Preto Medical School, University of S\u00e3o Paulo, S\u00e3o Paulo, Brazil"},{"name":"11Department of Hematology, Ribeir\u00e3o Preto Medical School, University of S\u00e3o Paulo, S\u00e3o Paulo, Brazil"},{"name":"12Department of Clinical Oncology, Ribeir\u00e3o Preto Medical School, University of S\u00e3o Paulo, S\u00e3o Paulo, Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9174-5803","authenticated-orcid":false,"given":"Mohsen","family":"Alzahrani","sequence":"additional","affiliation":[{"name":"13Department of Pediatric Hematology\/Oncology, King Abdulaziz Medical City, Ministry of National Guard Health Affairs, King Abdullah International Medical Research Center, King Saud Bin Abdulaziz University for Health Sciences, Riyadh, Saudi Arabia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8808-4266","authenticated-orcid":false,"given":"Michael J.","family":"Eckrich","sequence":"additional","affiliation":[{"name":"14Department of Pediatrics, Pediatric Transplant and Cellular Therapy, Wake Forest University School of Medicine, Atrium Health Levine Children's Hospital, Charlotte, 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