{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,29]],"date-time":"2026-06-29T22:56:03Z","timestamp":1782773763676,"version":"3.54.5"},"reference-count":16,"publisher":"American Society of Hematology","issue":"9","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2002,5,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>Infantile osteopetrosis (OP) carries an extremely poor prognosis unless treated early by hematopoietic stem cell transplantation. We explored the use of purified blood progenitor cells from HLA-haploidentical parents in 7 patients lacking suitable matched donors. Blood progenitor cells were purified by positive selection and by additional T-cell depletion using rosette formation. For conditioning, patients received busulfan, thiotepa, and either cyclophosphamide (5 patients) or fludarabine (2 patients). Stable donor engraftment developed in 6 of 7 patients. Graft-versus-host disease was not observed. Three of the 7 patients had no major complications and 4 of 7 had both veno-occlusive disease and respiratory failure. Five of 7 patients survive with complete cure of OP at a median of 4 years. Patients with OP lacking HLA-matched donors can be successfully treated by transplantation of purified blood progenitor cells from HLA-haploidentical donors.<\/jats:p>","DOI":"10.1182\/blood.v99.9.3458","type":"journal-article","created":{"date-parts":[[2002,10,11]],"date-time":"2002-10-11T14:33:52Z","timestamp":1034346832000},"page":"3458-3460","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":54,"title":["HLA-haploidentical blood progenitor cell transplantation in osteopetrosis"],"prefix":"10.1182","volume":"99","author":[{"given":"Ansgar S.","family":"Schulz","sequence":"first","affiliation":[{"name":"From the Departments of Pediatrics and Transfusion Medicine, University of Ulm, Ulm, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carl Friedrich","family":"Classen","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics and Transfusion Medicine, University of Ulm, Ulm, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Walther A.","family":"Mihatsch","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics and Transfusion Medicine, University of Ulm, Ulm, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Sigl-Kraetzig","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics and Transfusion Medicine, University of Ulm, Ulm, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Markus","family":"Wiesneth","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics and Transfusion Medicine, University of Ulm, Ulm, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Klaus-Michael","family":"Debatin","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics and Transfusion Medicine, University of Ulm, Ulm, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wilhelm","family":"Friedrich","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics and Transfusion Medicine, University of Ulm, Ulm, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susanna M.","family":"Mu\u0308ller","sequence":"additional","affiliation":[{"name":"From the Departments of Pediatrics and Transfusion Medicine, University of Ulm, Ulm, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","reference":[{"key":"2020021221134969300_B1","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1530\/eje.0.1340143","article-title":"Recent developments in the understanding of the pathophysiology of osteopetrosis.","volume":"134","author":"Felix","year":"1996","journal-title":"Eur J Endocrinol."},{"key":"2020021221134969300_B2","doi-asserted-by":"crossref","first-page":"343","DOI":"10.1038\/77131","article-title":"Defects in 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