{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,15]],"date-time":"2026-05-15T10:26:24Z","timestamp":1778840784573,"version":"3.51.4"},"reference-count":20,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2008,1,8]],"date-time":"2008-01-08T00:00:00Z","timestamp":1199750400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pediatric Transplantation"],"published-print":{"date-parts":[[2008,9]]},"abstract":"<jats:p><jats:bold>Abstract:\u2002<\/jats:bold> We evaluated the feasibility of UCBT from unrelated donors and a myeloablative preparative regimen that did not involve anti\u2010thymocyte globulin in five children with lysosomal and peroxisomal diseases. Patients with MPS II (n\u2003=\u20031), adrenoleukodystrophy (n\u2003=\u20031), metachromatic leukodystrophy (n\u2003=\u20032), and Krabbe disease (n\u2003=\u20031) received UCBT between December 2001 and September 2005. All patients received oral Bu (600\u2003mg\/m<jats:sup>2<\/jats:sup>), CY (200\u2003mg\/kg IV), and fludarabine (180\u2003mg\/m<jats:sup>2<\/jats:sup> IV). Prophylaxis for GVHD consisted of a combination of tacrolimus and a short methotrexate course. Neutrophil engraftment occurred a median of 24\u2003days (range, 21\u201325) after transplantation. None had graft rejection. One patient developed grade III acute GVHD and the other four patients had grade I acute GVHD; none had extensive chronic GVHD. One patient developed hemorrhagic cystitis. There were no treatment\u2010related deaths. Although one child with MPS II died of PTLD 10\u2003months after the UCBT, four of the five children are alive 14, 20, 31, and 55\u2003months after transplantation with complete donor chimerism. These results suggest the feasibility of the UCBT with Bu, fludarabine, and CY\u2010preparative regimen for patients with inherited metabolic diseases.<\/jats:p>","DOI":"10.1111\/j.1399-3046.2007.00876.x","type":"journal-article","created":{"date-parts":[[2008,8,7]],"date-time":"2008-08-07T01:30:29Z","timestamp":1218072629000},"page":"672-676","source":"Crossref","is-referenced-by-count":17,"title":["Umbilical cord\u2010blood transplantations from unrelated donors in patients with inherited metabolic diseases: Single\u2010institute experience"],"prefix":"10.1111","volume":"12","author":[{"given":"Sadao","family":"Tokimasa","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hideaki","family":"Ohta","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sachiko","family":"Takizawa","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shigenori","family":"Kusuki","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yoshiko","family":"Hashii","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Norio","family":"Sakai","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Masako","family":"Taniike","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Keiichi","family":"Ozono","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Junichi","family":"Hara","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2008,8,6]]},"reference":[{"key":"e_1_2_6_2_2","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1703839"},{"key":"e_1_2_6_3_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(04)00938-X"},{"key":"e_1_2_6_4_2","doi-asserted-by":"publisher","DOI":"10.1038\/nrg963"},{"key":"e_1_2_6_5_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00281-004-0166-2"},{"key":"e_1_2_6_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2003.09.003"},{"key":"e_1_2_6_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2005.09.016"},{"key":"e_1_2_6_8_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa032613"},{"key":"e_1_2_6_9_2","doi-asserted-by":"publisher","DOI":"10.1055\/s-2004-822629"},{"key":"e_1_2_6_10_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa042604"},{"key":"e_1_2_6_11_2","doi-asserted-by":"publisher","DOI":"10.1182\/asheducation-2004.1.354"},{"key":"e_1_2_6_12_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V99.11.4200"},{"key":"e_1_2_6_13_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1246-7820(01)00132-X"},{"key":"e_1_2_6_14_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.coi.2006.07.014"},{"key":"e_1_2_6_15_2","doi-asserted-by":"publisher","DOI":"10.1007\/BF02982017"},{"key":"e_1_2_6_16_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.trim.2005.03.009"},{"key":"e_1_2_6_17_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(04)16628-2"},{"key":"e_1_2_6_18_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V87.11.4894.bloodjournal87114894"},{"key":"e_1_2_6_19_2","first-page":"299","article-title":"The use of partially HLA\u2010mismatched donors for allogeneic transplantation in patients with mucopolysaccharidosis\u2010I","volume":"2","author":"Fleming DR","year":"1998","journal-title":"Pediatr Transplant"},{"key":"e_1_2_6_20_2","doi-asserted-by":"publisher","DOI":"10.1097\/01.pas.0000188030.63706.e7"},{"key":"e_1_2_6_21_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2006-03-011791"}],"container-title":["Pediatric Transplantation"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1111%2Fj.1399-3046.2007.00876.x","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/j.1399-3046.2007.00876.x","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,10,9]],"date-time":"2023-10-09T21:52:38Z","timestamp":1696888358000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/j.1399-3046.2007.00876.x"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2008,8,6]]},"references-count":20,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2008,9]]}},"alternative-id":["10.1111\/j.1399-3046.2007.00876.x"],"URL":"https:\/\/doi.org\/10.1111\/j.1399-3046.2007.00876.x","archive":["Portico"],"relation":{},"ISSN":["1397-3142","1399-3046"],"issn-type":[{"value":"1397-3142","type":"print"},{"value":"1399-3046","type":"electronic"}],"subject":[],"published":{"date-parts":[[2008,8,6]]}}}