{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,11]],"date-time":"2026-09-11T01:58:13Z","timestamp":1789091893402,"version":"build-2803163510"},"reference-count":29,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2006,10,6]],"date-time":"2006-10-06T00:00:00Z","timestamp":1160092800000},"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":[[2007,9]]},"abstract":"<jats:p><jats:bold>Abstract:\u2002<\/jats:bold> Transfusion\u2010dependent Diamond\u2010Blackfan anemia (DBA) patients opt for allogeneic hematopoietic stem cell transplantation (HSCT) as curative therapy. Clinical outcomes of 19 transplanted Japanese patients were analyzed. Prior to HSCT, 10 patients (53%) suffered hemosiderosis with organ dysfunction, and all eight with short stature (42%) had adverse effects of prednisolone. Median age at the time of HSCT was 56\u2003months. Transplantation sources were 13 bone marrow [six human leukocyte antigen (HLA)\u2010matched siblings, and six HLA\u2010matched and one HLA\u2010mismatched unrelated donors], five cord blood (two HLA\u2010matched siblings and three HLA\u2010mismatched unrelated donors), and one peripheral blood from haploidentical mother. All 13 patients with bone marrow transplantation (BMT) and two with sibling cord blood transplantation (CBT) had successful engraftment. Of three patients who underwent unrelated CBT, one died after engraftment, and the other two had graft failure but succeeded in a second BMT from an HLA\u2010disparate father and unrelated donor, respectively. One died shortly after haploidentical PBSCT. The five\u2010yr failure\u2010free survival rate after BMT was higher than CBT (100%: 40%, p\u2003=\u20030.002). Platelet recovery was slower in seven unrelated BMT than in six sibling BMT (p\u2003=\u20030.030). No other factors were associated with engraftment and survival. These results suggest that allogeneic BMT, but not unrelated CBT, is an effective HSCT for refractory DBA.<\/jats:p>","DOI":"10.1111\/j.1399-3046.2006.00616.x","type":"journal-article","created":{"date-parts":[[2006,10,6]],"date-time":"2006-10-06T15:06:26Z","timestamp":1160147186000},"page":"601-607","source":"Crossref","is-referenced-by-count":43,"title":["Hematopoietic stem cell transplantation for Diamond\u2010Blackfan anemia: A report from the Aplastic Anemia Committee of the Japanese Society of Pediatric Hematology"],"prefix":"10.1111","volume":"11","author":[{"given":"H.","family":"Mugishima","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"S.","family":"Ohga","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A.","family":"Ohara","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"S.","family":"Kojima","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"K.","family":"Fujisawa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"I.","family":"Tsukimoto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"for the Aplastic Anemia Committee of the Japanese Society of Pediatric 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