Hematopoietic stem cell transplantation for Diamond-Blackfan anemia: A report from the Aplastic Anemia Committee of the Japanese Society of Pediatric Hematology

H. Mugishima, S. Ohga, A. Ohara, S. Kojima, K. Fujisawa, I. Tsukimoto

研究成果: ジャーナルへの寄稿学術誌査読

35 被引用数 (Scopus)

抄録

Transfusion-dependent Diamond-Blackfan 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 months. Transplantation sources were 13 bone marrow [six human leukocyte antigen (HLA)-matched siblings, and six HLA-matched and one HLA-mismatched unrelated donors], five cord blood (two HLA-matched siblings and three HLA-mismatched 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-disparate father and unrelated donor, respectively. One died shortly after haploidentical PBSCT. The five-yr failure-free survival rate after BMT was higher than CBT (100%: 40%, p = 0.002). Platelet recovery was slower in seven unrelated BMT than in six sibling BMT (p = 0.030). 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.

本文言語英語
ページ(範囲)601-607
ページ数7
ジャーナルPediatric Transplantation
11
6
DOI
出版ステータス出版済み - 9月 1 2007

!!!All Science Journal Classification (ASJC) codes

  • 小児科学、周産期医学および子どもの健康
  • 移植

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