Authors: Mugishima, H.¹; Ohga, S.²; Ohara, A.³; Kojima, S.⁴; Fujisawa, K.⁵; Tsukimoto, I.⁶

Source: Pediatric Transplantation, Volume 11, Number 6, September 2007 , pp. 601-607(7)

Publisher: Wiley-Blackwell

Abstract:

Mugishima H, Ohga S, Ohara A, Kojima S, Fujisawa K, Tsukimoto I, for the Aplastic Anemia Committee of the Japanese Society of Pediatric Hematology. Hematopoietic stem cell transplantation for Diamond-Blackfan anemia: A report from the Aplastic Anemia Committee of the Japanese Society of Pediatric Hematology.

Pediatr Transplantation 2007: 11: 601-607. © 2006 Blackwell Munksgaard Abstract: 

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.

Keywords: hematopoietic stem cell transplantation; Diamond-Blackfan anemia

Document Type: Research article

DOI: http://dx.doi.org/10.1111/j.1399-3046.2006.00616.x

Affiliations: 1: Department of Advanced Medicine, Division of Cell Regeneration and Transplantation, Nihon University School of Medicine, Tokyo, Japan 2: Department of Pediatrics, Graduate School of Medical Sciences, Kyusyu University, Fukuoka, Japan 3: Department of Transfusion, Toho University School of Medicine, Tokyo, Japan 4: Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan 5: Department of Pediatrics, Jikei University School of Medicine, Tokyo, Japan 6: Department of Pediatrics, Toho University School of Medicine, Tokyo, Japan

Publication date: 2007-09-01

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