Allogeneic blood SCT for children with Hurler's syndrome: results from the German multicenter approach MPS-HCT 2005

被引:14
作者
Sauer, M. [1 ]
Meissner, B. [1 ]
Fuchs, D. [2 ]
Gruhn, B. [2 ]
Kabisch, H. [3 ]
Erttmann, R. [3 ]
Suttorp, M. [4 ]
Beilken, A. [1 ]
Luecke, T. [5 ]
Welte, K. [1 ]
Grigull, L. [1 ]
Sykora, K. W. [1 ]
机构
[1] Hannover Med Sch, Dept Pediat Hematol Oncol, D-3000 Hannover, Germany
[2] Univ Jena, Dept Pediat Hematol & Oncol, Jena, Germany
[3] Univ Childrens Hosp Hamburg Eppendorf, Dept Pediat Hematol & Oncol, Hamburg, Germany
[4] Carl Gustav Carus Univ, Dept Pediat Hematol & Oncol, Dresden, Germany
[5] Hannover Med Sch, Dept Pediat Neurol, D-3000 Hannover, Germany
关键词
Hurler's syndrome; mucopolysaccharidosis; SCT; preparative regimen; regimen-related toxicity; GVHD; BONE-MARROW-TRANSPLANTATION; CELL TRANSPLANTATION; MEGADOSE TRANSPLANTATION; UNRELATED DONORS; STEM-CELLS; FOLLOW-UP; BUSULFAN; IMMUNOTHERAPY; LEUKEMIA;
D O I
10.1038/bmt.2008.328
中图分类号
Q6 [生物物理学];
学科分类号
071011 ;
摘要
Hurler's syndrome is an inborn error of mucopolysaccharide metabolism leading to premature death in childhood. Allogeneic hematopoietic SCT can achieve long-term survival by correcting the enzymatic deficiency. In an attempt to improve long-term engraftment and to reduce regimen-related toxicity (RRT), a prospective multicenter approach was initiated in Germany using a fludarabine-based radiation-free preparative regimen. Between 2001 and 2008, 12 children were enrolled. Median age at SCT was 14 months (range, 4-31 months). The conditioning regimen contained fludarabine, BU, melphalan and antithymocyte globulin. CD34 positively selected PBSC were used in 10 children with a matched unrelated donor. Median cell dose was 24.6 x 10(6) CD34+ cells per kg (range 10.0-54.8). Two children with a matched sibling donor received non-manipulated BM. Donor lymphocyte infusions were given in 6/12 children for mixed hematopoietic chimerism. At a median follow-up of 29 months (range 2-85 months), all children engrafted and have either stabilized or improved neurological function. In total, 12/12 patients showed donor-derived engraftment with 9/12 having full and 3/12 having mixed hematopoiesis. One developed acute GVHD >= grade II. RRT >= grade II was observed in two patients.
引用
收藏
页码:375 / 381
页数:7
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