Outcomes after related and unrelated umbilical cord blood transplantation for hereditary bone marrow failure syndromes other than Fanconi anemia
Outcomes after related and unrelated umbilical cord blood transplantation for hereditary bone marrow failure syndromes other than Fanconi anemia
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DOI：
10.3324/haematol.2010.027839
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发表时间：
2011-01-01
期刊：
影响因子：
--
通讯作者：
EBMT, Eurocord SAA-WP
中科院分区：
文献类型：
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作者：
Bizzetto, Renata;Bonfim, Carmen;EBMT, Eurocord SAA-WP
异基因造血干细胞移植是治疗遗传性骨髓衰竭综合征的唯一选择。脐带血是一种替代来源的干细胞allogeneic transplantation.Design and MethodsThis多中心，回顾性研究是基于数据报告的Eurocord注册表遗传性骨髓衰竭综合征的患者进行脐带血transplantation.ResultsSixty-four遗传性骨髓衰竭综合征患者移植相关（n=20）或无关的捐助者（n=44）。诊断为Diamond-Blackfan贫血（21例）、先天性无巨核细胞性血小板减少症（16例）、先天性角化不良（8例）、Shwachman-Diamond综合征（2例）、重度先天性中性粒细胞减少症（16例）和未分类（1例）。在接受亲属供者移植的患者组中，除一名患者外，所有患者均接受了HLA匹配的同胞移植。输注的总有核细胞的中位数为5x 10（7）/kg。60天时中性粒细胞恢复的累积发生率为95%。2例患者发生II-IV级急性移植物抗宿主病，而慢性移植物抗宿主病的2年累积发生率为11%。3年总生存率为95%。在接受无关供体移植物的患者组中，86%的患者有HLA不匹配的移植物，3例患者接受了两个脐带血单位。输注的总有核细胞的中位数为6.1 × 10（7）/kg。该组第60天中性粒细胞恢复的累积发生率为55%。II-IV级急性移植物抗宿主病的100天累积发生率为24%，而慢性移植物抗宿主病的2年累积发生率为53%。3年总生存率为61%;年龄小于5岁的患者总体生存率更高（P=0.01）和输注6.1 × 10（7）/kg或更多的总有核细胞结论在遗传性骨髓衰竭综合征患者中，相关的脐带血移植与良好的结果相关，而增加细胞剂量和更好的HLA配型可能提供更好的结果。脐带血移植是一种非血缘关系的移植。
BackgroundAllogeneic stem cell transplantation is the only curative option for patients with hereditary bone marrow failure syndromes. Umbilical cord blood is an alternative source of stem cells for allogeneic transplantation.Design and MethodsThis multicenter, retrospective study is based on data reported to the Eurocord Registry about patients with hereditary bone marrow failure syndrome who underwent umbilical cord blood transplantation.ResultsSixty-four patients with hereditary bone marrow failure syndromes were transplanted from related (n=20) or unrelated donors (n=44). Diagnoses were Diamond-Blackfan anemia (21 patients), congenital amegakaryocytic thrombocytopenia (16 patients), dyskeratosis congenita (8 patients), Shwachman-Diamond syndrome (2 patients), severe congenital neutropenia (16 patients) and unclassified (1 patient). In the group of patients who received grafts from related donors, all patients but one received an HLA-matched sibling transplant. The median number of total nucleated cells infused was 5x10(7)/kg. The cumulative incidence of neutrophil recovery at 60 days was 95%. Two patients had grade II-IV acute graft-versus-host disease, while the 2-year cumulative incidence of chronic graft-versus-host disease was 11%. The 3-year overall survival rate was 95%. In the group of patients who received grafts from unrelated donors, 86% had HLA-mismatched grafts and three received two umbilical cord blood units. The median number of total nucleated cells infused was 6.1x10(7)/kg. The cumulative incidence of neutrophil recovery at day 60 in this group was 55%. The 100-day cumulative incidence of grade II-IV acute graft-versus-host disease was 24%, while the 2-year cumulative incidence of chronic graft-versus-host disease was 53%. The 3-year overall survival rate was 61%; better overall survival was associated with age less than 5 years (P=0.01) and 6.1x10(7)/kg or more total nucleated cells infused (P=0.05).ConclusionsIn patients with hereditary bone marrow failure syndromes, related umbilical cord blood transplantation is associated with excellent outcomes while increasing cell dose and better HLA matching might provide better results in unrelated umbilical cord blood transplantation.