Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning
Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning
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DOI：
10.1182/blood-2012-09-455238
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发表时间：
2013-05-09
期刊：
影响因子：
20.3
通讯作者：
Rocha, Vanderson
中科院分区：
文献类型：
--
作者：
Boelens, Jaap Jan;Aldenhoven, Mieke;Rocha, Vanderson
我们报告了1995年至2007年的骨髓性调节方案后258名跨流行综合征儿童（HS）的移植结果。移植的中位年龄为16.7个月，中位随访时间为57个月。第60天中性粒细胞恢复的累积发生率为91％，急性移植物抗宿主病（GVHD）（II-IV级）在第100天为25％，而慢性GVHD和5年为16％。 5年的总生存期和无事件生存期（EFS）分别为74％和63％。 HLA匹配的兄弟姐妹捐赠者（MSD）和6/6匹配的无关脐带血（CB）供体后的EFS相似，为81％，在10/10 HLA-Matched的无关供体（UD）后66％，5/6之后68％匹配的CB捐助者。在4/6匹配的无关CB（UCB）（57％; P = .031）和HLA不匹配的UD（41％; P = .007）中，EFS较低。与其他GRAFTS相比，CB移植后（分别为92％和98％）后，全偏嵌合体（P = .039）和正常酶水平（分别为92％和98％）更高（分别为92％和98％）（分别为69％和59％）。总之，HS的同种异体移植结果令人鼓舞，MSD之后的EFS速率相似，6/6匹配的UCB，5/6 UCB和10/10匹配的UD。不匹配的UD和4/6匹配的UCB的使用与较低的EFS相关。
We report transplantation outcomes of 258 children with Hurler syndrome (HS) after a myeloablative conditioning regimen from 1995 to 2007. Median age at transplant was 16.7 months and median follow-up was 57 months. The cumulative incidence of neutrophil recovery at day 60 was 91%, acute graft-versus-host disease (GVHD) (grade II-IV) at day 100 was 25%, and chronic GVHD and 5 years was 16%. Overall survival and event-free survival (EFS) at 5 years were 74% and 63%, respectively. EFS after HLA-matched sibling donor (MSD) and 6/6 matched unrelated cord blood (CB) donor were similar at 81%, 66% after 10/10 HLA-matched unrelated donor (UD), and 68% after 5/6 matched CB donor. EFS was lower after transplantation in 4/6 matched unrelated CB (UCB) (57%; P = .031) and HLA-mismatched UD (41%; P = .007). Full-donor chimerism (P = .039) and normal enzyme levels (P = .007) were higher after CB transplantation (92% and 98%, respectively) compared with the other grafts sources (69% and 59%, respectively). In conclusion, results of allogeneic transplantation for HS are encouraging, with similar EFS rates after MSD, 6/6 matched UCB, 5/6 UCB, and 10/10 matched UD. The use of mismatched UD and 4/6 matched UCB was associated with lower EFS.