Short-term growth hormone treatment in children with Hurler syndrome after hematopoietic cell transplantation.
Short-term growth hormone treatment in children with Hurler syndrome after hematopoietic cell transplantation.
复制标题
DOI：
10.1038/bmt.2009.31
复制
发表时间：
2009-09
影响因子：
4.8
通讯作者：
Petryk A
中科院分区：
文献类型：
--
作者：
Polgreen LE;Plog M;Schwender JD;Tolar J;Thomas W;Orchard PJ;Miller BS;Petryk A
患有Hurler综合征的儿童在造血细胞移植（HCT）后经历进行性生长衰竭。本研究的目的是回顾2005年至2008年期间生长激素（GH）治疗8例Hurler综合征患儿的安全性和有效性，这些患儿在我们的机构接受GH治疗身材矮小或GH缺乏症。开始GH治疗时的年龄为9.6 ± 2.3岁，自HCT的时间为7.5 ± 1.5年。平均GH剂量为0.32 mg/kg/周。基线生长速度为3.5 ± 1.5 cm/年（−2.6 ± 1.9 SDS），治疗1年后增加至5.2 ± 3.0 cm/年（−0.1 ± 3.6 SDS）。在6例有影像学数据的患者中，1例脊柱侧凸进展，1例脊柱后凸进展，1例脊柱外翻进展。无患者因骨骼疾病进展而停止治疗。1例患者因股骨头骨骺滑脱（SCFE）而停用GH。初步数据表明，一年的生长激素治疗可能会适度改善儿童赫尔勒综合征的生长速度。
Children with Hurler syndrome experience progressive growth failure after hematopoietic cell transplantation (HCT). The goal of this study was to review the safety and efficacy of growth hormone (GH) in eight children with Hurler syndrome who were treated at our institution with GH for short stature or GH deficiency between 2005 and 2008. The age at initiation of treatment with GH was 9.6 ± 2.3 years and time since HCT was 7.5 ± 1.5 years. Mean GH dose was 0.32 mg/kg/week. Baseline growth velocity was 3.5 ± 1.5 cm/yr (−2.6 ± 1.9 SDS) and increased to 5.2 ± 3.0 cm/yr (−0.1 ± 3.6 SDS) after 1 year of treatment. Of 6 patients with radiographic data there was 1 progression of scoliosis, 1 progression of kyphosis, and 1 progression of genu valgum. No patient discontinued treatment due to progression of skeletal disease. One patient discontinued GH due to slipped capital femoral epiphysis (SCFE). Preliminary data suggest that one year GH treatment may modestly improve growth velocity in children with Hurler syndrome.
登录
查看更多内容
影响因子：
8.8
作者：
Grewal, SS;Wynn, R;Peters, C
通讯作者：
Peters, C
影响因子：
20.3
作者：
HUMA, Z;BOULAD, F;SKLAR, C
通讯作者：
SKLAR, C
影响因子：
1.7
作者：
EXNER, GU
通讯作者：
EXNER, GU
影响因子：
4.8
作者：
Khanna, G.;Van Heest, A. E.;Orchard, P. J.
通讯作者：
Orchard, P. J.
影响因子：
3.8
作者：
Dusing, Stacey C.;Thorpe, Deborah;Escolar, Maria L.
通讯作者：
Escolar, Maria L.