Polgreen L E, Plog M, Schwender J D, Tolar J, Thomas W, Orchard P J, Miller B S, Petryk A
Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.
Bone Marrow Transplant. 2009 Sep;44(5):279-85. doi: 10.1038/bmt.2009.31. Epub 2009 Mar 2.
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/year (-2.6+/-1.9 s.d.), and it increased to 5.2+/-3.0 cm/year (-0.1+/-3.6 s.d.) after 1 year of treatment. Of the six patients with radiographic data, there was one progression of scoliosis, one progression of kyphosis and one progression of genu valgum. No patient discontinued treatment due to progression of skeletal disease. One patient discontinued GH due to slipped capital femoral epiphysis. Preliminary data suggest that 1-year GH treatment may modestly improve growth velocity in children with Hurler syndrome.
患有黏多糖贮积症I型（Hurler综合征）的儿童在造血干细胞移植（HCT）后会出现进行性生长发育迟缓。本研究的目的是回顾2005年至2008年间在我们机构接受生长激素（GH）治疗身材矮小或生长激素缺乏的8例黏多糖贮积症I型儿童使用生长激素的安全性和有效性。开始使用生长激素治疗时的年龄为9.6±2.3岁，造血干细胞移植后的时间为7.5±1.5年。生长激素的平均剂量为0.32mg/kg/周。基线生长速度为3.5±1.5cm/年（-2.6±1.9标准差），治疗1年后增加至5.2±3.0cm/年（-0.1±3.6标准差）。在有影像学数据的6例患者中，有1例脊柱侧弯进展，1例脊柱后凸进展，1例膝外翻进展。没有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而停用生长激素。初步数据表明，生长激素治疗1年可能会适度提高黏多糖贮积症I型儿童的生长速度。
