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.
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机译：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。这项研究的目的是回顾2005年至2008年间在我们机构接受过GH身材矮小或GH缺乏症治疗的8例Hurler综合征儿童的生长激素（GH）的安全性和有效性。 GH为9.6 +/- 2。3年，自HCT以来的时间为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 s.d.），并且在治疗1年后增加到5.2 +/- 3.0 cm /年（-0.1 +/- 3.6 s.d.）。在这6例具有影像学数据的患者中，脊柱侧弯进展为1个，后凸畸形为1个，外翻属为1个。没有患者因骨骼疾病的进展而中断治疗。一名患者因股骨骨epi滑脱而终止了GH。初步数据表明，一年的GH治疗可能会适度改善Hurler综合征儿童的生长速度。
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