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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We would like to recognize Dr William Krivit for his pioneering work in the field of transplant for MPS IH. We would also like to thank the outstanding nurses who have cared for these patients over the years, Eileen Hanson and Teresa Kivisto for their dedication in the care of the families and in obtaining the necessary data, and the support of the families in furthering our understanding of MPS IH. This study was partially funded by the Children’s Cancer Research Fund, Minneapolis, MN (JT and PJO), NIH T32-DK065519 (LEP), and the Minnesota Medical Foundation (JT and MP).