Short-term growth hormone treatment in children with Hurler syndrome after hematopoietic cell transplantation

Bone Marrow Transplant. 2009 Sep;44(5):279-85. doi: 10.1038/bmt.2009.31. Epub 2009 Mar 2.

Abstract

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.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Child
  • Cohort Studies
  • Female
  • Hematopoietic Stem Cell Transplantation*
  • Human Growth Hormone / adverse effects
  • Human Growth Hormone / deficiency
  • Human Growth Hormone / therapeutic use*
  • Humans
  • Male
  • Mucopolysaccharidosis I / drug therapy
  • Mucopolysaccharidosis I / therapy*
  • Recombinant Proteins / pharmacology
  • Retrospective Studies

Substances

  • Recombinant Proteins
  • Human Growth Hormone