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Growth patterns and the use of growth hormone in the mucopolysaccharidoses

L E Polgreen1, B S Miller

  • 1University of Minnesota, Department of Pediatrics, Division of Endocrinology, Minneapolis, MN, USA.

Journal of Pediatric Rehabilitation Medicine
|June 22, 2010
PubMed

Insights

Short stature is common in mucopolysaccharidosis (MPS). This review examines growth patterns and treatment impacts, including human growth hormone (hGH), for children with MPS.

Area of Science:

  • Pediatrics
  • Genetics
  • Endocrinology

Background:

  • Short stature is a hallmark of mucopolysaccharidosis (MPS) diseases.
  • Understanding natural growth patterns in MPS is crucial for managing skeletal dysplasias.
  • Current treatments like hematopoietic cell transplantation (HCT) and enzyme replacement therapy (ERT) have unknown long-term effects on growth.

Purpose of the Study:

  • To review published data on growth in children with MPS.
  • To describe preliminary data on human growth hormone (hGH) use in MPS patients.
  • To provide insights for individualized growth-promoting treatment decisions.

Main Methods:

  • Literature review of published growth data in MPS.
  • Analysis of preliminary data on hGH treatment in MPS.
  • Synthesis of information on growth patterns and treatment outcomes.

Main Results:

  • Published data on growth in MPS diseases is reviewed.
  • Preliminary findings on hGH therapy in MPS are presented.
  • The long-term impact of HCT and ERT on MPS growth remains under investigation.

Conclusions:

  • Accurate growth data is essential for MPS patient care.
  • hGH may offer potential for growth promotion in MPS, requiring further study.
  • Individualized treatment strategies are key for optimizing growth in children with MPS.

Keywords:
GrowthMPSendocrine diseasegrowth hormonemucopolysaccharidoses

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