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Growth patterns and the use of growth hormone in the mucopolysaccharidoses

Issue title: Promoting Bone Health in MPS VI (Maroteaux-Lamy Syndrome): Framing New Therapies, Part 1

Article type: Research Article

Authors: Polgreen, L.E. | Miller, B.S.

Affiliations: University of Minnesota, Department of Pediatrics, Division of Endocrinology, Minneapolis, MN, USA

Note: [] Address for correspondence: Lynda Polgreen, M.D., Assistant Professor, University of Minnesota, Pediatric Endocrinology, Minneapolis, MN, USA. Tel.: +1 (612) 624 4459; E-mail: polgr001@umn.edu

Keywords: Growth, growth hormone, mucopolysaccharidoses, MPS, endocrine disease

DOI: 10.3233/PRM-2010-0106

Journal: Journal of Pediatric Rehabilitation Medicine, vol. 3, no. 1, pp. 25-38, 2010

Received 21 December 2009
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Accepted 21 December 2009
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Published: 2010
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Abstract

Short stature is characteristic of patients with mucopolysaccharidosis (MPS) diseases. For children with skeletal dysplasias, such as MPS, it is important to know the natural history of growth. An understanding of the natural growth pattern in each MPS disease provides a measurement to which treatments can be compared, as well as data which can help families and providers make individualized decisions about growth promoting treatments. Multiple advancements have been made in the treatment of MPS with both hematopoietic cell transplantation (HCT) and enzyme replacement therapy (ERT). The long term benefit of these treatments on growth is unknown. This article will review the published data on growth in children with MPS, and describe preliminary data on the use of human growth hormone (hGH) in children with MPS.

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