Growth patterns in children with mucopolysaccharidosis I and II

Agnieszka Różdżyńska-Świątkowska1, Agnieszka Jurecka, Joachim Cieślik

  • 1Anthropology Laboratory, the Children's Memorial Health Institute, Warsaw, Poland, agnieszka.rozdzynska@gmail.com.

Insights

Children with Mucopolysaccharidosis (MPS) types I and II show distinct growth patterns. Early growth is similar, but height differences emerge, reflecting disease severity and aiding therapy evaluation.

Area of Science:

  • Pediatric endocrinology
  • Genetics and developmental biology
  • Metabolic disorders

Background:

  • Mucopolysaccharidoses (MPS) are rare genetic disorders disrupting growth and development.
  • MPS types I and II significantly impact children's physical development.

Purpose of the Study:

  • To investigate and compare the general growth patterns of children diagnosed with MPS type I (Hurler syndrome) and MPS type II.
  • To analyze anthropometric data and identify distinct growth trajectories in relation to disease severity.

Main Methods:

  • Retrospective analysis of anthropometric data from 76 patients with MPS I and II.
  • Plotting growth patterns onto Polish reference charts for comparison.
  • Longitudinal analysis to track age-related changes in growth parameters.

Main Results:

  • Infants with MPS I and II exhibited greater than average body length at birth.
  • Growth patterns were similar across all MPS groups until 30 months of age.
  • Significant divergence in growth patterns occurred post-30 months, with varying timelines for reaching below the 3rd percentile height based on MPS type and severity.

Conclusions:

  • Growth patterns in MPS I and II are distinct and correlate with clinical disease severity.
  • These growth differences can potentially aid in assessing the effectiveness of current MPS therapies.
Abstract

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