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Implantation and Monitoring by PET/CT of an Orthotopic Model of Human Pleural Mesothelioma in Athymic Mice
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Growth Patterns in MPS IVA and MPS IIIA: A Longitudinal Single-Center Study.

Lior Carmon1,2, Majd Nassar1,2, Daphna Idan2

  • 1Pediatrics Endocrinology Unit, Saban Children's Hospital, Soroka University Medical Center, Beer-Sheva 8410501, Israel.

Journal of Clinical Medicine
|June 12, 2026
PubMed
Summary
This summary is machine-generated.

Enzyme replacement therapy (ERT) with Elosulfase alfa did not prevent height loss in Mucopolysaccharidoses (MPS) IVA patients. Growth trajectories in MPS IVA and MPS IIIA show distinct patterns, with ERT not significantly altering height decline rates.

Keywords:
enzyme replacement therapy (ERT)growth impairmentlysosomal storage disordersmucopolysaccharidoses (MPS)skeletal dysplasia

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Area of Science:

  • Biochemistry and Genetics
  • Pediatric Endocrinology
  • Lysosomal Storage Disorders

Background:

  • Mucopolysaccharidoses (MPS) are genetic disorders causing progressive growth impairment due to impaired glycosaminoglycan degradation.
  • Limited longitudinal growth data exists for MPS IVA and MPS IIIA, particularly concerning enzyme replacement therapy (ERT) effects.
  • Understanding growth trajectories is crucial for managing these complex multisystemic conditions.

Purpose of the Study:

  • To characterize growth trajectories in patients with MPS IVA and MPS IIIA.
  • To assess the association of ERT with Elosulfase alfa on growth outcomes in MPS IVA patients.

Main Methods:

  • Retrospective analysis of growth data from 39 patients with MPS IIIA and IVA (2004-2024).
  • Calculation of height and weight standard deviation scores (SDS) using CDC references.
  • Modeling using linear mixed-effects models (LMM), including subgroup analysis for Elosulfase alfa treated MPS IVA patients.

Main Results:

  • Distinct growth trajectories observed: MPS IIIA showed height decline post-age six and weight loss; MPS IVA exhibited persistent short stature and overweight tendency.
  • In MPS IVA patients treated with Elosulfase alfa (n=16), height SDS significantly declined (-0.127 SDS/year, p < 0.001).
  • The rate of height SDS decline in MPS IVA was not significantly affected by the age of ERT initiation (interaction p = 0.53).

Conclusions:

  • ERT with Elosulfase alfa did not prevent progressive height loss relative to population norms in MPS IVA.
  • The timing of Elosulfase alfa initiation did not significantly influence the rate of height SDS decline.
  • Observational data limits causal conclusions regarding ERT efficacy on growth in MPS IVA.