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Growth hormone therapy, muscle thickness, and motor development in Prader-Willi syndrome: an RCT

Linda Reus1, Sigrid Pillen2, Ben J Pelzer3

  • 1Department of Rehabilitation, Pediatric Physical Therapy, Scientific Institute for Quality of Healthcare, lin.reus@gmail.com.

Pediatrics
|November 26, 2014
PubMed

Insights

Growth hormone (GH) combined with physical training significantly increased muscle thickness in infants with Prader-Willi syndrome (PWS). This improvement correlated with enhanced muscle strength and motor development, suggesting a training effect.

Area of Science:

  • Pediatrics
  • Endocrinology
  • Developmental Biology

Background:

  • Prader-Willi syndrome (PWS) is a complex genetic disorder affecting multiple body systems.
  • Infants with PWS often exhibit hypotonia, delayed motor development, and potential growth issues.
  • Growth hormone (GH) therapy is sometimes used in PWS management, but its impact on muscle development and function requires further investigation.

Purpose of the Study:

  • To determine the effect of physical training combined with growth hormone (GH) on muscle thickness in infants with Prader-Willi syndrome (PWS).
  • To explore the relationship between muscle thickness, muscle strength, and motor development in infants with PWS.
  • To assess if observed effects are attributable to GH, training, or a combination thereof.

Main Methods:

  • A 2-year randomized controlled trial involving 22 infants with PWS (mean age 12.9 months).
  • Comparison between a treatment group (n=10) receiving GH and physical training versus a waiting-list control group (n=12).
  • Measurements included muscle thickness (ultrasound), muscle strength (Infant Muscle Strength meter), and motor performance (Gross Motor Function Measurement). Statistical analyses included ANOVAs and multilevel modeling.

Main Results:

  • Growth hormone (GH) demonstrated a significant positive effect on muscle thickness in infants with PWS (P < .05).
  • Strong positive correlations were observed between muscle thickness and muscle strength (r = 0.61, P < .001).
  • Significant positive relationships were also found between muscle thickness and motor performance (r = 0.81, P < .001), and between muscle strength and motor performance (r = 0.76, P < .001).

Conclusions:

  • Growth hormone (GH) therapy, in conjunction with physical training, effectively increases muscle thickness in infants with Prader-Willi syndrome (PWS).
  • The observed increase in muscle thickness is directly associated with improvements in muscle strength and motor development.
  • Faster catch-up growth in frequently used muscles, independent of GH, suggests a significant training effect contributing to developmental gains.
Abstract

Keywords:
Prader-Willi syndromebody compositiongrowth hormoneinfantsmotor developmentmuscle development

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