Long-Term Growth Hormone Therapy Changes the Natural History of Body Composition and Motor Function in Children with Prader-Willi Syndrome

Carrel, Aaron L.; Myers, Susan E.; Whitman, Barbara Y.; Eickhoff, Jens; Allen, David B.

doi:10.1210/jc.2009-1389

Cited by 170 publications

(141 citation statements)

References 18 publications

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“…Most individuals with PWS, but not all, are deficient in GH and studies that were initially focused on improving growth have not only demonstrated this effect, but also improvements in metabolism, body composition, behavior and cognition (38)(39)(40)(41)(42)(43)(44). However, GH therapy on occasion can be associated with significant side effects, such as the stimulation of adenoid tissue leading to obstructive sleep apnea so it should be used carefully in the treatment of PWS.…”

Section: Clinicaltrialsgov [Nct]: Id Number Nct01725152)mentioning

confidence: 99%

The neurobiology of the Prader-Willi phenotype of fragile X syndrome

Muzar

Lozano

Kolevzon

et al. 2016

IRDR

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Section: Clinicaltrialsgov [Nct]: Id Number Nct01725152)mentioning

confidence: 99%

The neurobiology of the Prader-Willi phenotype of fragile X syndrome

Muzar

Lozano

Kolevzon

et al. 2016

IRDR

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“…39 Some studies reported that GH increases LBM (mainly determined by muscle mass) [20][21][22]26,29 ; however, in these studies, the interpretation of the reported results is problematic because the increase in LBM was not corrected for changes in height. Studies in children with PWS that reported height-corrected LBM have found that LBM normally decreases over time, but with GH treatment LBM stabilizes.…”

Section: Discussionmentioning

confidence: 99%

“…8,9 ,20 In children with PWS, GH treatment has a positive effect on agility and thoracic muscle strength. 21,26,29,30 To relate decreased muscle thickness in PWS to muscle weakness and motor developmental delay, it would be interesting to use a more direct measure of specific muscle groups and compare this with strength measurement to determine the direct relationship between structure and function.…”

mentioning

confidence: 99%

Growth Hormone Therapy, Muscle Thickness, and Motor Development in Prader-Willi Syndrome: An RCT

Reus

Pillen

Pelzer³

et al. 2014

Pediatrics

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WHAT'S KNOWN ON THIS SUBJECT: Infants with Prader-Willi syndrome suffer from hypotonia, muscle weakness, and motor developmental delay and have increased fat mass combined with decreased muscle mass. Growth hormone improves body composition and motor development.WHAT THIS STUDY ADDS: Ultrasound scans confirmed decreased muscle thickness in infants with Prader-Willi syndrome, which improved as result of growth hormone treatment. Muscle thickness was correlated to muscle strength and motor performance. Catch-up growth in muscle thickness was related to muscle use independent of growth hormone. abstract OBJECTIVE: To investigate the effect of physical training combined with growth hormone (GH) on muscle thickness and its relationship with muscle strength and motor development in infants with Prader-Willi syndrome (PWS). METHODS:In a randomized controlled trial, 22 infants with PWS (12.9 6 7.1 months) were followed over 2 years to compare a treatment group (n = 10) with a waiting-list control group (n = 12). Muscle thickness of 4 muscle groups was measured by using ultrasound. Muscle strength was evaluated by using the Infant Muscle Strength meter. Motor performance was measured with the Gross Motor Function Measurement. Analyses of variance were used to evaluate between-group effects of GH on muscle thickness at 6 months and to compare pre-and posttreatment (after 12 months of GH) values. Multilevel analyses were used to evaluate effects of GH on muscle thickness over time, and multilevel bivariate analyses were used to test relationships between muscle thickness, muscle strength, and motor performance. RESULTS:A significant positive effect of GH on muscle thickness (P , .05) was found. Positive relationships were found between muscle thickness and muscle strength (r = 0.61, P , .001), muscle thickness and motor performance (r = 0.81, P , .001), and muscle strength and motor performance (r = 0.76, P , .001).CONCLUSIONS: GH increased muscle thickness, which was related to muscle strength and motor development in infants with PWS. Catch-up growth was faster in muscles that are most frequently used in early development. Because this effect was independent of GH, it suggests a training effect.

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“…In the case of children with PWS, GH therapy can improve growth, body composition, muscle thickness, physical strength and agility, motor performance, fat utilization, and lipid metabolism [184][185][186]. The best response to GH in PWS patients is observed in the first 12 months of treatment.…”

Section: Treatment Options In Patients With Genetic Obesitymentioning

confidence: 99%

New Thoughts on Pediatric Genetic Obesity: Pathogenesis, Clinical Characteristics and Treatment Approach

Stagi¹,

Bianconi²,

Sammarco³

et al. 2017

Adiposity - Omics and Molecular Understanding

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Historically, some genetic syndromes and monogenic forms of obesity have been identified by clinical features and by sequencing candidate genes in patients with severe obesity. The phenotypic expression of genetic factors involved in obesity is variable, thereby allowing to distinguish several clinical pictures of obesity. Monogenic obesity is described as rare and severe early-onset obesity with abnormal feeding behavior and endocrine disorders. Many of the findings emerged from studying families who displayed a classical Mendelian pattern of inheritance. On the contrary, patients with syndromic obesity show a various degree of intellectual disability, different dysmorphic features, and organ-specific abnormalities. But to date, not all involved genes have been identified so far. New diagnostic tools, such as genome-wide studies, array CGH, and whole-exome sequencing, have highlighted more complex models of inheritance, and even more candidate genes were identified. This increase of knowledge may provide insights into the mechanisms involved in the regulation of body weight and finally lead to specific treatments. In these patients, hyperphagia is often a primary phenotypic component. Substantial gaps in understanding the molecular basis of inherited hyperphagia syndromes are present today with a lack of mechanistic targets that can serve as a basis for pharmacologic and behavioral treatments. We have evaluated retrospectively the literature data on weight, body mass index (BMI), clinical features, treatments, and treatment response in pediatric patients with forms of genetic obesity. However, this chapter provides an updated picture of emerging knowledge outlined by the more comprehensive genetic approaches, trying to outline more candidate genes for these forms of genetic obesity. Relevant papers will be identified through systematic searches of the PubMed, EMBASE and Cochrane databases. All published studies in the English language concerning these disorders will be evaluated. Keywords in the

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Long-Term Growth Hormone Therapy Changes the Natural History of Body Composition and Motor Function in Children with Prader-Willi Syndrome

Cited by 170 publications

References 18 publications

The neurobiology of the Prader-Willi phenotype of fragile X syndrome

The neurobiology of the Prader-Willi phenotype of fragile X syndrome

Growth Hormone Therapy, Muscle Thickness, and Motor Development in Prader-Willi Syndrome: An RCT

New Thoughts on Pediatric Genetic Obesity: Pathogenesis, Clinical Characteristics and Treatment Approach

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