Efficacy and Safety of Long-Term Continuous Growth Hormone Treatment in Children with Prader-Willi Syndrome

Wijngaarden, Roderick F. A. de Lind van; Siemensma, Elbrich P. C.; Festen, Dederieke; Otten, Barto J.; Mil, E.G.A.H. van; Rotteveel, Joost; Odink, Roelof J.; Heus, G. C. B. Bindels-de; Leeuwen, Mariettee van; Haring, D. A. J. P.; Bocca, Gianni; Houdijk, E. C. A. M.; Hoorweg-Nijman, J. J. G.; Vreuls, R. C. F. M.; Jira, Petr; Trotsenburg, A S Paul van; Bakker, Boudewijn; Schroor, Eelco J.; Pilon, Jan Willem; Wit, Jan M.; Drop, Stenvert L. S.; Hokken-Koelega, Anita

doi:10.1210/jc.2009-0454

Cited by 102 publications

(94 citation statements)

References 45 publications

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“…Studies in children with PWS that reported height-corrected LBM have found that LBM normally decreases over time, but with GH treatment LBM stabilizes. 24,27,40 In contrast to treatment in later childhood, GH treatment in infancy leads to an improvement in height-corrected LBM. 19 Our study results confirm these findings.…”

Section: Discussionmentioning

confidence: 99%

“…17,18,20 In children and infants with PWS, body fat percentages decrease as a result of GH treatment, 18,20,21,[24][25][26] although the fat:muscle ratio does not normalize. 27 Dual-energy radiograph absorptiometry revealed a positive GH effect on lean body mass (LBM), which mainly contains muscle tissue. [19][20][21][22]24,[26][27][28] In infants with PWS, GH positively influences motor development.…”

mentioning

confidence: 99%

“…27 Dual-energy radiograph absorptiometry revealed a positive GH effect on lean body mass (LBM), which mainly contains muscle tissue. [19][20][21][22]24,[26][27][28] In infants with PWS, GH positively influences motor development. 8,9 ,20 In children with PWS, GH treatment has a positive effect on agility and thoracic muscle strength.…”

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confidence: 99%

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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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confidence: 99%

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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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“…Although safety issues have been raised after case reports of worsening OSA during rhGH treatment have been reported, 76,80,89 and following improvement of obstructive symptoms after withdrawal of the therapy, 89 prospective studies with larger cohorts have failed to show an increased mortality risk. 77,90 In fact, one nocturnal sudden death was observed in studies by both Festen et al 77 (out of 53 patients) and de Lind van Wijngaarden et al 90 (out of 55 patients), and both of the patients had previous, near normal polysomnography. The effect of rhGH in young children with PWS was investigated in a prospective clinical case series study.…”

Section: Rhgh and Sleep Disordersmentioning

confidence: 92%

Endocrine management of children with Prader–Willi syndrome

Medeiros¹,

Bordallo²,

Souza³

et al. 2013

PHMT

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Prader-Willi syndrome is a rare genetic condition afflicting nearly 1/15,000 live births. Clinical features include neonatal hypotonia, poor weight gain in early infancy followed by binge eating from childhood to adulthood, severe obesity, developmental delay, short stature, and hypogonadism of both central and peripheral etiology. Central hypothyroidism and adrenal insufficiency may occur. Sleep disordered breathing, by obstruction of upper airways associated with central hypoventilation, is a common feature. Most of these characteristics are assumed to be the result of a hypothalamic dysfunction. The most important complication and the most difficult to manage is the obesity. This review aims at discussing the most recent strategies to manage the endocrine complications of Prader-Willi syndrome patients, with a special approach on the treatment of obesity, hypogonadism, and short stature. We summarize the indication and effects of recombinant human growth hormone therapy on growth, cognitive development, and body composition, and discuss the effects of recombinant human growth hormone therapy on the resulting sleep disorders.

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“…124 A recent multicenter study found that along with improving a number of physical characteristics previously mentioned before, no adverse effects of long-term GH treatment in a large group of prepubertal children was found. 125 Since July 2000, the United States Food and Drug Administration (FDA) approved GH for children with PWS regardless of the presence of growth hormone deficiency. However, in Tauber and colleagues' review of 64 cases of death in children with PWS, the authors suggest that the first 9 months of GH therapy has a higher risk period of death compared to later months.…”

Section: Growth Hormonementioning

confidence: 99%

Clinical management of behavioral characteristics of Prader–Willi syndrome

Dimitropoulos

2010

NDT

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Prader-Willi syndrome (PWS) is a complex neurodevelopmental disorder caused by an abnormality on the long arm of chromosome 15 (q11-q13) that results in a host of phenotypic characteristics, dominated primarily by hyperphagia and insatiable appetite. Characteristic behavioral disturbances in PWS include excessive interest in food, skin picking, difficulty with a change in routine, temper tantrums, obsessive and compulsive behaviors, and mood fluctuations. Individuals with PWS typically have intellectual disabilities (borderline to mild/moderate mental retardation) and exhibit a higher overall behavior disturbance compared to individuals with similar intellectual disability. Due to its multisystem disorder, family members, caregivers, physicians, dieticians, and speech-language pathologists all play an important role in the management and treatment of symptoms in an individual with PWS. This article reviews current research on behavior and cognition in PWS and discusses management guidelines for this disorder.

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Efficacy and Safety of Long-Term Continuous Growth Hormone Treatment in Children with Prader-Willi Syndrome

Cited by 102 publications

References 45 publications

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

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

Endocrine management of children with Prader–Willi syndrome

Clinical management of behavioral characteristics of Prader–Willi syndrome

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Efficacy and Safety of Long-Term Continuous Growth Hormone Treatment in Children with Prader-Willi Syndrome

Cited by 102 publications

References 45 publications

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

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

Endocrine management of children with Prader&ndash;Willi syndrome

Clinical management of behavioral characteristics of Prader&ndash;Willi syndrome

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Product

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Endocrine management of children with Prader–Willi syndrome

Clinical management of behavioral characteristics of Prader–Willi syndrome