Growth Hormone Therapy in Adults with Prader-Willi Syndrome

Vogt, Karen; Emerick, Jill E

doi:10.3390/diseases3020056

Cited by 13 publications

(16 citation statements)

References 52 publications

(91 reference statements)

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“… 7 , 12 Moreover, physical activity in patients with PWS has a substantial influence on the growth of lean body mass, 13 gross motor ability and general physical function, such as body composition, muscle strength, fitness levels, motor quality, and exercise tolerance; the use of GH treatment and physical training to improve these factors has been investigated. 14 – 19 …”

Section: Introductionmentioning

confidence: 99%

Hand strength and dexterity in patients with Prader–Willi syndrome: a pilot intervention study

et al. 2018

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ObjectiveThe study aim was to examine the hand function (hand strength and dexterity) and intervention effects of training in adults with Prader–Willi syndrome (PWS).MethodsSix adults with PWS (two females; mean age 26.14 years) underwent hand muscle strength and dexterity training for 3 months (2 hours per week). The following hand function tests were performed pre- and post-intervention: (1) hand grip, lateral pinch, and tip pinch hand strength tests, (2) the Box and Block test (BBT) for gross manual dexterity and (3) the Purdue Pegboard test for finger dexterity.ResultsBefore treatment, all subjects showed lower hand grip, lateral pinch, tip pinch strength, and poorer manual/finger dexterity relative to healthy adults. After training, hand function scores improved on many test items, but only the left hand tip pinch and the right hand BBT performance showed significant improvements.ConclusionsAll subjects showed lower hand strength and poorer manual/finger dexterity compared with healthy adults; this should be considered during physical training programs. Owing to limitations in the intervention intensity and possible subject behavioral deficits, further research is needed to clarify the effects of this intervention on hand function in PWS patients.

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Section: Introductionmentioning

confidence: 99%

Hand strength and dexterity in patients with Prader–Willi syndrome: a pilot intervention study

et al. 2018

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“…GH replacement therapy, the only Federal Drug Agency (FDA)‐approved treatment specifically for PWS, has been shown to normalize linear growth and improve motor function, psychomotor development, and body composition in children and adults with PWS . Specifically, GH treatment has been shown to decrease body fat and concomitantly increase lean muscle mass in children and adults with PWS. Mounting evidence suggests that continued GH therapy into adulthood increases muscle strength and exercise capacity in adult patients .…”

Section: Resultsmentioning

confidence: 99%

“…Specifically, GH treatment has been shown to decrease body fat and concomitantly increase lean muscle mass in children and adults with PWS. Mounting evidence suggests that continued GH therapy into adulthood increases muscle strength and exercise capacity in adult patients . A study found that cessation of GH upon completion of linear growth was associated with worsened BMI and increased visceral adipose tissue in patients with PWS (aged 14.0‐17.9 years) .…”

Section: Resultsmentioning

confidence: 99%

Current and emerging therapies for managing hyperphagia and obesity in Prader‐Willi syndrome: A narrative review

et al. 2019

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Summary In early childhood, individuals with Prader‐Willi syndrome (PWS) experience excess weight gain and severe hyperphagia with food compulsivity, which often leads to early onset morbid obesity. Effective treatments for appetite suppression and weight control are currently unavailable for PWS. Our aim to further understand the pathogenesis of PWS led us to carry out a comprehensive search of the current and emerging therapies for managing hyperphagia and extreme weight gain in PWS. A literature search was performed using PubMed and the following keywords: “PWS” AND “therapy” OR “[drug name]”; reference lists, pharmaceutical websites, and the ClinicalTrials.gov registry were also reviewed. Articles presenting data from current standard treatments in PWS and also clinical trials of pharmacological agents in the pipeline were selected. Current standard treatments include dietary restriction/modifications, exercise, and growth hormone replacement, which appear to have limited efficacy for appetite and weight control in patients with PWS. The long‐term safety and effectiveness of bariatric surgery in PWS remains unknown. However, many promising pharmacotherapies are in development and, if approved, will bring much needed choices into the PWS pharmacological armamentarium. With the progress that is currently being made in our understanding of PWS, an effective treatment may not be far off.

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“… 8 , 9 The most frequent adverse effects include lower extremity edema and altered glucose and insulin homeostasis. 61 In this context, only a slight increase in fasting glucose and trends toward hyperinsulinemia and higher insulin resistance are generally reported. 8 Nevertheless, GH treatment must be suspended in PWS adults with uncontrolled diabetes mellitus.…”

Section: Gh and Adulthoodmentioning

confidence: 99%

Growth hormone therapy for Prader–Willi syndrome: challenges and solutions

Grugni¹,

Sartório²,

Crinò³

2016

TCRM

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Prader–Willi syndrome (PWS) is characterized by a dysregulation of growth hormone (GH)/insulin-like growth factor I axis, as the consequence of a complex hypothalamic involvement. PWS’ clinical picture seems to resemble the classic non-PWS GH deficiency (GHD), including short stature, excessive body fat, decreased muscle mass, and impaired quality of life. GH therapy is able to ameliorate the phenotypic appearance of the syndrome, as well as to improve body composition, physical strength, and cognitive level. In this regard, however, some pathophysiologic and clinical questions still remain, representing a challenge to give the most appropriate care to PWS patients. Data about the prevalence of GHD in PWS children are not unequivocal, ranging from 40% to 100%. In this context, to establish whether the presence (or not) of GHD may have a different effect on clinical course during GH therapy may be helpful. In addition, the comparison of GH effects in PWS children diagnosed as small for gestational age with those obtained in subjects born appropriate for gestational age is of potential interest for future trials. Emerging information seems to demonstrate the maintenance of beneficial effects of GH therapy in PWS subjects after adolescent years. Thus, GH retesting after achievement of final height should be taken into consideration for all PWS patients. However, it is noteworthy that GH administration exerts positive effects both in PWS adults with and without GHD. Another critical issue is to clarify whether the genotype–phenotype correlations may be relevant to specific outcome measures related to GH therapy. Moreover, progress of our understanding of the role of GH replacement and concomitant therapies on bone characteristics of PWS is required. Finally, a long-term surveillance of benefits and risks of GH therapy is strongly recommended for PWS population, since most of the current studies are uncontrolled and of short duration.

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Growth Hormone Therapy in Adults with Prader-Willi Syndrome

Cited by 13 publications

References 52 publications

Hand strength and dexterity in patients with Prader–Willi syndrome: a pilot intervention study

Hand strength and dexterity in patients with Prader–Willi syndrome: a pilot intervention study

Current and emerging therapies for managing hyperphagia and obesity in Prader‐Willi syndrome: A narrative review

Growth hormone therapy for Prader–Willi syndrome: challenges and solutions

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Growth Hormone Therapy in Adults with Prader-Willi Syndrome

Cited by 13 publications

References 52 publications

Hand strength and dexterity in patients with Prader–Willi syndrome: a pilot intervention study

Hand strength and dexterity in patients with Prader–Willi syndrome: a pilot intervention study

Current and emerging therapies for managing hyperphagia and obesity in Prader‐Willi syndrome: A narrative review

Growth hormone therapy for Prader&ndash;Willi syndrome: challenges and solutions

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Product

Resources

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Growth hormone therapy for Prader–Willi syndrome: challenges and solutions