Time for a general approval of growth hormone treatment in adults with Prader–Willi syndrome

Höybye, Charlotte; Holland, Anthony; Driscoll, Daniel J.

doi:10.1186/s13023-020-01651-x

Cited by 15 publications

(18 citation statements)

References 21 publications

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“…Unfortunately, this may not always be possible as growth hormone treatment is not available for all adults with PWS. Based on our data on the combined effect of growth hormone treatment and multidisciplinary care, supported by previously reported beneficial effects of GH treatment alone in both children and adults [ 43 , 44 , 45 , 46 , 47 , 48 , 49 , 50 , 51 , 52 , 53 , 68 , 74 , 75 , 76 , 77 , 78 , 79 , 80 , 81 , 82 , 83 , 84 , 85 , 86 , 87 , 88 , 89 ], we support the pledge by Hoybye et al for general approval of growth hormone treatment in adults with PWS [ 94 ].…”

Section: Discussionsupporting

confidence: 85%

Effects of Childhood Multidisciplinary Care and Growth Hormone Treatment on Health Problems in Adults with Prader-Willi Syndrome

Pellikaan

Rosenberg

Davidse

et al. 2021

JCM

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Prader-Willi syndrome (PWS) is a complex hypothalamic disorder. Features of PWS include hyperphagia, hypotonia, intellectual disability, and pituitary hormone deficiencies. The combination of growth hormone treatment and multidisciplinary care (GHMDc) has greatly improved the health of children with PWS. Little is known about the effects of childhood GHMDc on health outcomes in adulthood. We retrospectively collected clinical data of 109 adults with PWS. Thirty-nine had received GHMDc during childhood and adolescence (GHMDc+ group) and sixty-three had never received growth hormone treatment (GHt) nor multidisciplinary care (GHMDc− group). Our systematic screening revealed fewer undetected health problems in the GHMDc+ group (10%) than in the GHMDc− group (84%). All health problems revealed in the GHMDc+ group had developed between the last visit to the paediatric and the first visit to the adult clinic and/or did not require treatment. Mean BMI and the prevalence of diabetes mellitus type 2 were significantly lower in the GHMDc+ group compared to the GHMDc− group. As all patients who received GHt were treated in a multidisciplinary setting, it is unknown which effects are the result of GHt and which are the result of multidisciplinary care. However, our data clearly show that the combination of both has beneficial effects. Therefore, we recommend continuing GHMDc after patients with PWS have reached adult age.

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Section: Discussionsupporting

confidence: 85%

Effects of Childhood Multidisciplinary Care and Growth Hormone Treatment on Health Problems in Adults with Prader-Willi Syndrome

Pellikaan

Rosenberg

Davidse

et al. 2021

JCM

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“…Recently, the global community of PWS experts has advocated for approval of GHt in adults with PWS ( 36 ). Although several review articles on GHt in PWS have been published, a meta-analysis is crucial to stress the importance of GHt in adults with PWS.…”

mentioning

confidence: 99%

Growth Hormone Treatment for Adults With Prader-Willi Syndrome: A Meta-Analysis

Rosenberg

Passone

Pellikaan

et al. 2021

The Journal of Clinical Endocrinology &Amp; Metabolism

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Context Features of Prader-Willi syndrome (PWS) overlap with features of growth hormone (GH) deficiency, like small hands and feet, short stature, increased body fat and low muscle mass and strength. In children with PWS, GH treatment (GHt) improves physical health and cognition. GHt has become standard of care in PWS children, but in adults this is not yet the case. Objective To provide an overview of the current knowledge on GHt in PWS adults. Data source Medline, Embase and Cochrane Central Register of Controlled Trials databases. Study selection Randomized controlled trials (RCTs) and non-randomized (un)controlled trials (NRCTs) that reported data for adults with PWS, who received GHt for at least six months. Data extraction Data on body composition, body mass index (BMI), cardiovascular endpoints, bone, cognitive function, quality of life and safety were extracted. Data synthesis Nine RCTs and 20 NRCTs were included. Body composition improved during 12 months of GHt with an increase in mean (95% CI) lean body mass of 1.95 kg (0.04 – 3.87 kg), and a reduction of mean (95% CI) fat mass of -2.23% (-4.10% to -0.36%). BMI, low-density lipoprotein cholesterol levels, fasting glucose levels and bone mineral density did not change during GHt. There were no major safety issues. Conclusion GHt appears to be safe and improves body composition in adults with PWS. As poor body composition is closely linked to the observed high incidence of cardiovascular morbidity in adults with PWS, improving body composition might reduce cardiovascular complications in this vulnerable patient group.

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Section: Growth Hormone Treatmentmentioning

confidence: 99%

“…Growth hormone (GH) treatment is for more than three decades a well-established and approved treatment in children with PWS in many countries (2,16,17). In contrast, PWS is an approved indication for GH treatment in the transition period or for adults only in a few countries (2,17). Therefore, in most countries the diagnosis of adult GH deficiency must be established according to guidelines before it can be used in the transition period and in adults with PWS.…”

Section: Growth Hormone Treatmentmentioning

confidence: 99%

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The transition from pediatric to adult care in individuals with Prader-Willi syndrome

Poitou

Holland

Höybye

et al. 2023

Endocrine Connections

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Prader-Willi syndrome (PWS), the most common form of syndromic obesity, is a complex neurodevelopmental genetic disorder including obesity with hyperphagia, endocrine and metabolic disorders and also psychiatric disorders. The most frequent endocrine disturbances include hypogonadism and growth hormone (GH) deficiency. Hypothyroidism and central adrenal insufficiency can also be observed but are less frequent. The transition of patients with PWS from adolescence to adult life is challenging because of multiple comorbidities and complex disability. Patients and caregivers face psychological, medical and social issues. This period of profound changes is thus prone to disruptions, the main risks being the worsening of the medical situation and loss to follow-up of the patients. Medical care may be poorly adapted to the needs of patients because of a lack of knowledge concerning the syndrome and also lack of the necessary specific skills. A multidisciplinary panel composed of several experts in PWS met in November 2021 during an endo-ERN webinar. They presented complementary aspects of PWS from the perspective of the transition including psychiatric, pediatrics and adult endocrinological and parent’s and patient’s point of view and shed light on the best way to approach this pivotal period.

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Time for a general approval of growth hormone treatment in adults with Prader–Willi syndrome

Cited by 15 publications

References 21 publications

Effects of Childhood Multidisciplinary Care and Growth Hormone Treatment on Health Problems in Adults with Prader-Willi Syndrome

Effects of Childhood Multidisciplinary Care and Growth Hormone Treatment on Health Problems in Adults with Prader-Willi Syndrome

Growth Hormone Treatment for Adults With Prader-Willi Syndrome: A Meta-Analysis

The transition from pediatric to adult care in individuals with Prader-Willi syndrome

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