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

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Charlotte Höybye ◽  
◽  
Anthony J. Holland ◽  
Daniel J. Driscoll
Keyword(s):  
Growth Hormone ◽  
Body Composition ◽  
Transition Period ◽  
Neurodevelopmental Disorder ◽  
Hormone Treatment ◽  
Psychomotor Development ◽  
Prader Willi Syndrome ◽  
Gh Treatment ◽  
Beneficial Effects ◽  
Positive Effects

AbstractPrader-Willi syndrome (PWS) is a complex, multi-system, neurodevelopmental disorder characterised by neonatal muscular hypotonia, short stature, high risk of obesity, hypogonadism, intellectual disabilities, distinct behavioural/psychiatric problems and abnormal body composition with increased body fat and a deficit of lean body mass. Growth hormone (GH) deficiency and other hormone deficiencies are common due to hypothalamic dysfunction. In children with PWS GH treatment has been widely demonstrated to improve body composition, normalise height and improve psychomotor development. In adults with PWS, GH’s main effects are to maintain normal body structure and metabolism. The positive effects of GH treatment on body composition, physical fitness and beneficial effects on cardiovascular risk markers, behaviour and quality of life in adults with PWS are also well established from several studies. GH treatment is approved for treatment of children with PWS in many countries, but until recently not as a treatment in young adults in the transition period or for adults in general. In this commentary we want to draw attention to the uneven global use of GH treatment, specifically in adults with PWS, and advocate for GH treatment to be approved internationally, not just for children, but also for adults with PWS and based only on the diagnosis of genetically confirmed PWS.

scholarly journals Growth hormone treatment for adults with Prader-Willi syndrome: another point of view

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Harry J. Hirsch ◽  
Varda Gross-Tsur
Keyword(s):  
Growth Hormone ◽  
Body Composition ◽  
Motor Development ◽  
Growth Hormone Treatment ◽  
Genetic Diagnosis ◽  
Hormone Treatment ◽  
Prader Willi Syndrome ◽  
Positive Effects ◽  
Long Term Treatment ◽  
Risk Of Cancer

AbstractGrowth hormone treatment for children with Prader Willi syndrome (PWS) has shown proven benefits not only in increasing final height but also with positive effects on body composition and motor development. In a recent letter to the editor, Hoybye and colleagues recommend growth hormone treatment for adults with PWS based exclusively on the genetic diagnosis and without regard for growth hormone secretory status. We question whether the benefits of growth hormone treatment in PWS adults, mainly improvement in body composition, are significant enough to justify the as yet unkown consequences of long-term treatment in an adult population. Morbidity and mortality in PWS are mainly due to complications of obesity, and growth hormone treatment does not result in a decrease in BMI or waist circumference. Increases in insulin-like factor-1 as a result of growth hormone treatment over the course of several decades in PWS adults raises concern over possible increase risk of cancer. Compliance with daily injections is likely to be poor. We suggest that efforts to provide appropriate dietary and exercise regimens may be more beneficial and cost-effective than advocating for growth hormone treatment for adults with PWS.

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

2021 ◽  
Vol 10 (15) ◽  
pp. 3250
Author(s):  
Karlijn Pellikaan ◽  
Anna G. W. Rosenberg ◽  
Kirsten Davidse ◽  
Anja A. Kattentidt-Mouravieva ◽  
Rogier Kersseboom ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Growth Hormone Treatment ◽  
Multidisciplinary Care ◽  
Hormone Treatment ◽  
Health Problems ◽  
Pituitary Hormone ◽  
Childhood And Adolescence ◽  
Prader Willi Syndrome ◽  
Adult Age ◽  
Beneficial Effects

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.

Effects of growth hormone treatment on growth and body composition in Prader-Willi syndrome: a preliminary report

1997 ◽  
Vol 86 (S423) ◽  
pp. 60-62 ◽  
Author(s):  
AC Lindgren ◽  
L. Hagenäs ◽  
J. Müller ◽  
S. Blichfeldt ◽  
M. Rosenborg ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Body Composition ◽  
Preliminary Report ◽  
Growth Hormone Treatment ◽  
Hormone Treatment ◽  
Prader Willi Syndrome

scholarly journals Cognitive functioning in children with Prader–Willi syndrome during 8 years of growth hormone treatment

2020 ◽  
Vol 182 (4) ◽  
pp. 405-411
Author(s):  
S H Donze ◽  
L Damen ◽  
E F Mahabier ◽  
A C S Hokken-Koelega
Keyword(s):  
Growth Hormone ◽  
Cognitive Development ◽  
Cognitive Functioning ◽  
Block Design ◽  
Hormone Treatment ◽  
Prader Willi Syndrome ◽  
Short Term ◽  
Intelligence Scale ◽  
Positive Effects

Objective Children with Prader–Willi syndrome (PWS) have mild to moderate cognitive impairment. Short-term studies showed positive effects of growth hormone (GH) on cognitive development. This study investigated the effects of 8 years of GH on cognitive development in children with PWS. We also investigated whether starting GH during infancy results in higher cognitive functioning after 8 years of GH. Design Longitudinal study in 43 children with PWS during 8 years of GH (median age at GH start 8.1 years). Cognitive functioning after 8 years was compared to another group of 22 children with PWS (median age at GH start 1.4 years). Methods Cognitive functioning was measured by Wechsler Intelligence Scale for Children. Vocabulary, Similarities and Block Design subtests were expressed as standard deviation scores (SDS) and total IQ (TIQ) calculated. Results Estimated mean (95%CI) Block Design SDS changed from −2.2 (−2.6; −1.8) at GH start to −1.8 (−2.2; −1.4) after 8 years of GH (P = 0.18), similarly SDS from −1.5 (−2.1; −0.9) to −1.3 (−1.9; −0.7, P = 0.66) and TIQ from 66 (60; 72) to 69 (63; 75, P = 0.57). Vocabulary SDS remained similar, being −1.9 (−2.3; −1.4) at GH start and −1.9 (−2.4; −1.5) after 8 years (P = 0.85). After 8 years of GH Vocabulary, SDS and TIQ were higher in the children who started GH during infancy, compared to those who started GH later in childhood (P < 0.01, P = 0.04, respectively). Conclusions Cognitive functioning in children with PWS remains similar during long-term GH and develops at the same pace as healthy peers.

O0055 IMPACT OF LONG TERM GROWTH HORMONE TREATMENT ON BODY COMPOSITION IN PRADER-WILLI SYNDROME (PWS)

2004 ◽  
Vol 39 (Supplement 1) ◽  
pp. S28-S29
Author(s):  
A. O. Scheimann ◽  
W. J. Klish ◽  
B. Hayslett ◽  
N. Lafuente ◽  
D. Orellana ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Body Composition ◽  
Growth Hormone Treatment ◽  
Hormone Treatment ◽  
Prader Willi Syndrome

Growth hormone treatment and bone mineral density in pediatric patients with Prader–Willi syndrome

2021 ◽  
Vol 0 (0) ◽  
Author(s):  
Yuji Oto ◽  
Nobuyuki Murakami ◽  
Takeshi Inoue ◽  
Keiko Matsubara ◽  
Sohei Saima ◽  
...  
Keyword(s):  
Bone Mineral Density ◽  
Growth Hormone ◽  
Bone Mineral ◽  
Pediatric Patients ◽  
Standard Deviation Score ◽  
Hormone Treatment ◽  
Prader Willi Syndrome ◽  
Mineral Density ◽  
Gh Treatment ◽  
Total Body

Abstract Objectives Previous reports indicate that growth hormone (GH) treatment for Prader–Willi syndrome (PWS) improves bone mineral density (BMD) only when initiated at a young age and not when initiated in adulthood. However, there are no data on BMD during long-term GH treatment of Japanese children and adolescents with PWS. Thus, this study aimed to investigate BMD changes among patients with PWS, who were undergoing GH treatment from childhood to adolescence. Methods Sixty-seven pediatric patients with PWS who had GH treatment initiated during childhood between January 2003 and June 2020 were evaluated. To avoid underestimation, we used total body BMD, which was evaluated using dual-X-ray absorptiometry adjusted for the BMD z-score using patient height, sex, and age. Results In both sexes, age was negatively correlated with the BMD-standard deviation score (SDS) (male: r=−0.156 [p=0.042]; female: r=−0.197 [p=0.043]), which started to decrease in childhood. Conclusions The BMD-SDS of patients with PWS decreases gradually despite GH treatment. As there are no clear recommendations about monitoring of bone health in patients with PWS, further studies are needed to improve the guidelines for screening of BMD and treatment of patients with PWS.

Beneficial Effects of Long-Term Growth Hormone Treatment on Adaptive Functioning in Infants With Prader-Willi Syndrome

2015 ◽  
Vol 120 (4) ◽  
pp. 315-327 ◽  
Author(s):  
Sin T. Lo ◽  
Dederieke A. M. Festen ◽  
Roderick F. A. Tummers-de Lind van Wijngaarden ◽  
Philippe J. L. Collin ◽  
Anita C. S. Hokken-Koelega
Keyword(s):  
Growth Hormone ◽  
Growth Hormone Treatment ◽  
Adaptive Functioning ◽  
Controlled Trial ◽  
Hormone Treatment ◽  
Repeated Measurements ◽  
Prader Willi Syndrome ◽  
Beneficial Effects ◽  
Lower Intelligence

Abstract The aim of this study was to investigate the effect of growth hormone treatment on adaptive functioning in children with Prader-Willi syndrome. Vineland Adaptive Behavior Scale (VABS) was assessed during a randomized controlled trial (RCT) and after 7 years of growth hormone treatment. In the RCT, 75 children (42 infants and 33 prepubertal children) with Prader-Willi syndrome were included. Subsequently, 53 children were treated with long-term growth hormone. Our study demonstrates a marked delay in adaptive functioning in infants and children with Prader-Willi syndrome, which was associated with older age and lower intelligence. Results of the repeated measurements show that the earlier growth hormone treatment was started during infancy, the better the adaptive skills were on the long-term.

scholarly journals Diurnal Variations in Twenty-Four-Hour Energy Expenditure During Growth Hormone Treatment of Adults with Pituitary Deficiency*

1997 ◽  
Vol 82 (4) ◽  
pp. 1255-1260
Author(s):  
Kaj Stenlöf ◽  
Jan-Ove Johansson ◽  
Lars Lönn ◽  
Lars Sjöström ◽  
Bengt-Åke Bengtsson
Keyword(s):  
Growth Hormone ◽  
Body Composition ◽  
Growth Factor ◽  
Energy Expenditure ◽  
Metabolic Rate ◽  
Hormone Treatment ◽  
Insulin Like Growth Factor ◽  
Free T4 ◽  
Gh Treatment ◽  
Procollagen Iii Peptide

Abstract The effects of growth hormone (GH) treatment on 24-h energy expenditure (EE) were studied in a open trial over a period of 4 weeks. Five subjects, four men and one woman, with a history of complete GH deficiency were included. All the subjects were examined on 2 consecutive days on baseline and, thereafter, at six occasions during a period of 1 month (days 1, 2, 5, 8, 15, and 30). The dose of GH was 0.25 U/kg·week, administered sc once a day in the evening. EE was determined in a chamber for indirect calorimetry. Body composition was determined with dual-energy x-ray absorptiometry and computed tomography using a four-scan technique. Blood samples were examined using well-established RIAs. During the first 2 weeks, 24-h EE increased by 6 ± 3% (range 1–8%) from 40.9 ± 4.8 to 42.9 ± 4.8 kcal/24 h·kg (P &lt; 0.05), sleeping metabolic rate by 14 ± 3% (range 10–18%) from 28.4 ± 1.9 to 32.9 ± 2.2 kcal/24h·kg (P &lt; 0.001), and basal metabolic rate by 11 ± 7% (range 0–18%) from 29.6 ± 2.4 to 33.3 ± 2.6 kcal/24h·kg (P &lt; 0.05). No change was found in daytime EE. The increase in EE covaried with changes in insulin-like growth factor 1, the free T3/free T4 ratio, insulin-like growth factor-binding protein-3, and the aminoterminal procollagen III peptide but not with changes in body composition. It is suggested that the stimulating effect of GH on EE occurs gradually during a 2-week period and is only detectable during night and morning hours, when significant levels of GH occur.

Sign in / Sign up

Export Citation Format

Share Document