Effects of 8 years of growth hormone treatment on scoliosis in children with Prader-Willi syndrome

2021 ◽  
Author(s):  
Lionne N. Grootjen ◽  
Joost P.h.j. Rutges ◽  
Layla Damen ◽  
Stephany H. Donze ◽  
Alicia F. Juriaans ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Lumbar Spine ◽  
Adverse Effects ◽  
Cobb Angle ◽  
Bone Mineral ◽  
Prader Willi Syndrome ◽  
X Rays ◽  
Mineral Density ◽  
Gh Treatment ◽  
Bone Mineral Apparent Density

Objective: Scoliosis is frequently seen in children with Prader-Willi syndrome (PWS). There is still concern that growth hormone (GH) treatment might increase the risk of onset or progression of scoliosis. Short-term data suggested no adverse effects of GH on scoliosis, but long-term effects of GH treatment on development of scoliosis in PWS are unknown. This study investigated the effects of 8 years of GH treatment on scoliosis in children with PWS. Design: Open-label, prospective cohort study in 103 children with PWS receiving GH for eight years. Prevalence and severity of scoliosis were compared to a group of 23 age-matched GH untreated children with PWS. Methods: Spine X-rays and DEXA-scans were performed, and Cobb angel was measured by two independent observers. Results: After 8 years of GH treatment, at median age of 10.8 years, prevalence of scoliosis was 77.7%. No difference in prevalence or severity of scoliosis was found between GH-treated and age-matched untreated children with PWS (P=0.409 and p=0.709, respectively). Height SDS and trunkLBM were significantly higher in GH-treated children. Higher bone mineral density of the lumbar spine was found in children without scoliosis after 8 years of GH. Bone mineral apparent density of lumbar spine (BMADLS) SDS was associated with lower Cobb angle (r=-0.270, p=0.008). Conclusions: Eight years of GH treatment has no adverse effects on prevalence and severity of scoliosis in children with PWS until 11 years of age. As BMADLS SDS is inversely associated with Cobb angle, it is pivotal to optimize BMD-status in children with PWS.

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.

Gaucher disease: alendronate disodium improves bone mineral density in adults receiving enzyme therapy

Blood ◽  
2004 ◽  
Vol 104 (5) ◽  
pp. 1253-1257 ◽  
Author(s):  
Richard J. Wenstrup ◽  
Laurie Bailey ◽  
Gregory A. Grabowski ◽  
Jay Moskovitz ◽  
Alan E. Oestreich ◽  
...  
Keyword(s):  
Bone Mineral Density ◽  
Lumbar Spine ◽  
Bone Mineral ◽  
Gaucher Disease ◽  
Bone Lesions ◽  
X Rays ◽  
Enzyme Therapy ◽  
Mineral Density ◽  
Focal Lesions ◽  
Enzyme Replacement

Abstract Symptomatic patients with Gaucher disease (GD) (acid β-glucosidase [Gcase] deficiency) are treated with injectable human recombinant GCase. Treatment results in significant decreases in lipid storage in liver, spleen, and bone marrow, but the generalized osteopenia and focal bone lesions present in many adult patients are refractory to treatment. A double-blind, 2-arm, placebo-controlled trial of alendronate (40 mg/d) was performed in adults with GD who had been treated with enzyme for at least 24 months. Primary therapeutic endpoints were improvements in (1) bone mineral density (BMD) and content (BMC) at the lumbar spine, and (2) focal lesions in x-rays of long bones assessed by a blinded reviewer. There were 34 patients with GD type 1 (age range, 18-50 years) receiving enzyme therapy who were randomized for this study. After 18 months, ΔBMD at the lumbar spine was 0.068 ± 0.21 and 0.015 ± 0.034 for alendronate and placebo groups, respectively (P = .001). Long-bone x-rays showed no change in focal lesions or bone deformities in any subject in either arm. Alendronate is a useful adjunctive therapy in combination with enzyme replacement therapy (ERT) for the treatment of GD-related osteopenia in adults, but it cannot be expected to improve focal lesions.

Bone Mineral Density during 3 years of Growth Hormone in previously GH-treated Young Adults with PWS

2021 ◽  
Author(s):  
Layla Damen ◽  
Lionne N. Grootjen ◽  
Stephany H. Donze ◽  
Laura C.g. de Graaff ◽  
Janielle A.e.m. van der Velden ◽  
...  
Keyword(s):  
Bone Mineral Density ◽  
Growth Hormone ◽  
Young Adults ◽  
Bone Mineral ◽  
Mineral Density ◽  
Open Label ◽  
Gh Treatment ◽  
Positive Effects ◽  
Total Body ◽  
One Year

Objective: In children with Prader-Willi syndrome (PWS), growth hormone (GH) treatment has positive effects on bone mineral density (BMD). Two one-year studies did not show a difference between GH or placebo on BMD in young adults with PWS. However, there are no studies investigating BMD during longer-term GH treatment in young adults with PWS. Design: Open-label, prospective study in 43 young adults with PWS. Methods: BMD of the total body (BMDTBSDS) and lumbar spine (BMADLSSDS) measured by DXA. Results: In the total group, estimated mean (95% CI) BMDTB remained similar during 3 years of GH, being -0.76 (-1.11 to -0.41) SDS at start and -0.90 (-1.27 to -0.54) SDS after 3 years (p=0.11), as did BMADLS, being -0.36 (-0.72 to 0.01) SDS and -0.46 (-0.77 to -0.16) SDS resp. (p=0.16). In men, there was a significant decrease in BMDTBSDS during 3 years of GH, while BMADLSSDS remained similar. In women, both BMDTBSDS and BMADLSSDS remained similar. BMDTBSDS was associated with female sex, lean body mass and age. The majority of patients received sex steroid replacement therapy (SSRT). Conclusions: During 3 years of combined GH and SSRT treatment, BMD remained stable in the normal range in young adults with PWS. However, men showed a decline in BMDTBSDS, probably due to insufficient SSRT. We recommend to continue GH treatment in young adults with PWS and to start SSRT during adolescence unless puberty progresses normally.

scholarly journals Changes in Bone Mineral Density, Body Composition, and Lipid Metabolism during Growth Hormone (GH) Treatment in Children with GH Deficiency1

1997 ◽  
Vol 82 (8) ◽  
pp. 2423-2428 ◽  
Author(s):  
Annemieke M. Boot ◽  
Melanie A. M. J. Engels ◽  
Geert J. M. Boerma ◽  
Eric P. Krenning ◽  
Sabine M. P. F. de Muinck Keizer-Schrama
Keyword(s):  
Bone Mineral Density ◽  
Growth Hormone ◽  
Body Composition ◽  
Lipid Metabolism ◽  
Bone Mineral ◽  
Mineral Density ◽  
Gh Treatment

Reduced bone mineral density in adults with growth hormone (GH) deficiency: increased bone turnover during 12 months of GH substitution therapy

1995 ◽  
Vol 133 (2) ◽  
pp. 180-188 ◽  
Author(s):  
Marie Degerblad ◽  
Bengt-Åke Bengtsson ◽  
Margareta Bramnert ◽  
Olof Johnell ◽  
Per Manhem ◽  
...  
Keyword(s):  
Bone Mineral Density ◽  
Growth Hormone ◽  
Bone Turnover ◽  
Bone Mineral ◽  
Gh Deficiency ◽  
Serum Markers ◽  
Substitution Therapy ◽  
Mineral Density ◽  
Gh Treatment ◽  
Total Body

Degerblad M, Bengtsson B-Å, Bramnert M, Johnell O, Manhem P, Rosén T, Thorén M. Reduced bone mineral density in adults with growth hormone (GH) deficiency: increased bone turnover during 12 months of GH substitution therapy. Eur J Endocrinol 1995;133:180–8. ISSN 0804–4643 To evaluate the consequences of growth hormone (GH) deficiency on bone mineral density and to evaluate the effects of GH substitution therapy, 68 adults (25 females and 43 males) aged 22–61 (mean 44.2 ± 1.2) years with GH deficiency (GHD) were studied. Fifty-eight patients had panhypopituitarism, three had isolated GHD and in seven patients at least one additional pituitary function was affected. Twenty-one patients had childhood onset GHD. The patients were randomized to receive either GH in daily injections (0.125 IU · kg−1 · week−1 for the first 4 weeks and subsequently 0.25 IU · kg−1 · week−1) or placebo for 6 months. The trial continued as an open study with GH treatment for 6 or 12 months, with data presented as compiled data of 12 months of GH treatment in 64 patients. Bone mineral density (BMD) was measured by dual energy x-ray absorptiometry and bone turnover was assessed by serum markers of bone metabolism (osteocalcin, procollagen I peptide, crosslinked telopeptide of type I collagen and alkaline phosphatase activity). In women with adult onset GHD (N = 19) and in men with childhood onset GHD (N = 15), total body, spine and hip BMD was significantly reduced at baseline compared to Swedish age- and sex-matched control material. In men with adult onset of GHD (N = 28), BMD did not differ from male controls. During the placebocontrolled period, GH induced decreased total body and spine BMD, probably due to an expansion of the remodelling space, whereas all serum markers of bone turnover increased. Compiled GH data showed similar results after 6 months of treatment. After 12 months of GH treatment, BMD did not differ from basal values except for total body BMD, which was lower, whereas the serum markers of bone metabolism were still increased as compared to basal values. Two-thirds of the patients experienced fluid retention with peripheral oedema and arthralgias on the higher GH dosage. One obese patient developed non-insulin-dependent diabetes mellitus and was withdrawn from the study. These results demonstrate that GHD has negative effects on BMD and that GH substitution induces increased bone turnover. Continued long-term observations will reveal if there is a positive effect of GH substitution on bone mass in the adult GHD patient. Marie Degerblad, Department of Endocrinology and Diabetology, Karolinska Hospital, S-17176 Stockholm, Sweden

scholarly journals Growth hormone treatment for adults with Prader-Willi syndrome: a meta-analysis

2021 ◽  
Author(s):  
Anna G W Rosenberg ◽  
Caroline De Gouveia Buff Passone ◽  
Karlijn Pellikaan ◽  
Durval Damiani ◽  
Aart J Van Der Lely ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Body Composition ◽  
Body Mass ◽  
Current Knowledge ◽  
Controlled Trials ◽  
Cochrane Central Register ◽  
Prader Willi Syndrome ◽  
Mineral Density ◽  
Gh Treatment ◽  
Hands And Feet

Abstract 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.

Sign in / Sign up

Export Citation Format

Share Document