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Lionne N. Grootjen, Joost P.h.j. Rutges, Layla Damen, Stephany H. Donze, Alicia F. Juriaans, Gerthe F Kerkhof, and Anita Cs Hokken-Koelega

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.

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Layla Damen, Lionne N Grootjen, Stephany H Donze, Laura C G de Graaff, Janielle A E M van der Velden, and Anita C S Hokken-Koelega


In children with Prader–Willi syndrome (PWS), growth hormone (GH) treatment has positive effects on bone mineral density (BMD). Two 1-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.


Open-label, a prospective study in 43 young adults with PWS.


BMD of the total body (BMDTBSDS) and lumbar spine (BMADLSSDS) measured by DXA.


In the total group, estimated mean (95% CI) of 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, respectively (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).


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 recommended to continue GH treatment in young adults with PWS and to start SSRT during adolescence unless puberty progresses normally.