Growth hormone therapy may improve sleep efficiency in Prader-Willi syndrome


With sleep apnea and abnormal sleep patterns characteristic of Prader-Willi syndrome, researchers in Sweden and Denmark have hypothesized that treatment with growth hormone may improve both for people with the disease. rare neurodevelopmental.

The rare, genetic, multisymptom neurodevelopmental disease, Prader-Willi syndrome (PWS) is characterized by muscle hypotonia, overeating, obesity, behavioral problems, cognitive impairment and endocrine deficiency, including hormone deficiency. growth (GH). Sleep is also affected by the rare disease.

Knowing the positive results of GH therapy on body composition, physical capacity and quality of life, and noting that PWS is characterized by abnormal body composition, the authors of a recent study hypothesized that the treatment could also have beneficial effects on sleep disturbances. associated with PWS, primarily sleep apnea and abnormal sleep patterns. Their results were published in The Journal of Clinical Endocrinology & Metabolism.

Their double-blind, randomized, placebo-controlled study lasted 1 year, after which all enrolled patients received 2 years of GH treatment. Initially, the 22 women and 15 men were randomized 1: 1 to receive treatment for 1 year with either GH (n = 19) or placebo (n = 18) and polysomnographic measurements were taken every 6 months. . Given the additional 2 years of GH treatment, the authors also used a mixed-effect regression model for comparison only among those who received GH for 3 years.

“There is little information available on sleep-related breathing disorders in adults with PWS and in particular with regard to treatment for GH,” the authors wrote. “The objectives of this study were to assess the effect of short and long term GH treatment on respiratory and sleep parameters.

At baseline, study participants had a median age of 29.5 (range, 16.0 to 41.6) years and classified as overweight, with a median body mass index of 27.0 (range, 17 , 9 to 41.9) kg / m2 for the placebo group and 27.3 (19.9-44.8) kg / m2 for the GH group. Median insulin-like growth factor 1 (IGF-1) levels were low, at 115 (range, 61.0-185.0) mcg / L; sleep efficiency (SE) for both groups was 89.0% (range, 41.0% -99.0%); and the median hypopnea apnea index (IAH) was 1.4 (range 0.0-13.9).

After one year, the following changes were noted:

  • IGF-1 jumped to 165 (range, 69-257) mcg / L for the GH group while remaining at the level for the placebo group (P = .013).
  • Respiration and sleep parameters (AHI, longest apnea, periodic limb movements, oxygen saturation, IGF-1 levels, total desaturations, REM sleep and latency, SE, delta sleep) did not differ between groups. .

After the extended 2-year period of treatment with GH for everyone, here are the results:

  • Among those who received GH for 3 years (main study + 2-year extension), IGF-1 levels increased from 104 mcg / L at baseline to 178 mcg / L, an increase of 71%.
  • The SE decreased from a median of 86.7% (range, 58.5% -99.9%) to 91.0% (range, 57.0% -100.0%).
  • The AHI tended to increase to 2.4 from 2.0, but was considered a significant intermittent association with baseline.
  • Desaturations increased inconsistently, despite adjusting body mass index.

Study treatment consisted of either GH or placebo, 0.3 or 0.4 mg / day if body weight was less or greater than 100 kg, during the first 4 weeks of the study; increasing the dosage to 0.6 or 0.8 mg / day, respectively, for people below or above 100 kg, and maintain it for the next 11 months; and for the 2-year period, titrating the dose of GH according to the IGF-1 levels of controls of the same age.

Overall, the SE increased steadily from the 6 month mark for those who received continuous GH treatment for 3 years, but other parameters did not change, the authors said, and no significant difference was observed in the breathing or sleep parameters for the 1st one-year trial period.

Noting that respiratory disease can be a common, sometimes sudden, cause of death in people with PWS, the authors noted that all adults with the disease may benefit from treatment with GH, suggesting that “treatment with GH increases muscle strength, which would result in less interrupted sleep and increased SE, ”and therapy can be safely initiated in children. Both groups would also need regular sleep analysis.

“GH treatment in adults with PWS has many positive effects, and our study showed that GH treatment is safe and concerns about a negative impact on sleep-related parameters could not be addressed. confirmed, ”they concluded. “Although further studies in this context are warranted, our results add to the confidence in the treatment of GH in adults with PWS. “


Shukur HH, Hussain-Alkhateeb L, FArholt T, Nørregaard O, Jøgensen AP, Hoybye C. Effects of growth hormone treatment on sleep parameters in adults with Prader-Willi syndrome. J Clin Endocrinol Metab. 2021; 106 (9): e3634-e3643. doi: 10.1210 / clinem / dgab300

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