GH therapy effective, safe for adults with Prader-Willi syndrome
Growth hormone therapy is safe for adults with Prader-Willi syndrome and leads to increased lean body mass and decreased fat mass, according to systemic review data published in Clinical Endocrinology.
“The use of GH therapy in adults has shown favorable outcomes, particularly in body composition, without significant changes in bone and cardiovascular health in the short term,” M. Guftar Shaikh, MD, FRCPCH, consultant pediatric endocrinologist and associate professor in the developmental endocrinology research group at the Royal Hospital for Children, University of Glasgow in Scotland, U.K., and colleagues wrote. “No major adverse events were seen, and therefore, overall, the use of GH therapy in adults has been shown to be beneficial and safe.”
Researchers conducted a systemic review of studies examining GH treatment in adults with Prader-Willi syndrome. Studies were obtained through a PubMed database search of literature published from 1960 to March 2020. Changes in bone health, CV health and body composition from baseline and during treatment to after treatment were analyzed. Researchers also looked at changes in lean body mass, BMI and fat mass.
The review included 20 studies with 364 unique patients (median age, 26.2 years). The median dose of GH in the study population was 0.8 mg per day. The median duration of treatment was 1 year, and the studies had a median follow-up time of 2 years.
Of 16 studies that included BMI, 13 showed no significant change in BVMI for GH-treated patients, two revealed an increase in BMI and one study showed lower BMI. Lean body mass was included in 15 studies, with all showing an increase with GH treatment. In 16 studies that assessed fat mass, 12 showed a decrease with GH.
In an analysis of CV health, two studies showed improvements in cholesterol levels with GH, whereas two others showed no improvement. Two studies reported an increase in cardiorespiratory exercise capacity. In nine studies that included blood pressure, none showed any significant changes. Five studies evaluating bone health showed no statistically significant changes. In 13 studies reporting on glucose control and diabetes, 10 showed no participants had impaired glucose or developed diabetes during the study period.
Of 13 studies in which adverse events were mentioned, seven reported events in 172 participants. None of the adverse events was major, and 43 minor adverse events were reported.
The researchers wrote that the review data show GH therapy is likely to result in favorable outcomes for adults with Prader-Willi syndrome, but longitudinal studies must be conducted to confirm findings for those receiving long-term treatment, as most of the studies included in the review had a short follow-up period.
“GH therapy appears to be well tolerated and should be considered in adults with Prader-Willi syndrome but only in specialist centers with access to a multidisciplinary team,” the researchers wrote. “Where GH therapy is used in adults, regular monitoring should be initiated, with particular emphasis on echocardiograms, oral glucose tolerance tests and insulin-like growth factor I and cholesterol measurements.”
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