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GH therapy continuation in Prader-Willi syndrome beneficial
Continuation of growth hormone therapy in young adults with Prader-Willi syndrome who have reached adult height can help to reduce fat mass and increase lean body mass, study data show.
“The current generation of young adults with Prader-Willi syndrome is treated with GH during childhood and this has substantially changed the phenotype of these patients,” Renske J. Kuppens, MD, of Erasmus University Medical Center in the Netherlands, told Endocrine Today. “During childhood, GH treatment is prescribed to improve body composition. GH counteracts the natural course of increasing obesity, which has resulting in a new generation of children with Prader-Willi syndrome.”
Kuppens and colleagues conducted a 2-year, randomized, double blind, placebo-controlled crossover study in 27 young adults (mean age, 17.2 years; eight men) with Prader-Willi syndrome to determine the effects of GH therapy compared with placebo on body composition.
All participants were treated with GH during childhood and attained adult height. Participants were treated with daily subcutaneous injections of 0.67 mg/m2 GH (Genotropin, Pfizer) or placebo for 1 year, and then were crossed over to the alternative treatment for another year. DXA was used to measure body composition.
“When young adults have attained adult height, they have to stop GH treatment, which deteriorates their body composition,” Kuppens told Endocrine Today. “Up until now, there were no GH studies in this new generation of patients and there were no effective treatment options for adults with Prader-Willi syndrome to counteract the natural course of increasing obesity and its concomitant comorbidity.”
Mean fat mass increased (4.1 kg; P < .001) and lean body mass decreased (–0.9 kg; P = .069) during placebo, whereas GH yielded lower mean fat mass (–2.9 kg; P = .004) and higher lean body mass (1.5 kg; P = .005). Fat mass percent of the limbs and trunk both increased (P < .001 for both) and limb lean body mass decreased (P = .004) during placebo. During GH, fat mass percent of limbs (P < .001) and trunk (P = .007) both decreased and lean body mass of limbs increased (P < .001).
No serious adverse events were reported during the GH periods, whereas two serious adverse events and 12 adverse events occurred during the placebo periods.
“In this first 2-year, randomized, double-blind, placebo-controlled cross-over trial, we demonstrate that discontinuation of GH deteriorates body composition, while GH treatment maintains the improve body composition without safety concerns,” Kuppens told Endocrine Today. “Thus, GH-treated young adults with Prader-Willi syndrome who have attained adult height benefit from continuation of GH treatment. Our findings indicate that GH during 1 year is not only an effective, but also a safe and well-tolerated treatment, although more research regarding the long-term effects and optimal dosing is required.” – by Amber Cox
For more information:
Renske J. Kuppens, MD, can be reached at info@kindengroei.nl.
Disclosure: Kuppens reports no relevant financial disclosures. One researcher reports receiving an independent research grant from Pfizer.