In the JournalsPerspective

Long-term GH therapy could slow course of Prader-Willi syndrome

Results from an 8-year study suggest that growth hormone therapy in children with Prader-Willi syndrome yielded long-term benefits, according to researchers.

“These results show that GH treatment in combination with a healthy lifestyle can counteract the clinical course of Prader-Willi syndrome [PWS], which is very important because development toward morbid obesity is a major threat to these patients,” Nienke Bakker, MD, of the Dutch Growth Research Foundation and Children’s Hospital Erasmus MC-Sophia in the Netherlands, and colleagues wrote.

The multicenter, prospective cohort study involved the examination of 60 prepubertal children, who were administered GH treatment (1 mg/m2 daily) between April 2002 and December 2005, enrolled in the Dutch PWS Cohort Study. Bakker and colleagues assessed body composition and anthropometric measurements to determine the effects of GH on the status of their condition.

After the first year of GH treatment, researchers reported a significant increase in lean body mass (P<.0001) that, for the next 7 years, remained stable at a level above baseline (P<.0001), they wrote.

BMI standard deviation score (SDS) decreased significantly after 8 years of GH treatment compared with baseline (P<.0001). Similarly after 8 years, height and head circumference SDS normalized.

"The clinical relevance of achieving normal head circumference is uncertain but parallels the positive effects of GH treatment on cognition," they wrote.

Additional data demonstrate significant decreases in SDS for total cholesterol (P=.005), LDL levels (P<.0001) and systolic blood pressure compared with baseline (P<.05).

Further, anthropometric measurements normalized after 8 years of treatment, they wrote.

The researchers reported an increase in insulin-like growth factor I SDS (from –1.83 to 2.36) during the first year (P<.0001). Similarly, this remained stable for the duration of the study.

Disclosure: This was an investigator-initiated study, supported by an independent research grant from Pfizer.

Results from an 8-year study suggest that growth hormone therapy in children with Prader-Willi syndrome yielded long-term benefits, according to researchers.

“These results show that GH treatment in combination with a healthy lifestyle can counteract the clinical course of Prader-Willi syndrome [PWS], which is very important because development toward morbid obesity is a major threat to these patients,” Nienke Bakker, MD, of the Dutch Growth Research Foundation and Children’s Hospital Erasmus MC-Sophia in the Netherlands, and colleagues wrote.

The multicenter, prospective cohort study involved the examination of 60 prepubertal children, who were administered GH treatment (1 mg/m2 daily) between April 2002 and December 2005, enrolled in the Dutch PWS Cohort Study. Bakker and colleagues assessed body composition and anthropometric measurements to determine the effects of GH on the status of their condition.

After the first year of GH treatment, researchers reported a significant increase in lean body mass (P<.0001) that, for the next 7 years, remained stable at a level above baseline (P<.0001), they wrote.

BMI standard deviation score (SDS) decreased significantly after 8 years of GH treatment compared with baseline (P<.0001). Similarly after 8 years, height and head circumference SDS normalized.

"The clinical relevance of achieving normal head circumference is uncertain but parallels the positive effects of GH treatment on cognition," they wrote.

Additional data demonstrate significant decreases in SDS for total cholesterol (P=.005), LDL levels (P<.0001) and systolic blood pressure compared with baseline (P<.05).

Further, anthropometric measurements normalized after 8 years of treatment, they wrote.

The researchers reported an increase in insulin-like growth factor I SDS (from –1.83 to 2.36) during the first year (P<.0001). Similarly, this remained stable for the duration of the study.

Disclosure: This was an investigator-initiated study, supported by an independent research grant from Pfizer.

    Perspective
    Moris A. Angulo

    Moris A. Angulo

    This study confirms again not only the growth promoting but the significant anabolic effect of GH treatment in children with PWS. It is somehow difficult in many children with PWS to find the right balance between GH dose, clinical response and serum IGF-1 levels. In the USA, GH has been approved for short stature/growth failure in children with PWS; therefore their response should be based on growth velocity and ideal body weight to calculate their GH dose. There is improvement on weight and body composition but to a lesser degree than growth velocity and final adult height, which normalizes after GH treatment. Considering the achievement of better weight and body composition as the main reason for GH treatment could mislead its use. As in any naïve GH-deficient child, GH response may vary regardless of their serum IGF-1 and extreme caution should be taken to prevent overtreatment and development of complications including type 2 diabetes and acromegaloid features.

    • Moris A. Angulo, MD
    • Director of Medical Genetics Associate Director of Pediatric Endocrinology Winthrop-University Hospital Mineola, N.Y.

    Disclosures: Angulo reports no relevant financial disclosures.

    See more from Spotlight on Prader-Willi syndrome