Meta-analysis confirms growth hormone therapy benefits in Prader–Willi syndrome

medwireNews: A meta-analysis confirms growth and body composition benefits for children with Prader–Willi syndrome (PWS) given recombinant human growth hormone (rhGH).

However, the researchers were unable to draw conclusions about its effects on cognition, because of inconsistent methods of assessing cognitive outcomes.

“To the best of our knowledge, this is the first meta-analysis that evaluated the use of rhGH in a large number of PWS patients while assessing different domains, allowing an overall vision of the impact of rhGH treatment”, say Caroline de Gouveia Buff Passone (Universidade de Sao Paulo, Brazil) and study co-authors.

The team identified 16 randomised, controlled trials involving 328 patients, as well as 20 nonrandomised studies (11 before and after studies and nine cohort studies) that provided 2909 patient–years of follow-up.

Patients taking rhGH in the randomised trials achieved an average improvement in height standard deviation score (SDS) of 1.67 relative to patients in the control group, and patients in the nonrandomised studies achieved an average 1.52 SDS improvement with rhGH treatment.

In addition, rhGH therapy resulted in an average BMI SDS reduction of 0.67 in the randomised trials. Patients in the nonrandomised trials had no change in BMI SDS, but the researchers stress that a stable BMI during follow-up is contrary to the natural disease course of PWS, which is characterised by increasing BMI.

Both randomised and nonrandomised studies reported a reduction in fat mass, by an average of 6.5% and 7.0%, respectively.

The study authors say that the height increase and BMI and fat reductions reported here are “not negligible in PWS daily life and can contribute [to] their fight against obesity and their complications.”

There was also some evidence for improvements in lean mass and head circumference in patients taking rhGH versus placebo. Reported adverse effects from the before and after studies, which had the longest follow-up, included sleep apnoea, initial headache and scoliosis.

The team had also wished to assess the impact of rhGH on patients’ mental development and IQ, but were unable to draw conclusions because of the inconsistent methods of assessing these endpoints.

“We are convinced that the elaboration of a specific questionnaire to evaluate the syndrome at each age would add great value for this analysis in future studies”, they write in BMJ Paediatrics Open.

However, they note that most studies reported improvements, especially for verbal and non-verbal IQ, and for vocabulary.

By Eleanor McDermid

medwireNews is an independent medical news service provided by Springer Healthcare. © 2020 Springer Healthcare part of the Springer Nature Group

BMJ Paedtr Open 2020; 4: e000630
Martin Savage
Programme Director
Sign up for eAlerts
Be the first to hear about new resources and content by signing up to receive our eAlerts.

We'd love your feedback!

Please complete this short 5-question survey to help improve the content offered on this website.
Please rank the following aspects of the program from best [1] to worst [4].