In this 6-minute video, Drs. Theresa Strong and Caroline Vrana-Diaz review data and findings from the PWS Weight Study conducted by the Foundation for Prader-Willi Research. This study has been published in the Orphanet Journal of Rare Diseases.
Understanding how weight changes over time in Prader-Willi syndrome is important for capturing the contemporary natural history of PWS as well as assessing the impact of new treatments for hyperphagia. The PWS Weight Study was a text message-based study that included 165 adolescents and adults with PWS to assess changes in weight and body mass index (BMI) over a six-month period.
There was considerable variability in weight across participants with 33% percent of the participants at a normal weight, 15% overweight and 52% obese. Weight was collected weekly, while changes in height, living situation, access to food, activity level, and medication were collected at three-month intervals.
Over the course of the 6-months of the study, small increases in weight and BMI were observed. Overall, participant weight increased over the study period by + 2.35%, while BMI was relatively stable, albeit high. Changes in living situation, activity, food access, and medication had limited impact on weight and BMI changes however, the percentage of life on growth hormone (GH) therapy statistically improved weight and BMI in an individual.
We anticipate that the results of this PWS research study will inform clinical trials for hyperphagia/obesity related therapies in PWS and provide a basis for understanding the efficacy of new therapies for hyperphagia in the real-world setting.