The Hyperphagia Questionnaire for Clinical Trials (HQ-CT) is typically completed by caregivers for individuals with PWS who are enrolled in clinical trials. The questionnaire is designed to evaluate changes in hyperphagia during the course of the trial. Despite the fact that the HQ-CT has been used in more than a dozen PWS clinical trials, the range of scores in the larger PWS population wasn’t known, nor could the PWS population be compared to typically developing individuals. So, what is a typical score, and how different is it compared to the scores of those with PWS?
To understand this, we needed caregivers to complete this questionnaire for their typically developing children and young adults, and in the true fashion of the PWS community, your support shined through! We asked you to share our request with family and friends, and we received a resounding response, which allowed us to publish a new paper examining hyperphagia scores in the PWS population and in typically developing individuals. The full paper can be found here.
The original hyperphagia questionnaire was developed by Dr. Elisabeth Dykens at Vanderbilt University and was adapted by Zafgen specifically for PWS clinical trials (HQ-CT), then donated to FPWR. In this study, we were able to collect HQ-CT scores from more than 400 individuals with PWS and more than 600 typically developing individuals, ages 5–26. A few key findings stand out. The study showed that individuals with PWS exhibit a wide range of scores and that scores are generally higher in older individuals. Scores for those with PWS were significantly greater compared to the typically developing population at all ages. Notably, scores of typically developing individuals were lower in older children and young adults, while older children and adults with PWS had significantly higher scores. In both those with PWS and those typically developing, individuals who were overweight or obese had higher HQ-CT scores.
We were also interested in understanding more about a small group of adults with PWS who consistently had low HQ-CT scores. Some previous reports in the medical literature suggested that hyperphagia may decrease in some older individuals with PWS, but other studies don’t support that finding. Here, we interviewed seventeen parents of adults with PWS in the ‘PATH for PWS’ study who had low HQ-CT scores. Caregivers were asked to share details about food-related activities such as meal preparation, attending social engagements with food, and food security measures.
We found there were many factors at play, but in many cases, these individuals followed very strict food routines. Caregivers indicated that the need for sameness in daily routines was critical for managing food-related behaviors, with one caregiver sharing, “Routine is the only thing that saves us.” Most caregivers also used food security measures such as ‘line of sight’ supervision, cameras, and/or locks. “If the opportunity [to get food] isn’t there, she is happier," said one caregiver. This suggests that, at least in most cases, it may not be that hyperphagia is necessarily decreasing as individuals get older, but that the longstanding controls and routine around food may decrease some of the hyperphagic behaviors measured on the HQ-CT scale.
When asked about the most challenging aspects of managing PWS, food security, behavior, and mental health management were commonly cited. One caregiver shared, “She is not compatible with the world. She is really smart, but she can’t control the food behavior, so that takes away all opportunity,” while another summarized the biggest challenge as “anxiety and behavior, transitioning, and unmet expectations." A little more than half of the caregivers interviewed reported that their loved one’s food-related behaviors had stayed about the same over time, while a few saw increased behaviors and others saw decreases. Those who observed a decrease in behaviors pointed out that changes in lifestyle, such as school endings, staying home more often, or the transition of siblings out of the home, contributed to things being more stable. Notably, most parents of low-scoring adults with PWS indicated they would still be interested in trying a drug that was safe and effective in treating hyperphagia if it became available, since it might increase the independence of their loved one with PWS and allow for a reduction in the intense caring needs required to maintain safety.
We want to sincerely thank the caregivers who participated in the interviews and generously gave of their time sharing deeply personal experiences about their lives with their loved ones with PWS. We are incredibly grateful for your commitment to advancing PWS research. We also want to thank the ‘PATH for PWS’ participants, the caregivers who completed the HQ-CT for their typically developing children, and those who shared the surveys with friends and family members, encouraging them to participate in our research. You have had a direct and meaningful impact on the development of normative data regarding food behaviors, which can now be used as comparison data for future clinical trials assessing treatments for hyperphagia. Your support continues to prove that our community will do whatever it takes to make a difference in the lives of each person with PWS.