Setmelanotide reduces hyperphagia, improves quality of life in Bardet-Biedl syndrome
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Key takeaways:
- All adults and caregivers of children receiving setmelanotide reported improvements with hyperphagia within 2 months of starting therapy.
- Setmelanotide was linked to improved physical and emotional well-being.
Treatment with the melanocortin-4 receptor agonist setmelanotide reduced hyperphagia and led to improvements in emotional well-being among children and adults with Bardet-Biedl syndrome, according to study findings.
“This study provides insights into less obvious consequences of living with insatiable hunger, such as the inability to focus on tasks or build relationships,” Claire Ervin, MPH, executive director for patient-centered outcomes assessment at RTI Health Solutions in North Carolina, and colleagues wrote in a study published in Advances in Therapy. “Treatment with setmelanotide facilitated improvements in hunger and promoted beneficial changes in patient and caregiver experiences. Thus, the impact of setmelanotide treatment in patients with Bardet-Biedl syndrome extends beyond clinically beneficial outcomes and includes health-related quality of life improvements that are deeply meaningful to patients and their families.”
Hyperphagia linked to intense hunger, negative emotions
Researchers conducted interviews with eight adults and 11 caregivers of children who participated in phase 2 and phase 3 setmelanotide (Imcivree, Rhythm Pharmaceuticals) trials for the treatment of Bardet-Biedl syndrome. Participants in the setmelanotide open-label, long-term extension were also invited to participate. Interviews took place between March and June 2021. Participants answered open-ended questions on experiences of hunger and their lives before treatment and changes in hunger, weight and function after starting setmelanotide. Participants rated their hunger on a scale of zero to 10 before treatment and after initiating setmelanotide. Caregivers answered the questions based on their observations of their children receiving setmelanotide.
Adult participants completed their interview a mean 29 months after participating in their first setmelanotide clinical trial, and caregivers were interviewed a mean 24 months after their child participated in a trial. Before taking setmelanotide, all participants described feelings of hyperphagia, with about half of the cohort reporting hunger that was intense throughout the day. All participants rated their peak hunger as an 8 or higher. Adult participants said any satisfaction they felt after eating was short-lived, whereas 81.8% of caregivers said their child never seemed satisfied after eating. A lack of control with eating was reported by 62.5% of adults and 90.9% of caregivers.
Negative effects directly attributed to hyperphagia were identified by 87.5% of adults and 90.9% of caregivers, with most of those effects related to emotional state. Participants also said hyperphagia had broad negative effects on relationships with family members. More than half of caregivers said hyperphagia negatively affected their child’s social participation. Impaired concentration and focus due to hyperphagia was reported by 72.7% of caregivers and 25% of adults.
Setmelanotide improves emotional and physical well-being
After starting setmelanotide, all adults and caregivers reported improvements in hyperphagia and satiety within 2 months. Peak hunger scores decreased by two to six points. All participants reported a large reduction in food consumption and greater control over how much food was eaten. None of the participants who reported hyperphagia behaviors before setmelanotide experienced them after starting treatment.
Of adult participants, 87.5% reported improvements in how they felt physically after starting setmelanotide. Caregivers observed physical improvements in their children. Improvements in emotional health were also consistently reported by participants. The improvements in hunger led to improvements in family dynamics for half of the adults and 54.5% of caregivers. All participants reported improved focus and concentration with setmelanotide, and 25% of adults and 63.6% of caregivers attributed the improved focus to improved performance at work or school.
Weight loss and associated health benefits were the most meaningful change with setmelanotide for 62.5% of adults and 54.5% of caregivers, with the other participants saying that the reduced obsessive focus on food was most meaningful. All but one participant said they were very satisfied with setmelanotide.
“Our results support further research on the long-term consequences of hyperphagia and obesity and treatment interventions that can alleviate their impact on health-related quality of life in patients with Bardet-Biedl syndrome,” the researchers wrote.
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