Division of Paediatric Endocrinology and Diabetes, Centre for Hormonal Disorders in Children and Adolescents, Department of Paediatrics and Adolescent Medicine, Ulm University Hospital, Eythstraße 24, 89075, Ulm, Germany.
RTI Health Solutions, Research Triangle Park, NC, USA.
Adv Ther. 2022 Apr;39(4):1772-1783. doi: 10.1007/s12325-022-02059-8. Epub 2022 Feb 22.
In patients with pro-opiomelanocortin (POMC) or leptin receptor (LEPR) deficiency, managing obesity and hyperphagia can be burdensome for patients and caretakers. The impacts on health-related quality of life are under-recognized and are not well characterized.
We conducted in-depth qualitative interviews in patients with POMC (n = 3) and LEPR (n = 2) deficiencies participating in an ongoing open-label extension of phase 3 clinical trials with the melanocortin receptor 4 agonist setmelanotide to describe the patient experience of hyperphagia and characterize changes following treatment with setmelanotide.
Prior to setmelanotide treatment, all five patients described abnormal sensations of hunger with none indicating feeling satiated after meals and also reported that the burden of hyperphagia impacted their families, emotions, and work and/or school functioning. Following setmelanotide treatment, all five patients reported consistent reductions in hunger and weight, decreased eating, and feeling satiated after meals in addition to substantial improvements in each area of functioning they had previously reported. All five patients indicated they were very satisfied with the impact of setmelanotide on their quality of life and would be upset if treatment was discontinued.
In patients with POMC or LEPR deficiency, hyperphagia and the inability to feel satiety negatively impacted quality of life. By reducing hunger and improving satiety, setmelanotide facilitated important changes in the lives of these patients. This qualitative research study suggests that the impact of setmelanotide goes beyond favorable clinical changes (e.g., weight and hunger) to also include quality of life improvements that are highly meaningful to patients.
在患有前阿黑皮素原（POMC）或瘦素受体（LEPR）缺乏症的患者中，管理肥胖和多食症可能会给患者和护理人员带来负担。其对健康相关生活质量的影响尚未得到充分认识，也未得到充分描述。
我们对参与正在进行的黑素皮质素受体 4 激动剂 setmelanotide 治疗 POMC（n = 3）和 LEPR（n = 2）缺乏症的 3 期临床试验的开放性扩展部分的患者进行了深入的定性访谈，以描述患者多食症的体验，并描述治疗后 setmelanotide 的变化。
在使用 setmelanotide 治疗之前，所有五名患者均描述了异常饥饿感，没有人表示饭后有饱腹感，并且还报告说，多食症的负担影响了他们的家庭、情绪以及工作和/或学业。在用 setmelanotide 治疗后，所有五名患者均报告饥饿感和体重持续减轻，进食减少，饭后有饱腹感，并且他们之前报告的每个功能领域均有实质性改善。所有五名患者均表示，他们对 setmelanotide 对生活质量的影响非常满意，如果停止治疗，他们会感到不安。
在 POMC 或 LEPR 缺乏症患者中，多食症和无法感到饱腹感会严重影响生活质量。通过减少饥饿感和改善饱腹感，setmelanotide 为这些患者的生活带来了重要变化。这项定性研究表明，setmelanotide 的影响不仅超出了有利的临床变化（例如体重和饥饿感），还包括对患者具有重要意义的生活质量改善。
