Howell Timothy A, Matza Louis S, Mallya Usha G, Goldstone Anthony P, Butsch W Scott, Lazarus Ethan
Patient-Centered Research Evidera Bethesda Maryland USA.
Value and Evidence Rhythm Pharmaceuticals, Inc Boston Massachusetts USA.
Obes Sci Pract. 2023 Feb 1;9(4):376-382. doi: 10.1002/osp4.652. eCollection 2023 Aug.
Rare genetic diseases of obesity typically present with hyperphagia, a pathologic desire to consume food. Cost-utility models assessing the value of treatments for these rare diseases will require health state utilities representing hyperphagia. This study estimated utilities associated with various hyperphagia severity levels.
Four health state vignettes were developed using published literature and clinician input to represent various severity levels of hyperphagia. Utilities were estimated for these health states in a time trade-off elicitation study in a UK general population sample.
In total, 215 participants completed interviews (39.5% male; mean age 39.1 years). Mean (SD) utilities were 0.98 (0.02) for no hyperphagia, 0.91 (0.10) for mild hyperphagia, 0.70 (0.30) for moderate hyperphagia, and 0.22 (0.59) for severe hyperphagia. Mean (SD) disutilities were -0.08 (0.10) for mild, -0.28 (0.30) for moderate, and -0.77 (0.58) for severe hyperphagia.
These data show increasing severity of hyperphagia is associated with decreased utility. Utilities associated with severe hyperphagia are similar to those of other health conditions severely impacting quality of life (QoL). These findings highlight that treatments addressing substantial QoL impacts of severe hyperphagia are needed. Utilities estimated here may be useful in cost-utility models of treatments for rare genetic diseases of obesity.
肥胖相关的罕见遗传病通常表现为食欲亢进，即一种病理性的进食欲望。评估这些罕见疾病治疗价值的成本效用模型将需要代表食欲亢进的健康状态效用值。本研究估计了与不同食欲亢进严重程度水平相关的效用值。
利用已发表的文献和临床医生的意见，开发了四个健康状态描述，以代表不同严重程度的食欲亢进。在一项针对英国普通人群样本的时间权衡诱导研究中，对这些健康状态的效用值进行了估计。
共有215名参与者完成了访谈（男性占39.5%；平均年龄39.1岁）。无食欲亢进状态的平均（标准差）效用值为0.98（0.02），轻度食欲亢进为0.91（0.10），中度食欲亢进为0.70（0.30），重度食欲亢进为0.22（0.59）。轻度食欲亢进的平均（标准差）负效用值为-0.08（0.10），中度为-0.28（0.30），重度为-0.77（0.58）。
这些数据表明，食欲亢进严重程度的增加与效用值降低相关。与重度食欲亢进相关的效用值与其他严重影响生活质量（QoL）的健康状况相似。这些发现突出表明，需要针对重度食欲亢进对生活质量产生的重大影响进行治疗。此处估计的效用值可能有助于肥胖相关罕见遗传病治疗的成本效用模型。
