Jason Shafrin, Nadine Zawadzki, Moises Marin, Ivana Audhya, Lauren E Sedita, Natasha Kulkarni, Alexa C Klimchak
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引用次数: 0
Abstract
Objectives: Quantify caregiver risk preferences to inform the "value of hope" for Duchenne muscular dystrophy (DMD) therapies affecting time to loss of ambulation (LoA).
Methods: Caregivers (medical decision-makers) of patients with DMD were surveyed to evaluate their preferences across 2 therapies with identical expected (average) time to LoA: 1 with variable (i.e. possibly longer or shorter than average) time to LoA and 1 with fixed (i.e. certain) time to LoA. Time to LoA with the fixed therapy was altered to determine the caregiver's indifference point. Study endpoints were (i) the share of caregivers who preferred the variable (vs fixed) time to LoA therapy; and (ii) the length of fixed time to LoA that would result in caregiver indifference between the variable and fixed therapies, calculated using parameter estimation by sequential testing. The base case examined therapy choice for a hypothetical ambulatory DMD patient aged 9 years; sensitivity analyses explored preferences for younger (aged 5) and older (aged 13) patients.
Results: Among 103 caregivers surveyed, 72 (69.9%) preferred the variable time to LoA therapy for a hypothetical 9-year-old patient with DMD (p < 0.001). Caregivers were willing to give up 11.5 months (p < 0.001) of certain time to LoA for a chance of longer time to LoA. Caregivers' preference for the variable therapy decreased with hypothetical patient age at treatment initiation, from 72.8% (75/103) for age 5 (p < 0.001) to 60.2% (62/103) for age 13 (p = 0.048).
Conclusions: Caregivers of patients with DMD demonstrated risk tolerance (positive value of hope) for therapies that could delay LoA.
目的：量化护理者的风险偏好，以告知Duchenne肌营养不良症（DMD）治疗的“希望价值”，影响行动能力丧失（LoA）的时间。方法：对DMD患者的护理人员（医疗决策者）进行调查，评估他们对两种治疗方法的偏好，两种治疗方法的预期（平均）时间相同：一种是可变的（即可能比平均更长或更短）到LoA的时间，一种是固定的（即一定的）到LoA的时间。通过改变固定疗法的LoA时间来确定照顾者的冷漠点。研究终点是(i)相对于LoA治疗，更喜欢可变（vs固定）时间的护理者的比例；（ii）到LoA的固定时间长度，该长度会导致变量和固定疗法之间的照顾者冷漠，使用序列检验的参数估计计算。基本病例检查了一名假想的9岁非卧床DMD患者的治疗选择；敏感性分析探讨了年龄较小（5岁）和较大（13岁）患者的偏好。结果：在103名接受调查的护理人员中，72名（69.9%）更倾向于对一名假设的9岁DMD患者进行可变时间的LoA治疗（P P P P = 0.048）。结论：DMD患者的护理人员对延迟LoA的治疗表现出风险耐受性（积极的希望值）。
期刊介绍：
Current Medical Research and Opinion is a MEDLINE-indexed, peer-reviewed, international journal for the rapid publication of original research on new and existing drugs and therapies, Phase II-IV studies, and post-marketing investigations. Equivalence, safety and efficacy/effectiveness studies are especially encouraged. Preclinical, Phase I, pharmacoeconomic, outcomes and quality of life studies may also be considered if there is clear clinical relevance
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