Caregiver risk preferences for delaying loss of ambulation in Duchenne muscular dystrophy in the United States

Quantify caregiver risk preferences to inform the "value of hope" for Duchenne muscular dystrophy (DMD) therapies affecting time to loss of ambulation (LoA). Caregivers (medical decision-makers) of patients with DMD were surveyed to evaluate their preferences across 2 therapies with identi...

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Vydáno v:Current medical research and opinion Ročník 41; číslo 8; s. 1499 - 1509
Hlavní autoři: Shafrin, Jason, Zawadzki, Nadine, Marin, Moises, Audhya, Ivana, Sedita, Lauren E., Kulkarni, Natasha, Klimchak, Alexa C.
Médium: Journal Article
Jazyk:angličtina
Vydáno: England 03.08.2025
Témata:
Adolescent
Adult
Caregivers - psychology
Child
Child, Preschool
Female
Humans
Male
Middle Aged
Muscular Dystrophy, Duchenne - physiopathology
Muscular Dystrophy, Duchenne - therapy
Time Factors
United States
Walking
United States
treatment preferences
loss of ambulation
value of hope
value assessment
Duchenne muscular dystrophy
ISSN:0300-7995, 1473-4877, 1473-4877
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Shrnutí:Quantify caregiver risk preferences to inform the "value of hope" for Duchenne muscular dystrophy (DMD) therapies affecting time to loss of ambulation (LoA). 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. Among 103 caregivers surveyed, 72 (69.9%) preferred the variable time to LoA therapy for a hypothetical 9-year-old patient with DMD (  < 0.001). Caregivers were willing to give up 11.5 months (  < 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 (  < 0.001) to 60.2% (62/103) for age 13 (  = 0.048). Caregivers of patients with DMD demonstrated risk tolerance (positive value of hope) for therapies that could delay LoA.
Bibliografie:ObjectType-Article-1
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ObjectType-Feature-2
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ISSN:0300-7995
1473-4877
1473-4877
DOI:10.1080/03007995.2025.2550347