Caregivers of boys with Duchenne muscular dystrophy (DMD) are more likely to choose a therapy that might extend the time before loss of ambulation (LoA) — but might also shorten it — over a therapy with a more fixed time to LoA, according to a report published in Current Medical Research and Opinion.
A survey of caregivers found that about 70% would choose a treatment with variable outcomes for a 9-year-old with DMD, even if it risked earlier LoA.
The authors conducted an online survey of 103 caregivers of patients with DMD in the United States to assess whether they would choose a hypothetical therapy with variable outcomes in terms of time to LoA or one with a more fixed time to LoA. In addition to the treatment choice, the authors also asked the caregivers whether those choices would change if the patient was younger (5 years old) or older (13 years old).
“Previous research has shown that patients often place a high value on ‘hopeful’ therapies,” the authors wrote. “While much of the research to date has focused on the value of hope as applied to survival gains, less empirical work has been applied to estimates of positively skewed non-fatal disease progression benefit.”
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The survey showed that on average, caregivers were willing to give up 11.5 months of guaranteed walking time in exchange for the chance of a longer delay to LoA. However, their preference for the variable therapy option declined as the child aged: 73% preferred the variable option for a 5-year-old, 70% for a 9-year-old and 60% for a 13-year-old.
Among caregivers of children who were still walking, most preferred the variable treatment compared with those whose children who were already non-ambulatory.
The authors noted that the findings highlight the value families and caregivers place on hope, even in the face of possible earlier decline. They also noted that this “value of hope” phenomenon has been observed in cancer care, and it may be important to consider when making treatment decisions regarding the benefits and risks of new DMD therapies.
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