Among caregivers and adults with Duchenne muscular dystrophy (DMD), willingness to accept the risks associated with gene therapy increases as the disease advances, according to a recent report published in the European Journal of Medical Genetics.
The study assessed the maximum risk of treatment-related death that people would accept to receive gene therapy. Gene therapy is non-curative and carries serious risks, including the possibility of death.
Participants were most willing to accept the risks of gene therapy during the last year a patient could lift their hand their mouth (representing the meaningful use of their arms).
“Although gene therapy offers a promising treatment approach for DMD, decisions about use are highly sensitive to how each person weighs the benefit/risk balance,” the authors wrote.
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The study built on a similar survey conducted in 2018. The latter survey, conducted in 2023, questioned 263 participants (241 caregivers and 22 adults with DMD). Most were from the United States and United Kingdom. Participants were asked if they would choose DMD gene therapy if the risk of death was one in 2,000 at four different points: when a child was a newborn, in the last year of a patient being able to raise a hand to their mouth, in the last year of walking well and “right now.”
The maximum acceptable risk was lowest for treatment in newborns, which was an average of 2.4% in the model the researchers developed to analyze the survey. The maximum acceptable risk was higher for treatment in the last year of walking well (4.7%), and highest in the last year of being able to lift one’s hands to one’s mouth (5.4%).
About 22%-33% of participants were willing to accept a greater than 10% risk of death at later disease stages. Participants indicated the progressive and serious nature of DMD, the lack of treatment alternatives and hopes for a better quality of life as reasons for accepting greater risk.
Those less willing to accept risk cited uncertainty about benefits and side effects, a focus on maintaining current quality of life, optimism regarding future therapies and concerns about the limited duration of gene therapy’s benefits.
The authors highlighted that since this survey was conducted, the first gene therapy for DMD, Elevidys, has received U.S. Food and Drug Administration approval. They expect that the overall risk-benefit profile will continue to evolve.
In the meantime, shared decision-making with care teams along with clear, balanced information about risks and benefits remain essential when weighing treatment options in DMD.
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