The lack of easily understandable information available for patients with Duchenne muscular distrophy (DMD) and their caregivers combined with the limited therapeutic options to treat the disease both impact shared decision making (SDM) between families and providers, according to a recently published study in the Orphanet Journal of Rare Diseases.
“The primary barrier to SDM is the limited number of treatment options available. As the treatment landscape evolves, clear, comprehensible information about DMD, clinical trials, and new therapies will be needed,” the study’s authors wrote.
The study used semi-structured interviews with healthcare professionals and caregivers of patients with DMD. Researchers did 18 interviews with physicians and 11 interviews with parents.
Over 80% of the interviewed parents’ children were receiving or had received corticosteroid treatment. Nine parents had adequate health literacy and the remaining two had marginal or low health literacy. Ten parents reported having a good or very good relationship with healthcare providers, and one parent reported a neutral relationship.
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Regarding SDM understanding, most parents were unfamiliar with the concept. Most healthcare professionals, on the other hand, were familiar with the concept but had trouble defining it. After receiving an explanation, all participants recognized the importance of SDM in DMD care.
Healthcare provider perceptions of SDM
Many healthcare providers said that their approach had evolved over time to move away from unilateral decision making without patient involvement. They reported that they currently emphasize the importance of patient approval and decision-making after thoroughly explaining the available treatment options.
However, their approach consists of explaining the risks and benefits of a particular treatment rather than offering a choice between different therapeutic alternatives. They highlighted the limited treatment options and strict eligibility criteria for clinical trials as significant barriers to SDM.
“Only a few patients have the luxury of choosing from a range of treatments,” noted one provider quoted in the study.
Caregiver perceptions of SDM
Several parents reported that corticosteroid treatment was frequently initiated in a unilateral manner. Many of them felt they hadn’t received sufficient information about adverse effects or the potential ramifications of not undergoing treatment. Furthermore, many caregivers desired more information on emerging DMD therapies.
The results highlight the need for readily available information not only about DMD itself, but also on emerging therapies and ongoing clinical trials.
“Most caregivers preferred explicit discussions about all clinical studies during consultations, including those not recommended or suited for their child, with explanations,” the study’s authors noted. “They viewed this as essential to SDM, preventing confusion from external information sources and ensuring a comprehensive understanding of all available options.”
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