DMD Patients, Caregivers Share Perspectives on Changing Care Goals

Marisa Wexler MS avatar

by Marisa Wexler MS |

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When it comes to making decisions about how care goals should change for adolescent and young adults with Duchenne muscular dystrophy (DMD) as the disease progresses, informal conversations among family members are more common than discussions with clinicians, according to a study based on interviews with DMD patients and caregivers.

The researchers suggest that “framing conversations in terms of [adolescent and adult] autonomy may increase motivation to engage in such discussions.”

“Engaging [these patients] to understand their priorities and values as individuals will enable clinicians to collaborate with them and assist them in making decisions from within their own priority system,” the team wrote, adding, “The importance of this is underscored by the emergent theme that [adolescents and young adults with DMD] were frequently unprepared to make health-related decisions due to lack of experience.”

The study, “Assessment of the relationship between disease progression and goals of care by individuals with Duchenne Muscular Dystrophy and their caregivers,” was published in the journal Muscle & Nerve.

With modern advances in treatment, people with DMD are living longer than has ever been possible before — nowadays, it is not uncommon for individuals with DMD to live into their 30s. Nonetheless, DMD remains a progressive disease, with symptoms that get gradually worse as time goes on.

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As patients age and DMD progresses, the physical changes that these individuals experience are likely to prompt alterations in the goals of their medical care. However, how people with DMD and their caregivers view changes in care goals with age remains poorly studied.

To find out more, a team of scientists in Ohio conducted a series of interviews — 13 with adolescents and young adults (AYA) with DMD, and 17 with caregivers of patients (mostly mothers).

“The primary goal of this study was to explore the perspectives of the maturing population of AYAs with DMD regarding their goals and care needs as they age and experience progressive physical changes,” the researchers wrote.

Several themes emerged from these interviews and were summarized by researchers in the study’s findings. First, informal conversations about care preferences among family members were generally more likely to happen than explicitly talking to healthcare providers about the goals of care.

“What the boys have done is that they’ve made sure that I know their wishes, and their sister [does],” said one caregiver, the mother of a 22-year-old with DMD.

Another theme that emerged is a tendency for both patients and caregivers to doubt the medical system and the advice of professional healthcare providers — often due to delays and difficulties in getting a DMD diagnosis in the first place. Such experiences engendered a lack of trust in both medical systems and providers, the researchers found. Indeed, mothers in the study reported that, when they first noticed something was wrong and took their child to the doctor, they were dismissed as “crazy” or “imagining things.”

“Family doctors or general practitioners aren’t educated enough,” said the mother of a 16-year-old patient.

Interviewees also noted that the progression of DMD tends to be quite gradual; sudden changes in symptom severity are uncommon. This slow progression forces families to continuously adapt to the current situation, which, many suggested, prompts a focus on managing the present rather than planning for the future.

I wouldn’t say there’s a time when we said, ‘Oh man, you know things have gotten you know a lot worse,’ and he’s in his chair a lot more and there’s a lot more equipment. I think everything was just so gradual,” one mother said. 

I’ve had to adapt all my life… different changes every stage,” said one patient older than 30. “And so I’m always adapting to the changes. I’ve adapted to my inability to move very much.”

As far as goals for medical care, maximizing lifespan was commonly noted as important. But both the adolescents and young adults and their caregivers also generally prioritized a desire to maximize life quality and the patients’ ability to spend meaningful time with their peers.

“This is why I’m here. This is the point of my life, is to be able to be with friends,” said a 15-year-old patient.

As long as he’s happy and somewhat healthy enough to just be a happy kid then you know then that’s all good,” said the mother of an 11-year-old. 

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Several patients also noted the importance of having autonomy over their medical choices, particularly as they age and transition into adulthood.

“It’s control over my own life … I’ll listen to parents, but in the end, it should be my decision because it’s my body,” said a 22-year-old patient. 

“I am an adult,” said another 22-year-old patient. “I shouldn’t have to rely on my parents all the time for me to do things.”

A final noted theme was that, when important decisions about care needed to be made, patients often were unprepared and had to rely on caregivers.

“I generally don’t make decisions by myself,” said one adult patient.

Notably, less than half of patients (44%) reported talking to healthcare providers about advanced directives, which are decisions about end-of-life care taken ahead of time. Even fewer, just 28%, reported having such directives established.

The researchers stressed a need for interventions that can help facilitate conversations about care directives with providers in a setting that is formally documented in medical records, to ensure that patients’ wishes are prioritized.

“The present-day focus exhibited by many participants may not represent avoidant behavior but rather a redefinition of their priorities in their own terms,” the researchers wrote.

“Framing goals-of-care conversations around patient autonomy may enhance patient engagement in such conversations,” they concluded.