Studies Highlight Heavy Burden on Caregivers of Patients With DMD

New research presented at the AMCP Nexus 2024 fall conference revealed the significant toll that caregiving for individuals with Duchenne muscular dystrophy (DMD) can take on work productivity and daily life.

DMD, a rare genetic disorder characterized by progressive muscle degeneration, primarily affects boys and requires increasingly intensive care as the disease advances and patients age. These studies underscored the challenges caregivers face, highlighting an unmet need for better support systems and treatments that can more effectively manage DMD symptoms. Both studies were led by researchers from Pfizer, UCSF Benioff Children’s Hospital, and Adelphi Real World.

Caring for Adults

The first study used data from the Adelphi Real World DMD Disease Specific Programme, a cross-sectional survey conducted in the US between October 2022 and November 2023.¹ In the survey, caregivers provided demographic information and responses to the Work, Productivity, and Activity Impairment (WPAI) questionnaire. The study also incorporated data from the 2013 US National Health and Wellness Survey, comparing caregivers’ work productivity and activity levels with those of noncaregivers.

Nearly 80% of the 27 included caregivers were women, with an average age of 51 years. The average adult male patient was 21 years old, with 93% of patients being nonambulatory. Most caregivers (89%) were caring for their own children, and 63% held part– or full-time jobs.

Caregivers reported a 49.3% impairment in their personal lives | Image credit: butsaya33 – stock.adobe.com

The data showed a striking loss in work productivity, with caregivers reporting 8.5% absenteeism, 35.3% presenteeism (working while impaired), and an overall productivity loss of 38.9%. Daily activities also suffered, with caregivers experiencing a 49.3% impairment in their personal lives. These figures are higher than those reported for caregivers of adults with other conditions, where work productivity loss averaged 27.4% and activity impairment was 27.1%. When looking at the 2013 survey results, caregivers experienced consistently higher rates of absenteeism, presenteeism, overall work productivity loss, and activity impairment.

“There is potential for this burden to be reduced with the provision of better support for the caregivers of adults with DMD and treatments that effectively control DMD symptoms,” the researchers said.

Caring for Children

A second study highlighted similar challenges among caregivers of pediatric patients with DMD 17 years and younger, with a mean patient age of 10.5 years and 69% still able to walk.² Data from 65 caregivers were again collected from the Adelphi Real World DMD Disease Specific Programme. The average caregiver age was 40 years, again with most being women with part- or full-time jobs.

Caregivers reported significant disruptions to their work and daily activities. Among the 43 caregivers who responded to work-related questions, absenteeism reached 12.8%, presenteeism was 35.6%, and overall productivity loss was 42.2%. Additionally, the 65 total caregivers reported a 40.9% reduction in their ability to engage in personal activities.

“This highlights the impact to caregivers when balancing their caregiving requirements with employment and personal activities, indicating an unmet need for better support for caregivers and treatments that effectively control DMD symptoms,” the research team said.

Compared with caregivers of children with other chronic conditions such as uncontrolled asthma or neurofibromatosis, those caring for children with DMD consistently reported higher levels of impairment across all WPAI measures.

As caregivers juggle employment, personal life, and the intensive demands of DMD care, these studies underscore the pressing need for policy reforms and community-based support programs. In addition to helping patients maintain mobility and independence longer, better treatments that manage DMD symptoms more effectively could also ease the burden on caregivers.

References

1. Strober J, Posner N, Cappelleri J, et al. Work, productivity, and activity impairment for caregivers of adults with Duchenne muscular dystrophy and other conditions in the United States: combined real-world evidence and literature review. Abstract presented at: AMCP Nexus; October 16, 2024; Las Vegas, NV. Abstract M23.
2. Strober J, Posner N, Cappelleri J, et al. Work, productivity, and activity impairment for caregivers of pediatric patients with Duchenne muscular dystrophy and other conditions in the United States: combined real-world evidence and literature review. Abstract presented at: AMCP Nexus; October 16, 2024; Las Vegas, NV. Abstract M24.