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Background <p>The rare genetic disease Duchenne muscular dystrophy (DMD) mostly affects males, impacting mobility and pulmonary/cardiac function from childhood. This study explored non-professional caregiving in DMD, comparing burden across regions and by care recipients’ ambulatory status.</p> Methods <p>Data were from the Adelphi Real World Disease Specific Programme™, a cross-sectional survey in Europe (France, Germany, Spain), the United States of America (USA), and Japan from October 2022–November 2023. Caregivers reported health status (their own and that of dependents with DMD) via the EQ-5D-5L, and their own productivity via the Work Productivity and Impairment Index (WPAI).</p> Results <p>Of 216 individuals with DMD, 45.8%, 41.7% and 12.5% were from Europe, USA and Japan, respectively; 54.4% were ambulatory, 68.4%, 52.2% and 11.1% in Europe, USA and Japan, respectively. Individuals with DMDs’ mean (standard deviation; SD) EQ-5D-5L index value was 0.19 (0.27), with no significant difference across regions, but was higher in ambulatory than non-ambulatory individuals (<i>p</i> &lt; 0.0001). Individuals with DMDs’ mean (SD) EQ-VAS score was 24.3 (27.9), differed across regions (<i>p</i> &lt; 0.0001), but did not differ by ambulatory status. In the USA, Europe and Japan, 38.9%, 23.5% and 7.4% of caregivers, respectively, worked full-time (<i>p</i> = 0.0020). Caregiver employment did not differ significantly between ambulatory/non-ambulatory dependents. Mean caregiver absenteeism, presenteeism and overall work impairment were 9.7%, 37.8% and 42.4%, respectively, with no significant differences between regions or ambulatory/non-ambulatory dependents. Mean activity impairment was 49.0%, (Japan: 67.6%, Europe: 49.4%, USA: 43.4%; <i>p</i> = 0.0002), and higher in caregivers of non-ambulatory than ambulatory dependents (<i>p</i> &lt; 0.0001). Most caregivers reported no problems with their own mobility, self-care, usual activities, or pain/discomfort. The severity of anxiety/depression varied across regions (<i>p</i> &lt; 0.0001), with no significant difference by dependents’ ambulatory status. Mean (SD) caregivers’ EQ-VAS score and index value were 26.9% (30.3%) and 0.83 (0.23), respectively. EQ-VAS score differed significantly across regions (USA: 52.1% [33.3%], Europe: 8.9% [1.7%], Japan: 8.9% [1.7%]; <i>p</i> &lt; 0.0001), but not by dependents’ ambulatory status. EQ-5D-5L index value did not differ significantly across regions or with dependents’ ambulatory status.</p> Conclusions <p>Non-professional caregiving in DMD presented a substantial burden, varying across regions, and greater for those caring for non-ambulatory than ambulatory dependents.</p>

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Caregiver burden in Duchenne muscular dystrophy in Europe, Japan, and the United States – a real-world study

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  • 发表日期：

    2026年1月10日

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  • Jonathan Strober,
  • Keiko Ishigaki,
  • Nate Posner,
  • Joseph C. Cappelleri,
  • Sherry Xiao,
  • Anna Talaga,
  • Stephen Dukacz,
  • Halima Iqbal,
  • Emma Chatterton,
  • Ella Morton,
  • Sarah Weatherby,
  • Jonathan de Courcy

摘要

Background

The rare genetic disease Duchenne muscular dystrophy (DMD) mostly affects males, impacting mobility and pulmonary/cardiac function from childhood. This study explored non-professional caregiving in DMD, comparing burden across regions and by care recipients’ ambulatory status.

Methods

Data were from the Adelphi Real World Disease Specific Programme™, a cross-sectional survey in Europe (France, Germany, Spain), the United States of America (USA), and Japan from October 2022–November 2023. Caregivers reported health status (their own and that of dependents with DMD) via the EQ-5D-5L, and their own productivity via the Work Productivity and Impairment Index (WPAI).

Results

Of 216 individuals with DMD, 45.8%, 41.7% and 12.5% were from Europe, USA and Japan, respectively; 54.4% were ambulatory, 68.4%, 52.2% and 11.1% in Europe, USA and Japan, respectively. Individuals with DMDs’ mean (standard deviation; SD) EQ-5D-5L index value was 0.19 (0.27), with no significant difference across regions, but was higher in ambulatory than non-ambulatory individuals (p < 0.0001). Individuals with DMDs’ mean (SD) EQ-VAS score was 24.3 (27.9), differed across regions (p < 0.0001), but did not differ by ambulatory status. In the USA, Europe and Japan, 38.9%, 23.5% and 7.4% of caregivers, respectively, worked full-time (p = 0.0020). Caregiver employment did not differ significantly between ambulatory/non-ambulatory dependents. Mean caregiver absenteeism, presenteeism and overall work impairment were 9.7%, 37.8% and 42.4%, respectively, with no significant differences between regions or ambulatory/non-ambulatory dependents. Mean activity impairment was 49.0%, (Japan: 67.6%, Europe: 49.4%, USA: 43.4%; p = 0.0002), and higher in caregivers of non-ambulatory than ambulatory dependents (p < 0.0001). Most caregivers reported no problems with their own mobility, self-care, usual activities, or pain/discomfort. The severity of anxiety/depression varied across regions (p < 0.0001), with no significant difference by dependents’ ambulatory status. Mean (SD) caregivers’ EQ-VAS score and index value were 26.9% (30.3%) and 0.83 (0.23), respectively. EQ-VAS score differed significantly across regions (USA: 52.1% [33.3%], Europe: 8.9% [1.7%], Japan: 8.9% [1.7%]; p < 0.0001), but not by dependents’ ambulatory status. EQ-5D-5L index value did not differ significantly across regions or with dependents’ ambulatory status.

Conclusions

Non-professional caregiving in DMD presented a substantial burden, varying across regions, and greater for those caring for non-ambulatory than ambulatory dependents.