Assessing the Socioeconomic Impact of Duchenne Muscular Dystrophy on Carer’s Life in Australia: A Cross-Sectional Study
Author(s)
Leonard Lee, MN1, Arun Jones, MS2, Melinda Spencer, BN2, Daniel Tan, BSc3, Hansoo Kim, BSc, MSc, PhD2.
1University of Sydney, Camperdown, Australia, 2Griffith University, Gold Coast, Australia, 3Roche, Sydney, Australia.
1University of Sydney, Camperdown, Australia, 2Griffith University, Gold Coast, Australia, 3Roche, Sydney, Australia.
OBJECTIVES: This study aimed to provide real-world evidence on the impact of Duchenne muscular dystrophy (DMD) on caregivers in Australia, with a focus on patient-reported outcome measures and severity of disease.
METHODS: A retrospective, cross-sectional, multi-site bottom-up prevalence study was conducted. The impact on caregiver’s health-related QoL (HRQoL) was measured through the Beck Depression Inventory (BDI), and the EQ-5D-5L questionnaire. A generalised linear model was developed to examine the relationship between patient reported outcomes and severity of disease. Disease severity was categorised based on the patient’s ability to transition from lying on their back to standing without aid. An inability to do so was categorised into severe.
RESULTS: Completed surveys were received from 91 carers. The mean age of the DMD patients were 10.57 (SD=6.21) years. The majority of the patients (56%) were able to get up from lying on their back to standing without aid or assistance (non-severe). HRQoL as measured by EQ-5D-5L was significantly higher for carers of patients with non-severe disease vs severe disease (0.56 vs 0.39, p<0.0001). The depression tool BDI similarly showed a significant difference with carers of non-severe disease patients reporting a mean score of 15.76 (10-18: mild depression) compared to carers of severe patients, with a score of19.46 (19-29: moderate depression) (p=0.0118).
CONCLUSIONS: This study shows that there is a significant burden with respect to quality of life for carers of patients with DMD. Specifically, carers of severe patients are estimated to have moderate depression vs mild depression for carers of non-severe patients.
METHODS: A retrospective, cross-sectional, multi-site bottom-up prevalence study was conducted. The impact on caregiver’s health-related QoL (HRQoL) was measured through the Beck Depression Inventory (BDI), and the EQ-5D-5L questionnaire. A generalised linear model was developed to examine the relationship between patient reported outcomes and severity of disease. Disease severity was categorised based on the patient’s ability to transition from lying on their back to standing without aid. An inability to do so was categorised into severe.
RESULTS: Completed surveys were received from 91 carers. The mean age of the DMD patients were 10.57 (SD=6.21) years. The majority of the patients (56%) were able to get up from lying on their back to standing without aid or assistance (non-severe). HRQoL as measured by EQ-5D-5L was significantly higher for carers of patients with non-severe disease vs severe disease (0.56 vs 0.39, p<0.0001). The depression tool BDI similarly showed a significant difference with carers of non-severe disease patients reporting a mean score of 15.76 (10-18: mild depression) compared to carers of severe patients, with a score of19.46 (19-29: moderate depression) (p=0.0118).
CONCLUSIONS: This study shows that there is a significant burden with respect to quality of life for carers of patients with DMD. Specifically, carers of severe patients are estimated to have moderate depression vs mild depression for carers of non-severe patients.
Conference/Value in Health Info
2025-09, ISPOR Real-World Evidence Summit 2025, Tokyo, Japan
Value in Health Regional, Volume 49S (September 2025)
Code
RWD99
Topic Subcategory
Health & Insurance Records Systems
Disease
SDC: Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)