OBJECTIVES

: Duchenne muscular dystrophy (DMD) is a progressive, severe genetic disease. Most patients with DMD lose the ability to walk by their early teenage years and rarely survive beyond their 20s. Due to the physical symptoms of DMD, patients experience reduced quality of life (QoL) that hinders their abilities to work. DMD impacts not only the patient, but the loss of functional independence also affects QoL and work productivity for family members. A patient’s loss of ambulation is reported as the most emotionally difficult time for parents.

METHODS

: A lifetime cost model was constructed based on a state transition cohort model (adapted from Landfeldt et al.) containing four disease stages of DMD: early ambulatory, late ambulatory, early non-ambulatory, and late non-ambulatory. DMD treated with corticosteroids and symptom management acts as the base case in the model, and the impact of a hypothetical treatment given in the early ambulatory stage, which reduces the risk of progression to later disease stages, is shown. The model predicts work years gained and increases in work productivity.

RESULTS

: In the base case, loss of ambulation occurs at age 13 (median). Reducing the risk of disease progression from early to later disease stages by 80-100% increases this to age 42-80. Compared to the base case, this results in a gain of 19-37 work years.

CONCLUSIONS

: Delaying progression into a non-ambulatory disease stage may improve the opportunity for DMD patients to maintain functional independence and attain future employment. This could in turn improve the QoL and work productivity for both patients and their family. Further research is needed to understand the broader impact of treatments that could delay the loss of ambulation in DMD on patients, family members, and society.