Poster Abstract #2: Surrogate Completion of the Treatment Satisfaction Questionnaire for Medication for a Trial of Corticosteroids in Duchenne Muscular Dystrophy

Introduction Satisfaction with treatment has become a useful outcome measure in oncologic studies, in which severe side effects must be balanced with tumor regression to determine if the treatment is of overall benefit to a patient. The use of corticosteroids in Duchenne muscular dystrophy (DMD) poses a similar dilemma: Does the improvement in strength outweigh the inevitable side effects and need for life-long medication? Background We are planning an international, randomized, double-blind trial in 300 DMD boys aged 4 to 7 years to compare three corticosteroid regimens with respect to function and patient–parent satisfaction over a 3- to 5-year follow-up period. Global treatment satisfaction will be measured by the Treatment Satisfaction Questionnaire for Medication (TSQM), which is a patient-centered assessment of satisfaction with treatment that is particularly relevant to a noncurative therapy applied in a chronic disease. The hypothesis is that a patient–parent satisfaction measure as a component of the primary outcome variable can yield firm evidence for changing practice and developing a standard of care. The questionnaire is designed to be completed by the individual with the chronic disease. Because the DMD boys are too young to complete the form, parents will serve as proxies, answering the questionnaire as they think their sons would. Pilot Study The TSQM has been validated only in adults with chronic disease, and only with the patient as responder. We asked 50 parents to complete the questionnaire from the perspective of their DMD sons taking corticosteroids. Parents also provided the son's age, walking ability, medications, and a rating of ease in completing the TSQM from the son's perspective. Results The results show that the parents felt that they could easily report on behalf of the sons. Associations between TSQM responses and demographic information (e.g., age, ambulatory status) will be analyzed for a future report. Sponsored in part by the National Institute of Neurological Disorders and Stroke, National Institutes of Health.