OBJECTIVES

To describe the types of healthcare and non-healthcare resource use and burden experienced by patients with SMA type I, II and III in the UK.

METHODS

Data were collected from patients and carers using separate (unmatched) online surveys designed to capture direct and indirect resource use and burden associated with SMA in the previous 12 months. Data were stratified by motor milestone achieved.

RESULTS

A total of 122 patient and 80 carer surveys were completed and included in the final analyses. The overall mean (SD) planned SMA-related outpatient visits was 7.5 (6.8). At least one type of planned surgery or procedure was reported by 37 (30%) patients. The overall mean (SD) number of carers reported per patient was 2.2 (1.0). The overall mean (SD) number of hours of unpaid care received by the patients with SMA was 102.9 (60.4) hours per week. Nearly half of all patients (n=54; 44%) were in education and received an average of 19 (14.5) hours of educational support per week. These patients took an average of 15.8 (12.3) days off from education. A total of 45/49 (92%) employed carers took time off from employment due to reasons relating to caring for a patient with SMA. The overall mean (SD) EQ-5D-3L crosswalk value for carers was 0.739 (0.205).

CONCLUSIONS

The management of SMA is associated with a high healthcare and non-healthcare resource use burden, particularly for those patients unable to walk or stand, as evidenced by frequent planned and unplanned healthcare visits and tests or procedures. SMA is also associated with a high level of social and emotional burden to both patient and carer. Patients require a considerable amount of educational support whilst carers take a large amount of time off employment and provide a vast amount of unpaid care to the patient with SMA.