OBJECTIVES

Standards of care for spinal muscular atrophy (SMA) has historically included rehabilitation therapy, nutritional support, and/or surgical interventions. There is limited real-world data on healthcare resource use (HRU) among adult patients with SMA prior to nusinersen approval.

METHODS

This retrospective, multicountry (United States, France, Germany, Italy, Spain, United Kingdom) study collected real-world natural history data on patients ≥18 years old, diagnosed with SMA prior to January 1, 2012 and managed by participating physicians from January 1, 2012 - December 31, 2016. HRU including surgical procedures, rehabilitation therapy, mobility devices, inpatient and outpatient visits were analyzed descriptively.

RESULTS

The study population included 339 adult patients with SMA [66% male; mean age (range) 31 (18-79) years] from 76 sites. Mean (range) follow-up was 11 (2-46) years from SMA diagnosis to most recent visit during which 16.2% of patients had a record of scoliosis surgery, 15.9% any tendon release, 8.0% gastrostomy procedure, 3.8% orthopedic hip surgery, and 3.5% tracheostomy. From time of diagnosis to most recent visit, physical, occupational, and respiratory therapy was recorded for 49.3%, 21.8%, and 21.5% of patients, respectively. From time of diagnosis to most recent visit, the following mobility devices were used: light wheelchair use (31.0%), motorized wheelchair use (32.2%), walking aids (31.6%), and standing aids (20.6%). At the most recent visit, 23.3% of patients could not use the restroom alone and 25.4% could not dress themselves alone. Between January 1, 2015 – December 31, 2016, 14.5% had ≥ 1 hospitalization or emergency room visit, and 78.2% had an outpatient visit related to SMA.

CONCLUSIONS

Based on a large sample size and diverse geographies, adult patients with SMA were found to require substantial treatment, caregiver support, procedures, and mobility aids prior to the availability of disease modifying treatment.