Johnson N, Paradis AD, Dave V, Macahilig C, Johnson C, Stephens JM, Atzinger C. Healthcare resource use and activities of daily living status among adult patients with spinal muscular atrophy: a natural history multicountry chart review study. Poster presented at the Virtual ISPOR Europe 2021; November 2021. [abstract] Value Health. 2021 Dec; 24(12):S2.

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.

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