Patient characteristics and hospitalisation costs of spinal muscular atrophy in Spain: a retrospective multicentre database analysis

OBJECTIVES: To analyse the characteristics of patients diagnosed with spinal muscular atrophy in Spain, and to revise data on disease management and use of resources in both public and private healthcare centres. DESIGN: A retrospective multicentre database analysis. SETTING: 870 admission records registered between 1997 and 2015 with a diagnosis of spinal muscular atrophy were extracted from a Spanish claims database that includes hospital inpatient and outpatient admissions from 313 public and 192 private hospitals in Spain. RESULTS: Admission files corresponded to 705 patients; 61.99% were males and 38.01% females. Average patient age was 37 years. Disease comorbidities registered during the admission consistently included hypertension, scoliosis and respiratory failures, all associated with the standard disease course. Regarding disease management at the hospital level, patients were mostly admitted through scheduled appointments (58.16%), followed by emergency admissions (41.72%), and into neurology services in 17% of the cases. Mean hospitalisation time was 10.45 days and in-hospital mortality reached 5.29%. The overall direct medical costs of spinal muscular atrophy were €291 525, excluding medication. The average annual cost per admission was €6274, with large variations likely to reflect disease complexity and that increases with length of stay. CONCLUSIONS: The rarity of the disease difficulties the study of demographics and management; yet, an analysis of patient characteristics provides necessary information that can be used by governments to establish more efficient healthcare protocols. This study reflects the impact that individual needs and disease severity can have in disease burden calculations. Forthcoming decision-making policies should take into account medical costs and its variability, as well as pharmaceutical expenses and indirect costs. To our knowledge, this is the first study evaluating the use of healthcare resources of patients with spinal muscular atrophy in Spain.