Key result
Early screening and treatment for spinal muscular atrophy resulted in lower total financial costs compared to treatment after symptom onset, although untreated patients had the lowest overall costs.
Why the study?
The study was conducted to compare the societal financial costs and quality of life among untreated patients with spinal muscular atrophy and treated patients identified by symptoms versus early testing.
Does early identification and treatment reduce societal financial costs and improve quality of life in patients with spinal muscular atrophy compared to treatment after symptom onset or no treatment?
Population
149 patients with spinal muscular atrophy
Comparison
Untreated vs treated after symptom presentation vs treated after early diagnosis
Design
Prospective cohort study combining data from two prospective collections
Authors
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May reduce costs with early SMA treatment versus delayed; leaves open cost-effectiveness versus no treatment and needs prospective confirmation.
Cohort (n=149)
Does early identification and treatment reduce societal financial costs and improve quality of life in patients with spinal muscular atrophy compared to treatment after symptom onset or no treatment?
Early screening and treatment for spinal muscular atrophy reduces direct financial and hospitalization costs compared to treatment initiated after symptom onset.
Dangouloff et al. (2022) conducted a cohort in spinal muscular atrophy (n=149). Early testing and treatment vs. Treatment after symptom onset or no treatment was evaluated on Societal financial costs and quality of life. Early screening and treatment for spinal muscular atrophy resulted in lower total financial costs compared to treatment after symptom onset, although untreated patients had the lowest overall costs.
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