期刊
DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY
卷 65, 期 1, 页码 67-77出版社
WILEY
DOI: 10.1111/dmcn.15286
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This study aims to compare the societal financial costs and quality of life (QoL) between untreated patients with spinal muscular atrophy (SMA) and treated patients identified by symptoms or early testing. The results show that the total costs are lower for untreated patients, but lower for patients identified by early testing. Early patient identification and treatment can reduce the overall societal costs of SMA.
Aim To compare the societal financial costs and quality of life (QoL) of untreated patients with spinal muscular atrophy (SMA) and treated patients identified because they presented symptoms or were identified by early testing (sibling or newborn screening). Method Data from two different sources were used: data collected prospectively in untreated patients from 2016 to 2018 and data collected during a prospective follow-up study from 2018 to 2021. Patients or their caregiver completed a questionnaire that included questions on direct medical and non-medical costs, indirect non-medical costs, and health-related QoL. Results Data (median; range) were available for 149 patients (93 untreated - 10 years; 2 years-59 years), 42 patients (6 years 3 months; 9 months-58 years) treated after presenting with symptoms, and 14 patients (1 year 7 months; 5 months-2 years) treated after early diagnosis. Total costs were lower in untreated patients due to the high cost of drugs used in treated patients. Costs were lower for treated patients who were identified by early testing than for treated patients identified because they presented with symptoms. In all groups, patients with two SMN2 copies had higher costs than those with more copies. Interpretation Early patient identification and treatment offer the opportunity to reduce the total societal costs of SMA where treatments are available for presymptomatic and postsymptomatic patients.
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