Natural History of Type 1 Spinal Muscular Atrophy in a Series of Argentinian Children
| Autor: | Hilda Verónica Aráoz, S Monges, J. Mozzoni, F. De Castro, Luis Pablo Gravina, V. Aguerre |
|---|---|
| Rok vydání: | 2020 |
| Předmět: |
medicine.medical_specialty
Argentina Spinal Muscular Atrophies of Childhood Cohort Studies 03 medical and health sciences 0302 clinical medicine Internal medicine medicine Humans 030212 general & internal medicine Respiratory system Survival analysis business.industry Infant Spinal muscular atrophy Hospitals Pediatric medicine.disease Respiration Artificial Natural history Neurology Respiratory failure Cohort Disease Progression Breathing Nusinersen Neurology (clinical) Respiratory Insufficiency business 030217 neurology & neurosurgery |
| Zdroj: | Journal of Neuromuscular Diseases. 7:453-458 |
| ISSN: | 2214-3602 2214-3599 |
| Popis: | Background: SMA1 natural history is characterized by early development of chronic respiratory failure. Respiratory interventions in type 1 SMA infants are subject to great practice variability. Nusinersen, has been recently approved in Argentina. The advent of novel treatments has highlighted the need for natural history studies reporting disease progression in type 1 SMA. Objective: To analyze the progression, respiratory interventions and survival based on the type of respiratory support in type 1SMA patients, in a third level pediatric hospital in Argentina. Methods: Cohort of SMA1 patients followed at the Interdisciplinary Program for the Study and Care of Neuromuscular Patients (IPNM). Patient survival was analyzed by using the Kaplan-Meier method. Log-rank test was performed to compare the survival curve for three respiratory intervention groups. Results: 59 patients. Mean age of symptom onset was 2.19 (±1.4) months, age at diagnosis was 3.9 (±2.1) months. Patients developed respiratory failure at 5.82 months (±2.32) and 13.8 months (±5.6) in Type 1B and Type 1C, respectively (p |
| Databáze: | OpenAIRE |
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