Survival of patients with spinal muscular atrophy type 1

Autor: G. Ottonello, Maria Beatrice Chiarini Testa, Elisabetta Bignamini, Cesare Gregoretti, Chiara Mastella, Lucilla Ravà, Aleksandar Veljkovic, Renato Cutrera
Přispěvatelé: Gregoretti, C., Ottonello, G., Testa, M., Mastella, C., Ravà, L., Bignamini, E., Veljkovic, A., Cutrera, R.
Rok vydání: 2013
Předmět:
Male
Palliative care
Databases
Factual
medicine.medical_treatment
Kaplan-Meier Estimate
Spinal Muscular Atrophies of Childhood
Severity of Illness Index
Cohort Studies
Retrospective Studie
Cause of Death
Palliative Care
Prognosis
Spinal muscular atrophy type 1
Child
Preschool
Home mechanical ventilation
Breathing
Female
Long survival
Survival Analysi
Pediatric palliative care
Human
medicine.medical_specialty
Prognosi
Mechanical assisted cough
Risk Assessment
Statistics
Nonparametric
Arts and Humanities (miscellaneous)
medicine
Respiratory muscle
Confidence Intervals
Humans
Decompensation
Survival analysis
Retrospective Studies
Mechanical ventilation
Noninvasive Ventilation
business.industry
Infant
Newborn
Oxygen Inhalation Therapy
Infant
Spinal muscular atrophy
medicine.disease
Respiration
Artificial
Survival Analysis
Confidence interval
Surgery
Pediatrics
Perinatology and Child Health
Cohort Studie
business
Confidence Interval
Zdroj: Pediatrics. 131(5)
ISSN: 1098-4275
Popis: BACKGROUND: Spinal muscular atrophy type 1 (SMA1) is a progressive disease and is usually fatal in the first year of life. METHODS: A retrospective chart review was performed of SMA1 patients and their outcomes according to the following choices: letting nature take its course (NT); tracheostomy and invasive mechanical ventilation (TV); continuous noninvasive respiratory muscle aid (NRA), including noninvasive ventilation; and mechanically assisted cough. RESULTS: Of 194 consecutively referred patients enrolled in this study (103 males, 91 females), NT, TV, and NRA were chosen for 121 (62.3%), 42 (21.7%), and 31 (16%) patients, respectively. Survival at ages 24 and 48 months was higher in TV than NRA users: 95% (95% confidence interval: 81.8%–98.8%) and 67.7% (95% confidence interval: 46.7%–82%) at age 24 months (P < .001) and 89.43% and 45% at age 48 months in the TV and NRA groups, respectively (P < .001). The choice of TV decreased from 50% (1992–1998) to 12.7% (2005–2010) (P < .005) with a nonstatistically significant increase for NT from 50% to 65%. The choice of NRA increased from 8.1% (1999–2004) to 22.7% (2005–2010) (P < .001). CONCLUSIONS: Long-term survival outcome is determined by the choice of the treatment. NRA and TV can prolong survival, with NRA showing a lower survival probability at ages 24 and 48 months.
Databáze: OpenAIRE
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