Trusted Resources: Education
Scientific literature and patient education texts
Comparative All-Cause Mortality Among a Large Population of Patients With Spinal Muscular Atrophy Versus Matched Controls
source: Neurology and therapy
authors: Viscidi E, Juneja M, Wang J, Wang N, Li L, Farwell W, Bhan I, Makepeace C, Laird K, Kupelian V, Eaton S, Dilley A, Hall Ssummary/abstract:
Introduction: There is little information about survival of spinal muscular atrophy (SMA) patients into adulthood, in particular from population-based samples. We estimated and compared age-specific, all-cause mortality rates in patients with SMA and matched controls in a large, retrospective cohort study using electronic health records (EHRs) from the pre-treatment era.
Methods: The US Optum® de-identified EHR database contains EHRs for ~ 104 million persons (study period: January 1, 2007-December 22, 2016). SMA cases were identified by one or more International Classification of Diseases, Ninth/Tenth Edition codes for SMA. Controls with no SMA diagnosis code were matched 10:1 to SMA cases based on birth year, gender, and first diagnostic code date. For both groups, >= 1 month of observation and (if deceased) a valid date of death were required for inclusion. Age-specific mortality rates per person-year (PY) and hazard ratios were calculated.
Results: Five thousand one hundred seventy-nine SMA cases and 51,152 controls were analyzed. The overall hazard ratio comparing cases with controls was 1.76 (95% CI 1.63-1.90). In patients with SMA type III diagnostic codes only, the all-age mortality rate was 1059/100,000 PYs in cases and 603/100,000 PYs in controls. In older age groups (13-20, 21-30, 31-40, 41-50, 51-60, and > 60 years), age-specific mortality rates for cases consistently exceeded those of controls. Limitations of this study included the inability to confirm the SMA diagnosis or SMA type by genetic or clinical confirmation.
Conclusion: Patients with SMA of all ages, including adults and type III patients, had a higher all-cause mortality rate as compared to age-matched controls during the pre-treatment era.organization: Biogen, USA; MJ Analytics Ltd, UK; Biogen, UK
Biogen Exercises Option With Ionis to Develop and Commercialize Investigational ASO for SMABiogen, Inc. and Ionis Pharmaceuticals, ...
New Treatment Can Help Fresno Infant With Rare Genetic DisorderA Fresno family is holding onto hope aft...
Scholar Rock Provides Corporate Update and Highlights Priorities for 2022Scholar Rock, a clinical-stage biopharma...
Muscular Dystrophy AssociationMDA's legacy of progress has always star...
Springfield Second-Grader Attends Class From Home Through Double RoboticsElliette “Ellie” Miller, 8, was diag...
Spinal Muscular Atrophy Patient iPSC-Derived Motor Neurons Display Altered Proteomes at Early Stages of Differentiat...Spinal muscular atrophy (SMA) is an auto...
Vitamin K Deficiency Caused Blood Clotting Problem in SMA TeenAn adolescent male with spinal muscular ...