December 22, 2021Neurology and TherapyOpen Access

Comparative All-Cause Mortality Among a Large Population of Patients with Spinal Muscular Atrophy Versus Matched Controls

Q: What is the clinical evidence from this study?

Study design: Cohort. Population: Spinal muscular atrophy (n=56331). Intervention: Spinal muscular atrophy (SMA) vs. Matched controls (no SMA diagnosis). Primary outcome: All-cause mortality rate per 100,000 person-years (HR 1.76, 95% CI 1.63-1.90).

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Key result

Patients with spinal muscular atrophy had a 1.76-fold higher all-cause mortality rate compared to matched controls (HR 1.76) during the pre-treatment era.

Why the study?

Little information was available regarding survival of spinal muscular atrophy patients into adulthood, particularly from population-based samples.

Does spinal muscular atrophy increase all-cause mortality compared to matched controls?

Comparison

SMA cases vs matched controls without an SMA diagnosis code

Design

Retrospective cohort study

Authors

EVEmma ViscidiModerna Therapeutics (United States)MJManeesh JunejaHemel Hempstead Hospital JWJin WangEast China University of Science and Technology

Discussion

Member takes

Implication

SMA associated with higher mortality; leaves open whether approved therapies alter survival in contemporary cohorts.

Study Design

Type

Cohort (n=56,331)

Multicenter

Yes

Structured PICO

Does spinal muscular atrophy increase all-cause mortality compared to matched controls?

Population

5,179 patients with spinal muscular atrophy (SMA) and 51,152 matched controls without SMA diagnosis code from an EHR database of ~104 million persons

Comparator

Controls with no SMA diagnosis code matched 10:1 to SMA cases based on birth year, gender, and first diagnostic code date

Outcome

Age-specific, all-cause mortality rateshard clinical

Main Result

Effect estimate: HR 1.76 (95% CI 1.63-1.90)

Absolute Event Rate: 1926% vs 1099%

Patients with spinal muscular atrophy have a significantly higher all-cause mortality rate compared to age-matched controls across all age groups.

Limitations

Inability to confirm the SMA diagnosis or SMA type by genetic or clinical confirmation
Lack of information on the specific SMA phenotype/SMA type
Lack of information on the age at first diagnosis of SMA
Did not evaluate individual causes of death
Possibility of misclassification of cases in the SMA group
inability to confirm the SMA diagnosis or SMA type by genetic or clinical confirmation

Cite This Study

Viscidi et al. (2021) conducted a cohort in Spinal muscular atrophy (n=56,331). Spinal muscular atrophy (SMA) vs. Matched controls (no SMA diagnosis) was evaluated on All-cause mortality rate per 100,000 person-years (HR 1.76, 95% CI 1.63-1.90). Patients with spinal muscular atrophy had a 1.76-fold higher all-cause mortality rate compared to matched controls (HR 1.76) during the pre-treatment era.

synapsesocial.com/papers/6a1b7eff6e13c4b6f1abd5cc https://doi.org/10.1007/s40120-021-00307-7

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Comparative All-Cause Mortality Among a Large Population of Patients with Spinal Muscular Atrophy Versus Matched Controls

Discussion

Member takes

Implication

Study Design

Structured PICO

Main Result

Limitations

Cite This Study

Comparative All-Cause Mortality Among a Large Population of Patients with Spinal Muscular Atrophy Versus Matched Controls

Discussion

Member takes

Implication

Study Design

Structured PICO

Main Result

Limitations

Cite This Study

Also Consider

Also Consider