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Aim: Nusinersen, administered by intrathecal injection at a dose of 12 mg, is indicated across all ages for the treatment of spinal muscular atrophy (SMA). Evidence on real-world healthcare resource use (HRU) and costs among patients taking nusinersen remains limited. This study aimed to evaluate real-world HRU and costs associated with nusinersen use through US claims databases. Patients & methods: Using the Merative™ MarketScan® Research Databases, patients with SMA receiving nusinersen were identified from commercial (January 2017 to June 2020) and Medicaid claims (January 2017 to December 2019). Those likely to have complete information on the date of nusinersen initiation and continuous enrollment 12 months pre- and post-index (first record of nusinersen treatment) were retained. Number and costs (US$ 2020) of inpatient admissions and emergency department (ED) visits, unrelated to nusinersen administration, were evaluated for 12 months pre- and post-nusinersen initiation and stratified by age: pediatric (<18 years) and adult (≥18 years). Results: Overall, 103 individuals treated with nusinersen were retained: 59 were pediatric (mean age [range]: 9 [1-17] years), and 44 were adults (30 [18-63] years). Inpatient admissions decreased by 41% for pediatrics and 67% for adults in the 12 months post-treatment versus the 12 months pre-treatment. Average inpatient admission costs per patient for the pediatric cohort decreased by 63% ($22,903 vs $8466) and by 79% ($13,997 vs $2899) for the adult cohort when comparing the 12 months pre-index with the 12 months post-index period. Total ED visits and ED visit costs decreased by 8% and 35%, respectively, for the overall cohort over the 12-month period pre- and post-index. Conclusion: Using US claims databases, nusinersen treatment in pediatric and adult patients was associated with reductions in HRU and costs over a 12-month period post-treatment initiation relative to the pre-treatment period.
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Importance: Patients with limited English proficiency (LEP) face multiple barriers and are at risk for worse health outcomes compared with patients with English proficiency (EP). In sepsis, a major cause of mortality in the US, the association of LEP with health outcomes is not widely explored. Objective: To assess the association between LEP and inpatient mortality among patients with sepsis and test the hypothesis that LEP would be associated with higher mortality rates. Design, Setting, and Participants: This retrospective cohort study of hospitalized patients with sepsis included those who met the Centers for Disease Control and Prevention's sepsis criteria, received antibiotics within 24 hours, and were admitted through the emergency department. Data were collected from the electronic medical records of a large New England tertiary care center from January 1, 2016, to December 31, 2019. Data were analyzed from January 8, 2021, to March 2, 2023. Exposures: Limited English proficiency, gathered via self-reported language preference in electronic medical records. Main Outcomes and Measures: The primary outcome was inpatient mortality. The analysis used multivariable generalized estimating equation models with propensity score adjustment and analysis of covariance to analyze the association between LEP and inpatient mortality due to sepsis. Results: A total of 2709 patients met the inclusion criteria, with a mean (SD) age of 65.0 (16.2) years; 1523 (56.2%) were men and 327 (12.1%) had LEP. Nine patients (0.3%) were American Indian or Alaska Native, 101 (3.7%) were Asian, 314 (11.6%) were Black, 226 (8.3%) were Hispanic, 38 (1.4%) were Native Hawaiian or Other Pacific Islander or of other race or ethnicity, 1968 (72.6%) were White, and 6 (0.2%) were multiracial. Unadjusted mortality included 466 of 2382 patients with EP (19.6%) and 69 of 327 with LEP (21.1%). No significant difference was found in mortality odds for the LEP compared with EP groups (odds ratio [OR], 1.12 [95% CI, 0.88-1.42]). When stratified by race and ethnicity, odds of inpatient mortality for patients with LEP were significantly higher among the non-Hispanic White subgroup (OR, 1.76 [95% CI, 1.41-2.21]). This significant difference was also present in adjusted analyses (adjusted OR, 1.56 [95% CI, 1.02-2.39]). No significant differences were found in inpatient mortality between LEP and EP in the racial and ethnic minority subgroup (OR, 0.99 [95% CI, 0.63-1.58]; adjusted OR, 0.91 [95% CI, 0.56-1.48]). Conclusions and Relevance: In a large diverse academic medical center, LEP had no significant association overall with sepsis mortality. In a subgroup analysis, LEP was associated with increased mortality among individuals identifying as non-Hispanic White. This finding highlights a potential language-based inequity in sepsis care. Further studies are needed to understand drivers of this inequity, how it may manifest in other diverse health systems, and to inform equitable care models for patients with LEP.