Dataset Information

Utilization and Treatment Patterns of Disease-Modifying Therapy in Pediatric Patients with Multiple Sclerosis in the United States.

ABSTRACT:

Background

The current landscape and treatment patterns of disease-modifying therapy (DMT) use in pediatric patients with multiple sclerosis (MS) are not yet well understood. This study examined DMT utilization and treatment patterns in pediatric patients newly diagnosed as having MS.

Methods

Pediatric patients (<18 years old) with two MS diagnosis claims from January 1, 2010, to December 31, 2016, were identified from the MarketScan Commercial Database. The index date was defined as the date of first MS diagnosis, and patients were followed up for 1 year post-index date. Outcomes evaluated included percentage of patients who initiated treatment after MS diagnosis, different DMTs initiated, treatment discontinuation, and switching treatment during follow-up.

Results

Of 182,057 patients newly diagnosed as having MS, 288 pediatric patients (mean age, 14 years; 61% female) were identified. Within the first year of diagnosis, 188 patients (65.3%) did not receive any DMT. The most common first-initiated treatments were interferons and glatiramer acetate (83%), but 28% of patients switched or discontinued from first-initiated treatment within 6 months of treatment initiation.

Conclusions

This study suggests that a considerable proportion of pediatric patients with MS remain untreated within 1 year of diagnosis. Patients most commonly initiated injectables as their first DMT. Overall, therapy failed early in approximately one in three patients. Thus, the study warrants urgency in treating these patients with currently approved treatment options.

SUBMITTER: Greenberg B

PROVIDER: S-EPMC8218586 | biostudies-literature |

REPOSITORIES: biostudies-literature

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Project description:IntroductionComparisons of healthcare utilization and expenditure among multiple sclerosis (MS) disease-modifying therapies (DMTs) are limited.MethodsIn this retrospective cohort study using commercial insurance claims data of a US population (2010-2019), we compared healthcare utilization and costs in MS across different DMTs. We assigned patients to different treatment arms: no DMT (ND), high-efficacy (HE) DMT (alemtuzumab, B cell depletion, cladribine, and natalizumab), and standard-efficacy (SE) DMT (dimethyl fumarate, glatiramer acetate, interferon beta, sphingosine-1-phosphate receptor modulator, and teriflunomide). We obtained healthcare costs and occurrences of healthcare services: outpatient visits, emergency room visits, hospitalizations, MS-related magnetic resonance imaging (MRI). We quantified relapses (based on MS-related hospitalizations, as well as outpatient visits with prescription of high-dose steroids) and medical complexity (based on unique drug classes of prescriptions). We calculated covariate-adjusted incidence rate ratio of healthcare services using negative binomial regression with ND as reference and covariate-adjusted mean cumulative healthcare costs using a generalized linear model with log-link function and gamma distribution.ResultsAmong the 25,932 patients with MS (mean age 52.8 years, 75.2% women), both HE (mean age 54.0 years, 76.2% women) and SE (mean age 43.9 years, 75.6% women) groups had more non-pharmacy healthcare utilization than ND (mean age 57.6 years, 75.4% women), including overall outpatient doctor visits, neurology visits, and MS-related MRIs as well as relapses and medical complexities. Relative to ND, both HE and SE groups had higher pharmacy costs and overall healthcare costs 12 months after treatment initiation, despite having lower or equivalent non-pharmacy medical costs. In patients on DMT, pharmacy costs accounted for up to 65% of overall healthcare costs with over 85% of pharmacy costs attributable to DMT costs.ConclusionDMT cost is a key driver of the overall healthcare expenditure in MS. Future comparative and cost-effectiveness studies integrating claims and electronic health records data with better balancing of patient characteristics are warranted.