Observing the Clinical Course of Duchenne Muscular Dystrophy in Medicaid Real-World Healthcare Data

Christina Qian, Alexa C. Klimchak, Shelagh M. Szabo, Evan Popoff, Susan T. Iannaccone, Katherine L. Gooch

Research output: Contribution to journalArticlepeer-review

Abstract

Introduction: Duchenne muscular dystrophy (DMD) is a rare, severe progressive neuromuscular disease. Health insurance claims allow characterization of population-level real-world outcomes, based on observed healthcare resource use. An analysis of data specific to those with Medicaid insurance is presently unavailable. The objective was to describe the real-world clinical course of DMD based on claims data from Medicaid-insured individuals in the USA. Methods: Individuals with DMD were identified from the MarketScan Multi-State Medicaid datasets (2013–2018). Diagnosis and procedure codes from healthcare claims were used to characterize the occurrence of DMD-relevant clinical observations; categories were scoliosis, cardiovascular-related, respiratory and severe respiratory-related, and neurologic/neuropsychiatric. Age-restricted analyses were conducted to focus on the ages at which DMD-relevant clinical observations were more likely to be captured, and to better understand the impact of both age and follow-up time. Results: Of 2007 patients with DMD identified, median (interquartile range) age at index was 14 (9–20) years, and median follow-up was 3.1 (1.6–4.7) years. Neurologic and neuropsychiatric observations were most frequently identified, among 49.3% of the cohort; followed by cardiovascular (48.5%), respiratory (38.1%), scoliosis (36.3%), and severe respiratory (25.0%). Prevalence estimates for each category were higher when analyzed within age-restricted subgroups; and increased as follow-up time increased. Conclusions: This study is the first to use diagnosis and procedure codes from real-world Medicaid claims to document the clinical course in DMD. Findings were consistent with previously published estimates from commercially insured populations and clinical registries; and contribute to the expanding body of real-world evidence around clinical progression of patients with DMD.

Original languageEnglish (US)
Pages (from-to)2519-2530
Number of pages12
JournalAdvances in Therapy
Volume41
Issue number6
DOIs
StatePublished - Jun 2024

Keywords

  • Claims data
  • DMD
  • Duchenne muscular dystrophy
  • Medicaid
  • Rare disease

ASJC Scopus subject areas

  • Pharmacology (medical)

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