Treatment utilization by race among Medicaid-insured males with Duchenne Muscular Dystrophy using U.S. real-world data


Clinical Management

Poster Number: 19


Nate Posner, MPH, Pfizer, Inc., Janna Manjelievskaia, PhD, Merative, Anna Talaga, PhD, Pfizer, Megan Richards, PhD, Merative, Carolyn R Lew, PhD, Merative, Valeria Merla, PhD, Pfizer Inc., Jose Ma J Alvir, DrPH, Pfizer Inc., Stanley F Nelson, MD, David Geffen School of Medicine at UCLA

Background: Data on the impact of race on treatment outcomes among patients with DMD are lacking. This study sought to describe the real-world treatment utilization among patients with DMD by race, in a Medicaid population.

Methods: This was a retrospective cohort study of patients with DMD in the Merative Multi-State Medicaid Database between January 2017- June 2021. Males with ≥2 DMD diagnoses (earliest diagnosis=index date), aged 40 years or younger, with at least 12 months of continuous enrollment prior to and 12 months (or evidence of death) following the index date were selected. A validated algorithm was applied to minimize inclusion of patients with BMD. Demographics, clinical characteristics, and treatment utilization were reported by race, and by ventilation/ambulation status in the 12-month follow-up period.

Results: 561 patients were included, of which 360 were White, 50 Black, 33 Hispanic, and 118 Other/Unknown. Median age on the index date was 16, 13, 14, and 15 years among the race categories, respectively. A third of White and Other/Unknown patients, 22% of Black patients, and 18% of Hispanic patients met the inclusion criteria for the assisted ventilation cohort. Approximately half of Black patients met the inclusion criteria for the ambulatory cohort, compared to 38-39% among the other races. Corticosteroids were the most commonly received treatment, with highest use among Hispanics (73%) and lowest among Blacks (52%). A third of patients treated with corticosteroids received deflazacort, with varying levels of utilization across groups (19-49%, with lowest use among Blacks). Almost half of White (47%) and Hispanic (46%) patients were treated with ACE-inhibitors, compared to 38% and 61% of Black and Other/Unknown patients, respectively. Exon-skipper use was rare, with <5% utilization overall, and highest among White patients (4.2%). Conclusions: Treatment utilization, ambulation status, and ventilation status differed by race among males with DMD.