Presentation (CTI)

OBJECTIVES: DMD is a severe and hereditary neuromuscular disease, caused by mutation in DMD genes which primarily affects males. In the US, this occurs approximately in one male out of every 3,500 - 5,000 male births. Understanding DMD through real-world data is essential to improve patient outcomes. Hence, this study aims to assess the disease progression and healthcare resource utilization in DMD patients over 5 years.
METHODS: This retrospective study identified the DMD patients with ICD-10 code:G71.01 by using Optum de-identified Market Clarity claims and EHR database from July 2017 through June 2019. Patients with either ≥2 outpatient-claims (≥30 days apart) or with ≥1 inpatient-claim and with continuous enrollment for 6 month prior and 5 years post index date (first DMD diagnosis) were included in the study. Patients diagnosed with DMD during the baseline period, those who received Nusinersen at any time during the study and above 7 years at index were excluded. Disease progression was assessed at 6 months of interval over the period of 5 years. Resource utilization included outpatient visits, in-patient visits, and emergency room visits.
RESULTS: A total of 129 DMD patients were identified. There was an increasing trend for HCRU among the DMD patients. At diagnosis, 100% of patients experienced musculoskeletal symptoms. Immune-related symptoms were the second most common, increasing from 55% at diagnosis to 60% after 4 years. Respiratory symptoms rose from 47% to 59%, while nervous symptoms increased from 40% to 54%. Cardiac symptoms also increased from 15% to 43%. Other symptoms showed varying prevalence over time.
CONCLUSIONS: Findings of this study showed elevated resource utilization and complications pattern in DMD patients. These insights can help improve patient care and support the development of treatments for the progressive nature of DMD.