Background
Myotonic dystrophy type 1 (DM1) is a rare, progressive, genetic disorder that affects multiple organ systems and leads to significant clinical and economic burden. Given the heterogeneous nature of the disease, this study evaluated the clinical factors most strongly associated with being an individual with DM1 incurring high cost of care (HCC).
Methods
Records from 01/01/2015 to 08/25/2023 in Clarivate Real-World Database, an integrated insurance claims and electronic health record database, were utilized. Eligibility criteria included: DM1 diagnosis (index date), data activity during ≥6 months pre- and ≥12 months post-index, and age ≥12 years at index date, excluding congenital DM. HCC individuals were defined as those in the top 25th percentile of total costs in the 12-month post-index period. Baseline demographic and clinical characteristics associated with being a HCC individual with DM1 were evaluated.
Results
The study included 301 HCC and 900 non-HCC individuals, with females comprising 56.1% and 56.5% of each group, respectively. HCC individuals were older (mean age 48.8 vs. 46.6 years, p=.047), and had higher Charlson Comorbidity Index scores (1.7 vs 0.7, p<.001) than non-HCC individuals. The largest differences were observed in congestive heart failure, chronic pulmonary disease, and cerebrovascular disease, with HCC individuals exhibiting prevalence rates 11%, 10%, and 8% higher than non-HCC individuals, respectively. At baseline, more HCC individuals had severe cardiac (22% vs 7%, p<.001) and severe respiratory (12% vs 2%, p<.001) complications compared to non-HCC individuals.
Conclusion
Individuals with DM1 incurring high cost of care are characterized in large part by severe cardiac and respiratory clinical complications. These results highlight the importance of early detection and integrated treatment of multi-systemic medical problems, including respiratory and cardiac complications among individuals with DM1.