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. 2024 Aug;14(4):e200312.
doi: 10.1212/CPJ.0000000000200312. Epub 2024 May 31.

Subspecialty Health Care Utilization in Pediatric Patients With Muscular Dystrophy in the United States

Susan E Matesanz  1 , Jonathan B Edelson  1 , Katherine A Iacobellis  1 , Erika Mejia  1 , John F Brandsema  1 , Carol A Wittlieb-Weber  1 , Oluwatimilehin Okunowo  1 , Heather Griffis  1 , Kimberly Y Lin  1
Affiliations

Subspecialty Health Care Utilization in Pediatric Patients With Muscular Dystrophy in the United States

Susan E Matesanz et al. Neurol Clin Pract. 2024 Aug.

Abstract

Background and objectives: Standards of care exist to optimize outcomes in Duchenne and Becker muscular dystrophy (DBMD), caused by alterations in the DMD gene; however, there are limited data regarding health care access in these patients. This study aims to characterize outpatient subspecialty care utilization in pediatric patients with DBMD.

Methods: This retrospective cohort study used administrative claims data from IBM MarketScan Medicaid and Commercial Claims and Encounters Research Databases (2013-2018). Male patients 1-18 years with an ICD-9/10 diagnosis code for hereditary progressive muscular dystrophy between January 1, 2013, and December 31, 2017, were included. Participants were stratified into 3 age cohorts: 1-6 years, 7-12 years, and 13-18 years. The primary outcome was rate of annual neurology visits. Secondary outcomes included annual follow-up rates in other subspecialties and proportion of days covered (PDC) by corticosteroids.

Results: A total of 1,386 patients met inclusion-347 (25.0%) age 1-6 years, 502 (36.2%) age 7-12 years, and 537 (38.7%) age 13-18 years. Heart failure, respiratory failure, and technology dependence increased with age (p for all<0.05). The rate of neurology visits per person-year was 0.36 and did not differ by age. Corticosteroid use was low; 30% of person-years (1452/4829) had a PDC ≥20%. Medicaid insurance was independently associated with a lower likelihood of annual neurology follow-up (OR 0.23; 95% CI 0.18-0.28).

Discussion: The rate of annual neurology follow-up and corticosteroid use in patients with DBMD is low. Medicaid insurance status was independently associated with a decreased likelihood of neurology follow-up, while age was not, suggesting that factors other than disease severity influence neurology care access. Identifying barriers to regular follow-up is critical in improving outcomes for patients with DBMD.

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Conflict of interest statement

S.E. Matesanz: Consultant for Sarepta Therapeutics and Novartis JFB - JFB: Consultant for Alexion, Audentes, AveXis, Biogen, Cytokinetics, Edgewise, Genentech, Janssen, Marathon, Momenta, NS Pharma, PTC Therapeutics, Sarepta, Scholar Rock, WaVe; Speaker for AveXis and Biogen; Medical advisory council member for Cure SMA; Site investigator for clinical trials with Alexion, Astella, AveXis, Biogen, Catabasis, CSL Behring, Cytokinetics, Fibrogen, Ionis, Pfizer, PTC Therapeutics, Sarepta, Summit, WaVe. All other authors report no disclosures relevant to the manuscript. Full disclosure form information provided by the authors is available with the full text of this article at Neurology.org/cp.

Figures

Figure 1
Figure 1. Cohort Identification Flow Diagram
aSee eTable 2 in the Supplement for complete list of ICD 9/10-CM exclusion codes.
Figure 2
Figure 2. Visit Rate of HPMD Patients
Figure 3
Figure 3. Patterns of Corticosteroid Use Across PDCa Categories
PDC = proportion of days covered. aPDC was calculated by adding the number of days patients were covered on steroids during each follow-up year and dividing by 365 days.
See this image and copyright information in PMC

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