Gaps in Follow Up and Sustained Clinical Care Observed in Pediatric Muscular Dystrophy

An outpatient subspecialty care utilization study recently published in Neurology showed a low rate of annual neurology follow-up among pediatric patients with Duchenne and Becker muscular dystrophy (DBMD), suggesting that identification of barriers to regular follow-up is important for improvement of patient outcomes.¹

Among 1386 patients with DBMD, results showed that the rate of neurology visits per person-year was 0.36 and did not differentiate by age. Despite this, authors noted that heart failure, respiratory failure, and technology dependence increased with age at the study entry (P for all <.05 additional="" findings="" showed="" that="" medicaid="" insurance="" was="" independently="" associated="" with="" a="" lower="" likelihood="" of="" annual="" neurology="" follow-up="" ci="" wp_automatic_readability="63.807471264368">

“Corticosteroids are the current standard of care treatment in those with Duchenne, but not Becker, muscular dystrophy. Care of patients with DBMD involves multiple subspecialists, with the neuromuscular neurologist frequently serving as the lead clinician and coordinating care across disciplines,” lead author Susan Matesanz, MD, a neurologist at Children’s Hospital of Philadelphia, and colleagues wrote.¹ “Our data show that neurology visit rates remain steady but low throughout childhood, which means that many patients may not be receiving comprehensive care and may be missing out on access to new therapies (such as the 4 commercially available exon-skipping therapies) or opportunities for intervention to maintain function.”

Investigators utilized administrative claims data from IBM MarketScan Medicaid and Commercial Claims and Encounters Research Databases (2013-2018). In this retrospective cohort study, researchers included male patients aged between 1 and 18 years with an International Classification of Diseases (ICD)-9/10 diagnosis code for hereditary progressive muscular dystrophy between January 1, 2013, and December 31, 2017. The patients were stratified into 3 age cohorts:1-6 years (n = 347), 7-12 years (n = 502), and 13-18 years (n = 537). The primary outcome was the rate of annual neurology visits, and the secondary outcomes were annual follow-up rates in other subspecialties and proportion of days covered (PDC) by corticosteroids.

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“While cardiology and pulmonary follow-up increased with patient age, in parallel with an increased rate of heart and respiratory failure in older patients, overall rates of follow-up with subspecialists were low, with only a quarter of patients regularly accessing annual subspecialty care, “ Matesanz, who also serves as an assistant professor of clinical neurology at University of Pennsylvania Perelman School of Medicine, and colleagues noted.¹ “Reasons for low subspecialty follow-up rates are likely multifactorial. The fact that neurology visits do not significantly change over time, whereas other specialty visits like cardiology and pulmonology increase with age and disease severity, may be due to lack of perceived benefit of neurology visits, or the gap between the need for neurologic services and the availability of practicing neurologists.”

Further results revealed that corticosteroid use was low; only 7.1% of person-years (345/4829) had a PDC of at least 80%, and 30% (1452 of4829) had a PDC of at least 20%. Among the cohort of patients aged 1–6 years, 2.8% of person-years (23 of 837) had PDC of at least 80% for corticosteroids, and 24.4% (204 of 837) had a PDC of at least 20%. In the 7–12 and 13–18 years cohorts, 7.9% (153 of1934) and 8.2% (169 of2058) of person-years had PDC of at least 80%, and 35.7% (690 of1934) and 27.1% (558 of 2058) had a PDC of at least 20%, respectively.

Although efforts were made to include only those with a likely DBMD diagnosis, some patients with alternative diagnoses may have been present in the cohort, which could have potentially influenced the observed rates. Authors also noted that the care guidelines primarily apply to DMD, not BMD, so the inclusion of patients with BMD may have also impacted outcomes. The lack of a specific ICD-10 code for DBMD before 2018 and differences in coding practices may have led to missed visits, according to the authors. Additionally, the study could not fully investigate the impact of race and region because of differences in available data between MarketScan Commercial and Medicaid databases. Finally, the databases only contained information for claims submitted, potentially missing some comorbidities and over-the-counter medications.

REFERENCES
1. Matesanz SE, Edelson JB, Iacobellis KA, et al. Subspecialty Health Care Utilization in Pediatric Patients With Muscular Dystrophy in the United States. Neurol Clin Pract. 2024;14(4):e200312. doi:10.1212/CPJ.0000000000200312