Treatment Patterns and Change in Pain Medication Utilization in Pediatric Patients Treated With Selumetinib: A US...

Selumetinib first received US FDA approval on April 10, 2020, for treatment of pediatric patients aged ≥2 years with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibroma (PN). This noninterventional, retrospective cohort study evaluated selumetinib treatment patterns and pain medication utilization in pediatric patients.

Merative MarketScan Research Databases were used to identify patients aged 2-18 years with ≥2 selumetinib prescription fills. Eligible patients had continuous enrollment 6-months prior to and following the first selumetinib prescription claim (index). Proportion of days covered (PDC; days covered/days in follow-up) was assessed to evaluate adherence (PDC ≥80%). Time to discontinuation and time to reinitiation of selumetinib (≥84 days post-discontinuation) were determined. Prescription pain medication utilization was assessed pre- and post-initiation.

Median (IQR) age was 13.0 (8.0-15.0) years, N = 89. Mean PDC was 83.5%, and patient adherence was 69.7%. At 6-months postindex, the probability of persistence was 85.4% (median time to discontinuation: 2.25 years). Approximately 20% of patients who discontinued treatment later reinitiated. Pain medication decreased marginally postindex (adjusted odds ratio [OR]: 0.62; 95% confidence interval [CI]: 0.30-1.28). In selumetinib-adherent patients (n = 62), utilization decreased (adjusted OR: 0.46; 95% CI: 0.18-1.16); there was no difference in utilization for nonadherent patients postindex (adjusted OR: 1.00; 95% CI: 0.30-3.37).

Most pediatric patients adhered to selumetinib and persisted with treatment for ≥6 months. There was a small reduction in pain medication utilization 6-months post-selumetinib initiation.