Impact of Selumetinib on Long-Term Pain Medication Utilization in Pediatric Patients: A Retrospective US Claims Database Study

Neurofibromatosis type 1 (NF1) is a heterogeneous, multisystem genetic condition associated with diverse clinical manifestations, including nerve sheath tumors called plexiform neurofibroma (PN), which can be extremely painful. Both chronic pain and regular pain medication use are associated with low overall quality of life in pediatric patients with NF1-PN. Previous short-term, real-world data demonstrate that selumetinib treatment reduces prescription pain medication utilization; however, there are no published long-term real-world data. This was a retrospective analysis of pediatric and young adult patients receiving selumetinib. Patients receiving selumetinib were investigated using a US retrospective claims database and were identified with relevant national drug codes. Patients were followed from selumetinib initiation to the end of available follow-up data (≤ 3 years). Patients had ≥ 3 selumetinib prescriptions filled, were aged 2–21 years at selumetinib initiation, and had ≥ 12 months (pre- and post-selumetinib initiation) of continuous enrollment and medical/pharmacy claims activity. Patients receiving selumetinib were indexed at the time of selumetinib initiation (April 1, 2020–January 31, 2025). The primary cohort comprised 220 patients (female patients: 46%; male patients: 54%), 24% of whom were receiving prescription pain medication at baseline of selumetinib initiation. At 3 years post-selumetinib initiation, this decreased to 9%. At baseline, 26/220 (12%) of patients were receiving gabapentin, half of whom discontinued gabapentin during the first year post-selumetinib initiation. At baseline, 17/220 (8%) patients were receiving oxycodone; 14/17 (82%) patients discontinued oxycodone in the first year post-selumetinib initiation. There was a 20.3% reduction in prescription pain medication utilization per year for patients receiving selumetinib (odds ratio = 0.797; p = 0.039). Selumetinib treatment was associated with a significant, consistent, and durable decline in prescription pain medication utilization over 3 years. Patients who continued taking pain medications experienced nominal dose reductions, and patients who received selumetinib continuously experienced a decrease in prescription pain medication utilization. Neurofibromatosis type 1 (NF1) is a genetic disorder that can cause nerve sheath tumors, called plexiform neurofibroma (PN), to grow. PN can be extremely painful, which can negatively affect a person’s quality of life. Previous short-term, real-world data show that selumetinib treatment reduces pain medication use; however, there are no published, long-term, real-world data. In this study, data from a research database of medical claims were evaluated to understand how selumetinib treatment affects prescription pain medication usage in children and young adults (aged 2–21 years when they started selumetinib treatment). The aim of this study was to understand whether continued use of selumetinib was linked to pain medication usage. This study showed that the number of children and young adults taking pain medication decreased after they started taking selumetinib. After 3 years of selumetinib treatment, prescription pain medication use was reduced by three times, compared with baseline. In people who stopped taking selumetinib, pain medication usage remained generally the same as before they started taking selumetinib. In people who continued selumetinib treatment, there was a consistent decrease in prescription pain medication usage over 3 years. Overall, these results reinforce the positive impact of selumetinib treatment on the lives of children and young adults with NF1-PN who are taking pain medication.