Evidence map›Paper›PMID 41999556›Full record

ArticlePain and therapy2026

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

Genevieve Lyons, Theresa Dettling, Alyssa Bowling, Ashwin Anand, Michael Sicilia, Wouter van der Pluijm, Benjamin Guikema, Julia Meade

Abstract read
In one paragraph

Article in Pain and therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Genevieve LyonsUS Health Economics and Outcomes Research, AstraZeneca Rare Disease, Alexion Pharmaceuticals, Inc., 121 Seaport Blvd, Boston, MA, 02210, USA. genevieve.lyons@alexion.com.
Theresa DettlingUS Health Economics and Outcomes Research, AstraZeneca Rare Disease, Alexion Pharmaceuticals, Inc., 121 Seaport Blvd, Boston, MA, 02210, USA.
Alyssa BowlingGlobal Medical Affairs, Alexion, AstraZeneca Rare Disease, Boston, MA, USA.
Ashwin AnandReal World Evidence, Forian Inc., Newton, PA, USA.
Michael SiciliaReal World Evidence, Forian Inc., Newton, PA, USA.
Wouter van der PluijmReal World Evidence, Forian Inc., Newton, PA, USA.
Benjamin GuikemaUS Medical Affairs, Alexion, AstraZeneca Rare Disease, Boston, MA, USA.
Julia MeadeDepartment of Pediatrics, UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionNeurofibromatosis 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.

methodsThis 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).

resultsThe 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).

conclusionSelumetinib 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.

Indexed as

Chronic painNeurofibromatosisPlexiform neurofibromaPrescription pain medication utilizationSelumetinib

Identifiers

PMID41999556
PMCPMC13172128

What Socratic holds

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.