ASSESSING THE FEASIBILITY OF INDIRECT COMPARISON OF LUVOMETINIB VERSUS SELUMETINIB IN THE TREATMENT FOR PEDIATRIC PATIENTS WITH NEUROFIBROMATOSIS TYPE 1 IN CHINA...
Author(s)
Lili Cheng, MBA1, Zigang Li, MSc2, Yang Yang, MSc2, Xin Chen, MSc2, Keruo Zhou, MSc2.
1Jiangsu Provincial Pharmacy Association (JPPA), Nanjing, China, 2Shanghai Fosun Pharmaceutical (Group) Co., Ltd., Shanghai, China.
1Jiangsu Provincial Pharmacy Association (JPPA), Nanjing, China, 2Shanghai Fosun Pharmaceutical (Group) Co., Ltd., Shanghai, China.
OBJECTIVES: Neurofibromatosis type 1 (NF1) is an autosomal-dominant neoplastic rare genetic disorder (prevalence ~1:3000). 30%-60% of NF1 patients develop plexiform neurofibromas (PNs). NF1-PN remains incurable with limited target therapies in China. This study aims to assess the feasibility of indirect comparison between two approved target therapies, luvometinib and selumetinib, for pediatric NF-1 patients.
METHODS: A targeted literature review was conducted in PubMed in March 2025 to examine studies on selumetinib for pediatric NF1-PN patients following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Trial design, eligibility criteria, baseline characteristics and outcomes were extracted and compared with that in the phase II trial of luvometinib (NCT04954001). Pre-adjustment with different baseline variables sets was conducted using R (version 4.1.0), to assess population matching feasibility. Effective sample size (ESS), sample loss and rescaled weight distribution plot were reported.
RESULTS: Two selumetinib studies were figured out, a Korean single-arm, single-center phase II trial, and the international pivotal clinical trial (SPRINT, NCT01362803). The Korea trial was unsuitable for indirect comparison due to differences in study design and outcome measures. The SPRINT trial was identified as the most appropriate choice in comparison. Luvometinib additionally covered patients aged 2-3 years than selumetinib. Both trials reported ORR, PR, DOR, PFS and safety, with minor outcome definition differences resolvable by simple statistical processing. Non-anchored matching-adjusted indirect comparison (MAIC) in the most suitable indirect comparison method regarding the single-arm design in both trials. Six common baseline characteristics were reported, and the pre-adjustment results show that target neurofibroma volume led to >90% sample loss. ESS under other variable combinations ranged from 4 to 34.
CONCLUSIONS: An unanchored MAIC is feasible between selumetinib and luvometinb for pediatric NF1-PN. However, further exploration is needed to confirm the optimal population-matched variable combination for subsequent analyses.
METHODS: A targeted literature review was conducted in PubMed in March 2025 to examine studies on selumetinib for pediatric NF1-PN patients following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Trial design, eligibility criteria, baseline characteristics and outcomes were extracted and compared with that in the phase II trial of luvometinib (NCT04954001). Pre-adjustment with different baseline variables sets was conducted using R (version 4.1.0), to assess population matching feasibility. Effective sample size (ESS), sample loss and rescaled weight distribution plot were reported.
RESULTS: Two selumetinib studies were figured out, a Korean single-arm, single-center phase II trial, and the international pivotal clinical trial (SPRINT, NCT01362803). The Korea trial was unsuitable for indirect comparison due to differences in study design and outcome measures. The SPRINT trial was identified as the most appropriate choice in comparison. Luvometinib additionally covered patients aged 2-3 years than selumetinib. Both trials reported ORR, PR, DOR, PFS and safety, with minor outcome definition differences resolvable by simple statistical processing. Non-anchored matching-adjusted indirect comparison (MAIC) in the most suitable indirect comparison method regarding the single-arm design in both trials. Six common baseline characteristics were reported, and the pre-adjustment results show that target neurofibroma volume led to >90% sample loss. ESS under other variable combinations ranged from 4 to 34.
CONCLUSIONS: An unanchored MAIC is feasible between selumetinib and luvometinb for pediatric NF1-PN. However, further exploration is needed to confirm the optimal population-matched variable combination for subsequent analyses.
Conference/Value in Health Info
2026-09, ISPOR Asia Pacific 2026, Bangkok, Thailand
Value in Health, Volume 55, Issue S1
Code
CO10
Topic
Clinical Outcomes
Topic Subcategory
Comparative Effectiveness or Efficacy
Disease
SDC: Pediatrics, SDC: Rare & Orphan Diseases