Dr McNall-Knapp on HRQOL Outcomes With Mirdametinib in NF1-PN

“There was significant [HRQOL] improvement [from baseline to week 13] in all patients [who received mirdametinib] when you looked at the adults and the parent reports of the childrens’ outcomes in the total score.”

Rene Y. McNall-Knapp, MD, pediatric hematologist-oncologist, Jimmy Everest Center, Oklahoma Children’s Hospital OU Health; professor, Department of Pediatrics, University of Oklahoma Health Sciences Center, discusses health-related quality of life (HRQOL) outcomes from phase 2b ReNeu trial (NCT03962543) investigating mirdametinib in patients with neurofibromatosis type 1–associated symptomatic inoperable plexiform neurofibroma (NF1-PN).

This analysis used the PedsQL 4.0 Generic Core Scales questionnaire to assess HRQOL in patients with NF1-PN. This questionnaire included an assessment of 4 functioning subscales: physical functioning, social functioning, emotional functioning, and school/work functioning. Investigators compared the HRQOL scores reported by patients and patient proxies at cycle 13 with those reported at baseline and observed a significant improvement PedsQL Total Score in all pediatric and adult patients who received mirdametinib. This improvement began as early as cycle 3 and was sustained at most time points through cycle 13. Overall, among patients who could have achieved a clinically meaningful improvement in HRQOL from baseline to week 13 with mirdametinib, 37%, 45%, and 47% of adult patients, pediatric patients (patient-reported), and pediatric patients (parent proxy–reported) achieved this clinically meaningful improvement.

Subscale QOL assessments showed significant improvements in physical functioning across all patient subgroups. Furthermore, significant improvements were seen in emotional functioning among pediatric patients by parent-proxy report and in school/work functioning were among adult patients.

An assessment of patients with a QOL detriment at baseline showed that approximately one-third of pediatric and adult patients who responded to mirdametinib treatment had a clinically meaningful response in each subscale. Additionally, approximately one-half of pediatric patients had clinically meaningful responses in each subscale. McNall-Knapp concluded by explaining that these subscale outcomes were likely caused by the disease only affecting a single facet of each patient’s life.

Disclosures: McNall-Knapp reports receiving institutional research funding from AstraZeneca; Incyte; Jazz Pharmaceuticals; Pfizer; and SpringWorks Therapeutics, Inc. The ReNeu trial was sponsored by SpringWorks Therapeutics, Inc.