QOLP-02. HEALTH-RELATED QUALITY OF LIFE (HRQOL) AMONG PEDIATRIC PATIENTS WITH NEUROFIBROMATOSIS TYPE 1 (NF1) AND PLEXIFORM NEUROFIBROMA (PN) IN THE UNITED STATES: PERSPECTIVES OF THE PATIENTS AND CAREGIVERS

Abstract

Abstract BACKGROUND PNs occur in 30-50% of pediatric patients with NF1, often resulting in debilitating pain, mobility limitations, significant disfigurement, and may be life-threatening. Real-world evidence on the HRQoL of pediatric NF1 PN patients is limited. METHODS Patients ages 8-18 years with NF1 PN (treatment-naïve/new on selumetinib [≤ 1 month of use]) and their caregivers were recruited through Children’s Tumor Foundation to participate in an online cross-sectional survey (12/2020-1/2021). HRQoL measures included Pediatric Quality of Life Inventory (PedsQL; Acute version), Pain Interference Index (PII), and Patient-Reported Outcomes Measurement Information System (PROMIS) mobility and upper extremity functioning subscales. Patients provided self-reported responses; caregivers provided proxy responses and patient characteristics. Agreement between patient-reports and caregiver proxies was assessed using intra-class correlation coefficients (ICCs). RESULTS 61 patient-caregiver dyads participated in the survey. Patients were mostly white/Caucasian (90.2%), 50.8% female, with a median (range) age of 13.0 (11-16) years. Most were treatment-naïve (96.7%), with NF1 and PN diagnosis for > 5 years (91.8%, 82.0%). On the PedsQL (range: 0-100; higher=better; healthy patient mean scores typically > 80), patient-reported mean scores were 50.3, 56.1, 60.7, and 63.7 for school, emotional, social, and physical functioning; mean total score= 58.5. Caregiver-proxy mean scores ranged from 49.9-61.5 across subscales; mean total score= 55.4. Among patients reporting pain in the last 7 days, PII mean scores (range: 0-6; higher=more interference) were 3.0 (patient-reports) and 2.9 (caregiver proxies). Among patients with movement difficulty in the past 7 days, mean PROMIS scales t-scores (distribution mean= 50; higher=better) were 40.2 and 39.5 (patient-reports), and 35.6 and 30.1 (caregiver proxies), for mobility and upper extremity functioning. ICCs were very good, ranging from 0.85-0.93 across HRQoL measures. CONCLUSION NF1 PN can have substantial negative impact on pediatric patients, as demonstrated across various HRQoL domains, highlighting the importance of collecting patient-centered outcomes to guide treatment decisions.