QOL-23ASSOCIATION BETWEEN QUALITY OF LIFE AND VESTIBULAR SCHWANNOMA VOLUME, WORD RECOGNITION SCORES, AND PATIENT- AND PHYSICIAN-REPORTED DISEASE SEVERITY IN A US POPULATION OF NF2 PATIENTS

Abstract

BACKGROUND: Neurofibromatosis type 2 (NF2) is associated with histologically benign but morbid tumors. Patient-reported outcomes (PROs) are important measures of patient function for this neuro-oncologic syndrome which impacts quality of life (QOL) due to multiple chronic tumors. Hence, NF2 serves as a model for developing PROs in neuro-oncology trials. The NFTI-QOL is a validated disease-specific QOL-instrument developed in the United Kingdom which has not been compared against historical clinical trial endpoints in NF2 including total vestibular schwannoma (VS) volume and best word recognition score (WRS). METHODS: An IRB-approved prospective observational study was conducted enrolling adult NF2 patients at Johns Hopkins. Measures included: NFTI-QOL, SF-36; total volume of bilateral VS, best WRS, provider- and patient-reported disease severity (ProvSev, PatSev) by institutionally-derived multi-item (e.g. symptom burden, age-of-onset, fatality-risk) and single-item Likert (mild, moderate, severe) scales. RESULTS: Twenty patients were enrolled; mean age at testing 46 + 21 years and age at diagnosis 32 + 18 years; 35% male. Prior treatment included surgery (50%), radiotherapy (25%), and systemic therapy (40%). Mean best WRS was 71.8 + 37% (n = 20). Two patients had no functional hearing and 10 had unilateral hearing. Mean total VS volume was 5.8 + 7.4 cc (n = 15). Additional lesions included meningioma (75%) and ependymoma (20%). Mean NFTI-QOL score was 8.5 + 5.1 and correlated well with all physical dimensions of the SF-36 (all p < 0.002). NFTI-QOL correlated well with PatSev (r = 0.60, 95%CI 0.21-0.82, p < 0.005) and multi-item ProvSev (r = 0.66, 0.31-0.85, p = 0.002) but not single-item ProvSev (r = 0.33, -0.13-0.68, p = 0.15). A trend toward positive correlation was observed between PatSev and multi-item ProvSev (r = 0.41, -0.04-0.72, p = 0.07). NFTI-QOL scores did not correlate with VS volume (r = 0.10, -0.44-0.58, p = 0.73), or best WRS (r = -0.40, -0.71-0.05, p-0.08). CONCLUSION: NFTI-QOL scores did not correlate with hearing function or tumor volume, but did correlate with patient and provider-reported disease severity underscoring the importance of patient-focused measures in therapeutic outcome assessment in NF2.