Functional outcome measures for NF1-associated optic pathway glioma clinical trials.

in Neurology by Michael J Fisher, Robert A Avery, Jeffrey C Allen, Simone L Ardern-Holmes, Larissa T Bilaniuk, Rosalie E Ferner, David H Gutmann, Robert Listernick, Staci Martin, Nicole J Ullrich, Grant T Liu,

TLDR

  • The Response Evaluation in Neurofibromatosis and Schwannomatosis Visual Outcomes Committee recommended visual acuity, optic disc pallor, and visual quality of life measures as key functional endpoints for NF1-OPG clinical trials.

Abstract

The goal of the Response Evaluation in Neurofibromatosis and Schwannomatosis Visual Outcomes Committee is to define the best functional outcome measures for future neurofibromatosis type 1 (NF1)-associated optic pathway glioma (OPG) clinical trials. The committee considered the components of vision, other ophthalmologic parameters affected by OPG, potential biomarkers of visual function, and quality of life measures to arrive at consensus-based, evidence-driven recommendations for objective and measurable functional endpoints for OPG trials. Visual acuity (VA) assessments using consistent quantitative testing methods are recommended as the main functional outcome measure for NF1-OPG clinical trials. Teller acuity cards are recommended for use as the primary VA endpoint, and HOTV as a secondary endpoint once subjects are old enough to complete it. The optic disc should be assessed for pallor, as this appears to be a contributory variable that may affect the interpretation of VA change over time. Given the importance of capturing patient-reported outcomes in clinical trials, evaluating visual quality of life using the Children's Visual Function Questionnaire as a secondary endpoint is also proposed. The use of these key functional endpoints will be essential for evaluating the efficacy of future OPG clinical trials.

Overview

  • The Response Evaluation in Neurofibromatosis and Schwannomatosis Visual Outcomes Committee aimed to define the best functional outcome measures for future neurofibromatosis type 1 (NF1)-associated optic pathway glioma (OPG) clinical trials.
  • The committee considered various components, including vision, ophthalmologic parameters, potential biomarkers, and quality of life measures to arrive at consensus-based recommendations.
  • The primary objective was to identify objective and measurable functional endpoints for OPG trials, which will help evaluate the efficacy of future clinical trials.

Comparative Analysis & Findings

  • The committee recommended visual acuity (VA) assessments using consistent quantitative testing methods as the main functional outcome measure for NF1-OPG clinical trials.
  • Teller acuity cards and HOTV assessments were recommended as primary and secondary endpoints, respectively, once subjects are old enough to complete them.
  • Assessing optic disc pallor and evaluating visual quality of life using the Children's Visual Function Questionnaire were also proposed as secondary endpoints.

Implications and Future Directions

  • The use of these key functional endpoints will be essential for evaluating the efficacy of future OPG clinical trials.
  • Capturing patient-reported outcomes will provide a comprehensive understanding of the impact of OPG on patients' quality of life.
  • Future research may focus on developing more biomarkers and refining the assessment methods to better capture the complexities of OPG and its effects on patients.
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