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

Neurology. 2013 Nov 19;81(21 Suppl 1):S15-24. doi: 10.1212/01.wnl.0000435745.95155.b8.


Objective: 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.

Methods: 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.

Results: 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.

Conclusions: The use of these key functional endpoints will be essential for evaluating the efficacy of future OPG clinical trials.

Trial registration: NCT00879034.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Child
  • Clinical Trials as Topic / methods
  • Clinical Trials as Topic / standards*
  • Consensus
  • Humans
  • Neurofibromatosis 1 / complications
  • Neurofibromatosis 1 / therapy*
  • Optic Nerve Glioma / complications
  • Optic Nerve Glioma / etiology
  • Optic Nerve Glioma / therapy*
  • Treatment Outcome*
  • Vision Disorders / diagnosis
  • Vision Disorders / etiology
  • Vision Disorders / therapy*
  • Vision Tests / methods
  • Vision Tests / standards*

Associated data