SC-4013: Developing Evidence-Based Criteria for Initiating Treatment for Neurofibromatosis Type 1 Associated Optic Pathway Glioma
SC4013-4013
What is the goal of the study?
Optic pathway gliomas (OPGs) are low-grade astrocytomas that occur commonly in children with neurofibromatosis type 1 (NF1). Although rarely life-threatening, these tumors can cause significant morbidity, including vision loss, disfiguring proptosis, and precocious puberty. Half to two-thirds of NF1-OPG are indolent with minimal tumor progression. When they do progress, progression tends to be slow. Given the indolent nature of many of these tumors, initial management entails close observation with serial phthalmologic and MRI examinations. Prior studies reveal a lack of correlation between radiographic and visual outcomes for NF1-OPG; therefore, visual acuity is recommended to be the primary outcome measure in future OPG clinical trials. Although we have learned a great deal about visual outcomes from a large retrospective series, several crucial questions remain. Specifically, there is a lack of information regarding the natural change in vision over time for NF1-OPG. It is unclear if radiographic tumor progression and visual decline are concurrent, or if the latter lags behind the former. Although most clinicians wait for visual decline prior to initiating treatment, there is no consensus regarding the reasons to treat. In order to minimize any visual loss and to limit treatment only to the subset of patients that truly require it, we need to define the treatment indications and factors (age, gender, OPG location, etc.) predictive of visual decline and tumor progression. Therefore, a prospective natural history study of NF1-OPG is needed with standardized visual assessment methods and clear definitions of visual outcomes and data acquisition time points. This prospective observational study will enroll 250 children (<18 years of age with nf1 who have newly diagnosed opg. subjects will be followed actively for a minimum of 3 years. however if a subject is started on treatment for their opg sometime during this 3 year follow up period they will be followed for an additional 3 years from the end of this initial treatment. the primary outcome measure is visual acuity. all evaluable subjects will make up the observation cohort. all subjects who start treatment while being actively followed will also be in the treatment cohort. decisions regarding treatment will be made by the local institution based on their clinical judgment. subjects will have regular surveillance mr imaging ophthalmology evaluations and oncology nf1 medical evaluations as per an accepted standard frequency see appendix i-iii. after conclusion of active follow-up all subjects will enter long term follow-up for an additional 10 years.>
Who can participate in the study?
Please contact the study team listed below to learn more.