Does Surveillance-Detected Disease Progression Yield Superior Patient Outcomes in High-Grade Glioma?

BACKGROUND: Standard follow-up care for patients with high-grade glioma (HGG) involves routine surveillance imaging to detect disease progression, assess treatment response, and monitor clinical symptoms. Although logical in nature, evidence supporting this practice is limited. We hypothesize patients with tumor recurrence detected on routine surveillance imaging will experience superior outcomes relative to symptomatic detection, using measures of survival and postrecurrence neurologic function. METHODS: Adult patients receiving treatment for HGG at our institution from 2004 to 2018 were identified, and data including tumor characteristics, imaging results, neurologic status, and survival were extracted from the medical records of patients meeting inclusion criteria. All participants were followed for a minimum of 12 months, or for survival duration. Survival and neurologic function differences were assessed using log rank and 2-sample t tests with 2-sided 0.05 alpha level of significance. RESULTS: Of the 74 patients meeting inclusion criteria, 47 (63.5%) had recurrence detected via routine surveillance imaging, and 27 (36.5%) had symptomatic detection outside of the surveillance schedule. Neither median overall survival (14.8 months for surveillance and 15.7 months for symptomatic; P = 0.600) nor postrecurrence neurologic function (assessed by Karnofsky Performance Scale Index and Eastern Cooperative Oncology Group) differed between the surveillance and symptomatic detection groups (P = 0.699 and P = 0.908, respectively). CONCLUSIONS: Recurrence detection occurring via routine surveillance imaging did not yield superior patient outcomes relative to symptomatic detection occurring outside of the standard surveillance schedule in patients with HGG. Further evaluation of surveillance imaging and alternative follow-up methods for this patient population may be warranted.

Dissecting Clinical Heterogeneity in Neurofibromatosis Type 1.

Neurofibromatosis type 1 (NF1) is a common neurogenetic disorder in which affected children and adults are predisposed to the development of benign and malignant nervous system tumors. Caused by a germline mutation in the NF1 tumor suppressor gene, individuals with NF1 are prone to optic gliomas, malignant gliomas, neurofibromas, and malignant peripheral nerve sheath tumors, as well as behavioral, cognitive, motor, bone, cardiac, and pigmentary abnormalities. Although NF1 is a classic monogenic syndrome, the clinical features of the disorder and their impact on patient morbidity are variable, even within individuals who bear the same germline NF1 gene mutation. As such, NF1 affords unique opportunities to define the factors that contribute to disease heterogeneity and to develop therapies personalized to a given individual (precision medicine). This review highlights the clinical features of NF1 and the use of genetically engineered mouse models to define the molecular and cellular pathogenesis of NF1-associated nervous system tumors.

Macrocephaly Is Not a Predictor of Optic Pathway Glioma Development or Treatment in Neurofibromatosis Type 1.

Neurofibromatosis type 1 is a common neurogenetic disorder characterized by significant clinical variability. As such, numerous studies have focused on identifying clinical, radiographic, or molecular biomarkers that predict the occurrence or progression of specific clinical features in individuals with neurofibromatosis type 1. One of these clinical biomarkers, macrocephaly, has been proposed as a prognostic factor for optic pathway glioma development. In the current study, the authors demonstrate that macrocephaly is not associated with the development of these brain tumors or the need to institute treatment for clinical progression. These findings suggest that macrocephaly is not a robust biomarker of optic pathway glioma formation or progression in children with neurofibromatosis type 1.