Single-arm phase II study of PC chemotherapy in patients with gliomatosis cerebri.
In this study, 35 patients were treated with PC chemotherapy. The study has been completed, analyzed, and published (Herrlinger et al., Ann Neurol 70:445-453,2011). With PC therapy alone, median progression-free survival was 14 months, and median overall survival was 30 months. Detection of an IDH-1 mutation and the absence of a bihemispheric symmetric growth pattern on MRI were found to be independent positive prognostic factors in a multivariate analysis. The NOA-05 study is the only prospective therapeutic study in gliomatosis cerebri to date and establishes PC therapy alone as a reasonable first-line treatment for gliomatosis cerebri.