Ketogenic Diet Treatment in Diffuse Intrinsic Pontine Glioma in Children: retrospective analysis of feasibility, safety and survival data

Background: Diffuse Intrinsic Pontine Glioma (DIPG) is one of the most devastating diseases amongst children with cancer, thus novel strategies are urgently needed. We retrospectively evaluated DIPG patients exposed to the carbohydrate restricted ketogenic diet (KD) with regards of feasibility, safety and overall survival (OS). Methods: Searches of MEDLINE and Embase identified four hits meeting the inclusion criteria (diagnosis of DIPG and exposure to KD ≥ 3 months). One additional case was identified by contact with experts. Individual patient data were extracted from publications or obtained from investigators. Feasibility was defined as number of patients who were able to use the KD for ≥ 3 months. OS was estimated by the Kaplan-Meier method. Results: Five DIPG patients (males, n=3; median age 4.4 years; range, 2.5-15 years) meeting the inclusion criteria were identified. Analysis of the available data showed that the KD is generally well tolerated. Only mild gastro-intestinal complaints, one borderline hypoglycemia (2.4 mmol/l) and one hyperketosis (max 7.2 mmol/l) were observed. Further analyses showed that the KD was feasible in DIPG patients (median KD duration, 7 months; range, 0.25-2 years). The median OS was 18.7 months. Conclusion: The KD appears to be feasible and safe, no serious adverse events were observed. The OS appeared to be favorable. These results may encourage further efforts on a larger scale; ideally through an observational prospective study assessing the most adequate nutritional composition of the KD and its impact on DIPG patients when started shortly after diagnosis