Abstract
BACKGROUND: Some newly diagnosed pediatric diffuse intrinsic brainstem glioma (DIPG) are with a wide range of infiltration, which may progress rapidly and cannot tolerate radiotherapy (RT). We define these DIPGs with symptoms rapidly deteriorated before or during radiotherapy with or without imaging progress as refractory DIPGs. New active treatment are required to treat these pediatric refractory DIPG. The purpose of the study was to assess the efficacy and toxicity of bevacizumab (BeV) combined with RT as salvage therapy in children with refractory DIPGs. METHODS: 14 patients younger than 14 years old with newly diagnosed DIPG were enrolled. Their symptoms rapidly deteriorated before or during radiotherapy with or without imaging progress. Bevacizumab combined with RT as salvage treatment was given. Their clinical features, MRI images and clinical test result was recorded and retrospectively analysed. The effect of combined treatment was evaluated according to the RANO criterion. The patient’s progression-free survival and overall survival time was assessed by Kaplan - Meier method. RESULT: With the combined treatment, 12 cases in 14 received radiographic and clinical symptoms improved and the total effective rate was 92.9%. The proportion of patients using dexamethasone was 100%(14/14) before combined treatment and decreased to 14.3%(2/14) 2 weeks later after that. Progression-free survival time was ranged from 0-14 months, median PFS time was 3 months. Overall survival time was ranged from 1 to 19 months, the median OS time was 6 months. Three cases underwent 1°myelosuppression, 1 case underwent 1°hypertension. There’s no hematuria, proteinuria, liver and kidney function damage and other side effects. CONCLUSION: Bevacizumab combined radiotherapy as a salvage treatment is effective for pediatric refractory DIPG, but does not improve the poor prognosis.