Convection-enhanced delivery of topotecan into diffuse intrinsic brainstem tumors in children

摘要: Convection-enhanced delivery (CED) for the treatment of malignant gliomas is a technique that can deliver chemotherapeutic agents directly into tumor and surrounding interstitium through sustained, low-grade positive-pressure infusion. This allows high local concentrations drug within while minimizing systemic levels often lead to dose-limiting toxicity. Diffuse intrinsic pontine (DIPGs) are universally fatal childhood tumors which there currently no effective treatment. In this report authors describe CED topoisomerase inhibitor topotecan DIPG in 2 children. As part pilot feasibility study, treated pediatric patients with DIPG. Stereotactic biopsy frozen section confirmation glial was followed by placement bilateral catheters during same procedure. The first patient underwent 210 days after initial diagnosis, radiation therapy at time recurrence, total dose 0.403 mg 6.04 ml over 100 hours. Her Karnofsky Performance Status (KPS) score 60 before 50 posttreatment. Serial MRI initially demonstrated modest reduction size edema, but progressed died 49 second 24 diagnosis prior 0.284 5.30 KPS 70 similarly an size. subsequently fractionated therapy, she 120 Topotecan delivered prolonged brainstem children technically feasible. both patients, infusion rates (> 0.12 ml/hr) volumes 2.8 ml) resulted new neurological deficits score, lower (< 0.04 were well tolerated. While serial showed moderate effect, did not prolong survival these patients. More studies needed improve selection determine optimal flow maximize safety efficacy. Clinical trial registration no.: NCT00324844.