Effective re-induction regimen for children with recurrent medulloblastoma

Abstract

Abstract Background There is no standard treatment for recurrence of medulloblastoma, the most common malignant childhood brain tumor, and prognosis remains dismal. In this study, we introduce a regimen that is well-tolerated and effective at inducing remission. Methods The primary objectives of this study were to assess tolerability of the regimen and overall response rate. A retrospective chart review of patients with recurrent medulloblastoma, treated at two institutions with a re-induction regimen of intravenous irinotecan and cyclophosphamide with oral temozolomide and etoposide, was performed. Demographic, clinicopathologic, toxicity and response data were collected and analyzed. Results Nine patients were identified. Median age was 5.75 years. Therapy was well-tolerated with no therapy-limiting toxicities and no toxic deaths. Successful stem cell collection was achieved in all five patients in whom it was attempted. Overall response rate after two cycles was 78%. Three patients had a complete response, four patients had a partial response, one patient had stable disease, and one patient had progressive disease. Four patients are alive with no evidence of disease (NED), two patients are alive with disease, two patients have died of disease, and one patient died of toxicity related to additional therapy (NED at time of death). Conclusion This regimen is well-tolerated and effective. Tumor response was noted in the majority of cases, allowing patients to proceed to additional treatment with no or minimal disease. Further study of this regimen in a clinical trial setting is an important next step.