Outcome of children less than three years old at diagnosis with non‐metastatic medulloblastoma treated with chemotherapy on the “Head Start” I and II protocols

作者：Girish Dhall, Howard Grodman, Lingyun Ji, Stephen A. Sands, Sharon L. Gardner, Ira J. Dunkel, Geoffrey McCowage, Blanca Diez, Jeffrey C. Allen, Anjali Gopalan, Albert Cornelius, Amanda Termuhlen, Minnie Abromowitch, Richard Sposto, Jonathan L. Finlay · 发表于：Pediatric Blood & Cancer · 年份：2008 · DOI：10.1002/pbc.21525 · 被引用次数：242 · 研究领域：Glioma Diagnosis and Treatment、Brain Metastases and Treatment、Neuroblastoma Research and Treatments

PURPOSE: To determine the survival of infants and young children with non-metastatic medulloblastoma using intensive myeloablative chemotherapy and autologous hematopoietic progenitor cell rescue (AuHCR). METHODS: Twenty-one children less than 3 years old at diagnosis with non-metastatic medulloblastoma were enrolled on two identical serial studies, "Head Start" I and "Head Start" II. After surgery, patients received five cycles of induction chemotherapy consisting of vincristine, cisplatin, cyclophosphamide and etoposide. Following induction, all patients underwent myeloablative chemotherapy using carboplatin, thiotepa and etoposide with AuHCR. Irradiation was used only at relapse. RESULTS: The 5-year event-free (EFS) and overall survival (OS) rates (+/-SE) for all patients, patients with gross total resection, and patients with residual tumor were 52 +/- 11% and 70 +/- 10%, 64 +/- 13% and 79 +/- 11%, and 29 +/- 17% and 57 +/- 19%, respectively. The 5-year EFS and OS ( +/- SE) for patients with desmoplastic and classical medulloblastoma were 67 +/- 16% and 78 +/- 14%, and 42 +/- 14 and 67 +/- 14%, respectively. There were four treatment related deaths. The majority of survivors (71%) avoided irradiation completely. Mean intellectual functioning and quality of life (QoL) for children surviving without irradiation was within average range for a majority of survivors tested. CONCLUSION: This strategy of brief intensive chemotherapy for young children with non-metastatic medullo...