Efficacy of temozolomide for recurrent embryonal brain tumors in children.
OBJECTIVE
The salvage therapy of recurrent embryonal brain tumors in children is disappointing. Temozolomide is a newly developed chemotherapeutic agent in central nervous system tumors. This study analyzed the efficacy of temozolomide on the treatment of recurrent embryonal brain tumors in children.


METHODS
There were eight patients, including four with medulloblastoma (MB), three with atypical teratoid/rhabdoid tumor (AT/RT) and one with supratentorial primitive neuroectodermal tumor, whose tumors recurred after surgery and radiotherapy, with or without conventional intravenous cisplatin-based chemotherapy. They all received once daily oral temozolomide (150 mg/m(2)/day) for five consecutive days in a 28-day cycle. The responsiveness of the tumors to temozolomide was judged by magnetic resonance imaging (MRI) during regular follow-up.


RESULTS
The median treatment cycles received by these eight patients were 17 (range from two to 59 cycles). The follow-up MRI showed no tumor progression in five patients at 6 months and four patients at 12 months. The median progression-free survival (PFS) of the eight patients was 15.7 months (range from 0 to 59 months). Complete response was achieved in one patient with MB accompanying with a long period of PFS for 26 months. Another patient with AT/RT showed partial response accompanying with a long period of PFS for 59 months. The observed adverse effects of temozolomide included nausea, vomiting, headache, constipation, mild marrow suppression, and decreased activity; none of them was severe enough to discontinue the treatment. No patient experienced moderate or severe marrow suppression in this series.


CONCLUSIONS
In this preliminary study, oral temozolomide shows promising results on recurrent embryonal brain tumors in children. The adverse effects of temozolomide are mild and tolerable. When conventional chemotherapy fails and/or the adverse response is too severe to tolerate, temozolomide is a reasonable alternative. However, a further well-designed, controlled study and more long-term follow-up are needed to assess the exact role of temozolomide in children with embryonal tumors in brain.
Wang
Chung-Hao
CH
Department of Pediatrics, Taipei Veterans General Hospital, 201, Section 2, Shih-Pai Road, Taipei, 112 Taiwan, Republic of China. wjorden@gmail.com
Hsu
Ting-Rong
TR
Wong
Tai-Tong
TT
Chang
Kai-Ping
KP
D000284:Administration, Oral; D000293:Adolescent; D018906:Antineoplastic Agents, Alkylating / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000188:drug therapy*; D003606:Dacarbazine / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006261:Headache / Q000139:chemically induced; D006801:Humans; D008279:Magnetic Resonance Imaging; D008297:Male; D008527:Medulloblastoma / Q000188:drug therapy* / Q000532:radiotherapy / Q000601:surgery; D009325:Nausea / Q000139:chemically induced; D018242:Neuroectodermal Tumors, Primitive / Q000188:drug therapy* / Q000532:radiotherapy / Q000601:surgery; D018335:Rhabdoid Tumor / Q000188:drug therapy* / Q000532:radiotherapy / Q000601:surgery; D000077204:Temozolomide; D013724:Teratoma / Q000188:drug therapy* / Q000532:radiotherapy / Q000601:surgery; D016896:Treatment Outcome; D014839:Vomiting / Q000139:chemically induced
D018906:Antineoplastic Agents, Alkylating; D003606:Dacarbazine; D000077204:Temozolomide