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Progressive disease in children with medulloblastoma/PNET during preradiation chemotherapy
A Tornesello1, S Mastrangelo, D Piciacchia
1Division of Pediatric Oncology, Catholic University, Rome, Italy.
Journal of Neuro-Oncology
|April 25, 2000
Summary
Intensive chemotherapy for high-risk medulloblastoma (MB/PNET) showed carboplatin effectiveness but high progression rates with cyclophosphamide. Further research is needed to reduce progression and improve survival in pediatric MB/PNET patients.
Area of Science:
- Pediatric Oncology
- Neuro-oncology
- Chemotherapy Regimens
Background:
- Prognosis for pediatric medulloblastoma/primitive neuroectodermal tumors (MB/PNET) has stagnated.
- Intensive neoadjuvant chemotherapy requires further exploration for high-risk cases.
Purpose of the Study:
- To evaluate short-term clinical outcomes of an intensive chemotherapy regimen in high-risk, newly diagnosed MB/PNET children.
- To assess the efficacy and safety of a neoadjuvant chemotherapy sequence before radiation therapy.
Main Methods:
- Twelve high-risk MB/PNET patients received high-dose carboplatin followed by high-dose cyclophosphamide and vincristine.
- Treatment was administered in cycles after surgery and prior to radiation therapy.
- Disease response (CR, PR, MR, PD) was evaluated using neuroimaging of the entire neuraxis.
Main Results:
- High-dose carboplatin showed effectiveness, with 1 complete response (CR), 4 partial responses (PR), and 2 minor responses (MR) after two cycles.
- Subsequent high-dose cyclophosphamide yielded additional responses in 4 patients but also showed progressive disease (PD) in 4 patients.
- An unacceptable incidence of PD was observed during cyclophosphamide therapy, particularly when radiotherapy was delayed.
Conclusions:
- Two courses of high-dose carboplatin are effective in high-risk MB/PNET.
- High-dose cyclophosphamide in this neoadjuvant setting led to an unacceptable rate of progressive disease.
- Optimizing drug combinations for "sandwich chemotherapy" is crucial to reduce PD and improve long-term survival in pediatric MB/PNET.