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Very-high-dose cisplatin and etoposide in children with untreated advanced neuroblastoma
O Hartmann1, C R Pinkerton, T Philip
1Pediatric Department, Institut Gustave-Roussy, Villejuif, France.
Insights
This pilot study found that high-dose cisplatin and etoposide chemotherapy in children with advanced neuroblastoma showed encouraging response rates with acceptable toxicity, despite manageable side effects like myelosuppression and electrolyte imbalances.
Area of Science:
- Pediatric Oncology
- Cancer Chemotherapy
Background:
- Advanced neuroblastoma presents a significant challenge in pediatric oncology.
- Effective treatment strategies are crucial for improving outcomes in young patients.
Purpose of the Study:
- To evaluate the toxicity and response rates of a high-dose cisplatin (CPDD) and etoposide (VP-16) regimen in children with advanced neuroblastoma.
- To assess the safety and efficacy of this short-term chemotherapy protocol.
Main Methods:
- A pilot study involving 17 children with advanced neuroblastoma (stages III and IV).
- Administration of two courses of high-dose cisplatin (200 mg/m2) and etoposide (500 mg/m2).
- Monitoring of toxicity, including renal function, hearing, neuropathy, electrolytes, and myelosuppression, alongside response assessment.
Main Results:
- Partial response (PR) was observed in 12 of 17 patients.
- Complete clearing of disease in bone marrow was achieved in 6 of 15 patients.
- Severe but brief myelosuppression occurred, with 7 patients requiring platelet transfusions and 7 readmitted for febrile neutropenia; however, no treatment-related deaths were reported.
Conclusions:
- The high-dose cisplatin and etoposide regimen demonstrated encouraging response rates and acceptable toxicity in advanced neuroblastoma.
- Careful supervision is essential for managing the associated toxicities, which were comparable to longer-term cisplatin administration.
- The rapid and significant response warrants further investigation of this treatment approach.
Abstract:
Between January and December 1985, 17 children with advanced neuroblastoma who were greater than 1 year old (16 stage IV, one stage III) were administered cisplatin (CPDD, 200 mg/m2) and etoposide (VP-16, 500 mg/m2) as a pilot study of toxicity and response rates for the European Neuroblastoma Study Group (ENSG). The study was designed to assess toxicity of two courses of treatment, and evaluate response rates after this short therapy. The creatinine clearance declined in seven of 15 patients. No patient experienced clinically significant hearing loss, but formal audiometric assessment of nine children revealed characteristic high tone loss in seven patients. Peripheral neuropathy was not seen. Asymptomatic hypomagnesemia (less than 0.7 microEq/L) was frequent, despite routine supplementation. Asymptomatic electrolyte imbalances occurred frequently, but were generally transient. Myelosuppression was severe, but brief. Seven patients required platelet transfusions and seven were readmitted between courses due to febrile episodes while neutropenic. There were no treatment-related deaths. According to strictly defined criteria, 12 of 17 patients showed a partial response (PR), and extensive marrow evaluation showed complete clearing of disease in six of 15 patients. This high-dose regimen, if carefully supervised, is associated with acceptable toxicity, comparable to that seen when the dose of CPDD is spread over several months. The rapidity and degree of response was encouraging and merits further evaluation.