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Pediatric phase II cancer chemotherapy trials: a Pediatric Oncology Group study
S Weitman1, S Ochoa, J Sullivan
1University of Texas Health Science Center, San Antonio 78284-7810, USA.
Purpose:
This study reviewed the Pediatric Oncology Group experience with phase II clinical trials in children (< 21 years of age) with refractory tumors.
Patients And Methods:
Patients registered in Pediatric Oncology Group phase II studies were evaluated. Patients had to be < 21 years of age with recurrent and refractory measurable disease. Tumor types and response rates were determined. Death on therapy from either drug toxicity, progressive disease, infection, or hemorrhage was measured. Tumor-specific, disease-free survival curves were calculated by Kaplan-Meier analysis.
Results:
Between 1984 and 1994, 2,465 patient entries were made on 45 phase II trials. Malignancies registered included acute lymphocytic leukemia (ALL) (16.7%), acute myeloid leukemia (AML) (12.0%), osteogenic sarcoma (7.8%), neuroblastoma (7.2%), astrocytoma (7.2%), medulloblastoma (7.1%), glioma (6.7%), ependymoma (6.1%), and others (29.2%). The overall response rate was 19.6% (CR + PR) for children entered on phase II trials. Tumor-specific response rates ranged from 62.1% (23/37) for children with Hodgkin's disease to no responses (0/23) in patients with hepatoblastoma. When comparing single versus multiagent trials, a significantly better initial response rate was seen in the latter studies. However, 5-year survival was comparable. Progression-free survival for all tumor histologies were 12.9% and 9.2% at 2 and 5 years, respectively. Death on study was seen in 11.6% of the patients; however, only three deaths were directly related to drug toxicity. There were no significant gender differences in regards to response, progressive disease, or death on study.
Conclusion:
Phase II studies conducted in children offer a considerable likelihood of therapeutic benefit without exposing these patients to untoward toxicity.
Insights
Pediatric Oncology Group phase II trials for refractory pediatric tumors show a 19.6% overall response rate. These studies offer therapeutic benefit with low drug toxicity in children with cancer.
Area of Science:
- Pediatric Oncology
- Clinical Trials
- Cancer Research
Background:
- Refractory pediatric tumors present significant treatment challenges.
- Phase II clinical trials are crucial for evaluating novel therapies in pediatric oncology.
Purpose of the Study:
- To review the Pediatric Oncology Group's experience with phase II clinical trials.
- To evaluate outcomes in children (< 21 years) with refractory tumors.
Main Methods:
- Analysis of patient data from 45 Pediatric Oncology Group phase II trials (1984-1994).
- Inclusion criteria: < 21 years, recurrent/refractory measurable disease.
- Assessment of tumor types, response rates, survival, and treatment-related deaths.
Main Results:
- 2,465 patient entries across various pediatric malignancies including leukemia, sarcoma, and brain tumors.
- Overall response rate of 19.6% (CR + PR); tumor-specific rates varied widely.
- Multiagent trials showed better initial response rates, but 5-year survival was comparable.
- Progression-free survival at 5 years was 9.2%; only 3 deaths were directly drug-related.
Conclusions:
- Phase II trials in pediatric oncology provide a substantial chance for therapeutic benefit.
- These trials demonstrate acceptable toxicity profiles for pediatric cancer patients.