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Children's Oncology Group's 2013 blueprint for research: rare tumors
Carlos Rodriguez-Galindo1, Mark Krailo, Lindsay Frazier
1Dana-Farber/Children's Hospital Cancer Center, Harvard Medical School, Boston, MA 02115, USA. carlos_rodriguez-galindo@dfci.harvard.edu
Abstract:
In the US, approximately 2,000 children are diagnosed with rare cancers each year, with 5-year survival ranging from <20% for children with advanced carcinomas to >95% for children with intraocular retinoblastoma or localized germ cell tumors. During the last years, 12 clinical studies have been successfully completed in children with retinoblastoma, liver tumors, germ cell tumors, and infrequent malignancies, including therapeutic, epidemiologic, and biologic studies. Current efforts are centered in the development of large international collaborations to consolidate evidence-based definitions and risk stratifications that will support international Phase 3 clinical trials in germ cell tumors, hepatoblastoma, and other rare cancers.
Insights
Approximately 2,000 US children annually face rare cancers. Recent studies focused on retinoblastoma and germ cell tumors, with ongoing international collaborations aiming for advanced clinical trials in rare pediatric malignancies.
Area of Science:
- Pediatric Oncology
- Rare Cancers Research
- Clinical Trials
Background:
- 2,000 US children diagnosed annually with rare cancers.
- Survival rates vary widely, from <20% to >95%.
- Significant disparities exist in outcomes for pediatric malignancies.
Purpose of the Study:
- Summarize recent clinical studies in pediatric rare cancers.
- Highlight progress in understanding and treating these diseases.
- Outline future directions for international research collaborations.
Main Methods:
- Review of 12 completed clinical studies.
- Inclusion of therapeutic, epidemiologic, and biologic research.
- Focus on retinoblastoma, liver tumors, germ cell tumors, and other rare cancers.
Main Results:
- Successful completion of 12 clinical studies in rare pediatric cancers.
- Demonstrated feasibility of therapeutic, epidemiologic, and biologic investigations.
- Established a foundation for evidence-based definitions and risk stratifications.
Conclusions:
- International collaborations are crucial for rare pediatric cancers.
- Future Phase 3 trials will focus on germ cell tumors, hepatoblastoma, and other rare malignancies.
- Consolidated evidence and risk stratification will improve treatment outcomes.
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