期刊
CANCER DISCOVERY
卷 11, 期 11, 页码 2764-2779出版社
AMER ASSOC CANCER RESEARCH
DOI: 10.1158/2159-8290.CD-21-0094
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资金
- German Cancer Aid [111234]
- German Childhood Oncology Foundation [DKS 2014.12, DKS 2018.18]
- German Federal Ministry of Health [ZMVI1-2520IGW004]
- German Federal Ministry of Education and Research [01KX2025]
- Structure-and Innovation Fund, BadenWurttemberg
- German Cancer Consortium (DKTK, Heidelberg, Germany) via the German Cancer Research Center (DKFZ)
- German Cancer Research Center (DKFZ)
- Heidelberg University Hospital, Heidelberg, Germany
The INFORM registry collected clinical and molecular data of pediatric cancer patients and tested a target prioritization algorithm in a real-world setting, identifying subgroups of patients benefiting from matched targeted treatment, leading to improved progression-free survival, refinement of diagnosis, and identification of hereditary cancer predisposition syndromes.
INFORM is a prospective, multinational registry gathering clinical and molecular data of relapsed, progressive, or high-risk pediatric patients with cancer. This report describes long-term follow-up of 519 patients in whom molecular alterations were evaluated according to a predefined seven-scale target prioritization algorithm. Mean turnaround time from sample receipt to report was 25.4 days. The highest target priority level was observed in 42 patients (8.1%). Of these, 20 patients received matched targeted treatment with a median progression-free survival of 204 days [95% confidence interval (CI), 99-not applicable], compared with 117 days (95% CI, 106-143; P = 0.011) in all other patients. The respective molecular targets were shown to be predictive for matched treatment response and not prognostic surrogates for improved outcome. Hereditary cancer predisposition syndromes were identified in 7.5% of patients, half of which were newly identified through the study. Integrated molecular analyses resulted in a change or refinement of diagnoses in 8.2% of cases. SIGNIFICANCE: The pediatric precision oncology INFORM registry prospectively tested a target prioritization algorithm in a real-world, multinational setting and identified subgroups of patients benefiting from matched targeted treatment with improved progression-free survival, refinement of diagnosis, and identification of hereditary cancer predisposition syndromes.
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