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Precision medicine for poor-prognosis paediatric patients

Prof. Cornelis van Tilburg, Hopp Children’s Cancer Center, Germany
ASCO 2020

Featured video: INFORM study examining use of precision medicine for pediatric cancers with poor prognosis.


In-depth tumour molecular characterisation of children and adolescents who have relapsed after initial therapy, and for whom there are no established treatment concepts available, can offer diagnostic insight and potential novel therapeutic approaches.

Prof. Cornelis van Tilburg (Hopp Children’s Cancer Center, Germany) presented the INdividualized therapy FOr children with Relapsed Malignancies (INFORM) registry study [1]. INFORM evaluates the clinical potential of precision oncology in children by identifying molecular targets of (off-label) treatment, identifying potential biomarkers for clinical trials or other uses, and providing additional diagnostic precision (diagnostic refinement). INFORM gives children a chance to benefit from off-label (adult oncology) targeted therapies and enrolment in biomarker-driven clinical trials. This presentation is the first time a clinical outcome in a real-world setting of a large multi-national personalised paediatric oncology platform was assessed.

INFORM included 72 sites in 8 countries. All tumour material was molecularly analysed in Heidelberg and discussed by a central board together with the treating paediatric oncologist. An algorithm developed by the project assigned patients to a 7-step, pre-defined priority scale, based on draggability, alteration type, and specific evidence/relevance. Treatment was decided by the treating physician, considering the available clinical trials, off-label options suggested by the algorithm, and other (conventional) therapy. Documentation and clinical follow-up were centrally documented by INFORM.

A total of 526 children were analysed, of which 149 received matching targeted drugs; the algorithm was unable to match 377 patients to any targeted drug. Twenty patients qualified as the highest priority level and were more likely to receive subsequent treatment. Seventeen children (3.2%) were included in clinical trials. Forty children (7.6%) were identified to have potential cancer predisposition syndromes; consequently, their families were offered genetic counselling. Among the children with brain tumours, molecular analyses provided diagnostic refinement in 8%.

In a subgroup analysis of children whose tumours ranked “very high priority level” for a matching targeted drug, an extended and significant progression-free survival was observed (204 days vs 114 days; P=0.0093, see Figure), indicating the algorithm accurately predicts responders to a given therapy.

Figure. Progression-free survival (PFS) of children whose tumours ranked “very high priority level” for a matching targeted drug [1]













Although this study is limited in size and consists of a highly heterogeneous cohort, it suggests a clinical value for accurate molecular profiling in a subset of poor prognosis paediatric patients.

    1. Van Tilburg CM, et al. ASCO Virtual Meeting, 29-31 May 2020,Abstract LBA10503.

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