Prognosis for pediatric patients with refractory or relapsed tumor disease is unfavorable. Significant improvements in 5-year survival rates were achieved by optimizing intensifying chemotherapy concepts since the 1970s. However in the last two decades, the 5-year survival rats in most tumor entities have stagnated.
Advanced genetic characterization of tumor cells and activated signaling pathways reveal more and more targets for targeted antineoplastic therapy. The efficacy and value of these new therapies is currently poorly evaluated and requires testing in early phase clinical trials.
Pediatric patients with refractory or relapsed tumors are rare. In addition, this patient group is extremely heterogeneous with respect to genetic basis, diagnoses, and existing toxicity after first-line treatment. For this reason, clinical trials with a gain of knowledge for all participants are only possible in a multicenter setting.