“If you work on frequent cancers, do randomized trials! If you work on rare cancers – FIND FRIENDS!” This is exactly the spirit that led to the foundation of the European Cooperative Study Group for Pediatric Rare Tumors (EXPeRT) in June 2008, which is officially supported by the International Society of Peadiatric Oncology – Europe (SIOP-E).
Children with very rare tumors have not benefited to the same extent from the enormous advances made in pediatric oncology. This is why several national groups specifically focusing on rare cancers in childhood have been founded in Italy, France, the UK, Poland and Germany in the new millennium and joined forces in EXPeRT. These initiatives have contributed to increase awareness and improve knowledge, treatment and outcome of rare pediatric tumors.
The definition of very rare tumors in children and adolescents is generally based on an annual incidence of <2 in a million and includes a heterogeneous assortment of neoplasms (e.g. pancreatoblastoma, pleuropulmonary blastoma, malignant melanoma). In most instances, these very rare tumors are not in the focus of cooperative clinical studies aiming for better diagnostic assessment and therapy of childhood cancers.
EXPeRT aims to enhance the knowledge on very rare pediatric tumors by exchanging data and performing analyses, to develop evidence-based and internationally recognized guidelines and to enforce EXPeRT consultation (Expo-r-Net) in order to assist in clinical decisions, and to empower the clinical and molecular genetic research on very rare pediatric tumors. By collaboration of the European countries and thus larger numbers of patients, experience can be expanded significantly. Thus, each patient with a very rare tumor does not have to remain a ‘first case’, but can benefit from experience gained from other patients with the same diagnosis.