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Paediatric Oncology Very Rare Tumours in Paediatric Age – From ‘Tumori Rari in Età Pediatrica’ to the European Cooperative Study Group for Paediatric Rare Tumours Gianni Bisogno, 1 Giovanni Cecchetto 2 and Andrea Ferrari 3 1. Paediatric Consultant and Head, Solid Tumour Unit, Haematology/Oncology Division, Department of Paediatrics, University Hospital of Padua; 2. Associate Professor, Division of Paediatric Surgery, Department of Paediatrics, University Hospital of Padua; 3. Oncologist Consultant and Head, Outpatient Service, Paediatric Oncology Unit, National Tumour Institute, IRCCS, Milan, Italy Abstract Very rare tumours (VRTs) in paediatric age are a heterogeneous group of cancers very rarely encountered in daily practice, even in large paediatric oncology centres. Some of them are typical of paediatric age, such as pleuropulmonary blastoma or pancreatoblastoma; others are typically found in adulthood, such as carcinomas and melanoma. With the objective of improving the research on, and management of, paediatric VRTs, a national study group was founded and the Tumori Rari in Età Pediatrica (Rare Tumours in Paediatric Age [TREP]) project was launched in Italy in 2000. For the purposes of this project, VRTs have been defined as “any solid malignancy characterized by an annual incidence of <2 cases/million children and not considered in other clinical trials”. From January 2000 to December 2011, 652 patients <18 years of age were registered in the TREP database. This article presents the experience gathered so far and underlines the need to develop international collaborations dedicated to paediatric VRTs. With this aim, national groups from Italy, Germany, France, Poland and the UK have created, in June 2008, a new collaborative group named European Cooperative Study Group for Paediatric Rare Tumours (EXPeRT). Keywords Very rare tumours, paediatric patients, pleuropulmonary blastoma, nasopharyngeal carcinoma, pancreatic tumours, thyroid carcinoma, melanoma, Tumori Rari in Età Pediatrica (TREP) project, European Cooperative Study Group for Paediatric Rare Tumours (EXPeRT) Disclosure: The authors have no conflicts of interest to declare Acknowledgement: The Tumori Rari in Età Pediatrica (TREP) project has been supported by a grant from the Fondazione Città della Speranza, Padova, and the Fondazione Cassa di Risparmio di Padova e Rovigo. Received: 16 January 2012 Accepted: 6 February 2012 Citation: European Oncology & Haematology, 2012; 8(2):130–4 Correspondence: Gianni Bisogno, Haematology/Oncology Division, Department of Paediatrics, University Hospital of Padua, Via Giustiniani 3, 35128 Padova, Italy. E: gianni.bisogno@unipd.it. Malignant tumours are relatively uncommon in children, especially considering the incidence of cancer in adults. The difficulty of performing meaningful studies due to the limited numbers of patients treated at each paediatric oncology centre has encouraged oncologists to embark on national and international collaboration schemes in order to perform significant research. The positive results achieved by these schemes seem evident from the gradual improvement in the survival rates in nearly all paediatric tumours, to a point where nearly three in four children diagnosed in developed countries can currently be expected to be ‘cured’. Unfortunately, not all children benefit from these efforts; for those affected by exceptionally rare tumours, the very low incidence of their disease has often limited the interest in research programmes capable of collecting meaningful clinical and biological data. As a consequence, it is extremely difficult to produce evidence-based treatment guidelines for these patients and physicians are forced to treat them on an individual basis. Paediatric very rare tumours (VRTs) comprise a variety of entities. Some of them are typical of paediatric age, such as pleuropulmonary 130 blastoma or pancreatoblastoma, and are very rarely encountered in daily practice, even in large paediatric oncology centres. Others are typically found in adulthood, such as thyroid carcinoma, colorectal carcinoma, melanoma, thymic tumours and renal carcinoma. So far, the paediatric oncology community has shown little interest in this group of tumours, which partially explains our lack of understanding of the biology of these disorders and the difficulties encountered in conducting clinical trials. In fact, survival rates have not changed substantially over the years and have remained unsatisfactory for some histotypes. In several European countries, national groups focusing specifically on rare cancers in children have recently been founded to deal with these issues. The ‘Tumori Rari in Età Pediatrica’ Project The Tumori Rari in Età Pediatrica (Rare Tumours in Paediatric Age [TREP]) project was launched in Italy in 2000 and represents a model of network dedicated to rare paediatric tumours. It has not only created a registry for collecting cases, but has also established treatment guidelines for various rare tumours and a network of medical experts who can help clinicians manage their patients. 1 © TOUCH BRIEFINGS 2012