The introduction of multidisciplinary treatment strategies constitutes a major advance to the improvement of survival of children with cancer. For more common childhood cancers, multimodal treatment is centrally coordinated by national or multinational cooperative groups and reference centers. Thus, an optimized orchestration of different therapeutic modalities is achieved with the best possible and risk adapted stratification, scheduling and dosing. In addition, patients with particularly problematic cancers can be referred to experts, both for consultation and for treatment.
Most cooperative groups and reference centers offer medical/treatment guidelines to the collaborating centres, that address these highly specific questions. Central review of pathologic diagnosis is often offered and, in some instances , multidisciplinary evaluation of difficult cases is provided. This consulting activity is organized in different ways and may provide different levels of assistance depending on available local resources and organization. In some, but not all of these activities, the consultation takes place within an interdisciplinary tumour board orchestrated by the responsible study coordinators.
Based on these experiences the constitution of officially recognized well organized tumour boards is considered an essential component of excellence in paediatric cancer care, as they may coordinate a broad range of medical experts in different disciplines, allowing for discussions and decisions on how to best care for each single patient with cancer and thereby assuring quality of care.
While such initiatives have already been established for more common childhood cancers, such infrastructure is unavailable for very rare paediatric tumours (VRT). In total numbers, all paediatric cancers might be considered “rare”. However, in the “uncommon” rare paediatric tumours, the problems are not only infrequent events but the complexity and heterogeneity of diagnoses. Indeed, the panel of diagnoses may include adult cancers, such as epithelial cancers, that rarely occur in the pediatric age (e.g. colon carcinoma, adenocarcinoma, melanoma etc.), and specific pediatric entities with extremely low incidence (e.g. pancreatoblastoma, pleuropulmonary blastoma etc.).
As a result, histopathological diagnosis may be uncertain and too unspecific, and the responsible physicians at a clinical center may never have treated a patient with this particular diagnosis before. Sometimes, it may also be difficult to find an expert on a particularly rare tumour – even on a national basis. The extreme rarity of these diagnoses also leads to the obvious lack of scientific evidence with regard to optimal treatment.
Thus, the experience in the management of VRT is low and may impact the quality of care in these patients, too. In contrast to more frequent tumour entities, no national study centers with the appropriate infrastructure have been established, and the visibility of very rare tumour study groups may be less compared to study groups of more common paediatric tumours.
Central consultation at reference centers is of critical importance for the care of patients with very rare cancers, and even more than for more frequent cancer types, in which clinical experience is more widespread.
Therefore, the establishment of a central virtual tumour board (VTB) is essential to create a network of experts in different tumours that can discuss the best management of patients with VRT. This VTB has to be established in an international setting that includes experts with outstanding expertise. Recent advances in telecommunication, such as modern broadband telemedicine technology, have made possible the establishment of such tumour boards. These technologies can overcome physical limits and may connect teams of specialists from different centers and even different nations or continents in a VTB.
This VTB can be contacted for requests regarding verification of diagnosis and optimal treatment. To ensure high quality of consultation, standard requirements for clinical consultation will be established, including pathology and surgical reports, imaging and clinical documentation.
The discussion, decision and clinical data on treatment and outcome will be documented centrally. Lastly, a tracking system for follow-up of each patient will be included, allowing for expanding the knowledge of outcomes in these rare tumours. Thus, a central VTB may become a formidable consultation platform to increase the experience of the experts and transfer knowledge to the paediatric oncology community.
The implementation of VTB has also been included in the EU Directive 2011/24/EU of patients’ rights in cross-border healthcare as a tool to increase the capacity of healthcare providers and fundamental to create European Network of references.
VTB can provide medical advice, allowing the patient to receive appropriate treatment in the local Centre, but can also facilitate access to Centres with adequate expertise throughout Europe if a particular treatment is needed.
During the last decade, several national groups have been established that specifically focus on VRT in children and adolescents. These initiatives have increased the awareness of the problem of VRT. In June 2008 this led to the formation of a new cooperative group, European Cooperative Study Group for Pediatric Rare Tumours (EXPeRT). The primary aim of this group is to empower the clinical and biological research on VRT by promoting collaboration between the founder national groups: Italy, France, United Kingdom, Poland and Germany. It is expected that additional European countries will join the group in the short term.
The establishment of a VRT-VTB is supported by the experience gained by EXPeRT in the recent years and would represent an important step in the further development of EXPeRT activities. The establishment of the VRT-VTB in coordination with other EXPeRT activities will also provide a framework and possibly human resources to sustain this initiative after the conclusion of ExPO-r-NetT.