Very Rare Tumours (VRTs) are, by their nature, a major challenge for paediatric oncologists. The low incidence of VRTs has been a key factor in the limited research on these diseases and the collection of clinical and biological data. It is particularly difficult to produce evidence-based treatment guidelines for patients with VRTs and consequently, physicians are forced to treat patients on an individual basis.
Until recently, the paediatric oncology community has shown little interest in this group of tumours, which partially explains the lack of understanding of these disorders and the difficulties encountered in conducting clinical trials.
Some VRTs arise at the paediatric age, other tumours develop during adulthood, but they only rarely develop in children and adolescents. There is growing evidence that at least some of them are biologically and clinically distinct from their adult counterparts. Additionally, a substantial group of “borderline” tumours with uncertain clinical behaviour and no standard treatments are usually also included in the group of VRTs.
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Diagnosis and treatment guidelines adopted for children are often created from those used to treat adults. However, the peculiar biological characteristics of the diseases and the more severe long-term consequences of radiotherapy and chemotherapy in children have to be considered.
These factors emphasise the need for a tailored therapeutic approach and recognition and treatment of patients pose a challenge for paediatric oncologists: it may take many years to establish diagnostic guidelines and treatment concepts.
Even at European level, some diseases are unfortunately too rare to provide sufficient number of cases to conduct clinical trials leading to evidence-based treatment guidelines. Therefore, the need to develop international collaboration on paediatric VRTs is crucial.
A dedicated international VRT network denominated EXPeRT was established in 2008 by five countries (Italy, France, Germany, Poland, United Kingdom) with the aim of improving treatment and research for children with VRTs.
This effort has been supported by the European paediatric oncology community, including all SIOP Europe members, which recognised the need to develop projects specifically dedicated to rare paediatric tumours.
The ExPeRT group was instrumental in successfully applying for the EU funded PARTNER project which is aligned with aims of the European Reference Network for Paediatric Cancer (ERN PaedCan).
"The PARTNER project is expected to strengthen collaboration between countries and stimulate the formation of similar groups in other European countries"
The PARTNER project will create a Paediatric Rare Tumour European Registry dedicated to children and adolescents with VRTs, develop clinical guidelines and support LHEAR (Low Health Expenditure Average Rate) countries to be part of this network.
The three-year PARTNER project will link existing national registries and provide a VRT registry for those countries that currently do not have one.
VRTs are clearly orphan diseases, therefore a European registry will provide oversight and improve the care of VRT patients. Data collection will also help optimise and improve patient consultations and reduce inequalities in cancer outcomes across the EU.
A European registry will be a fundamental tool in sustaining a European network dedicated to VRTs and will allow experts to increase clinical research and share their knowledge. The Registry will be part of a platform that can also provide virtual consultations to clinicians allowing information to travel, rather than the patients.
By cooperating and exchanging life-saving knowledge at European level, patients across the EU should be able to gain access to the best expertise available.
The public health value of the PARTNER project is based on its capacity of connecting international experts, care providers, hospital and family/children with rare tumours and providing the necessary framework for collecting information on VRT, generate new guidance recommendations and delivering clinical advice with immediate benefit for the patient.
Innovation will be embedded in the new platform: original IT tools will link existing databases assuring interoperability and pseudonymisation as well as anonymisation of patients’ data with respect to the secondary use of data as planned by respecting relevant data protection rules. This may serve well as a model for data set integration at EU level, thus, paving the way for the integration of registries in the near future across the globe.
In conclusion, the PARTNER project is expected to strengthen collaboration between countries and stimulate the formation of similar groups in other European countries. In addition, collaboration with already existing disease-oriented registries and collaborative groups will be actively sought. This will improve research quality and treatment outcomes for children who have, until recently, been rather neglected.