The challenge of very rare childhood cancers in developed and developing countries

ABSTRACT

Introduction: The improvements made in the field of pediatric oncology in the last decades due to the propensity to develop national and international cooperative protocols have not been historically seen for a number of very rare pediatric neoplasms whose common denominator lies in their having been treated as orphan diseases. For several years now, this situation has fortunately been gradually changing, and various projects dedicated to these rare diseases have been developed in several countries.

Areas covered: This paper describes the schemes dedicated to rare pediatric tumors in countries with different development levels, with a particular reference to the EXPeRT (European Cooperative Study Group for Pediatric Rare Tumors) project.

Expert opinion: Experience gained in recent years on rare tumors in childhood underscores the importance of cooperation and networking. Further efforts are now needed to extend research and improve the quality of patients care. The pediatric study groups that have invested their resources in this area must now strive to obtain institutional recognition and to seek new partnerships with adult medical oncology centers, organizations concerned with biological-genetic studies, and partners such as pharmaceutical industries, regulatory authorities, and international funding commissions.

KEYWORDS:

• Rare tumors
• childhood cancers
• cooperative studies
• tumor board
• EXPeRT
• developing countries

Article highlights

• Among tumors of children and adolescents, there is a list very rare pediatric neoplasms whose common denominator lies in their having been treated as orphan diseases and consequently in their having failed to benefit from the advances made over the years by pediatric oncologists sharing their expertise and networkingIn the last decade, various projects specifically dedicated to very rare pediatric tumors have been developed in several countries. In Europe, different national initiatives have merged in the EXPeRT (European Cooperative Study Group for Pediatric Rare Tumors) projectThe involvement in the ExPO-r-Net project (European Expert Paediatric Oncology Reference Network for Diagnostics and Treatment) have given to the EXPeRT the necessary support to implement its activities, and in particular to develop a specific website to inform the non-scientific community and to set up a virtual tumor board and advisory desk for professionalsMulti-level international cooperation is warranted to deal with the disadvantage historically borne by children with rare tumors due to the very rarity of their disease and the shortage of clinical and biological informationFurther efforts are needed to obtain institutional recognition, to increase the resources available (in particular for tumor sample collection and biology studies), to seek new partnerships, e.g. with adult medical oncology organizations, pharmaceutical industries, regulatory authorities, and international funding commissions

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Acknowledgment

The content represents the views of the author and is his sole responsibility and it can in no way be taken to reflect the views of European Union bodies. The European Commission and/or Chafea do not accept responsibility for any use that may be made of the information it contains.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Additional information

Funding

Part of this publication arises from the project ExPO-r-Net which has received funding from the European Union in the framework of the Health Programme (2008-2013), grant agreement nr. 2013 12 07.