This proposal is to support the delivery of ERN-RARE-LIVER, the European Reference Network (ERN) in rare hepatological diseases. Liver disease is a major and growing problem in the European population. The expansion in clinical need because of increasing disease impact has not been matched by expansion in the clinical workforce leading to a relative lack of expertise. The challenge is particularly acute in rare liver disease; a setting in which Europe leads the world in terms of academic innovation. The ERN model is particularly attractive in the setting of rare liver disease as it will foster the development of ‘virtual critical mass’ across European centres with benefits in terms of clinical care deliver, teaching and training and research capacity. Supported by the relevant professional societies (The European Association for the Study of the Liver EASL) and the European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN), the community of our field. Covering all ranges, from paediatric to adult through transitional care, ERN-RARE-LIVER has 3 themes reflecting important disease groupings (Autoimmune Liver Disease; Metabolic, Biliary Atresia and Related Disease; Structural Liver Disease) with a coherent plant to increase both our disease scope and geographical coverage. We will improve patient care directly through our work on guideline optimisation, care pathway development and the implementation of a model of “TELE-BOARDS” with multi-centre discussion of complex and challenging cases. We will also facilitate improvement in diagnostics capacity through development of quality assurance programmes in serology and histopathology, and case referral pathways to support centres lacking diagnostic technology. Our training and research programmes will enhance care delivery through increase in the trained workforce and knowledge respectively.
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This proposal is to support the delivery of ERN-RARE-LIVER, the European Reference Network (ERN) in rare hepatological
diseases. Liver disease is a major and growing problem in the European population. The expansion in clinical need
because of increasing disease impact has not been matched by expansion in the clinical workforce leading to a relative lack
of expertise. The challenge is particularly acute in rare liver disease; a setting in which Europe leads the world in terms of
academic innovation. The ERN model is particularly attractive in the setting of rare liver disease as it will foster the
development of ‘virtual critical mass’ across European centres with benefits in terms of clinical care deliver, teaching and
training and research capacity. We are also supported by the relevant professional societies, the European Association for the Study of
the Liver (EASL) and the European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN), who are the
communities of our field. Covering all ranges, from paediatric to adult through transitional care, ERN-RARE-LIVER has 3
themes reflecting important disease groupings (Autoimmune Liver Disease; Metabolic, Biliary Atresia and Related Disease;
Structural Liver Disease) with a coherent plant to increase both our disease scope and geographical coverage. We will
improve patient care directly through our work on guideline optimisation, care pathway development and the implementation
of a model of “TELE-BOARDS” with multi-centre discussion of complex and challenging cases. We will also facilitate
improvement in diagnostics capacity through development of quality assurance programmes in serology and histopathology,
and case referral pathways to support centres lacking diagnostic technology. Our training and research programmes will
enhance care delivery through increase in the trained workforce and knowledge respectively.
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This proposal supports the European Reference Network (ERN) in rare hepatological disease. Liver disease is a major and growing problem in the European population. The expansion in clinical need because of increasing disease impact has not been matched by expansion in the clinical workforce leading to a relative lack of expertise. The ERN model is attractive for rare liver disease as it fosters the development of "virtual critical mass" across European centers with benefits in terms of clinical care delivery, teaching, training and research capacity. The ERN covers both adult and paediatric patents in the disease groupings (Autoimmune Liver Disease; Metabolic Biliary Atresia and related diseases; Structural Liver Disease) with a coherent plan to increase both disease scope and geographical coverage. Patient care will be improved through work on guidelines, care pathway development and the use of the Clinical Patient Management System for multi-centre discussion of complex cases. We also facilitate improvement in diagnostics capacity through development of quality assurance programmes in serology and hisopathology and case referral pathways to support centres lacking diagnostic technology. Our training and research programmes will enhance care delivery through increase in the trained workforce and knowledge respectively.
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This proposal is to support the creation of a European Reference Network (ERN) in rare hepatological diseases. Liver disease is a major and growing problem in the European population. The expansion in clinical need because of increasing disease impact has not been matched by expansion in the clinical workforce leading to a relative lack of expertise. The challenge is particularly accute in rare liver diseaese; a setting in which Europe leads the world in terms of academic innovation. The ERN model is particularly attractive in the setting of rare liver disease as it will foster the development of "virtual critical mass" across European centres with benefits in terms of clinical care deliver, teaching and training and research capacity. Supported by the relevant professional societies (The European Association for the Study of the Liver (EASL) and the European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN), the community of clinicians and patient groups interested in rare liver dsiease have come to together to develop a network which is unique in our field. Covering all ranges, from paediatric to adult through transitional care, RARE-LIVER has 3 themes reflecting important disease groupings (Autoimmune Liver Diseease; Metabolic, Biliary Atresia and Related Disease; Structural Liver Disease) with a coherent plan to increase both our disease scope and geographical coverage. We will improve patient care directly through our work on guideline optimisation, care pathway development and the implementation of a model of "TELE-BOARDS" with multi-centre discussion of complex and challenging cases. We will also facilitate improvement in diagnostics capcity through development of quality assurance programmes in serology and histopathology, and case referral pathways to support centres lacking diagnostic techology. Our training and research programmes will enhance care delivery through increase in the trained workforce and knowledge respectively.
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ERNs should link existing highly specialised healthcare providers, as expertise in rare or lowprevalence
complex disease or conditions is scarce and dispersed. ERNs will facilitate timely access
to care by centralising knowledge and experience, research and training for these diseases and
conditions. We propose TRANSplantCHILD based on the understanding of transplantation in children as
a low-prevalence complex medical condition that requires highly specialised expertise and
resources.
Paediatric transplantation (PT), both solid organ (SOT) and hematopoietic stem cell transplantation
(HSCT), is the only curative procedure for several rare diseases causing the highest mortality rate in
European children. Paediatric surgical procedures allow transplantation of virtually all organs, but a
clear gap yet exists, as proper post-transplant care constitutes a challenge that demands the
multidisciplinary coordinated efforts of organ-focused specialists, especially when more than one
organ is transplanted, with other specialised clinical and laboratory resources that support the
transplantation procedure in common processes for SOT and HSCT as immunosuppression,
immune reconstitution, rejection, tolerance, risk of infection, second malignancies, etc.
In addition, other areas of non-medical supportive care need to be in place for children and their
families, due to severe psychological and socio-economic issues that extend to adulthood. Costeffective
measures will clearly be beneficial in PT when compared to those obtained in adults as life
expectancy after transplantation is much longer.
Transplanted individuals shift their primary disease to a chronic condition of immunosuppresion to
avoid rejection, requiring proper monitoring and care of post-transplant complications. This state is
even more life-conditioning for children, demanding even more expertise in reference centres
connecting multidisciplinary medical expertise, transfer of knowledge and innovative medicine as
children
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