Sickle cell disease landscape and challenges in the EU: the ERN-EuroBloodNet perspective

Publikation: Bidrag til tidsskrift › Review › Forskning › fagfællebedømt

  • María del Mar Mañú Pereira
  • Raffaella Colombatti
  • Federico Alvarez
  • Pablo Bartolucci
  • Celeste Bento
  • Angelo Loris Brunetta
  • Elena Cela
  • Soteroula Christou
  • Anna Collado
  • Mariane de Montalembert
  • Laurence Dedeken
  • Pierre Fenaux
  • Frédéric Galacteros
  • Victoria Gutiérrez Valle
  • Antonis Kattamis
  • Joachim Kunz
  • Stephan Lobitz
  • Corrina McMahon
  • Mariangela Pellegrini
  • Sara Reidel
  • Giovanna Russo
  • Miriam Santos Freire
  • Eduard van Beers
  • Petros Kountouris
  • Béatrice Gulbis
Sickle cell disease is a hereditary multiorgan disease that is considered rare in the EU. In 2017, the Rare Diseases Plan was implemented within the EU and 24 European Reference Networks (ERNs) were created, including the ERN on Rare Haematological Diseases (ERN-EuroBloodNet), dedicated to rare haematological diseases. This EU initiative has made it possible to accentuate existing collaborations and create new ones. The project also made it possible to list all the needs of people with rare haematological diseases not yet covered health-care providers in the EU to allow optimised care of individuals with rare pathologies, including sickle cell disease. This Viewpoint is the result of joint work within 12 EU member states (ie, Belgium, Cyprus, Denmark, France, Germany, Greece, Ireland, Italy, Portugal, Spain, Sweden, and The Netherlands), all members of the ERN-EuroBloodNet. We describe the role of the ERN-EuroBloodNet to improve the overall approach to and the management of individuals with sickle cell disease in the EU through specific on the pooling of expertise, knowledge, and best practices; the development of training and education programmes; the strategy for systematic gathering and standardisation of clinical data; and its reuse in clinical research. Epidemiology and research strategies from ongoing implementation of the Rare Anaemia Disorders European Epidemiological Platform is depicted.

OriginalsprogEngelsk
TidsskriftThe Lancet Haematology
Vol/bind10
Udgave nummer8
Sider (fra-til)e687-e694
Antal sider8
ISSN2352-3026
DOI
StatusUdgivet - 2023

Bibliografisk note

Funding Information:
We thank Anne Sophie Adam, Patrick Ducoroy, Bichr Allaf, Ana Marcao, and Marelle Bouva for gathering information about the status of national neonatal screening programmes on sickle cell disease ERN-EuroBloodNet actions are carried out thanks to the European Reference Network on Rare Haematological Diseases members and European patient advocacy groups. ERN-EuroBloodNet is partly funded by the EU within the framework of the Fourth EU Health Programme (ERN-EuroBloodNet GA101085717). ENROL is cofunded by the European Commission—Consumers, Health, Agriculture and Food Executive Agency under the call for proposals HPPJ-2019 on Rare Disease Registries for the European Reference Networks (ENROL GA947670). Editorial note: The Lancet Group takes a neutral position with respect to territorial claims in published maps.

Funding Information:
We thank Anne Sophie Adam, Patrick Ducoroy, Bichr Allaf, Ana Marcao, and Marelle Bouva for gathering information about the status of national neonatal screening programmes on sickle cell disease ERN-EuroBloodNet actions are carried out thanks to the European Reference Network on Rare Haematological Diseases members and European patient advocacy groups. ERN-EuroBloodNet is partly funded by the EU within the framework of the Fourth EU Health Programme (ERN-EuroBloodNet GA101085717). ENROL is cofunded by the European Commission—Consumers, Health, Agriculture and Food Executive Agency under the call for proposals HPPJ-2019 on Rare Disease Registries for the European Reference Networks (ENROL GA947670).

Publisher Copyright:
© 2023 Elsevier Ltd

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