TY - JOUR

T1 - Sickle cell disease landscape and challenges in the EU

T2 - the ERN-EuroBloodNet perspective

AU - Mañú Pereira, María del Mar

AU - Colombatti, Raffaella

AU - Alvarez, Federico

AU - Bartolucci, Pablo

AU - Bento, Celeste

AU - Brunetta, Angelo Loris

AU - Cela, Elena

AU - Christou, Soteroula

AU - Collado, Anna

AU - de Montalembert, Mariane

AU - Dedeken, Laurence

AU - Fenaux, Pierre

AU - Galacteros, Frédéric

AU - Glenthøj, Andreas

AU - Gutiérrez Valle, Victoria

AU - Kattamis, Antonis

AU - Kunz, Joachim

AU - Lobitz, Stephan

AU - McMahon, Corrina

AU - Pellegrini, Mariangela

AU - Reidel, Sara

AU - Russo, Giovanna

AU - Santos Freire, Miriam

AU - van Beers, Eduard

AU - Kountouris, Petros

AU - Gulbis, Béatrice

N1 - Publisher Copyright: © 2023 Elsevier Ltd

PY - 2023

Y1 - 2023

N2 - 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.

AB - 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.

U2 - 10.1016/S2352-3026(23)00182-5

DO - 10.1016/S2352-3026(23)00182-5

M3 - Review

C2 - 37451300

AN - SCOPUS:85166442729

VL - 10

SP - e687-e694

JO - The Lancet Haematology

JF - The Lancet Haematology

SN - 2352-3026

IS - 8

ER -