Document is current
Any future updates will be listed below
-
Authors
Velasco Puyo, Pablo https://orcid.org/0000-0001-7331-9483
Christou, Soteroula
Campisi, Saveria
Rodríguez-Sánchez, Maria A.
Reidel, Sara
Perez-Hoyo, Santiago
Mota, Miriam
Savvidou, Irene
Rekleiti, Anna
Salvo, Alessandra
Voi, Vincenzo
Ferrero, Giovanni Battista
Mandrile, Giorgia
Gaglioti, Carmen Maria
Cela, Elena
Ponce-Salas, Beatriz
Bardón-Cancho, Eduardo J.
Flevari, Pagona
Voskaridou-Dimoula, Ersi
Nur, Erfan
Biemond, Bart J.
Delaporta, Polynexi
Beneitez-Pastor, David
Collado Gimbert, Anna
Spasiano, Anna
Besse-Hammer, Tatiana
Lafiatis, Ioannis G.
Dedeken, Laurence
Raso, Simona
Ruiz-Llobet, Anna
Bagnato, Sabrina
Labarque, Veerle
Glenthøj, Andreas
Ruffo, Giovan Battista
Guerzoni, Maria Elena
Hafraoui, Kaoutar
Pistoia, Laura
Rosso, Rosamaria
Tagliaferri, Laura
Gonzalez-Urdiales, Paula
Benghiat, Fleur Samantha
de Montalembert, Mariane
Teles, Maria Jose
Vanderfaeillie, Anna
Bertoni, Elisa
Cuzzubbo, Daniela
Ferreira, Teresa
Saunders, Christopher J.
Stiakaki, Eftichia
Van de Velde, Ann L.
Diamantidis, Michael D.
Kerkhoffs, Jean-Louis H.
Oliveira, Marisa I.
Quota, Alessandra
Russo, Roberta
Van Damme, An
Argüello Marina, María
Lorite Reggiori, Mikael
Rijneveld, Anita W.
Rodríguez Gallego, Alexis
Colombatti, Raffaella
Iolascon, Achille
Taher, Ali
Gulbis, Béatrice
Roy, Noémi B. A.
Mañú-Pereira, María del Mar
-
Funding
Funding for this research was provided by:
Fourth EU Health Programme
- License Information
-
More Information
Article History
Received: 4 January 2025
Accepted: 19 March 2025
First Online: 16 April 2025
Declarations
:
: The Research Ethics Committee of Vall d'Hebron Hospital has confirmed that this exceptional case justifies the waiver of informed consent (Ethics Committee approval by University Hospital Vall d'Hebron dated 7 th April 2020 (PR(AG)215/2020/VHI-ERN- 2020 - 00)), and it has subsequently been approved in the various countries and centers involved in the registry.
: Not applicable.
: Andreas Glenthøj is part of the Advisory board of Agios, Bluebird bio, Bristol Myers Squibb, Novartis, Novo Nordisk, Pharmacosmos and Vertex Pharmaceuticals and also provides Research support to Agios, Bristol Myers Squibb, Novo Nordisk, Saniona and Sanofi. Mariane de Montalembert is part of the Steering Committtes of Addmedica, Vertex and Novartis. Raffaella Colombatti is part of the Advisory Board of NovoNordisk, Novartis, AddMedica, Pfizer, Forma Therapeutics, Global Blood Therapeutics and Vertex. Ali Taher performs consultancy activities and received research funding from Novartis Pharmaceuticals, Bristol-Myers Squibb (Celgene), Vifor, Pharmacosmos and Agios. Béatrice Gulbis has a Contract with EuroBloodNet Association (RADeep): the payments are made to her institution. For the remaining authors, no relevant conflicts of interest were declared. Mar Mañu is an editor of the Orphanet Journal of Rare Diseases.
