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Clinical-genomic profiling of MDS to inform allo-HCT: recommendations from an international panel on behalf of the EBMT

2025-05-01Letter Open access
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dc.contributor.authorGurnari, Carmelo
dc.contributor.authorRobin, Marie
dc.contributor.authorAdès, Lionel
dc.contributor.authorAljurf, Mahmoud
dc.contributor.authorAlmeida, António M.
dc.contributor.authorDuarte, Fernando Barroso
dc.contributor.authorBernard, Elsa
dc.contributor.authorCutler, Corey
dc.contributor.authorPorta, Matteo Giovanni Della
dc.contributor.authorWitte, Theo De
dc.contributor.authorDeZern, Amy
dc.contributor.authorDrozd-Sokolowska, Joanna
dc.contributor.authorDuncavage, Eric
dc.contributor.authorFenaux, Pierre
dc.contributor.authorGagelmann, Nico
dc.contributor.authorGarcia-Manero, Guillermo
dc.contributor.authorHaferlach, Claudia
dc.contributor.authorHaferlach, Torsten
dc.contributor.authorHasserjian, Robert
dc.contributor.authorHellström-Lindberg, Eva
dc.contributor.authorJacoby, Meagan
dc.contributor.authorKulasekararaj, Austin
dc.contributor.authorLindsley, R. Coleman
dc.contributor.authorMaciejewski, Jaroslaw P.
dc.contributor.authorMakishima, Hideki
dc.contributor.authorMalcovati, Luca
dc.contributor.authorMittelman, Moshe
dc.contributor.authorMyhre, Anders E.
dc.contributor.authorOgawa, Seishi
dc.contributor.authorOnida, Francesco
dc.contributor.authorPapaemmanuil, Elli
dc.contributor.authorPassweg, Jakob
dc.contributor.authorPlatzbecker, Uwe
dc.contributor.authorPleyer, Lisa
dc.contributor.authorRaj, Kavita
dc.contributor.authorSantini, Valeria
dc.contributor.authorSureda, Anna
dc.contributor.authorTobiasson, Magnus
dc.contributor.authorVoso, Maria Teresa
dc.contributor.authorYakoub-Agha, Ibrahim
dc.contributor.authorZeidan, Amer
dc.contributor.authorWalter, Matthew
dc.contributor.authorKröger, Nicolaus
dc.contributor.authorMcLornan, Donal P.
dc.contributor.authorCazzola, Mario
dc.date.accessioned2025-08-05T11:50:52Z
dc.date.available2025-08-05T11:50:52Z
dc.date.issued2025-05-01
dc.description.abstractFor patients with myelodysplastic neoplasm/syndrome (MDS), allogeneic hematopoietic cell transplantation (allo-HCT) represents the only potentially curative treatment, capable of eradicating disease-related mutant hematopoietic cells and establishing normal donor hematopoiesis. Biologic-assignment clinical trials have indicated that in eligible patients, allo-HCT is associated with superior clinical outcomes compared with nontransplant therapy. However, this therapeutic option is only available to a subset of patients, and the outcome is influenced by multiple factors inherent to the patient, the MDS subtype, and the allo-HCT procedure itself. In 2017, the European Society for Blood and Marrow Transplantation (EBMT) published recommendations for allo-HCT in MDS to guide practical decision-making. In the contemporary era, genomic profiling has become routine clinical practice in many centers, and the most recent classification systems include MDS entities that are defined by genetic abnormalities. In particular, the molecular International Prognostic Scoring System offers more precise prognostication across all clinical end points and currently represents the standard tool for estimating patient survival in the absence of disease-modifying treatment. Evidence from multiple sources increasingly indicates that allo-HCT should be considered at the time of diagnosis in all eligible patients with MDS. Therefore, genomic profiling for somatic mutations and testing for germ line predisposition variants are integral to determining a patient's eligibility for transplantation. Although all patients with higher-risk MDS are potential candidates for immediate transplantation, a subset of those with lower-risk MDS may also derive benefit from this procedure at an earlier disease stage. Comprehensive recommendations on behalf of an expert international panel for clinical practice and future clinical studies of relevance are presented.eng
dc.identifier.doi10.1182/blood.2024025131
dc.identifier.eid105000927075
dc.identifier.issn0006-4971
dc.identifier.other961ddacb-d8dd-4f74-b70c-b1b4f27abd24
dc.identifier.pmid39970324
dc.identifier.urihttp://hdl.handle.net/10400.14/54160
dc.identifier.wos001485476200001
dc.language.isoeng
dc.peerreviewedno
dc.rights.uriN/A
dc.subjectAllogeneic hematopoietic cell transplantation
dc.subjectMyelodysplastic syndrome/neoplasm
dc.subjectPatient outcomes
dc.titleClinical-genomic profiling of MDS to inform allo-HCT: recommendations from an international panel on behalf of the EBMTeng
dc.typeletter
dspace.entity.typePublication
oaire.citation.issue18
oaire.citation.startPage1987
oaire.citation.titleBlood
oaire.citation.volume145
oaire.versionhttp://purl.org/coar/version/c_970fb48d4fbd8a85

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