TY - JOUR
T1 - Clinical-genomic profiling of MDS to inform allo-HCT
T2 - recommendations from an international panel on behalf of the EBMT
AU - Gurnari, Carmelo
AU - Robin, Marie
AU - Ades, Lionel
AU - Aljurf, Mahmoud
AU - Almeida, Antonio
AU - Duarte, Fernando Barroso
AU - Bernard, Elsa
AU - Cutler, Corey
AU - Della Porta, Matteo Giovanni
AU - De Witte, Theo
AU - Dezern, Amy
AU - Drozd-Sokolowska, Joanna
AU - Duncavage, Eric
AU - Fenaux, Pierre
AU - Gagelmann, Nico
AU - Garcia-Manero, Guillermo
AU - Haferlach, Claudia
AU - Haferlach, Torsten
AU - Hasserjian, Robert
AU - Hellstroem-Lindberg, Eva
AU - Jacoby, Meagan
AU - Kulasekararaj, Austin
AU - Lindsley, R. Coleman
AU - Maciejewski, Jaroslaw P.
AU - Makishima, Hideki
AU - Malcovati, Luca
AU - Mittelman, Moshe
AU - Myhre, Anders E.
AU - Ogawa, Seishi
AU - Onida, Francesco
AU - Papaemmanuil, Elli
AU - Passweg, Jakob
AU - Platzbecker, Uwe
AU - Pleyer, Lisa
AU - Raj, Kavita
AU - Santini, Valeria
AU - Sureda, Anna
AU - Tobiasson, Magnus
AU - Voso, Maria Teresa
AU - Yakoub-Agha, Ibrahim
AU - Zeidan, Amer
AU - Walter, Matthew
AU - Kroeger, Nicolaus
AU - Mclornan, Donal P.
AU - Cazzola, Mario
N1 - Pleyer: Third Medical Department with Hematology, Medical Oncology, Hemostaseology, Rheumatology and Infectiology, Oncologic Center, Paracelsus Medical University, Salzburg, Austria
PY - 2025/5/1
Y1 - 2025/5/1
N2 - For 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 influ-enced 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.
AB - For 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 influ-enced 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.
KW - Stem-cell transplantation
KW - Acute myeloid-leukemia
KW - Bone-marrow-transplantation
KW - Prognostic scoring system
KW - Myelodysplastic syndrome
KW - Allogeneic transplantation
KW - Biologic-assignment
KW - Donor availability
KW - Comorbidity index
KW - Decision-analysis
UR - https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=pmu_pure&SrcAuth=WosAPI&KeyUT=WOS:001485476200001&DestLinkType=FullRecord&DestApp=WOS_CPL
U2 - 10.1182/blood.2024025131
DO - 10.1182/blood.2024025131
M3 - Original Article
C2 - 39970324
SN - 0006-4971
VL - 145
SP - 1987
EP - 2001
JO - BLOOD
JF - BLOOD
IS - 18
ER -