Clinical and Genomic-Based Decision Support System to Define the Optimal Timing of Allogeneic Hematopoietic Stem-Cell Transplantation in Patients With Myelodysplastic Syndromes
Tentori, C.A. (Department of Biomedical Sciences. Humanitas University)
Gregorio, C. (Department of Neurobiology. Care Sciences and Society. Aging Research Center. Karolinska Institutet)
Robin, M. (Department of Hematology and Bone Marrow Transplantation. Hôpital Saint-Louis/Assistance Publique-Hôpitaux de Paris (AP-HP). University Paris 7)
Gagelmann, N. (University Medical Center Hamburg-Eppendorf)
Gurnari, C. (Policlinico Tor Vergata. Department of Biomedicine and Prevention. Tor Vergata University)
Ball, S. (Vanderbilt University School of Medicine. Vanderbilt-Ingram Cancer Center)
Berrocal, J.C.C. (Hospital Universitario de Salamanca)
Lanino, Luca (Department of Biomedical Sciences. Humanitas University)
D'Amico, Saverio (Humanitas Clinical and Research Center-IRCCS)
Spreafico, M. (Mathematical Institute. Leiden University)
Maggioni, G. (Department of Biomedical Sciences. Humanitas University)
Travaglino, E. (Fondazione IRCCS Policlinico San Matteo (Itàlia))
Sauta, Elisabetta (Humanitas Clinical and Research Center-IRCCS)
Meggendorfer, M. (MLL Munich Leukemia Laboratory)
Zhao, Lin-Pierre (Department of Hematology and Bone Marrow Transplantation. Hôpital Saint-Louis/Assistance Publique-Hôpitaux de Paris (AP-HP). University Paris 7)
Campagna, A. (Department of Biomedical Sciences. Humanitas University)
Savevski, V. (Humanitas Clinical and Research Center-IRCCS)
Santoro, Armando (Department of Biomedical Sciences. Humanitas University)
Al Ali, N. (Moffitt Cancer Center (Tampa, Estats Units d'Amèrica))
Sallman, D. (Moffitt Cancer Center (Tampa, Estats Units d'Amèrica))
Sole, F (Institut Germans Trias i Pujol. Institut de Recerca contra la Leucèmia Josep Carreras)
Garcia-Manero, Guillermo (The University of Texas MD Anderson Cancer Center)
Germing, U. (Department of Hematology. Oncology. and Clinical Immunology. Heinrich-Heine-University. University Clinic)
Kroger, N. (University Medical Center Hamburg-Eppendorf)
Kordasti, Shahram (Hematology Department & Stem Cell Transplant Unit. DISCLIMO Università Politecnica delle Marche)
Santini, Valeria (MDS Unit. Azienda Ospedaliero-Universitaria Careggi. University of Florence)
Sanz, Guillermo (Hospital Universitari i Politècnic La Fe (València))
Kern, Wolfgang (MLL Munich Leukemia Laboratory)
Platzbecker, Uwe (Medical Clinic and Policlinic 1. Hematology and Cellular Therapy. University Hospital Leipzig)
Diez-Campelo, María (Hospital Universitario de Salamanca)
Maciejewski, Jaroslaw (Department of Translational Hematology and Oncology Research. Taussig Cancer Institute. Cleveland Clinic Foundation)
Arenillas, Leonor (Department of Hematology and Bone Marrow Transplantation. Hôpital Saint-Louis/Assistance Publique-Hôpitaux de Paris (AP-HP). University Paris 7)
Fenaux, P. (Department of Hematology and Bone Marrow Transplantation. Hôpital Saint-Louis/Assistance Publique-Hôpitaux de Paris (AP-HP). University Paris 7)
Haferlach, T. (MLL Munich Leukemia Laboratory)
Zeidan, A.M. (Section of Hematology. Department of Internal Medicine. Yale University School of Medicine. Yale Cancer Center)
Castellani, Gastone (Experimental. Diagnostic and Specialty Medicine-DIMES)
Komrokji, Rami (Moffitt Cancer Center (Tampa, Estats Units d'Amèrica))
Ieva, F. (HDS. Health Data Science Center. Human Technopole)
Della Porta, Matteo Giovanni (Department of Biomedical Sciences. Humanitas University)
Bernardi, M. (IRCCS San Raffaele Scientific Institute. University Vita-Salute San Raffaele)
Di Grazia, C. (IRCCS Ospedale Policlinico San Martino (Gènova, Itàlia))
Vago, L. (IRCCS San Raffaele Scientific Institute. University Vita-Salute San Raffaele)
Rivoli, G. (IRCCS Ospedale Policlinico San Martino (Gènova, Itàlia))
Borin, L. (IRCCS Ospedale San Gerardo)
Chiusolo, P. (IRCCS Fondazione Policlinico Universitario Gemelli. Università Cattolica del Sacro Cuore)
Giaccone, L. (Department of Molecular Biotechnology and Health Sciences. University of Turin)
Voso, M.T. (Memorial Sloan Kettering Cancer Center)
Bewersdorf, J.P. (Memorial Sloan Kettering Cancer Center)
Nibourel, O. (Laboratory of Hematology. CHU Lille)
Beyá, M.D. (Hospital Clínic i Provincial de Barcelona)
Jerez, Andrés (Universidad de Murcia)
Hernández, F. (Hospital Universitario Virgen de las Nieves (Granada))
Kennedy, K.V. (Hospital Universitario Ramón y Cajal (Madrid))
Xicoy, Blanca (Universitat Autònoma de Barcelona)
Ubezio, M. (Humanitas Clinical and Research Center-IRCCS)
Russo, A. (Humanitas Clinical and Research Center-IRCCS)
Todisco, G. (Department of Biomedical Sciences. Humanitas University)
Mannina, D. (Humanitas Clinical and Research Center-IRCCS)
Bramanti, S. (Humanitas Clinical and Research Center-IRCCS)
Zampini, M. (Humanitas Clinical and Research Center-IRCCS)
Riva, E. (Humanitas Clinical and Research Center-IRCCS)
Bicchieri, M. (Humanitas Clinical and Research Center-IRCCS)
Asti, G. (Humanitas Clinical and Research Center-IRCCS)
Viviani, F. (Humanitas Clinical and Research Center-IRCCS)
Buizza, A. (Humanitas Clinical and Research Center-IRCCS)
Tinterri, B. (Department of Biomedical Sciences. Humanitas University)
Kubasch, A.S. (Medical Clinic and Policlinic 1. Hematology and Cellular Therapy. University Hospital Leipzig)
Bacigalupo, A. (IRCCS Fondazione Policlinico Universitario Gemelli. Università Cattolica del Sacro Cuore)
Raiola, A.M. (IRCCS Ospedale Policlinico San Martino (Gènova, Itàlia))
Rambaldi, A. (Department of Oncology and Hemato-Oncology. University of Milan)
Passamonti, F. (Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (Milà, Itàlia))
Ciceri, F. (IRCCS San Raffaele Scientific Institute. University Vita-Salute San Raffaele)

Data:	2024
Resum:	PURPOSE Allogeneic hematopoietic stem-cell transplantation (HSCT) is the only potentially curative treatment for patients with myelodysplastic syndromes (MDS). Several issues must be considered when evaluating the benefits and risks of HSCT for patients with MDS, with the timing of transplantation being a crucial question. Here, we aimed to develop and validate a decision support system to define the optimal timing of HSCT for patients with MDS on the basis of clinical and genomic information as provided by the Molecular International Prognostic Scoring System (IPSS-M). PATIENTS AND METHODS We studied a retrospective population of 7,118 patients, stratified into training and validation cohorts. A decision strategy was built to estimate the average survival over an 8-year time horizon (restricted mean survival time [RMST]) for each combination of clinical and genomic covariates and to determine the optimal transplantation policy by comparing different strategies. RESULTS Under an IPSS-M based policy, patients with either low and moderate-low risk benefited from a delayed transplantation policy, whereas in those belonging to moderately high-, high- and very high-risk categories, immediate transplantation was associated with a prolonged life expectancy (RMST). Modeling decision analysis on IPSS-M versus conventional Revised IPSS (IPSS-R) changed the transplantation policy in a significant proportion of patients (15% of patient candidate to be immediately transplanted under an IPSS-R-based policy would benefit from a delayed strategy by IPSS-M, whereas 19% of candidates to delayed transplantation by IPSS-R would benefit from immediate HSCT by IPSS-M), resulting in a significant gain-in-life expectancy under an IPSS-M-based policy (P 5. 001). CONCLUSION These results provide evidence for the clinical relevance of including genomic features into the transplantation decision making process, allowing personalizing the hazards and effectiveness of HSCT in patients with MDS.
Drets:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, i la comunicació pública de l'obra, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original. No es permet la creació d'obres derivades.
Llengua:	Anglès
Document:	Article ; recerca ; Versió publicada
Matèria:	Adult ; Aged ; Decision Support Systems, Clinical ; Decision Support Techniques ; Female ; Genomics ; Hematopoietic Stem Cell Transplantation ; Humans ; Male ; Middle Aged ; Myelodysplastic Syndromes ; Retrospective Studies ; Risk Assessment ; Time Factors ; Transplantation, Homologous ; Young Adult
Publicat a:	Journal of clinical oncology, Vol. 42 Núm. 24 (20 2024) , p. 2873-2886, ISSN 1527-7755

DOI: 10.1200/JCO.23.02175
PMID: 38723212

17 p, 1.8 MB

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