Clinical and Genomic-Based Decision Support System to Define the Optimal Timing of Allogeneic Hematopoietic Stem-Cell Transplantation in Patients With Myelodysplastic Syndromes

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 b...

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Autores: Tentori, C.A., Gregorio, C.|||0000-0002-8163-1634, Robin, M., Gagelmann, N.|||0000-0002-0891-1744, Gurnari, C.|||0000-0001-6829-5544, Ball, S., Berrocal, J.C.C.|||0000-0001-5269-521X, Lanino, Luca|||0000-0003-2404-8829, D'Amico, Saverio|||0000-0003-1877-8497, Spreafico, M.|||0000-0002-7773-9976, Maggioni, G., Travaglino, E., Sauta, Elisabetta|||0000-0002-7830-988X, Meggendorfer, M., Zhao, Lin-Pierre|||0000-0001-5035-4470, Campagna, A., Savevski, V., Santoro, Armando, Al Ali, N., Sallman, D.|||0000-0003-0504-8233, Sole, F.|||0000-0002-3251-2161, Garcia-Manero, Guillermo|||0000-0002-3631-2482, Germing, U., Kroger, N.|||0000-0002-2961-4183, Kordasti, Shahram|||0000-0002-0347-4207, Santini, Valeria|||0000-0002-5439-2172, Sanz, Guillermo|||0000-0002-2767-8191, Kern, Wolfgang|||0000-0002-6452-2874, Platzbecker, Uwe|||0000-0003-1863-3239, Diez-Campelo, María|||0000-0002-1467-6779, Maciejewski, Jaroslaw|||0000-0002-6837-4346, Arenillas, Leonor|||0000-0002-9020-8766, Fenaux, Pierre, Haferlach, T.|||0000-0003-0196-2837, Zeidan, A.M.|||0000-0001-7017-8160, Castellani, Gastone|||0000-0003-4892-925X, Komrokji, Rami|||0000-0002-1876-5269, Ieva, F., Della Porta, Matteo Giovanni|||0000-0002-6915-5970, Bernardi, Mauro|||0000-0002-1308-5440, Di Grazia, C., Vago, Luca|||0000-0003-4247-3175, Rivoli, G., Borin, L., Chiusolo, P., Giaccone, L., Voso, Maria Teresa|||0000-0002-6164-4761, Bewersdorf, J.P., Nibourel, O., Beyá, M.D., Jerez, Andrés|||0000-0002-2079-6638, Hernández, F., Kennedy, K.V., Xicoy, Blanca|||0000-0002-0295-1307, Ubezio, M., Russo, A., Todisco, G., Mannina, D., Bramanti, S., Zampini, M., Riva, E., Bicchieri, M., Asti, G., Viviani, F., Buizza, A., Tinterri, B., Kubasch, A.S., Bacigalupo, A., Raiola, A.M., Rambaldi, Alessandro|||0000-0002-3739-7502, Passamonti, Francesco|||0000-0001-8068-5289, Ciceri, Fabio|||0000-0003-0873-0123
Tipo de recurso: artículo
Fecha de publicación:2024
País:España
Institución:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:309344
Acceso en línea:https://ddd.uab.cat/record/309344
https://dx.doi.org/urn:doi:10.1200/JCO.23.02175
Access Level:acceso abierto
Palabra clave: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
Descripción
Sumario: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.