Personalized Timing for Allogeneic Stem-Cell Transplantation in Hematologic Neoplasms: A Target Trial Emulation Approach Using Multistate Modeling and Microsimulation

作者：Caterina Gregorio, M. Spreafico, S. D'amico, Elisabetta Sauta, G. Asti, L. Lanino, C. Tentori, U. Platzbecker, T. Haferlach, M. Díez-Campelo, Pierre Fenaux, R. Komrokji, M. D. Della Porta, F. Ieva · 发表于：JCO Clinical Cancer Informatics · 年份：2024 · DOI：10.1200/CCI.23.00205 · 被引用次数：5 · 研究领域：Medicine

PURPOSE Decision about the optimal timing of a treatment procedure in patients with hematologic neoplasms is critical, especially for cellular therapies (most including allogeneic hematopoietic stem-cell transplantation [HSCT]). In the absence of evidence from randomized trials, real-world observational data become beneficial to study the effect of the treatment timing. In this study, a framework to estimate the expected outcome after an intervention in a time-to-event scenario is developed, with the aim of optimizing the timing in a personalized manner. METHODS Retrospective real-world data are leveraged to emulate a target trial for treatment timing using multistate modeling and microsimulation. This case study focuses on myelodysplastic syndromes, serving as a prototype for rare cancers characterized by a heterogeneous clinical course and complex genomic background. A cohort of 7,118 patients treated according to conventional available treatments/evidence across Europe and United States is analyzed. The primary clinical objective is to determine the ideal timing for HSCT, the only curative option for these patients. RESULTS This analysis enabled us to identify the most appropriate time frames for HSCT on the basis of each patient's unique profile, defined by a combination relevant patients' characteristics. CONCLUSION The developed methodology offers a structured framework to address a relevant clinical issue in the field of hematology. It makes several valuable contributi...