A sex-informed approach to improve the personalised decision making process in myelodysplastic syndromes: a multicentre, observational cohort study

作者：G. Maggioni, M. Bersanelli, E. Travaglino, Ana Alfonso Piérola, A. Kasprzak, Arnan Sangerman Montserrat, Elisabetta Sauta, C. Sala, T. Matteuzzi, M. Meggendorfer, Matteo Gnocchi, Lin-Pierre Zhao, C. Tentori, K. Nachtkamp, Daniele Dall’Olio, E. Mosca, M. Ubezio, A. Campagna, A. Russo, G. Rivoli, M. Bernardi, L. Borin, M. Voso, M. Riva, E. Oliva, M. Zampini, E. Riva, Elena Saba, S. D'Amico, L. Lanino, Benedetta Tinterri, Francesca Re, Marilena Bicchieri, L. Giordano, G. Angelotti, Pierandrea Morandini, A. Kubasch, F. Passamonti, A. Rambaldi, V. Savevski, A. Santoro, A. A. van de Loosdrecht, A. Brogi, V. Santini, S. Kordasti, G. Sanz, F. Solé, N. Gattermann, W. Kern, U. Platzbecker, L. Adès, P. Fenaux, T. Haferlach, G. Castellani, U. Germing, M. Díez-Campelo, M. D. della Porta · 发表于：The Lancet Haematology · 年份：2022 · DOI：10.1016/s2352-3026(22)00323-4 · 被引用次数：22 · 研究领域：Medicine

Summary Background Sex is a major source of diversity among patients and a sex-informed approach is becoming a new paradigm in precision medicine. We aimed to describe sex diversity in myelodysplastic syndromes in terms of disease genotype, phenotype, and clinical outcome. Moreover, we sought to incorporate sex information into the clinical decision-making process as a fundamental component of patient individuality. Methods In this multicentre, observational cohort study, we retrospectively analysed 13 284 patients aged 18 years or older with a diagnosis of myelodysplastic syndrome according to 2016 WHO criteria included in the EuroMDS network (n=2025), International Working Group for Prognosis in MDS (IWG-PM; n=2387), the Spanish Group of Myelodysplastic Syndromes registry (GESMD; n=7687), or the Düsseldorf MDS registry (n=1185). Recruitment periods for these cohorts were between 1990 and 2016. The correlation between sex and genomic features was analysed in the EuroMDS cohort and validated in the IWG-PM cohort. The effect of sex on clinical outcome, with overall survival as the main endpoint, was analysed in the EuroMDS population and validated in the other three cohorts. Finally, novel prognostic models incorporating sex and genomic information were built and validated, and compared to the widely used revised International Prognostic Scoring System (IPSS-R). This study is registered with ClinicalTrials.gov, NCT04889729. Findings The study included 7792 (58·7%) men and 5492...