Francesca Palandri MD, PhD, Filippo Branzanti MSc, Massimiliano Bonifacio MD, PhD, Elena M. Elli MD, Erika Morsia MD, Mirko Farina MD, Mario Tiribelli MD, PhD, Giulia Benevolo MD, Eloise Beggiato MD, Bruno Martino MD, Giovanni Caocci MD, Novella Pugliese MD, Alessia Tieghi MD, Monica Crugnola MD, Gianni Binotto MD, Francesco Cavazzini MD, Elisabetta Abruzzese MD, Alessandro Isidori MD, Alessandra Dedola MD, Emilia Scalzulli MD, Andrea Duminuco MD, PhD, Luca Tosoni MD, Alda Strazimiri MD, Roberto M. Lemoli MD, PhD, Daniela Cilloni MD, PhD, Monica Bocchia MD, Fabrizio Pane MD, PhD, Chiara Sartor MD, PhD, Florian H. Heidel MD, PhD, Massimo Breccia MD, PhD, Giuseppe A. Palumbo MD, PhD, Andrew T. Kuykendall MD
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引用次数: 0
Abstract
Background
The response to ruxolitinib after 6 months (RR6) model allows early identification of ruxolitinib-treated myelofibrosis (MF) patients with poorer overall survival (OS); however, it is less applicable to lower-risk patients.
Methods
To further explore this, the authors performed a subanalysis of the “RUX-MF” study (NCT06516406) with an aim to validate the RR6 and to develop a score specific for intermediate-1 DIPSS/MYSEC-PM risk patients.
Results
Among the 776 evaluable patients, 34.4%, 47.8%, and 17.8% were at low, intermediate, and high RR6 risk, with 5-year OS of 64.1%, 51.8%, and 44.5%, respectively (p < .001). In the 428 intermediate-1 patients, the RR6 model did not discriminate between intermediate and low-risk patients (5-year OS: 74.4% vs. 72.0%, p = .24). The intermediate-1 specific RR6 (iRR6) model was therefore developed by incorporating new variables: underdosed ruxolitinib with respect to platelet count at one or more time points (hazard ratio [HR], 3.91; p < .001), absence of palpable spleen reduction by ≥50% at 6 months (HR, 1.45; p = .02), and red blood cell transfusion requirement at all time points (HR, 1.85; p = .01). The iRR6 model stratified patients into three risk categories: low (score 0, 20.3%), intermediate (score 1–2, 45.8%), and high-risk (score >2, 33.9%), with 5-year OS of 84.8%, 76.4%, and 56.6%, respectively (p < .0001). The iRR6 model was validated in a cohort of 95 intermediate-1 risk patients from the Moffitt Cancer Center, yielding stratification into the same three risk categories, with 5-year OS of 83.3% (low-risk), 71.7% (intermediate-risk), and 54.5% (high-risk) (p = .01).
Conclusions
The iRR6 model provides a more refined tool for the identification of intermediate-1 MF patients who may benefit from early therapy shift.
期刊介绍:
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