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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Kuykendall MD","doi":"10.1002/cncr.70062","DOIUrl":null,"url":null,"abstract":"<div>\n \n \n <section>\n \n <h3> Background</h3>\n \n <p>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.</p>\n </section>\n \n <section>\n \n <h3> Methods</h3>\n \n <p>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.</p>\n </section>\n \n <section>\n \n <h3> Results</h3>\n \n <p>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 (<i>p</i> < .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%, <i>p</i> = .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; <i>p</i> < .001), absence of palpable spleen reduction by ≥50% at 6 months (HR, 1.45; <i>p</i> = .02), and red blood cell transfusion requirement at all time points (HR, 1.85; <i>p</i> = .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 (<i>p</i> < .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) (<i>p</i> = .01).</p>\n </section>\n \n <section>\n \n <h3> Conclusions</h3>\n \n <p>The iRR6 model provides a more refined tool for the identification of intermediate-1 MF patients who may benefit from early therapy shift.</p>\n </section>\n </div>","PeriodicalId":138,"journal":{"name":"Cancer","volume":"131 17","pages":""},"PeriodicalIF":5.1000,"publicationDate":"2025-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://acsjournals.onlinelibrary.wiley.com/doi/epdf/10.1002/cncr.70062","citationCount":"0","resultStr":"{\"title\":\"Revised “iRR6” model in intermediate-1 risk myelofibrosis patients treated with ruxolitinib\",\"authors\":\"Francesca Palandri MD, PhD, Filippo Branzanti MSc, Massimiliano Bonifacio MD, PhD, Elena M. 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引用次数: 0
摘要
鲁索利替尼6个月后反应（RR6）模型允许早期识别鲁索利替尼治疗的总生存期（OS）较差的骨髓纤维化（MF）患者；然而，它不太适用于低风险患者。为了进一步探讨这一点，作者对“RUX-MF”研究（NCT06516406）进行了一项亚分析，目的是验证RR6，并开发针对中-1 DIPSS/MYSEC-PM风险患者的特异性评分。结果776例可评估患者中，低、中、高RR6风险分别为34.4%、47.8%和17.8%，5年OS分别为64.1%、51.8%和44.5% （p < .001）。在428例中-1患者中，RR6模型没有区分中危和低危患者（5年OS: 74.4% vs. 72.0%, p = 0.24）。因此，中-1特异性RR6 （iRR6）模型通过纳入新的变量来建立：鲁索利替尼在一个或多个时间点的血小板计数不足（风险比[HR]， 3.91; p < 0.001）， 6个月时未见明显脾脏缩小≥50%（风险比，1.45;p = 0.02），以及所有时间点的红细胞输血需求（风险比，1.85;p = 0.01）。iRR6模型将患者分为低危（评分0,20.3%）、中危（评分1-2,45.8%）和高危（评分>；2, 33.9%）三个风险类别，5年OS分别为84.8%、76.4%和56.6% （p < 0.0001）。iRR6模型在Moffitt癌症中心的95名中危患者队列中得到验证，得到相同的三个风险类别，5年OS为83.3%（低危），71.7%（中危）和54.5%（高危）（p = 0.01）。结论：iRR6模型为识别可能从早期治疗转变中受益的中-1 MF患者提供了更精细的工具。
Revised “iRR6” model in intermediate-1 risk myelofibrosis patients treated with ruxolitinib
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：
The CANCER site is a full-text, electronic implementation of CANCER, an Interdisciplinary International Journal of the American Cancer Society, and CANCER CYTOPATHOLOGY, a Journal of the American Cancer Society.
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