Dosing and clinical outcomes of ruxolitinib in patients with myelofibrosis in a real-world setting: Interim results of the Italian observational study (ROMEI)
Massimo Breccia MD, Francesca Palandri MD, PhD, Maurizio Martelli MD, Francesco Mendicino MD, Alessandra Malato MD, PhD, Giuseppe A. Palumbo MD, PhD, Silvia Sibilla MD, Nicola Di Renzo MD, Elisabetta Abruzzese MD, PhD, Sergio Siragusa MD, Monica Crugnola MD, Carmine Selleri MD, Fabrizio Pane MD, Paolo Sportoletti MD, Bruno Martino MD, Stefana Impera MD, Alessandra Ricco MD, Maria Langella MD, Paolo Ditonno MD, Giuseppe Carli MD, Federico Itri MD, Anna Marina Liberati MD, Tiziana Urbano MD, Agostino Tafuri MD, Vita Polizzi MD, Domenico Pastore MD, Erika Morsia MD, Giulia Benevolo MD, Giorgia Micucci MD, Gabriella Farina MD, Massimiliano Bonifacio MD, Elena Maria Elli MD, Angelo Gardellini MD, Valerio De Stefano MD, Giovanni Caocci MD, Antonietta Pia Falcone MD, Daniele Vallisa MD, Marco Brociner MD, Mario Tiribelli MD, Gianni Binotto MD, Barbara Pocali MD, Francesco Cavazzini MD, Simona Tomassetti MD, Francesca Lunghi MD, Mauro Di Ianni MD, Alessandro Allegra MD, Barbara Anaclerio MD, Serena Mazzotta MD, Nicola Orofino MD, Filippo Gherlinzoni MD, Chiara Castiglioni PharmD, Marina Landoni PharmD, Diletta Valsecchi PharmD, Michela Magnoli MSc, Paola Guglielmelli MD, PhD, Francesco Passamonti MD
{"title":"Dosing and clinical outcomes of ruxolitinib in patients with myelofibrosis in a real-world setting: Interim results of the Italian observational study (ROMEI)","authors":"Massimo Breccia MD, Francesca Palandri MD, PhD, Maurizio Martelli MD, Francesco Mendicino MD, Alessandra Malato MD, PhD, Giuseppe A. Palumbo MD, PhD, Silvia Sibilla MD, Nicola Di Renzo MD, Elisabetta Abruzzese MD, PhD, Sergio Siragusa MD, Monica Crugnola MD, Carmine Selleri MD, Fabrizio Pane MD, Paolo Sportoletti MD, Bruno Martino MD, Stefana Impera MD, Alessandra Ricco MD, Maria Langella MD, Paolo Ditonno MD, Giuseppe Carli MD, Federico Itri MD, Anna Marina Liberati MD, Tiziana Urbano MD, Agostino Tafuri MD, Vita Polizzi MD, Domenico Pastore MD, Erika Morsia MD, Giulia Benevolo MD, Giorgia Micucci MD, Gabriella Farina MD, Massimiliano Bonifacio MD, Elena Maria Elli MD, Angelo Gardellini MD, Valerio De Stefano MD, Giovanni Caocci MD, Antonietta Pia Falcone MD, Daniele Vallisa MD, Marco Brociner MD, Mario Tiribelli MD, Gianni Binotto MD, Barbara Pocali MD, Francesco Cavazzini MD, Simona Tomassetti MD, Francesca Lunghi MD, Mauro Di Ianni MD, Alessandro Allegra MD, Barbara Anaclerio MD, Serena Mazzotta MD, Nicola Orofino MD, Filippo Gherlinzoni MD, Chiara Castiglioni PharmD, Marina Landoni PharmD, Diletta Valsecchi PharmD, Michela Magnoli MSc, Paola Guglielmelli MD, PhD, Francesco Passamonti MD","doi":"10.1002/cncr.35801","DOIUrl":null,"url":null,"abstract":"<div>\n \n \n <section>\n \n <h3> Background</h3>\n \n <p>Myelofibrosis (MF) significantly impacts patients’ overall survival (OS) and quality of life (QOL). This prospective study analyzed ruxolitinib dosing patterns and associated clinical outcomes in patients with MF over 12 months.</p>\n </section>\n \n <section>\n \n <h3> Methods</h3>\n \n <p>ROMEI, a multicenter, observational, ongoing study, enrolled 508 adult patients with MF treated with ruxolitinib. For the current interim analysis, eligible patients with baseline platelet values were categorized into two groups based on ruxolitinib starting dosage: as expected (AsEx, <i>n</i> = 174) and lower than expected (LtEx, <i>n</i> = 132); ruxolitinib dose changes, interruptions and time to permanent discontinuation were analyzed, along with symptoms response, health-related QOL scores, spleen response, OS, and safety.</p>\n </section>\n \n <section>\n \n <h3> Results</h3>\n \n <p>Forty-three percent of patients started at a lower-than-expected dose. Both groups showed reduction in average daily ruxolitinib doses over 12 months. Symptoms response rate was similar in both groups at week 48 (40.8% AsEx vs 40.9% LtEx). The AsEx group demonstrated higher spleen response rates at both 24 weeks (50.0% vs 30.2%) and 48 weeks (57.7% vs 45.8%) with a shorter median time to first response (3.3 vs 11.1 months, <i>p</i> = .019) when compared to the LtEx group. Both groups showed upward trends in health-related QOL values. Estimated median OS was not reached for the AsEx group versus 4.7 years in the LtEx group (<i>p</i> = .014). Adverse events were reported in 87.4% and 84.9% of patients in the AsEx and LtEx groups, respectively.</p>\n </section>\n \n <section>\n \n <h3> Conclusions</h3>\n \n <p>The ROMEI study demonstrated the importance of optimal ruxolitinib dosage in patients with MF for maximum effectiveness and improved OS, with manageable safety.</p>\n </section>\n </div>","PeriodicalId":138,"journal":{"name":"Cancer","volume":"131 7","pages":""},"PeriodicalIF":5.1000,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11925231/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Cancer","FirstCategoryId":"3","ListUrlMain":"https://acsjournals.onlinelibrary.wiley.com/doi/10.1002/cncr.35801","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"ONCOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Background
Myelofibrosis (MF) significantly impacts patients’ overall survival (OS) and quality of life (QOL). This prospective study analyzed ruxolitinib dosing patterns and associated clinical outcomes in patients with MF over 12 months.
Methods
ROMEI, a multicenter, observational, ongoing study, enrolled 508 adult patients with MF treated with ruxolitinib. For the current interim analysis, eligible patients with baseline platelet values were categorized into two groups based on ruxolitinib starting dosage: as expected (AsEx, n = 174) and lower than expected (LtEx, n = 132); ruxolitinib dose changes, interruptions and time to permanent discontinuation were analyzed, along with symptoms response, health-related QOL scores, spleen response, OS, and safety.
Results
Forty-three percent of patients started at a lower-than-expected dose. Both groups showed reduction in average daily ruxolitinib doses over 12 months. Symptoms response rate was similar in both groups at week 48 (40.8% AsEx vs 40.9% LtEx). The AsEx group demonstrated higher spleen response rates at both 24 weeks (50.0% vs 30.2%) and 48 weeks (57.7% vs 45.8%) with a shorter median time to first response (3.3 vs 11.1 months, p = .019) when compared to the LtEx group. Both groups showed upward trends in health-related QOL values. Estimated median OS was not reached for the AsEx group versus 4.7 years in the LtEx group (p = .014). Adverse events were reported in 87.4% and 84.9% of patients in the AsEx and LtEx groups, respectively.
Conclusions
The ROMEI study demonstrated the importance of optimal ruxolitinib dosage in patients with MF for maximum effectiveness and improved OS, with manageable safety.
背景：骨髓纤维化（Myelofibrosis， MF）显著影响患者的总生存期（OS）和生活质量（QOL）。这项前瞻性研究分析了鲁索利替尼给药模式和MF患者超过12个月的相关临床结果。方法：ROMEI是一项多中心、观察性、正在进行的研究，纳入了508名接受鲁索利替尼治疗的成年MF患者。在目前的中期分析中，基线血小板值符合条件的患者根据鲁索利替尼起始剂量分为两组：符合预期（AsEx, n = 174）和低于预期（LtEx, n = 132）；分析ruxolitinib剂量变化、中断和永久停药时间，以及症状反应、健康相关的QOL评分、脾脏反应、OS和安全性。结果：43%的患者开始时的剂量低于预期。两组在12个月内平均每日鲁索利替尼剂量均有所减少。两组在第48周的症状缓解率相似（AsEx 40.8% vs LtEx 40.9%）。与LtEx组相比，AsEx组在24周（50.0% vs 30.2%）和48周（57.7% vs 45.8%）时均表现出更高的脾脏缓解率，且到达首次缓解的中位时间（3.3 vs 11.1个月，p = 0.019）更短。两组的健康相关生活质量值均呈上升趋势。AsEx组的估计中位生存期未达到，而LtEx组为4.7年（p = 0.014）。AsEx组不良事件发生率为87.4%，LtEx组不良事件发生率为84.9%。结论：ROMEI研究表明，对于MF患者，最佳鲁索利替尼剂量对于最大有效性和改善OS的重要性，并且具有可管理的安全性。
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.
CANCER publishes interdisciplinary oncologic information according to, but not limited to, the following disease sites and disciplines: blood/bone marrow; breast disease; endocrine disorders; epidemiology; gastrointestinal tract; genitourinary disease; gynecologic oncology; head and neck disease; hepatobiliary tract; integrated medicine; lung disease; medical oncology; neuro-oncology; pathology radiation oncology; translational research
京公网安备 11010802042870号
京ICP备2023020795号-1
分享
求助内容：
应助结果提醒方式：
扫码关注我们
