在莱纳利多米德耐火性多发性骨髓瘤中使用Cilta-cel或标准护理
Jesús San-Miguel1, Binod Dhakal1, Kwee Yong1
1From the Cancer Center Clínica Universidad de Navarra, Centro Investigación Medica Aplicada, Instituto de Investigación de Navarra, Centro Investigación Biomédica en Red Cáncer (CIBERONC), Pamplona (J.S.-M.), the University Hospital of Salamanca, Instituto de Investigación Biomédica de Salamanca, Centro Investigación del Cáncer, CIBERONC, Salamanca (M.-V.M.), the Amyloidosis and Myeloma Unit, Department of Hematology, Hospital Clínic of Barcelona, Institut d'Investigacions Biomèdiques August Pi i Sunyer, University of Barcelona, Barcelona (C.F.L.), the Hematological Malignancies Clinical Research Unit, Hospital 12 de Octubre Universidad Complutense, Centro Nacional de Investigaciones Oncológicas, CIBERONC, Madrid (J.M.-L.), and the Institut Català d'Oncologia and Institut Josep Carreras, Hospital Germans Trias i Pujol, Badalona (A.O.) - all in Spain; the Medical College of Wisconsin, Milwaukee (B.D.); the University College London Cancer Institute (K.Y.) and University College London Hospitals, NHS Foundation Trust (R.P.), London, University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (J. Griffin), and Janssen Research and Development, High Wycombe (J. Gilbert) - all in the United Kingdom; Monash University (A. Spencer) and the Peter MacCallum Cancer Centre and Royal Melbourne Hospital, Sir Peter MacCallum Department of Oncology, University of Melbourne (S.J.H.), Melbourne, VIC, and Royal Prince Alfred Hospital and the University of Sydney, Sydney (P.J.H.) - all in Australia; the Vaccine and Infectious Disease Institute, University of Antwerp, Antwerp, and the Division of Hematology and Center for Cell Therapy and Regenerative Medicine, Antwerp University Hospital, Edegem - both in Belgium (S.A.); the Department of Hematology, University Hospital Hôtel-Dieu, Nantes (P.M., C.T.), and Service d'Hématologie et Thérapie Cellulaire, Hôpital La Milétrie, and Centre d'investigation Clinique INSERM 1402, Poitiers University Hospital, Poitiers (X.L.) - both in France; Tel Aviv Sourasky Medical Center (I.A., Y.C.C.) and the Sackler Faculty of Medicine, Tel Aviv University (Y.C.C.) - both in Tel Aviv, Israel; IRCCS Azienda Ospedaliero-Universitaria di Bologna, Seràgnoli Institute of Hematology, and the Department of Medical and Surgical Sciences, Bologna University School of Medicine, Bologna, Italy (M.C.); the Department of Hematology, Japanese Red Cross Medical Center, Tokyo (T.I.); the Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea (S.J.K.); the Department of Hematology, University Medical Center Groningen, Groningen (W.R.), and Amsterdam University Medical Center, Vrije Universiteit Amsterdam, Amsterdam (N.W.C.J.D.) - both in the Netherlands; Poznan University of Medical Sciences, Poznan, Poland (D.D.); Stanford University School of Medicine, Stanford, CA (S.S.); the University of Alabama at Birmingham, Birmingham (L.J.C.); Ohio State University Comprehensive Cancer Center, Columbus (A.M.K.); Janssen Research and Development, Raritan (N.L., J.M.S., C.C.J., K.C., T.Y., S.N.), and Legend Biotech, Somerset (E.F., L.P., N.P.) - both in New Jersey; Janssen, Buenos Aires (C.L.); Cilag International, Zug, Switzerland (A. Slaughter); Janssen Research and Development, Spring House, PA (K.L., E.Z.); Janssen China Research and Development, Shanghai (D.C.); and Universitätsklinikum Würzburg, Medizinische Klinik und Poliklinik II, Würzburg, Germany (H.E.).
在患有勒那利多米德耐药多发性骨髓瘤的患者中,Ciltacabtagene自杀细胞 (cilta-cel) 显著改善了无进展的存活率. 这种CAR T细胞疗法与早期治疗线的标准护理相比,显示出更高的疗效.
科学领域:
- 血液学 血液学 血液学
- 在瘤学瘤学.
- 免疫治疗是一种免疫疗法.
背景情况:
- Ciltacabtagene autoleucel (cilta-cel) 是一种BCMA指导的CAR T细胞疗法,有效治疗严重预先治疗的多发性骨髓瘤.
- 在较早的治疗线路中研究了cilta-cel,用于勒纳利多米德耐药多发性骨髓瘤患者.
研究的目的:
- 为了评估cilta-cel与标准治疗相比,在勒纳利多米德耐药多发性骨髓瘤患者中的疗效.
- 在3期随机试验中,评估无进展生存率作为主要结局.
主要方法:
- 第三阶段,随机化,开放标签试验,涉及419名勒纳利多米德耐药多发性髓瘤患者.
- 患者在一到三个之前的治疗线之后接受了cilta-cel或医生选择的标准护理.
- 主要结局是无进展生存期.
主要成果:
- 治疗西尔塔细胞的中位数没有达到无进展生存期,而标准治疗的中位数为11.8个月 (HR,0.26;P<0.001).
- 在12个月的无进展生存率为75.9%的cilta-cel对比48.6%的标准治疗.
- 较高的整体响应 (84.6% vs. 67.3%) 和完全响应或更好的 (73.1% vs. 21.8%) 率观察到西尔塔细胞.
- 报告了包括细胞因子释放综合征和神经毒性在内的不良事件,其中大多数是低级的.
结论:
- 与标准护理相比,单次细胞输液显著降低了疾病进展或死亡的风险.
- 在较早的治疗环境中,Cilta-cel为勒那利多米德耐药的多发性骨髓瘤患者提供了更有效的治疗选择.
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