CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CAR-T(CAR-T)细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Saudi experts statement on advancing multiple myeloma treatment: the evolving role of bispecific antibodies.
Saudi experts statement on advancing multiple myeloma treatment: the evolving role of bispecific antibodies.
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BsAbs 是三类药物难治性多发性骨髓瘤患者的有效治疗选择,本文提供了一个框架,以支持其在沙特阿拉伯的最佳使用。
**引言:**多发性骨髓瘤(MM)的治疗格局快速演进,但沙特阿拉伯复发/难治性(RR)MM患者仍存在显著未满足需求。尽管CAR-T 疗法已在当地获批用于骨髓瘤,但可及性仍有限;高龄、合并症、疾病进展迅速及物流障碍等因素也使许多患者不符合治疗条件。双特异性抗体(BsAb)在沙特阿拉伯逐渐成为有前景的选择,但缺少地区特异性临床证据,因此亟须制定适合当地、以证据为基础的临床指导,以优化其应用。 **方法:**一组沙特阿拉伯MM专家依据临床经验和文献分析证据,就BsAb临床最佳应用方式达成共识。研究采用改良Delphi法,预先设定70%的共识阈值,形成专家建议。 **结果:**35项陈述中有30项(86%)达成共识,涉及患者选择、治疗顺序与剂量、监测及疗效结局四个方面。专家同意将BsAb用于三类药物耐药MM。BsAb临床试验显示总体缓解率较高、无进展生存期和总生存期延长,凸显选择已证实有效BsAb的重要性。CAR-T 疗法近期才在沙特获批用于MM;考虑到CAR-T 产品制备需要时间,对于疾病进展迅速的患者,专家更倾向选择BsAb。 **结论:**BsAb是三类药物耐药MM的有效治疗选择,本文提出的框架旨在支持其在沙特阿拉伯的优化应用。
A group of experts in MM based in Saudi Arabia convened to align on optimal use of BsAbs in clinical practice based on their experience and evidence from a literature analysis. A modified Delphi methodology with prespecified acceptance threshold of 70% was used to generate expert recommendations.
Agreement was achieved in 30 of 35 statements (86%) across four broad areas: patient selection, sequencing and dosing, monitoring, and efficacy outcomes. Experts agreed that BsAbs should be positioned in triple-class refractory MM. Positive clinical trial outcomes observed with BsAbs, including high overall response rates, prolonged progression-free survival and overall survival underscore the importance of selecting BsAbs with proven efficacy. While CAR T-cell therapy is only recently approved for MM in Saudi Arabia, BsAbs are preferred for patients with rapidly progressing disease due to CAR T-cell product manufacturing delays.
BsAbs are an effective treatment option for those with triple-class refractory MM and this manuscript provides a framework to support their optimal use in Saudi Arabia.
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