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靶向 GPRC5D 的 CAR-T 细胞治疗复发/难治性多发性骨髓瘤:病例报告与文献综述

英文原题:Targeting GPRC5D With CAR-T Cells in Relapse/Refractory Multiple Myeloma: Case Report and Literature Review.

PubMed 2025/12/15(内容时间) Case Rep Hematol Q4 · IF 0.7(JCR 2025)

研究概要

靶向 GPRC5D 的 CAR-T 细胞疗法对 RRMM 患者,尤其是具有高危因素的患者,高效且耐受性良好。

中文摘要

多发性骨髓瘤(MM)是第二常见的血液系统恶性肿瘤,复发/难治性多发性骨髓瘤(RRMM)患者面临治疗选择有限和预后不良的困境。近年来,靶向G蛋白偶联受体C类第5组成员D(GPRC5D)的CAR-T细胞疗法在临床前研究中显示出良好的疗效和安全性,为RRMM患者带来了新的希望。我们报告了一例48岁女性复发/难治性非分泌型MM患者的成功治疗。该患者具有高危因素,包括1q21扩增和TP53缺失,在接受了包括自体造血干细胞移植、蛋白酶体抑制剂、免疫调节剂、PD-1抑制剂和CD38单克隆抗体在内的七线治疗后复发。她还出现了髓外病变。最终,她接受了靶向GPRC5D的CAR-T细胞治疗,髓外病灶完全消失,并获得了持续长达17个月的完全缓解。总之,靶向GPRC5D的CAR-T细胞疗法在RRMM患者中,尤其是具有高危因素的患者中,具有高度有效性和良好的耐受性。仍需更大规模队列和更长随访期的进一步研究来验证GPRC5D靶向CAR-T细胞疗法在RRMM中的临床应用,特别是对于BCMA靶向治疗失败的患者。

展开英文摘要原文

Multiple myeloma (MM) is the second most common hematologic malignancy, and patients with relapsed/refractory MM (RRMM) face limited treatment options and a poor prognosis. Recently, CAR-T cell therapy targeting G-protein-coupled receptor, class C group 5 member D (GPRC5D) has shown promising efficacy and safety in preclinical studies, offering new hope for patients with RRMM. We report the successful treatment of a 48-year-old female patient with relapsed/refractory nonsecretory MM. The patient had high-risk factors, including 1q21 amplification and TP53 deletion, and had relapsed after seven lines of therapy, including autologous hematopoietic stem cell transplantation, proteasome inhibitors, immunomodulatory agents, PD-1 inhibitors, and CD38 monoclonal antibodies. She also developed extramedullary disease. Eventually, she received CAR-T cell therapy targeting GPRC5D, which led to the complete disappearance of extramedullary lesions and a sustained complete remission lasting up to 17 months. In conclusion, CAR-T cell therapy targeting GPRC5D is highly effective and well-tolerated in patients with RRMM, especially those with high-risk factors. Further studies with larger cohorts and longer follow-up periods are needed to validate the clinical application of GPRC5D-targeted CAR-T cell therapy in RRMM, particularly for patients who have failed BCMA-targeted therapies.

论文信息

作者
Yan S、Ming X、Wu J、Peng D、Zhou M、Xiao Y
单位
Department of Hematology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei, China, hust.edu.cn.China
期刊
Case reports in hematology2025
原文标识
PubMed 41439209 · DOI 10.1155/crh/5900306