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Journal of Zhejiang University SCIENCE B 2024 Vol.25 No.8 P.719-722

http://doi.org/10.1631/jzus.B2300835


Waldenström macroglobulinemia: a challenging case treated with anti-CD19 CAR-T cell therapy


Author(s):  Yang YANG, Xiaolin GU, Jingsong HE, Yongxian HU, Zhen CAI

Affiliation(s):  Bone Marrow Transplantation Center, Department of Hematology, the First Affiliated Hospital, School of Medicine, Zhejiang University,Hangzhou310006,China; more

Corresponding email(s):   caiz@zju.edu.cn

Key Words:  Waldenström macroglobulinemia, Chimeric antigen receptor T cell therapy, Relapsed and refractory


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Yang YANG, Xiaolin GU, Jingsong HE, Yongxian HU, Zhen CAI. Waldenström macroglobulinemia: a challenging case treated with anti-CD19 CAR-T cell therapy[J]. Journal of Zhejiang University Science B, 2024, 25(8): 719-722.

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%T Waldenström macroglobulinemia: a challenging case treated with anti-CD19 CAR-T cell therapy
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%A Xiaolin GU
%A Jingsong HE
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Abstract: 
Waldenström macroglobulinemia (WM) is characterized by lymphoplasmacytic lymphoma associated with large amounts of monoclonal immunoglobulin M (IgM) protein (Owen et al., 2003). Common signs and symptoms include fatigue due to anemia, lymph node enlargement, hepatosplenomegaly, thrombocytopenia, symptoms related to high viscosity, and peripheral neuropathy, among others. Despite significant advances in WM treatment, this type of indolent lymphoma remains incurable, with a wide array of patient outcomes (Ruan et al., 2020). In recent years, chimeric antigen receptor T (CAR-T) cell therapy targeting cluster of differentiation 19 (CD19) has shown unprecedented response rates and durability in the treatment of B-cell malignancies. In this report, we describe a challenging case of WM that involved multiple extramedullary sites, relapsed, and was refractory to chemotherapy, immunotherapy, and targeted therapy. After anti-CD19 CAR-T cell therapy, the tumor burden significantly decreased and the patient’s condition remained stable at the writing of this report.

华氏巨球蛋白血症:抗CD19嵌合抗原受体T细胞疗法治疗的挑战性病例

杨杨1,顾晓琳1,2,何静松1,胡永仙1,蔡真1
1浙江大学医学院第一附属医院血液科骨髓移植中心,中国杭州市,310006
2浙江省舟山市普陀人民医院血液科,中国舟山市,316100
摘要:华氏巨球蛋白血症(WM)是一种罕见的小淋巴细胞、浆细胞样淋巴细胞和浆细胞肿瘤,会累及骨髓,甚至累及淋巴结和脾,通常与克隆性IgM球蛋白血症相关。尽管WM通常伴有惰性临床病程,但却无法治愈。本研究报告了一个具有挑战性的WM案例。该患者在化疗、免疫治疗和布鲁顿酪氨酸激酶(BTK)抑制剂治疗后出现复发/难治性病变,并累及髓外,但在CD19定向嵌合抗原受体T(CAR-T)细胞治疗后病情得到改善。此外,我们还回顾了文献中报道的CAR-T细胞治疗WM病例。本案例中的患者已接受一年多的随访,其病情仍保持良好的部分缓解状态,这表明CAR-T细胞疗法具有作为复发/难治性WM替代方案的潜力。人们迫切希望进行更大规模的临床研究来评估这种疗法的有效性和安全性。

关键词:华氏巨球蛋白血症;嵌合抗原受体T(CAR-T)细胞治疗;复发和难治性

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