Bone Marrow Transplantation Center, Department of Hematology, the First Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou 310006, China.
Department of Hematology, Putuo People's Hospital, Zhoushan 316100, China.
J Zhejiang Univ Sci B. 2024 Aug 15;25(8):719-722. doi: 10.1631/jzus.B2300835.
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（WM）的特征是与大量单克隆免疫球蛋白 M（IgM）蛋白相关的淋巴浆细胞淋巴瘤（Owen 等人，2003 年）。常见的体征和症状包括因贫血引起的疲劳、淋巴结肿大、肝脾肿大、血小板减少症、与高粘度相关的症状以及周围神经病等。尽管 WM 治疗取得了重大进展，但这种惰性淋巴瘤仍然无法治愈，患者的预后差异很大（Ruan 等人，2020 年）。近年来，针对分化抗原 19（CD19）的嵌合抗原受体 T（CAR-T）细胞疗法在治疗 B 细胞恶性肿瘤方面显示出前所未有的反应率和持久性。在本报告中，我们描述了一例涉及多个髓外部位的 WM 挑战性病例，该病例复发且对化疗、免疫疗法和靶向治疗均耐药。接受抗 CD19 CAR-T 细胞治疗后，肿瘤负荷显著降低，截至本报告撰写时患者病情仍保持稳定。
