Department of Internal Medicine, Capital Health Medical Center, Trenton, NJ, USA.
University of Cincinnati, Cincinnati, OH, USA.
Ann Hematol. 2024 Jun;103(6):1859-1876. doi: 10.1007/s00277-023-05345-9. Epub 2023 Jul 6.
Waldenström macroglobulinemia (WM) is a chronic B-cell lymphoproliferative disorder characterized by lymphoplasmacytic cell overgrowth in the bone marrow and increased secretion of IgM immunoglobulins into the serum. Patients with WM have a variety of clinical outcomes, including long-term survival but inevitable recurrence. Recent advances in disease knowledge, including molecular and genetic principles with the discovery of MYD88 and CXCR4 mutations, have rapidly increased patient-tolerable treatment options. WM patients may benefit from chemotherapy regimens that include rituximab-based regimens, alkylating drugs, proteasome inhibitors, monoclonal antibodies, and drugs targeting Bruton tyrosine kinase inhibitors. In light of these advancements, patients can now receive treatment customized to their specific clinical characteristics, focusing on enhancing the depth and durability of their response while limiting the adverse effects. Despite the rapidly developing therapeutic armament against WM, a lack of high-quality evidence from extensive phase 3 trials remains a significant challenge in the research. We believe clinical outcomes will keep improving when new medicines are introduced while preserving efficacy and minimizing toxicity.
华氏巨球蛋白血症(WM)是一种慢性 B 细胞淋巴增殖性疾病,其特征是骨髓中淋巴浆细胞过度生长,并导致 IgM 免疫球蛋白分泌到血清中。WM 患者具有多种临床结局,包括长期生存但不可避免的复发。近年来,疾病知识取得了快速进展,包括分子和遗传原则的发现,如 MYD88 和 CXCR4 突变,这极大地增加了患者可耐受的治疗选择。WM 患者可能受益于包括利妥昔单抗在内的化疗方案、烷化剂、蛋白酶体抑制剂、单克隆抗体和针对布鲁顿酪氨酸激酶抑制剂的药物。鉴于这些进展,现在可以根据患者的具体临床特征为其制定治疗方案,重点是增强反应的深度和持久性,同时限制不良反应。尽管针对 WM 的治疗方法不断发展,但缺乏来自广泛的 3 期试验的高质量证据仍然是研究中的一个重大挑战。我们相信,随着新药的推出,临床结果将不断改善,同时保持疗效并最大限度地减少毒性。