Rituximab-based chemo-immunotherapy is currently the standard first-line treatment for Waldenstrom macroglobulinaemia (WM), while ibrutinib has emerged as an alternative. In the absence of randomised trials (RCTs) comparing these regimens, the optimal first-line treatment for WM remains uncertain. In this systematic review and meta-analysis, we sought to assess the efficacy and safety of first-line treatment regimens for WM. We searched key databases from January 2007 to March 2023, including phase II and III trials, including treatment-naïve WM patients treated with rituximab-based regimens or ibrutinib. Response rates, progression-free survival (PFS), overall survival (OS), and toxicities were evaluated. Four phase III and seven phase II trials were included among 736 unique records. Pooled response rates from all comparative and non-comparative trials were 46%, 33% and 26% for bendamustine rituximab (BR), bortezomib-dexamethasone, cyclophosphamide, rituximab (BDRC) and ibrutinib rituximab (IR), respectively. Two-year pooled PFS was 89%, 81% and 82% with BR, BDRC and IR, respectively. Neuropathy was more frequent with bortezomib, while haematologic and cardiac toxicities were more common with chemo-immunotherapy and ibrutinib-based regimens respectively. Our findings suggest that BR yields higher response rates than bortezomib or ibrutinib-based combinations. RCTs comparing BR against emerging therapies, including novel Bruton Tyrosine Kinase Inhibitors, are warranted.
基于利妥昔单抗的化学免疫治疗是目前华氏巨球蛋白血症（WM）的标准一线治疗方案，而伊布替尼已成为替代选择。由于缺乏比较这些方案的随机对照试验（RCT），WM的最佳一线治疗仍不明确。在本系统综述与荟萃分析中，我们旨在评估WM一线治疗方案的有效性和安全性。我们检索了2007年1月至2023年3月的主要数据库，纳入包含使用利妥昔单抗方案或伊布替尼治疗的初治WM患者的II期和III期试验。评估指标包括缓解率、无进展生存期（PFS）、总生存期（OS）及毒性反应。从736条独立记录中，共纳入4项III期试验和7项II期试验。所有比较性和非比较性试验的汇总缓解率显示，苯达莫司汀联合利妥昔单抗（BR）、硼替佐米-地塞米松-环磷酰胺-利妥昔单抗（BDRC）及伊布替尼联合利妥昔单抗（IR）的缓解率分别为46%、33%和26%。BR、BDRC和IR的两年汇总PFS分别为89%、81%和82%。神经病变在硼替佐米治疗中更常见，而血液学毒性和心脏毒性分别在化学免疫治疗方案和基于伊布替尼的方案中更为常见。我们的研究结果表明，BR相比基于硼替佐米或伊布替尼的联合方案可获得更高的缓解率。有必要开展随机对照试验比较BR与新兴疗法（包括新型布鲁顿酪氨酸激酶抑制剂）的疗效。
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