华氏巨球蛋白血症的临床特征及预后:单中心临床数据报道

Clinical characteristics and prognosis of Waldenström's macroglobulinemia:a clinical data report from a single center

目的总结51例华氏巨球蛋白血症(WM)患者的临床特征及预后,重点评估伊布替尼治疗WM的疗效和不良反应,为WM诊疗提供经验和借鉴。方法回顾性分析2008年11月至2019年10月海军军医大学附属长征医院收治的51例初诊WM患者的基线临床特征、疗效及预后。结果51例患者男女比例为2.64∶1,中位年龄65(46~84)岁。ISSWM危险分层:低危组9例(18%)、中危组21例(41%)、高危组21例(41%),27例(73%)存在MYD88L265P突变。中位随访时间38.6(0.3~120.0)个月,中位无进展生存时间为46.4个月,中位总生存时间未达到。接受伊布替尼治疗的16例患者总体缓解率为87%,主要缓解率为80%,起效速度快,达到至少部分缓解的中位时间为8周,与其他药物组相比差异有统计学意义(P<0.05)。结论WM多见于老年男性,多数患者合并MYD88L265P突变。伊布替尼治疗WM疗效确切,即使是高龄、ISSWM高危患者,也有较高的总体缓解率、主要缓解率。伊布替尼起效速度较快,严重不良反应少见,是一种安全、有效的治疗方案。

Objective To summarize the clinical characteristics and prognosis of 51 patients with Waldenström’s macroglobulinemia(WM)and evaluate the efficacy and adverse reactions of ibrutinib in the treatment of WM.Methods We carried out a single-center retrospective study,including 51 patients with WM of our single center from November 2008 to October 2019.Results The median age at diagnosis was 65 years with a male-to-female ratio of 2.64∶1.There were 9(18%),21(41%),and 21(41%)ISSWM stage low-,intermediate-and high-risk patients identified,respectively.A total of 27(73%)patients harbored MYD88L265P mutation.The median follow-up time was 38.6(0.3-120.0)months,the median progression free survival was 46.4 months,and the median overall survival was not reached.The overall remission and major remission rates of patients who received ibrutinib were 87%and 80%,respectively.The median time to achieve at least partial remission of patients treated with ibrutinib was 8 weeks,which was earlier than those treated with other drugs(P<0.05).Conclusion WM is often seen in elderly men.MYD88L265P had a high frequency in WM.The findings of our study validate the efficacy of ibrutinib monotherapy.Even in patients with advanced age and at high risk of ISSWM,the overall remission rate and major remission rate are high.Ibrutinib is a safe and effective therapy because of its rapid onset and rare serious adverse reactions.