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

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 MYD88(L265P) 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. MYD88(L265P) 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.