Objective:lymphoplasmacytic lymphoma/Waldenström Macroglobulinemia(LPL/WM) is a rare and incurable lymphoid malignancy. The treatment outcomes in Chinese patients had never been studies. In this study, we systematically reviewed the treatment outcome in a consecutive cohort from a single center in the past 20 years to explore the efficacy of different treatment regimens in real word in China.

Methods:The clinical data of 269 newly diagnosis patients with LPL/WM admitted to our hospital in the past 20 years (2000-2020) were retrospectively analyzed. Therapy efficacy and survival were compared between different groups.

Results:The median age of 269 patients was 62 years (range 32-87) and the male to female ratio was 2.7(196/73). Nearly half patients were high risk group of ISSWM (48%). Of 269 patients,192 patients received combined chemotherapy.The most common symptoms for initial chemotherapy wereanemia(75.7%), thrombocytopenia (36.9%), followed by IgM-related (33.7%) and constitutional symptoms(21.2%).Of the 192 patients, 109 patients received the novel agents for first-line therapy,83 patients received traditional chemotherapy.180 of192 patients would evaluated. The overall response rate(ORR) and major response rate(MRR) in the novel agent group were significantly higher than the traditional group(ORR:81.7% vs 61.8%,P=0.003; MRR:68.3% vs 48.7%,P=0.008) .In the traditional group, 41 patients received oral alkylating agent-based therapy and 42 patients received conventional intravenous cytotoxicity agents-basedtherapy. The ORR and MRR in the intravenous group were higher than oral group(ORR:75% vs 47.2%,P=0.013;MRR:62.5% vs33.3%,P=0.011).With a median observation time of 36 months, the median overall survival (OS) and progression-free survival (PFS) were 60 months and not reached in the novel agent groups which were also significantly higher than traditional groups(PFS: 60 vs 42,P=0.009,OS:NR vs 89,P=0.000). Intriguingly, In the traditional group, the PFS and OS were no significantly differentbetween conventional intravenous and oralgroup(PFS oral vs intravenous: 42 vs 40,P=0.849,OS oral vs intravenous:62vs 114,P=0.260). In novel agent group, 36 patients had received RCHOP regiment,20 patients received DRC,21 patients received BCD,8 patients received BRD,16 patients received TCD and 8 patients received RFC regiment. Patients who received RCHOP,DRC,BCD,BDR or RFC had similar ORR (82%-87.5%)which were slightly higher than TCD (58.3%)regiment. The median PFS and OS in different subgroups were also similar. But BDR regiment showed a trend of better OS(5 year 100%) and TCD showed a worse OS(5 year 46.4%). For all the patients, 35 patients received maintenance therapy, including 16 patients with rituximab maintenance and 19 patients with thalidomide maintenance. Maintenance was associated with better PFS(60 vs 47,p=0.083) and OS(5 year OS were 96.3% and 64.8%,p=0.006) and patients who received rituximab maintenance had better survival than thalidomide maintenance. In novel agent subgroup, maintenance with rituximab also had a trend of better PFS and OS. But maintenance thalidomide had no significantly different with no maintenance group.

Conclusion:Combined with novel agent can tremendously improve the short-term and long-term therapeutic effects in Chinese LPL/WM patients. Different novel agent regiment might had similar efficacy, BRD regiment had a trend of better survival. Maintenance with rituximab would further improve the effect even in novel agent age.


No relevant conflicts of interest to declare.

Author notes


Asterisk with author names denotes non-ASH members.

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