A real-world 10-year follow-up analysis of survival and safety of CD20-targeted therapy in patients with Waldenstrom macroglobulinemia

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Objective: To evaluate the long-term survival and safety outcomes of CD20-targeted chemoimmunotherapy regimens in patients with Waldenstrom macroglobulinemia (WM) in a real-world single-center cohort from China. Methods: We conducted a retrospective analysis of 128 patients with WM who received CD20-based regimens at a single center from 2014 to 2024. Baseline characteristics, treatment efficacy, survival outcomes (OS and PFS) and safety profiles were compared among the four treatment groups. Results: < 0.01). The RTX group showed the poorest outcomes, with a mortality rate of 75% within 12 months after relapse. ORR and consolidation therapy rates were similar across groups. The FCR group had the highest rate of grade ≥3 adverse events (60%), mainly leukopenia and thrombocytopenia. The R-CHOP group had a higher infection risk, while RTX was the safest. Conclusion: In this 10-year single-center real-world cohort from China, CD20-targeted chemoimmunotherapy achieved durable disease control with acceptable long-term safety in patients with WM. Differences in efficacy and toxicity profiles among FCR, BR, R-CHOP, and rituximab monotherapy support an individualized treatment approach that takes into account patient age, comorbidities, and tolerance. Because standardized quality-of-life instruments were not used in this retrospective study, our findings should be interpreted as reflecting survival and treatment-related toxicity rather than validated health-related quality of life.

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