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

## TL;DR

A 10-year study in China shows that CD20-targeted therapy for Waldenstrom macroglobulinemia has durable disease control and acceptable safety, with treatment choices affecting survival and side effects.

## Contribution

This study provides real-world long-term data on CD20-targeted therapies for Waldenstrom macroglobulinemia in a Chinese cohort, highlighting individualized treatment approaches.

## Key findings

- The FCR group had the longest overall survival (75.86 ± 22.05 months) among CD20-targeted therapies.
- Rituximab monotherapy (RTX) showed the poorest outcomes with a 75% mortality rate within 12 months after relapse.
- FCR therapy had the highest rate of grade ≥3 adverse events (60%), mainly leukopenia and thrombocytopenia.

## Abstract

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.

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.

Significant differences were found in age, disease burden, and bone marrow infiltration. Patients in the R-CHOP group had higher IgM levels and bone marrow infiltration rates. The FCR group achieved the longest OS (75.86 ± 22.05 months, P < 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.

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.