[Clinical Characteristics and Prognosis of Lymphoplasmacytic Lymphoma/Waldenström Macroglobulinemia]

To explore the clinical features, treatment strategies, and prognostic factors of patients with new diagnosed Lymphoplasmacytic lymphoma/Waldenström macroglobulinemia (LPL/WM), thereby enhancing the diagnostic and therapeutic understanding of this disease.

Comprehensive clinical data were collected from 35 newly diagnosed LPL/WM patients at our hospital between December 2015 and June 2024. A systematic analysis was conducted on baseline characteristics, laboratory parameters, treatment regimens, and follow-up information. Survival analysis was performed using the Kaplan-Meier method, and a Cox regression model was applied to assess prognostic factors.

A total of 35 LPL/WM patients were enrolled, with a male predominance (91.4%) and a median age at diagnosis of 69 years (range: 32-83). The most common clinical manifestations were fatigue (45.7%), lower limb edema (28.5%), and lymphadenopathy (62.9%). Laboratory findings revealed anemia in 88.6% of patients. The vast majority (94.3%) secreted monoclonal IgM, one patient secreted monoclonal IgG, and one patient had both monoclonal IgM and IgG. The light chain type was predominantly kappa (77.1%). Molecular genetic testing showed a MYD88 L265P mutation rate of 82.4% (28/34), while the CXCR4 mutation rate was lower (17.6%, 3/17). The overall response rate was 82.4% in the treatment group containing Bruton's tyrosine kinase inhibitors (BTKi) and 66.7% in the non-BTKi treatment group. With a median follow-up of 70 months, one patient was lost to follow-up and 14 patients died. The median overall survival (OS) for the entire cohort was 71 months. Univariate analysis identified β₂-microglobulin ≥4 mg/L at diagnosis was associated with OS (P =0.036) and PFS (P =0.021). Multivariate analysis confirmed β₂-microglobulin ≥4 mg/L at diagnosis as an independent adverse prognostic factor for OS (HR =3.854, P =0.025) and PFS (HR=3.201, P =0.030), while receiving BTKi-containing therapy was identified as a protective factor for OS (HR=0.312, P =0.047).

This study confirms that elevated β₂-microglobulin levels are an independent adverse prognostic factor in patients with LPL/WM. In our center's cohort, patients receiving BTKi therapy achieve higher response rates and longer OS.