Epidemiology, Real-World Treatment, and Economic Burden of Waldenström Macroglobulinemia: A Comprehensive Analysis...

This study aimed to provide evidence on the epidemiology, real-world treatment patterns, overall survival, and economic burden of Waldenström macroglobulinemia (WM).

A retrospective analysis of an anonymized large German claims database from January 1, 2010, to June 30, 2022, identified incident WM cases based on a 12-month diagnosis-free period before the first confirmed WM diagnosis (ICD-10-GM code C88.0). WM diagnosis was validated through at least 2 confirmed outpatient diagnoses in different quarters within 12 months or 1 inpatient diagnosis (further sensitivity scenarios were tested). Treatment patterns were analyzed based on the follow-up period after diagnosis, with line of treatment derived by an algorithm using outpatient prescription/inpatient procedure information. Incremental costs were derived by comparing patients with WM with a propensity-score-matched control group.

A total of 593 incident WM cases were identified (mean age: 72.7 years; 46.5% female). In 2021, standardized cumulative incidence ranged from 1.39 to 1.90 per 100 000 persons (males: 1.98 to 2.60; females: 0.73 to 1.25). Median overall survival was 7.9 years. Nearly 70% of patients initially followed a watch-and-wait approach, with less than one-third starting therapy after 2 years. Rituximab, often combined with bendamustine or unspecified inpatient chemotherapy, was the most common treatment. Recently, ibrutinib has been used more frequently in later lines. Incremental cost analysis showed substantial care costs, mainly driven by medications and inpatient care.

The study indicates an increasing incidence of WM in Germany, associated with significant economic burden, despite the majority of newly diagnosed patients initially adopting a watch-and-wait approach.