OBJECTIVES: This study aimed to give a general overview of the treatment pathway and unmet needs of Waldenström Macroglobulinemia (WM) in Portugal.

METHODS: Two interviews with Hematologists were conducted to ascertain the treatment pathway of WM patients. Subsequently, a web-based survey was administered, in November 2022, to characterize the percentage of patients with WM per step of the treatment pathway, including watch-and-wait and in-treatment according to patient fitness and line of treatment. The main therapeutic unmet needs were also assessed. All answers were self-reported and were based on the personal clinical experience of each Hematologist.

RESULTS: A total of 19 Hematologists, from distinct geographical regions answered the web-based survey. On average, each Hematologist diagnosed 6 new patients in the last 12 months (average age: 69 years) and has currently 5 patients in-treatment. Approximately 61% of diagnosed patients stay in watch-and-wait over three years, and 55% are considered fit for intensive therapy when initiating treatment. 1L therapy consists essentially of immunochemotherapy, while only 6% are treated with targeted therapy. This percentage increases to 13% for unfit patients in 1L. On average, 40% of patients are eligible for 2L treatment, approximately 22 months after 1L, of which 30% are considered fit. In 2L, 24% and 26% of fit and unfit patients are treated with targeted therapy, respectively. Patients with WM are submitted to 2 lines of treatment, on average. The main identified unmet needs were related with cure, duration of therapeutic response, lower treatment toxicity, more effective therapeutic alternatives specially for older and unfit patients and exclusively oral therapies for 1L.

CONCLUSIONS: WM is a rare disease with scarce treatment pattern information in Portugal. This exploratory study showed that a low percentage of patients is treated with targeted therapy, with the lack of targeted therapeutic alternatives and treatment toxicity pointed as the main therapeutic unmet needs.