Bruton's tyrosine kinase inhibitors (BTKi) demonstrated efficacy in multiple hematologic malignancies; however, limited RW evidence exists on treatment patterns and outcomes in patients (pts) treated with these drugs in middle and lower income countries. This study assessed characteristics, treatment patterns, and the economic impact of pts treated with BTKi as part of routine clinical care in a large Brazilian private healthcare database (728.245 lives).

The objective of the study is to better characterize, from a clinical and economic point of view, based on real-life data, the current usage of Bruton's tyrosine kinase inhibitors in the Brazilian supplementary health system.

This retrospective, multisite, cohort study included pts with a diagnosis of any hematologic neoplasms who were initially prescribed BTKi on or after 01/01/22. Eligible pts were ≥ 18 years at the time of prescription and had ≥ 3 months (mo) of follow-up (unless < 3 mo due to death). All clinical data was extracted from pre-authorization files and medical records of eligible patients into electronic case report forms. Economic impact was calculated from real pricing of the drugs acquired for the treatment of the eligible patients. Results were summarized using descriptive statistics.

Data from 71 pts (65% male; 35% female; median age at BTKi prescription =72 years [range 41 - 86 yrs]) were collected from 17 different practices. Chronic Lymphocytic Leukemia was the most common indication (56%), followed by Mantle Cell Lymphoma (41%) and Waldenstrom Macroglobulinemia (3%). Across all pts, 35 (49,2%) were initially prescribed Ibrutinib, 29 (40.8%) Acalbrutinib and 7 (9%) Zanubrutinib respectively. Most patients had a BTKi prescribed for recurrent or refractory disease (62%) and were previously treated with chemo/immunotherapy. The median time on treatment for all the cohort was 12 months and the main reasons for discontinuation were death or disease progression, although 6 pts (6/35 - 17%) initially treated with Ibrutinib and 1 pt (1/7 - 14%) treated with Zanubrutinib switched to another agent due to major toxicity. The estimated total cost of BTKi use in the cohort was R$ 49.417.112 (USD 9.079.702) and the mean cost of treatment per patient was estimated at R$ 696.015 (USD 127.883). Ibruinitib had a significantly higher cost per patient than Acalabrutinib or Zanubrutinib (R$ 950.366 × R$ 450.953 × R$ 439.520).

Our study provides important RW evidence for BTKi usage in a resource limited setting. Further actions can be derived from the data found in order to improve outcomes, quality of care and limit the economic impact of hematologic neoplasms in lower and middle income countries such as Brazil.