Long-term outcomes and treatment patterns in Waldenström macroglobulinemia patients who discontinue Bruton tyrosine kinase inhibitor (BTKi) therapy

This is the authors' abstract. We don't add key points for this paper.

Since receiving regulatory approval in 2015, oral Bruton tyrosine kinase (BTK) inhibitors have revolutionized the treatment of Waldenström macroglobulinemia (WM) in both the frontline and relapsed/refractory settings [ 1 , 2 ]. Covalent BTK inhibitors (BTKis), including ibrutinib, acalabrutinib, and zanubrutinib, have demonstrated robust response rates and prolonged progression-free survival, especially for patients with MYD88 MUT WM [ 3 ]. However, BTKi treatment duration can be limited by adverse events (AEs), such as bleeding and cardiac toxicities, as well as drug resistance [ 4 , 5 ]. Although long-term follow-up has been presented from some trials, there is a dearth of real-world data examining the outcomes of patients post-BTKi therapy [ 6 , 7 ]. The objectives of this study were to evaluate the outcomes of patients with WM who previously received BTKi therapy (BTKi-discontinued), looking at their response to, and duration of, therapy, reasons for BTKi discontinuation, next-line treatment patterns, and associated outcomes. Furthermore, we sought to compare their outcomes with those of patients who remained on BTKi therapy (BTKi-active) at the time of data cutoff.