Khwaja, Jahanzaib;
Uppal, Encarl;
Baker, Robert;
Trivedi, Kajal;
Rismani, Ali;
Gupta, Rajeev;
Proctor, Ian;
... D'Sa, Shirley; + view all
(2022)
Bortezomib‐based therapy is effective and well tolerated in frontline and multiply pre‐treated Waldenström macroglobulinaemia including BTKi failures: A real‐world analysis.
eJHaem
10.1002/jha2.597.
(In press).
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Abstract
Waldenström macroglobulinemia (WM) is a rare, incurable low grade lymphoma following a relapsing trajectory. Management strategies have evolved with the introduction of targeted therapy including new classes of Bruton tyrosine kinase inhibitor (BTKi). Treatment may however be limited particularly at relapse by a lack of drug availability and tolerability. We assessed the real-world efficacy and tolerability of bortezomib-containing regimens in patients with WM at frontline and relapse including those with prior BTKi resistance. Forty-one patients were identified with 44 bortezomib-containing regimens administered (n = 12 frontline, n = 32 relapse). Of patients treated at relapse, the median prior lines of therapy was 3 (range 1–7). 24% (10/41) of the cohort were refractory or intolerant to BTKi prior to bortezomib delivery. The median follow-up after bortezomib administration was 34 months (range 0-131). Overall response rate was 88%; 2-year overall survival and progression-free survival were 90% (95% confidence interval [CI] 73–96) and 76% (95% CI 55–87), respectively. Median time-to-next-treatment was 66 months. Neuropathy (grade 1–2) occurred in 24% (8/34) and did not result in treatment cessation in any case. Gastrointestinal disturbance occurred in 7% (3/41). Treatment discontinuations were rare (1/44; 2%), suggesting a manageable safety profile. Major response rate was comparable in those with prior BTKi compared with those without (75% [6/8] vs 84% [27/32], p = 0.61). Bortezomib should be considered as a treatment modality particularly in those who are refractory to BTKi.
| Type: | Article |
|---|---|
| Title: | Bortezomib‐based therapy is effective and well tolerated in frontline and multiply pre‐treated Waldenström macroglobulinaemia including BTKi failures: A real‐world analysis |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1002/jha2.597 |
| Publisher version: | https://doi.org/10.1002/jha2.597 |
| Language: | English |
| Additional information: | © 2022 The Authors. eJHaem published by British Society for Haematology and John Wiley & Sons Ltd. This is an open access article under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/). |
| Keywords: | bortezomib, BTKi, Waldenström macroglobulinaemia, lymphoplasmacytic lymphoma |
| UCL classification: | UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10157920 |
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