Research progress of Bruton’s tyrosine kinase inhibitors in the treatment of Waldenström macroglobulinemia_West China Medical Journal

Authors：

XIONG Yanqiu ,  ZHANG Li

Department of Hematology, West China Hospital, Sichuan University, Chengdu, Sichuan 610041, P. R. China;

Corresponding author：

ZHANG Li, Email: drzhangli2014@sina.com

Keywords：

Bruton’s tyrosine kinase inhibitors; Waldenström macroglobulinemia; treatment; progress

DOI：

10.7507/1002-0179.202008274

Video：

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Waldenström macroglobulinemia (WM) is a rare indolent lymphoplasmacytic lymphoma. Bruton’s tyrosine kinase (BTK) plays a key role in the signaling pathway of WM, which has changed the way of treatment of WM. As a first-generation BTK inhibitor, ibrutinib is an early-stage treatment and a salvage treatment that can control toxicity characteristics. However, in order to overcome the resistance of the first-generation BTK inhibitors, reduce the adverse reactions caused by off-target effects, and improve the efficacy, the research and development of new BTK inhibitors has become a hot topic. This article discusses the clinical studies of the first generation and new BTK inhibitors for WM, aiming to provide a certain basis for the more rational application of BTK inhibitors in WM.

Citation： XIONG Yanqiu, ZHANG Li. Research progress of Bruton’s tyrosine kinase inhibitors in the treatment of Waldenström macroglobulinemia. West China Medical Journal, 2022, 37(7): 1098-1102. doi: 10.7507/1002-0179.202008274 Copy

1.	Gertz MA. Waldenström macroglobulinemia: 2019 update on diagnosis, risk stratification, and management. Am J Hematol, 2019, 94(2): 266-276.
2.	Abeykoon JP, Yanamandra U, Kapoor P. New developments in the management of Waldenström macroglobulinemia. Cancer Manag Res, 2017, 9: 73-83.
3.	Olszewski AJ, Treon SP, Castillo JJ. Evolution of management and outcomes in waldenström macroglobulinemia: a population-based analysis. Oncologist, 2016, 21(11): 1377-1386.
4.	Castillo JJ, Olszewski AJ, Kanan S, et al. Overall survival and competing risks of death in patients with Waldenström macroglobulinaemia: an analysis of the surveillance, epidemiology and end results database. Br J Haematol, 2015, 169(1): 81-89.
5.	Leblond V, Kastritis E, Advani R, et al. Treatment recommendations from the Eighth International Workshop on Waldenström’s Macroglobulinemia. Blood, 2016, 128(10): 1321-1328.
6.	Buske C, Seymour JF. Immunochemotherapy in Waldenström macroglobulinemia - still the backbone of treatment. Leuk Lymphoma, 2015, 56(9): 2489-2490.
7.	de Weerdt I, Koopmans SM, Kater AP, et al. Incidence and management of toxicity associated with ibrutinib and idelalisib: a practical approach. Haematologica, 2017, 102(10): 1629-1639.
8.	Bradshaw JM. The Src, Syk, and Tec family kinases: distinct types of molecular switches. Cell Signal, 2010, 22(8): 1175-1184.
9.	da Cunha-Bang C, Niemann CU. Targeting Bruton’s tyrosine kinase across B-cell malignancies. Drugs, 2018, 78(16): 1653-1663.
10.	Seda V, Mraz M. B-cell receptor signalling and its crosstalk with other pathways in normal and malignant cells. Eur J Haematol, 2015, 94(3): 193-205.
11.	Yu X, Li W, Deng Q, et al. MYD88 L265P mutation in lymphoid malignancies. Cancer Res, 2018, 78(10): 2457-2462.
12.	Yang G, Buhrlage SJ, Tan L, et al. HCK is a survival determinant transactivated by mutated MYD88, and a direct target of ibrutinib. Blood, 2016, 127(25): 3237-3252.
13.	Castillo JJ, Palomba ML, Advani R, et al. Ibrutinib in Waldenström macroglobulinemia: latest evidence and clinical experience. Ther Adv Hematol, 2016, 7(4): 179-186.
14.	Treon SP, Xu L, Hunter Z. MYD88 mutations and response to ibrutinib in Waldenström’s macroglobulinemia. N Engl J Med, 2015, 373(6): 584-586.
15.	Smith MR. Ibrutinib in B lymphoid malignancies. Expert Opin Pharmacother, 2015, 16(12): 1879-1887.
16.	Advani RH, Buggy JJ, Sharman JP, et al. Bruton tyrosine kinase inhibitor ibrutinib (PCI-32765) has significant activity in patients with relapsed/refractory B-cell malignancies. J Clin Oncol, 2013, 31(1): 88-94.
17.	Treon SP, Gustine J, Meid K, et al. Ibrutinib monotherapy in symptomatic, treatment-naïve patients with Waldenström macroglobulinemia. J Clin Oncol, 2018, 36(27): 2755-2761.
18.	Treon SP, Tripsas CK, Meid K, et al. Ibrutinib in previously treated Waldenström’s macroglobulinemia. N Engl J Med, 2015, 372(15): 1430-1440.
19.	Treon SP, Meid K, Gustine J, et al. Long-term follow-up of ibrutinib monotherapy in symptomatic, previously treated patients with Waldenström macroglobulinemia. J Clin Oncol, 2021, 39(6): 565-575.
20.	Buske C, Tedeschi A, Trotman J, et al. Ibrutinib treatment in Waldenström’s macroglobulinemia: follow-up efficacy and safety from the iNNOVATETM study. Blood, 2018, 132(Suppl 1): 149.
21.	Dimopoulos MA, Tedeschi A, Trotman J, et al. Phase 3 trial of ibrutinib plus rituximab in Waldenström’s macroglobulinemia. N Engl J Med, 2018, 378(25): 2399-2410.
22.	Castillo JJ, Gustine JN, Keezer A, et al. Response and survival outcomes of Waldenstrom macroglobulinemia patients on ibrutinib therapy on and off clinical trial: PF492. HemaSphere, 2019, 3(S1): 198-199.
23.	Castillo JJ, Xu L, Gustine JN, et al. CXCR4 mutation subtypes impact response and survival outcomes in patients with Waldenström macroglobulinaemia treated with ibrutinib. Br J Haematol, 2019, 187(3): 356-363.
24.	Hunter ZR, Yang G, Xu L, et al. Genomics, signaling, and treatment of Waldenström macroglobulinemia. J Clin Oncol, 2017, 35(9): 994-1001.
25.	Cao Y, Hunter ZR, Liu X, et al. The WHIM-like CXCR4(S338X) somatic mutation activates AKT and ERK, and promotes resistance to ibrutinib and other agents used in the treatment of Waldenstrom’s macroglobulinemia. Leukemia, 2015, 29(1): 169-176.
26.	Xu L, Tsakmaklis N, Yang G, et al. Acquired mutations associated with ibrutinib resistance in Waldenström macroglobulinemia. Blood, 2017, 129(18): 2519-2525.
27.	Treon SP, Meid K, Hunter ZR, et al. Phase 1 study of ibrutinib and the CXCR4 antagonist ulocuplumab in CXCR4-mutated Waldenström macroglobulinemia. Blood, 2021, 138(17): 1535-1539.
28.	Lampson BL, Yu L, Glynn RJ, et al. Ventricular arrhythmias and sudden death in patients taking ibrutinib. Blood, 2017, 129(18): 2581-2584.
29.	Gustine JN, Meid K, Dubeau TE, et al. Atrial fibrillation associated with ibrutinib in Waldenström macroglobulinemia. Am J Hematol, 2016, 91(6): E312-E313.
30.	Byrd JC, Furman RR, Coutre SE, et al. Three-year follow-up of treatment-naïve and previously treated patients with CLL and SLL receiving single-agent ibrutinib. Blood, 2015, 125(16): 2497-2506.
31.	Wang M, Rule S, Zinzani PL, et al. Acalabrutinib in relapsed or refractory mantle cell lymphoma (ACE-LY-004): a single-arm, multicentre, phase 2 trial. Lancet, 2018, 391(10121): 659-667.
32.	Owen RG, McCarthy H, Rule S, et al. Acalabrutinib monotherapy in patients with Waldenström macroglobulinemia: a single-arm, multicentre, phase 2 study. Lancet Haematol, 2020, 7(2): e112-e121.
33.	AlphaMed Press. Zanubrutinib shows clinical benefits in Waldenström macroglobulinemia. Oncologist, 2020, 25(Suppl 1): S16-S17.
34.	Tam CS. Zanubrutinib: a novel BTK inhibitor in chronic lymphocytic leukemia and non-Hodgkin lymphoma. Clin Adv Hematol Oncol, 2019, 17(1): 32-34.
35.	Trotman J, Opat S, Gottlieb D, et al. Zanubrutinib for the treatment of patients with Waldenström macroglobulinemia: 3 years of follow-up. Blood, 2020, 136(18): 2027-2037.
36.	Tam CS, Opat S, D’Sa S, et al. A randomized phase 3 trial of zanubrutinib vs ibrutinib in symptomatic Waldenström macroglobulinemia: the ASPEN study. Blood, 2020, 136(18): 2038-2050.
37.	Tam CS, LeBlond V, Novotny W, et al. A head-to-head Phase Ⅲ study comparing zanubrutinib versus ibrutinib in patients with Waldenström macroglobulinemia. Future Oncol, 2018, 14(22): 2229-2237.
38.	Sekiguchi N, Rai S, Munakata W, et al. A multicenter, open-label, phase Ⅱ study of tirabrutinib (ONO/GS-4059) in patients with Waldenström’s macroglobulinemia. Cancer Sci, 2020, 111(9): 3327-3337.

1. Gertz MA. Waldenström macroglobulinemia: 2019 update on diagnosis, risk stratification, and management. Am J Hematol, 2019, 94(2): 266-276.
2. Abeykoon JP, Yanamandra U, Kapoor P. New developments in the management of Waldenström macroglobulinemia. Cancer Manag Res, 2017, 9: 73-83.
3. Olszewski AJ, Treon SP, Castillo JJ. Evolution of management and outcomes in waldenström macroglobulinemia: a population-based analysis. Oncologist, 2016, 21(11): 1377-1386.
4. Castillo JJ, Olszewski AJ, Kanan S, et al. Overall survival and competing risks of death in patients with Waldenström macroglobulinaemia: an analysis of the surveillance, epidemiology and end results database. Br J Haematol, 2015, 169(1): 81-89.
5. Leblond V, Kastritis E, Advani R, et al. Treatment recommendations from the Eighth International Workshop on Waldenström’s Macroglobulinemia. Blood, 2016, 128(10): 1321-1328.
6. Buske C, Seymour JF. Immunochemotherapy in Waldenström macroglobulinemia - still the backbone of treatment. Leuk Lymphoma, 2015, 56(9): 2489-2490.
7. de Weerdt I, Koopmans SM, Kater AP, et al. Incidence and management of toxicity associated with ibrutinib and idelalisib: a practical approach. Haematologica, 2017, 102(10): 1629-1639.
8. Bradshaw JM. The Src, Syk, and Tec family kinases: distinct types of molecular switches. Cell Signal, 2010, 22(8): 1175-1184.
9. da Cunha-Bang C, Niemann CU. Targeting Bruton’s tyrosine kinase across B-cell malignancies. Drugs, 2018, 78(16): 1653-1663.
10. Seda V, Mraz M. B-cell receptor signalling and its crosstalk with other pathways in normal and malignant cells. Eur J Haematol, 2015, 94(3): 193-205.
11. Yu X, Li W, Deng Q, et al. MYD88 L265P mutation in lymphoid malignancies. Cancer Res, 2018, 78(10): 2457-2462.
12. Yang G, Buhrlage SJ, Tan L, et al. HCK is a survival determinant transactivated by mutated MYD88, and a direct target of ibrutinib. Blood, 2016, 127(25): 3237-3252.
13. Castillo JJ, Palomba ML, Advani R, et al. Ibrutinib in Waldenström macroglobulinemia: latest evidence and clinical experience. Ther Adv Hematol, 2016, 7(4): 179-186.
14. Treon SP, Xu L, Hunter Z. MYD88 mutations and response to ibrutinib in Waldenström’s macroglobulinemia. N Engl J Med, 2015, 373(6): 584-586.
15. Smith MR. Ibrutinib in B lymphoid malignancies. Expert Opin Pharmacother, 2015, 16(12): 1879-1887.
16. Advani RH, Buggy JJ, Sharman JP, et al. Bruton tyrosine kinase inhibitor ibrutinib (PCI-32765) has significant activity in patients with relapsed/refractory B-cell malignancies. J Clin Oncol, 2013, 31(1): 88-94.
17. Treon SP, Gustine J, Meid K, et al. Ibrutinib monotherapy in symptomatic, treatment-naïve patients with Waldenström macroglobulinemia. J Clin Oncol, 2018, 36(27): 2755-2761.
18. Treon SP, Tripsas CK, Meid K, et al. Ibrutinib in previously treated Waldenström’s macroglobulinemia. N Engl J Med, 2015, 372(15): 1430-1440.
19. Treon SP, Meid K, Gustine J, et al. Long-term follow-up of ibrutinib monotherapy in symptomatic, previously treated patients with Waldenström macroglobulinemia. J Clin Oncol, 2021, 39(6): 565-575.
20. Buske C, Tedeschi A, Trotman J, et al. Ibrutinib treatment in Waldenström’s macroglobulinemia: follow-up efficacy and safety from the iNNOVATETM study. Blood, 2018, 132(Suppl 1): 149.
21. Dimopoulos MA, Tedeschi A, Trotman J, et al. Phase 3 trial of ibrutinib plus rituximab in Waldenström’s macroglobulinemia. N Engl J Med, 2018, 378(25): 2399-2410.
22. Castillo JJ, Gustine JN, Keezer A, et al. Response and survival outcomes of Waldenstrom macroglobulinemia patients on ibrutinib therapy on and off clinical trial: PF492. HemaSphere, 2019, 3(S1): 198-199.
23. Castillo JJ, Xu L, Gustine JN, et al. CXCR4 mutation subtypes impact response and survival outcomes in patients with Waldenström macroglobulinaemia treated with ibrutinib. Br J Haematol, 2019, 187(3): 356-363.
24. Hunter ZR, Yang G, Xu L, et al. Genomics, signaling, and treatment of Waldenström macroglobulinemia. J Clin Oncol, 2017, 35(9): 994-1001.
25. Cao Y, Hunter ZR, Liu X, et al. The WHIM-like CXCR4(S338X) somatic mutation activates AKT and ERK, and promotes resistance to ibrutinib and other agents used in the treatment of Waldenstrom’s macroglobulinemia. Leukemia, 2015, 29(1): 169-176.
26. Xu L, Tsakmaklis N, Yang G, et al. Acquired mutations associated with ibrutinib resistance in Waldenström macroglobulinemia. Blood, 2017, 129(18): 2519-2525.
27. Treon SP, Meid K, Hunter ZR, et al. Phase 1 study of ibrutinib and the CXCR4 antagonist ulocuplumab in CXCR4-mutated Waldenström macroglobulinemia. Blood, 2021, 138(17): 1535-1539.
28. Lampson BL, Yu L, Glynn RJ, et al. Ventricular arrhythmias and sudden death in patients taking ibrutinib. Blood, 2017, 129(18): 2581-2584.
29. Gustine JN, Meid K, Dubeau TE, et al. Atrial fibrillation associated with ibrutinib in Waldenström macroglobulinemia. Am J Hematol, 2016, 91(6): E312-E313.
30. Byrd JC, Furman RR, Coutre SE, et al. Three-year follow-up of treatment-naïve and previously treated patients with CLL and SLL receiving single-agent ibrutinib. Blood, 2015, 125(16): 2497-2506.
31. Wang M, Rule S, Zinzani PL, et al. Acalabrutinib in relapsed or refractory mantle cell lymphoma (ACE-LY-004): a single-arm, multicentre, phase 2 trial. Lancet, 2018, 391(10121): 659-667.
32. Owen RG, McCarthy H, Rule S, et al. Acalabrutinib monotherapy in patients with Waldenström macroglobulinemia: a single-arm, multicentre, phase 2 study. Lancet Haematol, 2020, 7(2): e112-e121.
33. AlphaMed Press. Zanubrutinib shows clinical benefits in Waldenström macroglobulinemia. Oncologist, 2020, 25(Suppl 1): S16-S17.
34. Tam CS. Zanubrutinib: a novel BTK inhibitor in chronic lymphocytic leukemia and non-Hodgkin lymphoma. Clin Adv Hematol Oncol, 2019, 17(1): 32-34.
35. Trotman J, Opat S, Gottlieb D, et al. Zanubrutinib for the treatment of patients with Waldenström macroglobulinemia: 3 years of follow-up. Blood, 2020, 136(18): 2027-2037.
36. Tam CS, Opat S, D’Sa S, et al. A randomized phase 3 trial of zanubrutinib vs ibrutinib in symptomatic Waldenström macroglobulinemia: the ASPEN study. Blood, 2020, 136(18): 2038-2050.
37. Tam CS, LeBlond V, Novotny W, et al. A head-to-head Phase Ⅲ study comparing zanubrutinib versus ibrutinib in patients with Waldenström macroglobulinemia. Future Oncol, 2018, 14(22): 2229-2237.
38. Sekiguchi N, Rai S, Munakata W, et al. A multicenter, open-label, phase Ⅱ study of tirabrutinib (ONO/GS-4059) in patients with Waldenström’s macroglobulinemia. Cancer Sci, 2020, 111(9): 3327-3337.

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