WALDENSTRÖM MACROGLOBULINEMIA - A STATE-OF-THE-ART REVIEW: PART 2- FOCUS ON THERAPY
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Abstract: The diagnosis and treatment of Waldenstrom macroglobulinemia (WM) are the subjects of this two-part review, which aims to provide current and thorough knowledge of these topics. The first portion of the study, previously published, investigated the epidemiology, etiology, clinicopathological aspects, differential diagnosis, prognostic factors, and impact on WM-specific groups. Specifically, this second section examines both the standard consolidated method and the new therapeutic strategy to handle the complex topic of the treatment of WM.
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References
REFERENCES:
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1. Kumar SK, Callander NS, Adekola K, Anderson LD Jr, Baljevic M, Baz R, Campagnaro E, Castillo JJ, Costello C, D'Angelo C, Derman B, Devarakonda S, Elsedawy N, Garfall A, Godby K, Hillengass J, Holmberg L, Htut M, Huff CA, Hultcrantz M, Kang Y, Larson S, Lee H, Liedtke M, Martin T, Omel J, Robinson T, Rosenberg A, Sborov D, Schroeder MA, Sherbenou D, Suvannasankha A, Valent J, Varshavsky-Yanovsky AN, Snedeker J, Kumar R. Waldenström Macroglobulinemia/Lymphoplasmacytic Lymphoma, Version 2.2024, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2024 Jan;22(1D):e240001. doi: 10.6004/jnccn.2024.0001. PMID: 38244272.
2. Bibas M, Sarosiek S, Castillo JJ. Waldenström Macroglobulinemia - A State-of-the-Art Review: Part 1: Epidemiology, Pathogenesis, Clinicopathologic Characteristics, Differential Diagnosis, Risk Stratification, and Clinical Problems. Mediterr J Hematol Infect Dis. 2024 Jul 1;16(1):e2024061. doi: 10.4084/MJHID.2024.061. PMID: 38984103; PMCID: PMC11232678.
3. Castillo JJ, Olszewski AJ, Cronin AM, et al. Survival trends in Waldenstrom macroglobulinemia: an analysis of the Surveillance, Epidemiology and End Results database. Blood 2014;123:3999–4000.
4.Kyle RA, Benson JT, Larson DR, et al. Progression in smoldering Waldenstrom macroglobulinemia: long-term results. Blood 2012;119:4462–4466.
5.Kyle RA, Larson DR, Therneau TM, et al. Long-term follow-up of monoclonal gammopathy of undetermined significance. N Engl J Med 2018;378:241–249.
6.Kastritis E, Morel P, Duhamel A, Gavriatopoulou M, Kyrtsonis MC, Durot E, Symeonidis A, Laribi K, Hatjiharissi E, Ysebaert L, Vassou A, Giannakoulas N, Merlini G, Repousis P, Varettoni M, Michalis E, Hivert B, Michail M, Katodritou E, Terpos E, Leblond V, Dimopoulos MA. A revised international prognostic score system for Waldenström's macroglobulinemia. Leukemia. 2019 Nov;33(11):2654-2661. doi: 10.1038/s41375-019-0431-y. Epub 2019 May 22. PMID: 31118465.
7.Bustoros M, Sklavenitis-Pistofidis R, Kapoor P, et al. Progression risk stratification of asymptomatic Waldenstroom macroglobulinemia. J Clin Oncol 2019;37:1403–1411.
8.Kastritis E, Leblond V, Dimopoulos MA, et al. Waldenstroom’s macroglobulinaemia:ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol 2019;30:860–862.
9.Castillo JJ, Advani RH, Branagan AR, et al. Consensus treatment recommendations from the tenth International Workshop for Waldenstroom Macroglobulinaemia. Lancet Haematol 2020;7:e827–837.
10.Pratt G, El-Sharkawi D, Kothari J, et al. Diagnosis and management of Waldenstroom macroglobulinaemia—a British Society for Haematology guideline. Br J Haematol 2022;197:171–187.
11.Grunenberg A, Buske C. How to manage waldenström's macroglobulinemia in 2024. Cancer Treat Rev. 2024 Apr;125:102715. doi: 10.1016/j.ctrv.2024.102715. Epub 2024 Mar 5. PMID: 38471356.
12.Ghafoor B, Masthan SS, Hameed M, Akhtar HH, Khalid A, Ghafoor S, Allah HM, Arshad MM, Iqbal I, Iftikhar A, Husnain M, Anwer F. Waldenström macroglobulinemia: a review of pathogenesis, current treatment, and future prospects. Ann Hematol. 2024 Jun;103(6):1859-1876. doi: 10.1007/s00277-023-05345-9. Epub 2023 Jul 6. PMID: 37414960.
13.Treon SP, Cao Y, Xu L, et al. Somatic mutations in MYD88 and CXCR4 are determinants of clinical presentation and overall survival in Waldenstrom macroglobulinemia. Blood 2014;123:2791–2796.
14.Treon SP, Tripsas CK, Meid K, et al. Ibrutinib in previously treated Waldenström’s macroglobulinemia. N Engl J Med 2015;372:1430–1440.
15. Treon SP, Gustine J, Meid K, et al: Ibrutinib monotherapy in symptomatic, treatment-naıve patients with Waldenstroom macroglobulinemia. J Clin Oncol 36: 2755-2761, 2018
16. Castillo JJ, Meid K, Gustine JN, et al. Long-term follow-up of ibrutinib monotherapy in treatment-naive patients with Waldenstrom macroglobulinemia. Leukemia 2022;36:532–539.
17. Sarosiek S, Gustine JN, Flynn CA, et al. Dose reductions in patients with Waldenström macroglobulinaemia treated with ibrutinib. Br J Haematol 2023;201:897–904.
18. Shadman M, Flinn IW, Levy MY, et al. Zanubrutinib in patients with previously treated B-cell malignancies intolerant of previous Bruton tyrosine kinase inhibitors in the USA: a phase 2, open-label, single-arm study. Lancet Haematol 2023;10:e35–45.
19. Buske C, Tedeschi A, Trotman J, et al. Ibrutinib Plus rituximab versus placebo plus rituximab for Waldenstr€om’s macroglobulinemia: final analysis from the randomized phase III iNNOVATE study. J Clin Oncol 2022;40:52–62.
20. Castillo JJ, Meid K, Gustine JN, et al. Long-term follow-up of ibrutinib monotherapy in treatment-naive patients with Waldenstrom macroglobulinemia. Leukemia 2022;36:532–539.
21. Dimopoulos MA, Tedeschi A, Trotman J, et al. Phase 3 trial of ibrutinib plus rituximab in Waldenstroom’s macroglobulinemia. N Engl J Med 2018;378:2399–2410.
22. Tam CS, Opat S, D’Sa S, et al. A randomized phase 3 trial of zanubrutinib vs ibrutinib in symptomatic Waldenstroom macroglobulinemia: the ASPEN study. Blood 2020;136:2038–2050.
23. Dimopoulos MA, Opat S, D’Sa S, et al. Zanubrutinib versus ibrutinib in symptomatic Waldenstroom macroglobulinemia: final analysis from the randomized phase III ASPEN study. J Clin Oncol 2023;41:5099–5106
24. Rummel MJ, Niederle N, Maschmeyer G, et al. Bendamustine plus rituximab versus CHOP plus rituximab as first-line treatment for patients with indolent and mantle-cell lymphomas: an open-label, multicentre, randomised, phase 3 non-inferiority trial. Lancet 2013;381:1203–1210.
25. Greil R, Wupperfeld J, Hinke A, et al. Two years rituximab maintenance vs. observation after first line treatment with bendamustine plus rituximab (B-R) in patients with Waldenstr€om’s Macroglobulinemia (MW): Results of a Prospective, Randomized, Multicenter Phase 3 Study (the StiL NHL7- 2008 MAINTAIN trial). Blood 2019;134(Suppl 1):Abstract 343.
26. Treon SP, Ioakimidis L, Soumerai JD, et al. Primary therapy of Waldenstroom macroglobulinemia with bortezomib, dexamethasone, and rituximab: WMCTG clinical trial 05-180. J Clin Oncol 2009;27:3830–3835.
27. Dimopoulos MA, Garcıa-Sanz R, Gavriatopoulou M, et al. Primary therapy of Waldenstrom macroglobulinemia (WM) with weekly bortezomib,low-dose dexamethasone, and rituximab (BDR): long-term results of a phase 2 study of the European Myeloma Network (EMN). Blood 2013;122:3276–3282.
28. Treon SP, Tripsas CK, Meid K, et al. Carfilzomib, rituximab, and dexamethasone (CaRD) treatment offers a neuropathy-sparing approach for treating Waldenstr€om’s macroglobulinemia. Blood 2014;124:503–510.
29. Meid K, Dubeau T, Severns P, et al. Long-term follow-up of a prospective clinical trial of carfilzomib, rituximab and dexamethasone (CaRD) in Waldenstrom’s macroglobulinemia. Blood 2017;130(Suppl 1):Abstract 623.
30. Castillo JJ, Meid K, Gustine JN, et al. Prospective clinical trial of ixazomib, dexamethasone, and rituximab as primary therapy in Waldenstr€om macroglobulinemia. Clin Cancer Res 2018;24:3247–3252.
31. Castillo JJ, Meid K, Flynn CA, et al. Ixazomib, dexamethasone, and rituximab in treatment-naive patients with Waldenstrom macroglobulinemia:long-term follow-up. Blood Adv 2020;4:3952–3959.
32. Dimopoulos MA, Anagnostopoulos A, Kyrtsonis MC, et al. Primary treatment of Waldenstr€om macroglobulinemia with dexamethasone, rituximab, and cyclophosphamide. J Clin Oncol 2007;25:3344–3349.
33. Kastritis E, Gavriatopoulou M, Kyrtsonis MC, et al. Dexamethasone, rituximab, and cyclophosphamide as primary treatment of Waldenstrom macroglobulinemia: final analysis of a phase 2 study. Blood 2015;126:1392–1394.
34. Paludo J, Abeykoon JP, Kumar S, et al. Dexamethasone, rituximab and cyclophosphamide for relapsed and/or refractory and treatment-naïve patients with Waldenstrom macroglobulinemia. Br J Haematol 2017;179:98–105.
35. Ioakimidis L, Patterson CJ, Hunter ZR, et al. Comparative outcomes following CP-R, CVP-R, and CHOP-R in Waldenstroom’s macroglobulinemia. Clin Lymphoma Myeloma 2009;9:62–66.
36. Treon SP. How I treat Waldenstr€om macroglobulinemia. Blood 2015; 126:721–732
37. Ghobrial IM, Fonseca R, Greipp PR, et al. Initial immunoglobulin M ‘flare’ after rituximab therapy in patients diagnosed with Waldenstrom macroglobulinemia: an Eastern Cooperative Oncology Group Study.Cancer 2004;101:2593–2598.
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Feb 27, 2025
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Bibas, M., Sarosiek , S. and Castillo, J. (2025) “WALDENSTRÖM MACROGLOBULINEMIA - A STATE-OF-THE-ART REVIEW: PART 2- FOCUS ON THERAPY”, Mediterranean Journal of Hematology and Infectious Diseases, 17(1), p. e2025015. doi: 10.4084/MJHID.2025.015.
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