761
Views
11
CrossRef citations to date
0
Altmetric
Review

Current treatment options and investigational drugs for Waldenstrom’s Macroglobulinemia

, , &
Pages 197-205 | Received 15 Jul 2016, Accepted 19 Dec 2016, Published online: 03 Jan 2017

Bibliography

  • Swerdlow SH, Campo E, Harris NL, et al. WHO classification of tumours of haematopoietic and lymphoid tissues. Lyon, France: IARC Press; 2008.
  • Leblebjian H, Agarwal A, Ghobrial i. Novel treatment options for Waldenström macroglobulinemia. Clin Lymphoma Myeloma Leuk. 2013;13(Suppl 2):S310–S316.
  • Gertz MA. Waldenstrom macroglobulinemia: 2013 update on diagnosis, risk stratification, and management. Am J Hematol. 2013;88:703–711.
  • Ghobrial IM, Witzig TE. Waldenstrom macroglobulinemia. Curr Treat Options Oncol. 2004;5(3):239–247.
  • Treon SP. How I treat Waldenstrom macroglobulinemia. Blood. 2009;114(12):2375–2385.
  • Dimopoulos MA,Kyle RA, Anagnostopoulos A, et al. Diagnosis and management of Waldenstrom’s macroglobulinemia. J Clin Oncol. 2005;23(7):1564–1577.
  • Jemal A, Murray T, Ward E, et al. Cancer statistics. CA Cancer J Clin. 2005;55(1):10–30.
  • Buske C, Leblond V. How to manage Waldenstrom’s macroglobulinemia. Leukemia. 2013;27:762–772.
  • Leblond V, Kastritis E, Advani R, et al. Treatment recommendations from the eighth international workshop on Waldenström’s macroglobulinemia. Blood. 2016 Sep 8;128(10):1321–1328. DOI:10.1182/blood-2016-04-711234. Epub 2016 Jul 18.
  • 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.
  • Garcia-Sanz R, Montoto S, Torrequebrada A, et al. Waldenstrom macroglobulinaemia: presenting features and outcome in a series with 217 cases. Br J Haematol. 2001;115:575–582.
  • NCCN Clinical Practice Guidelines in Oncology. Waldenstrom’s macroglobulinemia/lymphoplasmacytic lymphoma, version 2; 2015 [cited 2014 Oct 17]. Available from: http://www.nccn.org/professionals/physician_gls/pdf/waldenstroms.pdf
  • Vijay A, Gertz MA. Waldenstrom macroglobulinemia. Blood. 2007;109(12):5096–5103.
  • Ghobrial IM, Fonseca R, Gertz MA, et al. Prognostic model for disease-specific and overall mortality in newly diagnosed symptomatic patients with Waldenstrom macroglobulinaemia. Br J Haematol. 2006;133(2):158–164.
  • Gobbi PG, Baldini L, Broglia C, et al. Prognostic validation of the international classification of immunoglobulin M gammopathies: a survival advantage for patients with immunoglobulin M monoclonal gammopathy of undetermined significance. Clin Cancer Res. 2005;11:1786–1790.
  • Alexanian R, Weber D, Delasalle K, et al. Asymptomatic Waldenstrom’s macroglobulinemia. Semin Oncol. 2003;30:206–210.
  • Ansell SM, Kyle RA, Reeder CB, et al. Diagnosis and management of Waldenstrom macroglobulinemia: mayo stratification of macroglobulinemia and risk-adapted therapy (mSMART) guidelines. Mayo Clin Proc. 2010;85:824–833.
  • Treon SP, Xu L, Yang G, et al. MYD88 L265P somatic mutation in Waldenstrom’s macroglobulinemia. N Engl J Med. 2012;367:826–833.
  • 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.
  • Kyle RA, Therneau TM, Rajkumar SV, et al. Long-term follow-up of IgM monoclonal gammopathy of undetermined significance. Blood. 2003;102(10):3759–3764.
  • Treon SP, Hunter ZR, Aggarwal A, et al. Characterization of familial Waldenstrom’s macroglobulinemia. Ann Oncol. 2006;17:488–494.
  • Kristinsson SY, Bjorkholm M, Goldin LR, et al. Risk of lymphoproliferative disorders among first-degree relatives of lymphoplasmacytic lymphoma/Waldenström macroglobulinemia patients: a population-based study in Sweden. Blood. 2008;112:3052–3056.
  • RITUXAN® (rituximab). US Prescribing Information. Biogen Idec, Inc. and Genentech, Inc; 2014 Aug.
  • Dimopoulos MA, Zervas C, Zomas A, et al. Extended rituximab therapy for previously untreated patients with Waldenström’s macroglobulinemia. Clin Lymphoma. 2002;3:163–166.
  • Dimopoulos MA, Anagnostopoulos A, Kyrtsonis MC, et al. Primary treatment of Waldenström macroglobulinemia with dexamethasone, rituximab, and cyclophosphamide. J Clin Oncol. 2007;25:3344–3349.
  • Sp T, Hanzis C, Rj M, et al. Maintenance rituximab is associated with improved clinical outcome in rituximab naïve patients with Waldenstrom macroglobulinaemia who respond to a rituximab-containing regimen. Br J Haematol. 2011;154:357–362.
  • Dimopoulos MA, Roussou M, Kastritis E, et al. Primary treatment of Waldenstrom’s macroglobulinemia with dexamethasone, rituximab and cyclophosphamide (DRC): final analysis of a phase II study. Blood ASH Annu Meet Abstr. 2012;12021:Abstract 438. Atlanta, Georgia.
  • Rummel MJ, Von Gruenhagen U, Niederle N et al. Bendamustine plus rituximab versus CHOP plus rituximab in the first-line treatment of patients with Waldenstrom’s macroglobulinemia: first interim results of a randomized phase III study of the Study Group Indolent Lymphomas (StiL) [abstract]. Presented at the 5th International Workshop on Waldenstrom’s macroglobulinemia; 2008 Oct 15–18; Stockholm, Sweden. Abstract 139.
  • Treon SP, Hanzis C, Tripsas C, et al. Bendamustine therapy in patients with relapsed or refractory Waldenstrom’s macroglobulinemia. Clin Lymphoma Myeloma Leuk. 2011;11:133–135.
  • Rummel MJ, Al-Batran SE, Kim SZ, et al. Bendamustine plus rituximab is effective and has a favorable toxicity profile in the treatment of mantle cell and low-grade non-Hodgkin’s lymphoma. J Clin Oncol. 2005;23:3383–3389.
  • Buske C, Hoster E, Dreyling M, et al. The addition of rituximab to front-line therapy with CHOP (R-CHOP) results in a higher response rate and longer time to treatment failure in patients with lymphoplasmacytic lymphoma: results of a randomized trial of the German Low-Grade Lymphoma Study Group (GLSG). Leukemia. 2009;23:153–161.
  • Gupta IV, Jewell RC. Ofatumumab, the first human anti-CD20 monoclonal antibody for the treatment of B cell hematologic malignancies. Ann N Y Acad Sci. 2012;1263:43–56.
  • Furman RR, Eradat H, DiRenzo CG, et al. A phase II trial of ofatumumab in subjects with Waldenstrom’s macroglobulinemia. Blood (ASH Annu Meet Abstr). 2011;118:Abstract 3701. San Diego, California.
  • CAMPATH® (alemtuzumab). US Prescribing Information. Genzyme Corporation; 2009
  • Treon SP, Kelliher A, Keele B, et al. Expression of serotherapy target antigens in Waldenstrom’s macroglobulinemia: therapeutic applications and considerations. Semin Oncol. 2003;30:248–252.
  • Treon SP, Soumerai JD, Hunter ZR, et al. Long-term follow-up of symptomatic patients with lymphoplasmacytic lymphoma/Waldenstrom macroglobulinemia treated with the anti-CD52 monoclonal antibody alemtuzumab. Blood. 2011;118:276–281.
  • Panwalkar A, Verstovsek S. Giles F Nuclear factor-kappaB modulation as a therapeutic approach in hematologic malignancies. Cancer. 2004;100:1578–1589.
  • VELCADE® (bortezomib). US Prescribing Information. Millenium Pharmaceuticals, Inc; 2014 Oct
  • Roccaro AM, Leleu X, Sacco A, et al. Dual targeting of the proteasome regulates survival and homing in Waldenstrom macroglobulinemia. Blood. 2008;111:4752–4763.
  • Dimopoulos MA, Garcia-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.
  • Treon SP, Ioakimidis L, Soumerai JD, et al. Primary therapy of Waldenstrom macroglobulinemia with bortezomib, dexamethasone, and rituximab: WMCTG clinical trial 05-180. J Clin Oncol. 2009 Aug 10;27:3830–3835.
  • Palumbo A, Bringhen S, Rossi D, et al. Overall survival benefit for bortezomib-melphalan-prednisone-thalidomide followed by maintenance with bortezomib-thalidomide (VMPT-VT) versus bortezomib-melphalan-prednisone (VMP) in newly diagnosed multiple myeloma patients. ASH Annu Meet Abstr. 2012;120:200. Atlanta, Georgia.
  • Mateos MV, Oriol A, Martinez-Lopez J. Bortezomib/melphalan/prednisone (VMP) versus Bortezomib/Thalidomide/Prednisone (VTP) as induction therapy followed by maintenance treatment with Bortezomib/Thalidomide (VT) versus Bortezomib/Prednisone (VP): A randomized trial in elderly untreated patients with multiple myeloma older than 65 years. Lancet Oncol. 2010;11:934–941.
  • Moreau P, Pylypenko H, Grosicki S, et al. Subcutaneous versus intravenous administration of bortezomib in patients with relapsed multiple myeloma: a randomized, phase 3, non-inferiority study. Lancet Oncol. 2011;12:431–440.
  • Ghobrial IM, Hong F, Padmanabhan S, et al. Phase II trial of weekly bortezomib in combination with rituximab in relapsed or relapsed and refractory Waldenstrom macroglobulinemia. J Clin Oncol. 2010;28:1422–1428.
  • Dimopoulos MA, Gertz MA, Kastritis E, et al. Update on treatment recommendations from the fourth international workshop on Waldenstrom’s macroglobulinemia. J Clin Oncol. 2009;27:120–126.
  • KYPROLIS™ (carfilzomib).US Prescribing Information. Onyx Pharmaceuticals, Inc; 2012 July
  • Treon SP, Tripsas CK, Meid K, et al. Carfilzomib, rituximab, and dexamethasone (CaRD) treatment offers a neuropathy-sparing approach for treating Waldenström’s macroglobulinemia. Blood. 2014;124:503–510.
  • Siegel DS, Kaufman JL, Raje NS, et al. Updated results from a multicenter, open-label, dose-escalation phase 1b/2 study of single-agent oprozomib in patients with Waldenström macroglobulinemia (WM). Blood. 2014;124:1715.
  • Dimopoulos MA, Zomas A, Viniou NA, et al. Treatment of Waldenstrom’s macroglobulinemia with thalidomide. J Clin Oncol. 2001;19:3596–3601.
  • Treon SP, Soumeral JD, Branagan AR, et al. Thalidomide and rituximab in Waldenstrom macroglobulinemia. Blood. 2008;112:4452–4457.
  • Treon SP, Soumerai JD, Branagan AR, et al. Lenalidomide and rituximab in Waldenstrom’s macroglobulinemia. Clin Cancer Res. 2009;15:355–360.
  • Fouquet G, Guidez S, Petillon MO, et al. Lenalidomide is safe and active in Waldenstrom macroglobulinemia. Am J Hematol. 2015 Nov;90(11):1055–1059. DOI:10.1002/ajh.24175. Epub 2015 Oct 6.
  • Leleu X, Jia X, Runnels J, et al. The Akt pathway regulates survival and homing in Waldenstrom macroglobulinemia. Blood. 2007;110:4417–4426.
  • Vesole S, Jia X, Roccaro A, et al. RAD001 exerts anti-tumor activity in Waldenstrom macroglobulinemia [abstract]. Blood ASH Annu Meet Abstr. 2009;114:abstract 3732. New Orleans, Louisiana.
  • Treon SP, Tripsas CK, Meid K, et al. Prospective, multicenter study of the mTor inhibitor everolimus (RAD001) as primary therapy in Waldenstrom’s macroglobulinemia. Blood. 2013;122:Abstract 1822. New Orleans, Louisiana.
  • Ghobrial IM, Witzig TE, Gertz M, et al. Long-term results of the phase II trial of the oral mTOR inhibitor everolimus (RAD001) in relapsed or refractory Waldenstrom macroglobulinemia. Am J Hematol. 2014;89:237–242.
  • Ghobrial IM, Boswell EN, Banwait R, et al. Phase I trial of everolimus and rituximab or everolimus, bortezomib and rituximab in relapsed or relapsed/refractory Waldenstrom’s macroglobulinemia [abstract]. Blood ASH Annu Meet Abstr. 2011;118:abstract 2705. San Diego, California.
  • Kastritis E, Terpos E. Dimopoulos MA emerging drugs for Waldenström’s macroglobulinemia. Expert Opin Emerg Drugs. 2011;16:45–57.
  • Ghobrial IM, Roccaro A, Hong F, et al. Clinical and translational studies of a phase II trial of the novel oral Akt inhibitor perifosine in relapsed or relapsed/refractory Waldenstrom’s macroglobulinemia. Clin Cancer Res. 2010;16:1033–1041.
  • Ghobrial IM, Siegel D, Vij R, et al. MLN0128 (INK128), an investigational oral dual TORC1/2 inhibitor, in patients (pts) with relapsed or refractory multiple myeloma (MM), non-Hodgkin’s lymphoma (NHL), or Waldenstrom macroglobulinemia (WM): preliminary results from a phase 1 Dose-Escalation Study [abstract]. Blood ASH Annu Meet Abstr. 2012;120:abstract 4038. Atlanta, Georgia.
  • Ghobrial IM, Moreau P, Harris B, et al. A multicenter phase II study of single-agent enzastaurin in previously treated Waldenstrom macroglobulinemia. Clin Cancer Res. 2012;18(18):5043–5050.
  • Kahl BS, Byrd J, Flinn IW, et al. Clinical Safety and Activity in a Phase 1 Study of CAL-101, An Isoform-Selective Inhibitor of Phosphatidylinositol 3-Kinase P110δ, In Patients with Relapsed or Refractory Non-Hodgkin Lymphoma [abstract]. Blood ASH Annu Meet Abstr. 2010;116:abstract 1777. Orlando, Florida.
  • Yang G, Zhou Y, Liu X, et al. A mutation in MYD88 (L265P) supports the survival of lymphoplasmacytic cells by activation of Bruton tyrosine kinase in Waldenström macroglobulinemia. Blood. 2013;122:1222–1232.
  • Treon SP, Tripsas CK, Meid K, et al. Ibrutinib in previously treated Waldenström’s macroglobulinemia. N Engl J Med. 2015;372:1430–1440.
  • IMBRUVICA™ (ibrutinib). US Prescribing Information. Pharmacyclics, Inc; 2015 Jan
  • AbbVie. IMBRUVICA® (ibrutinib) now approved to treat Waldenstrom’s macroglobulinemia in Europe; 2015 July 10 [cited 2015 Oct 10]. Available from: http://abbvie.mediaroom.com/2015-07-10-IMBRUVICA-ibrutinib-Now-Approved-to-Treat-Waldenstroms-Macroglobulinemia-in-Europe
  • Roccaro AM, Sacco A, Jia X, et al. microRNA-dependent modulation of histone acetylation in Waldenstrom macroglobulinemia. Blood. 2010;116:1506–1514.
  • Grant S. Targeting Waldenstrom macroglobulinemia with histone deacetylase inhibitors. Leuk Lymphoma. 2011;52(9):1623–1625.
  • Ghobrial IM, Campigotto F, Murphy TJ, et al. Results of a phase 2 trial of the single-agent histone deacetylase inhibitor panobinostat in patients with relapsed/refractory Waldenström macroglobulinemia. Blood. 2013;121:1296–1303.
  • Ghobrial IM, Poon T, Rourke M, et al. Phase II Trial of Single Agent Panobinostat (LBH589) In relapsed or relapsed/refractory Waldenstrom macroglobulinemia [abstract]. Blood (ASH Annu Meet Abstr). 2010;116:abstract 3952. Orlando, Florida.
  • Tamburini J, Levy V, Chaleteix C, et al. Fludarabine plus cyclophosphamide in Waldenstrom’s macroglobulinemia: results in 49 patients. Leukemia. 2005;19:1831–1834.
  • Laurencet FM, Zulian GB, Guetty-Alberto M, et al. Cladribine with cyclophosphamide and prednisone in the management of low-grade lymphoproliferative malignancies. Br J Cancer. 1999;79:1215–1219.
  • Van Den Neste E, Louviaux I, Michaux JL, et al. Phase I/II study of 2-chloro-2ʹ-deoxyadenosine with cyclophosphamide in patients with pretreated B cell chronic lymphocytic leukemia and indolent non-Hodgkin’s lymphoma. Leukemia. 2000;14:1136–1142.
  • Treon SP, Branagan AR, Ioakimidis L, et al. Long-term outcomes to fludarabine and rituximab in Waldenstrom macroglobulinemia. Blood. 2009;113:3673–3678.
  • Tam CS, Wolf M, Prince HM, et al. Fludarabine, cyclophosphamide, and rituximab for the treatment of patients with chronic lymphocytic leukemia or indolent non-Hodgkin lymphoma. Cancer. 2006;106:2412–2420.
  • Leblond V, Johnson S, Chevret S, et al. Results of a randomized trial of chlorambucil versus fludarabine for patients with untreated Waldenstrom macroglobulinemia, marginal zone lymphoma, or lymphoplasmacytic lymphoma. J Clin Oncol. 2013;31:301–307.
  • Dhodapkar MV, Jacobson JL, Gertz MA, et al. Prognostic factors and response to fludarabine therapy in patients with Waldenstrom macroglobulinemia: results of United States intergroup trial (Southwest Oncology Group S9003). Blood. 2001;98:41–48.
  • Carney DA, Westerman DA, Tam CS, et al. Therapy-related myelodysplastic syndrome and acute myeloid leukemia following fludarabine combination chemotherapy. Leukemia. 2010;24:2056–2062.
  • Laszlo D, Andreola G, Rigacci L, et al. Rituximab and subcutaneous 2-chloro-2ʹ-deoxyadenosine combination treatment for patients with Waldenstrom macroglobulinemia: clinical and biologic results of a phase II multicenter study. J Clin Oncol. 2010;28:2233–2238.
  • ClinicalTrials.gov. Study of the glutaminase inhibitor CB-839 in hematological tumors; [ cited 2015 Jun 10]. Available from: https://clinicaltrials.gov/ct2/show/NCT02071888
  • Davids MS, Seymour JF, Gerecitano JF, et al. Phase I study of ABT-199 (GDC-0199) in patients with relapsed/refractory (R/R) non-Hodgkin lymphoma (NHL): responses observed in diffuse large B-cell (DLBCL) and follicular lymphoma (FL) at higher cohort doses. J Clin Oncol. 2014;32(Suppl. 5):Abstract 8522.
  • Cao Y, Yang G, Hunter ZR, et al. The BCL2 antagonist ABT-199 triggers apoptosis, and augments ibrutinib and idelalisib mediated cytotoxicity in CXCR4 Wild-type and CXCR4 WHIM mutated Waldenstrom macroglobulinemia cells. Br J Haematol. 2015 Jul;170(1):134–138. DOI:10.1111/bjh.13278
  • Treon SP, Yang G, Hanzis C, et al. Attainment of complete/very good partial response following rituximab-based therapy is an important determinant to progression-free survival, and is impacted by polymorphisms in FCGR3A in Waldenstrom macroglobulinaemia. Br J Haematol. 2011;154:223–228.
  • Weber D, Treon SP, Emmanouilides C, et al. Uniform response criteria in Waldenstrom’s macroglobulinemia: consensus panel recommendations from the Second International Workshop on Waldenstrom’s macroglobulinemia. Semin Oncol. 2003;30:127–131.
  • Owen RG, Kyle RA, Stone MJ, et al. Response assessment in Waldenstrom macroglobulinaemia: update from the VIth international workshop. Br J Haematol. 2013;160:171–176.
  • Gertz MA, Reeder CB, Kyle RA, et al. Stem cell transplant for Waldenstrom macroglobulinemia: an underutilized technique. Bone Marrow Transplant. 2012;47:1147–1153.
  • Gertz MA, Rue M, Blood E, et al. Multicenter phase 2 trial of rituximab for Waldenstrom macroglobulinemia (WM): an Eastern Cooperative Oncology Group Study (E3A98). Leuk Lymphoma. 2004;45:2047–2055.
  • Usmani S, Sexton R, Crowley J, et al. Autologous stem cell transplantation as a care option in Waldenstrom’s macroglobulinemia. Clin Lymphoma Myeloma Leuk. 2011;11:139–142.
  • Kyriakou C, Canals C, Cornelissen JJ, et al. Allogeneic stem-cell transplantation in patients with Waldenstrom macroglobulinemia: report from the Lymphoma Working Party of the European Group for Blood and Marrow Transplantation. J Clin Oncol. 2010; 28:4926–4934
  • Castillo JJ, Gustine J, Meid K, et al. Histological transformation to diffuse large B-cell lymphoma in patients with Waldenstrom macroglobulinemia. Am J Hematol. 2016 Oct;91(10):1032–1035. DOI:10.1002/ajh.24477. Epub 2016 Jul 27.
  • Xu L, Hunter ZR, Tsakmaklis N, et al. Clonal architecture of CXCR4 WHIM-like mutations in Waldenstrom macroglobulinemia. Br J Haematol. 2016 Mar;172(5):735–744. DOI:10.1111/bjh.13897. Epub 2015 Dec 13.
  • Poulain S, Roumier C, Venet-Caillault A, et al. Genomic Landscape of CXCR4 Mutations in Waldenstrom macroglobulinemia. Clin Cancer Res. 2016 Mar 15;22(6): 1480–1488. DOI:10.1158/1078-0432.CCR-15-0646. Epub 2015 Oct 21.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.