How i treat Waldenström macroglobulinemia

Blood

Volumn 126, Issue 6, 2015, Pages 721-732

  Treon, Steven P ^a  

^a HARVARD MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BENDAMUSTINE; BORTEZOMIB; CARFILZOMIB; CD27 ANTIGEN; CD70 ANTIGEN; CHEMOKINE RECEPTOR CXCR4; CHIMERIC ANTIGEN RECEPTOR; CLADRIBINE; CYCLOPHOSPHAMIDE; DEXAMETHASONE; DOXORUBICIN; EVEROLIMUS; FAMOTIDINE; FLUDARABINE; HEMOGLOBIN; IBRUTINIB; IDELALISIB; IMMUNOGLOBULIN M; IMO 8400; IXAZOMIB; MYELOID DIFFERENTIATION FACTOR 88; OFATUMUMAB; OPROZOMIB; PREDNISONE; PROTEASOME INHIBITOR; PROTEIN BCL 2; RITUXIMAB; ULOCUPLUMAB; UNCLASSIFIED DRUG; VENETOCLAX; VINCRISTINE; ANTINEOPLASTIC AGENT; BORONIC ACID DERIVATIVE; CHLORMETHINE DERIVATIVE; CXCR4 PROTEIN, HUMAN; MONOCLONAL ANTIBODY; MYD88 PROTEIN, HUMAN; OLIGOPEPTIDE; PYRAZINE DERIVATIVE; PYRAZOLE DERIVATIVE; PYRIMIDINE DERIVATIVE; RAPAMYCIN; VIDARABINE;

AMYLOIDOSIS; ARTICLE; ASYMPTOMATIC DISEASE; AUTOLOGOUS STEM CELL TRANSPLANTATION; CELL THERAPY; CLINICAL FEATURE; CRYOGLOBULINEMIA; DISEASE CONTROL; DRUG SAFETY; DRUG TARGETING; HERPES ZOSTER; HUMAN; HYPERVISCOSITY SYNDROME; IMMUNE DEFICIENCY; IMMUNOGLOBULIN BLOOD LEVEL; IMMUNOGLOBULIN DEFICIENCY; INFECTION; LYMPHADENOPATHY; MAINTENANCE THERAPY; METASTASIS; MONOTHERAPY; NEUROPATHY; OVERALL SURVIVAL; PARAPROTEINEMIA; PERIPHERAL NEUROPATHY; PLASMAPHERESIS; PRIORITY JOURNAL; PROGRESSION FREE SURVIVAL; PROPHYLAXIS; SIGNAL TRANSDUCTION; SOMATIC MUTATION; T LYMPHOCYTE; THROMBOCYTE COUNT; TREATMENT OUTCOME; TREATMENT RESPONSE; WALDENSTROEM MACROGLOBULINEMIA; ADULT; AGED; ANALOGS AND DERIVATIVES; AUTOTRANSPLANTATION; B LYMPHOCYTE; CASE REPORT; DRUG EFFECTS; GENE EXPRESSION; GENETIC PREDISPOSITION; GENETICS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; MALE; METABOLISM; MIDDLE AGED; MOLECULARLY TARGETED THERAPY; PATHOLOGY; WALDENSTROM MACROGLOBULINEMIA;

ADULT; AGED; ANTIBODIES, MONOCLONAL, MURINE-DERIVED; ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; B-LYMPHOCYTES; BORONIC ACIDS; CLADRIBINE; CYCLOPHOSPHAMIDE; GENE EXPRESSION; GENETIC PREDISPOSITION TO DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; IMMUNOGLOBULIN M; MALE; MIDDLE AGED; MOLECULAR TARGETED THERAPY; MYELOID DIFFERENTIATION FACTOR 88; NITROGEN MUSTARD COMPOUNDS; OLIGOPEPTIDES; PLASMAPHERESIS; PYRAZINES; PYRAZOLES; PYRIMIDINES; RECEPTORS, CXCR4; SIROLIMUS; TRANSPLANTATION, AUTOLOGOUS; VIDARABINE; WALDENSTROM MACROGLOBULINEMIA;

EID: 84940050343     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2015-01-553974     Document Type: Article

References (122)

1. Owen RG, Treon SP, Al-Katib A, et al. Clinicopathological definition of Waldenstrom's macroglobulinemia: consensus panel recommendations from the Second International Workshop on Waldenstrom's Macroglobulinemia. Semin Oncol. 2003;30(2):110-115.
2. World Health Organization. Classification of Tumours of Haematopoietic and Lymphoid Tissues. Lyon, France: IARC Press; 2008.
3. Treon SP, Xu L, Yang G, et al. MYD88 L265P somatic mutation in Waldenström's macroglobulinemia. N Engl J Med. 2012;367(9):826-833.
4. Xu L, Hunter ZR, Yang G, et al. MYD88 L265P in Waldenström macroglobulinemia, immunoglobulin M monoclonal gammopathy, and other B-cell lymphoproliferative disorders using conventional and quantitative allele-specific polymerase chain reaction. Blood. 2013;121(11):2051-2058.
5. Varettoni M, Arcaini L, Zibellini S, et al. Prevalence and clinical significance of the MYD88 (L265P) somatic mutation in Waldenstrom's macroglobulinemia and related lymphoid neoplasms. Blood. 2013;121(13):2522-2528.
6. Jiménez C, Sebastián E, Chillón MC, et al. MYD88 L265P is a marker highly characteristic of, but not restricted to, Waldenström's macroglobulinemia. Leukemia. 2013;27(8):1722-1728.
7. Poulain S, Roumier C, Decambron A, et al. MYD88 L265P mutation in Waldenstrom macroglobulinemia. Blood. 2013;121(22):4504-4511.
8. Ansell SM, Hodge LS, Secreto FJ, et al. Activation of TAK1 by MYD88 L265P drives malignant B-cell Growth in non-Hodgkin lymphoma. Blood Cancer J. 2014;4:e183.
9. Hunter ZR, Xu L, Yang G, et al. The genomic landscape of Waldenstrom macroglobulinemia is characterized by highly recurring MYD88 and WHIM-like CXCR4 mutations, and small somatic deletions associated with B-cell lymphomagenesis. Blood. 2014;123(11):1637-1646.
10. Roccaro AM, Sacco A, Jimenez C, et al. C1013G/CXCR4 acts as a driver mutation of tumor progression and modulator of drug resistance in lymphoplasmacytic lymphoma. Blood. 2014;123(26):4120-4131.
11. Schmidt J, Federmann B, Schindler N, et al. MYD88 L265P and CXCR4 mutations in lymphoplasmacytic lymphoma identify cases with high disease activity [published online ahead of print March 29, 2015]. Br J Haematol.
12. Poulain S, Roumier C, Doye E, et al. Genomic landscape of CXCR4 mutations in Waldenstrom's macroglobulinemia. Blood. 2014;124:1627.
13. Treon SP, Cao Y, Xu L, Yang G, Liu X, Hunter ZR. Somatic mutations in MYD88 and CXCR4 are determinants of clinical presentation and overall survival in Waldenstrom macroglobulinemia. Blood. 2014;123(18):2791-2796.
14. Treon SP, Tripsas CK, Meid K, et al. Ibrutinib in previously treated Waldenström's macroglobulinemia. N Engl J Med. 2015;372(15):1430-1440.
15. Treon SP, Hunter ZR, Matous J, et al. Multicenter clinical trial of bortezomib in relapsed/refractory Waldenstrom's macroglobulinemia: results of WMCTG Trial 03-248. Clin Cancer Res. 2007;13(11):3320-3325.
16. Ghobrial IM, Gertz M, Laplant B, et al. Phase II trial of the oral mammalian target of rapamycin inhibitor everolimus in relapsed or refractory Waldenstrom macroglobulinemia. J Clin Oncol. 2010;28(8):1408-1414.
17. 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(8):1296-1303.
18. Merlini G, Farhangi M, Osserman EF. Monoclonal immunoglobulins with antibody activity in myeloma, macroglobulinemia and related plasma cell dyscrasias. Semin Oncol. 1986;13(3):350-365.
19. Farhangi M, Merlini G. The clinical implications of monoclonal immunoglobulins. Semin Oncol. 1986;13(3):366-379.
20. Marmont AM, Merlini G. Monoclonal autoimmunity in hematology. Haematologica. 1991;76(6):449-459.
21. Treon SP, Hunter ZR, Aggarwal A, et al. Characterization of familial Waldenstrom's macroglobulinemia. Ann Oncol. 2006;17(3):488-494.
22. McMaster ML, Csako G, Giambarresi TR, et al. Long-term evaluation of three multiple-case Waldenstrom macroglobulinemia families. Clin Cancer Res. 2007;13(17):5063-5069.
23. Kristinsson SY, Björkholm M, Goldin LR, McMaster ML, Turesson I, Landgren O. Risk of lymphoproliferative disorders among first-degree relatives of lymphoplasmacytic lymphoma/Waldenstrom macroglobulinemia patients: a population-based study in Sweden. Blood. 2008;112(8):3052-3056.
24. Treon SP, Tripsas C, Hanzis C, et al. Familial disease predisposition impacts treatment outcome in patients with Waldenström macroglobulinemia. Clin Lymphoma Myeloma Leuk. 2012;12(6):433-437.
25. Santini GF, Crovatto M, Modolo ML, et al. Waldenström macroglobulinemia: a role of HCV infection? Blood. 1993;82(9):2932.
26. Silvestri F, Barillari G, Fanin R, et al. Risk of hepatitis C virus infection, Waldenström's macroglobulinemia, and monoclonal gammopathies. Blood. 1996;88(3):1125-1126.
27. Leleu X, O'Connor K, Ho AW, et al. Hepatitis C viral infection is not associated with Waldenström's macroglobulinemia. Am J Hematol. 2007;82(1):83-84.
28. Treon SP. How I treat Waldenström macroglobulinemia. Blood. 2009;114(12):2375-2385.
29. Kyle RA, Treon SP, Alexanian R, et al. Prognostic markers and criteria to initiate therapy in Waldenstrom's macroglobulinemia: consensus panel recommendations from the Second International Workshop on Waldenstrom's Macroglobulinemia. Semin Oncol. 2003;30(2):116-120.
30. Anderson KC, Alsina M, Bensinger W, et al. Waldenström's macroglobulinemia/lymphoplasmacytic lymphoma, version 1. J Natl Compr Canc Netw. 2013;11(1):11-7.32.
31. Ciccarelli BT, Patterson CJ, Hunter ZR, et al. Hepcidin is produced by lymphoplasmacytic cells and is associated with anemia in Waldenström's macroglobulinemia. Clin Lymphoma Myeloma Leuk. 2011;11(1):160-163.
32. Treon SP, Tripsas CK, Ciccarelli BT, et al. Patients with Waldenström macroglobulinemia commonly present with iron deficiency and those with severely depressed transferrin saturation levels show response to parenteral iron administration. Clin Lymphoma Myeloma Leuk. 2013;13(2):241-243.
33. Dimopoulos MA, Kastritis E, Owen RG, et al. Treatment recommendations for patients with Waldenström macroglobulinemia (WM) and related disorders: IWWM-7 consensus. Blood. 2014;124(9):1404-1411.
34. th International Workshop on Waldenstrom's macroglobulinemia; October 15-19, 2008; Stockholm, Sweden. Abstract 50.
35. Leleu X, Soumerai J, Roccaro A, et al. Increased incidence of transformation and myelodysplasia/acute leukemia in patients with Waldenström macroglobulinemia treated with nucleoside analogs. J Clin Oncol. 2009;27(2):250-255.
36. Leleu X, Tamburini J, Roccaro A, et al. Balancing risk versus benefit in the treatment of Waldenström's Macroglobulinemia patients with nucleoside analogue-based therapy. Clin Lymphoma Myeloma. 2009;9(1):71-73.
37. Rakkhit R, Delasalle KB, Gavino MB, et al. Incidence of transformation to large cell lymphoma and to second malignancies in symptomatic patients with Waldenstrom's macroglobulinemia (WM) treated with cladribine (2-CdA) combination induction [abstract]. Blood. 2008;112(11). Abstract 3065.
38. Treon SP, Branagan AR, Hunter Z, Santos D, Tournhilac O, Anderson KC. Paradoxical increases in serum IgM and viscosity levels following rituximab in Waldenstrom's macroglobulinemia. Ann Oncol. 2004;15(10):1481-1483.
39. Ghobrial IM, Fonseca R, Greipp PR, et al Eastern Cooperative Oncology Group. Initial immunoglobulin M 'flare' after rituximab therapy in patients diagnosed with Waldenstrom macroglobulinemia: an Eastern Cooperative Oncology Group Study. Cancer. 2004;101(11):2593-2598.
40. Ghobrial IM, Uslan DZ, Call TG, Witzig TE, Gertz MA. Initial increase in the cryoglobulin level after rituximab therapy for type II cryoglobulinemia secondary to Waldenström macroglobulinemia does not indicate failure of response. Am J Hematol. 2004;77(4):329-330.
41. Noronha V, Fynan TM, Duffy T. Flare in neuropathy following rituximab therapy for Waldenstrom's macroglobulinemia. J Clin Oncol. 2006;24(1):e3.
42. Broglio L, Lauria G. Worsening after rituximab treatment in anti-mag neuropathy. Muscle Nerve. 2005;32(3):378-379.
43. Gertz MA, Rue M, Blood E, Kaminer LS, Vesole DH, Greipp PR. Multicenter phase 2 trial of rituximab for Waldenström macroglobulinemia (WM): an Eastern Cooperative Oncology Group Study (E3A98). Leuk Lymphoma. 2004;45(10):2047-2055.
44. Dimopoulos MA, Zervas C, Zomas A, et al. Treatment of Waldenström's macroglobulinemia with rituximab. J Clin Oncol. 2002;20(9):2327-2333.
45. Treon SP, Emmanouilides C, Kimby E, et al Waldenström's Macroglobulinemia Clinical Trials Group. Extended rituximab therapy in Waldenström's macroglobulinemia. Ann Oncol. 2005;16(1):132-138.
46. Furman RR, Eradat H, Switzky JC, et al. A phase II trial of ofatumumab in subjects with Waldenstrom's macroglobulinemia [abstract]. Blood. 2011;118(21). Abstract 3701.
47. Kanan S, Meid K, Treon SP, et al. Clinical characteristics of rituximab intolerance in patients with Waldenstrom's macroglobulinemia [abstract]. Blood. 2014; 124(21): Abstract 2610.
48. Treon SP, Ioakimidis L, Soumerai JD, et al. Primary therapy of Waldenström macroglobulinemia with bortezomib, dexamethasone, and rituximab: WMCTG clinical trial 05-180. J Clin Oncol. 2009;27(23):3830-3835.
49. Dimopoulos MA, García-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(19):3276-3282.
50. 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(4):503-510.
51. Moreau P, Pylypenko H, Grosicki S, et al. Subcutaneous versus intravenous administration of bortezomib in patients with relapsed multiple myeloma: a randomised, phase 3, non-inferiority study. Lancet Oncol. 2011;12(5):431-440.
52. Rummel MJ, Niederle N, Maschmeyer G, et al; Study group indolent Lymphomas (StiL). 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(9873):1203-1210.
53. Dimopoulos MA, Anagnostopoulos A, Kyrtsonis MC, et al. Primary treatment of Waldenström macroglobulinemia with dexamethasone, rituximab, and cyclophosphamide. J Clin Oncol. 2007;25(22):3344-3349.
54. Buske C, Hoster E, Dreyling M, et al German Low-Grade Lymphoma Study Group. 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(1):153-161.
55. Ioakimidis L, Patterson CJ, Hunter ZR, et al. Comparative outcomes following CP-R, CVP-R, and CHOP-R in Waldenström's macroglobulinemia. Clin Lymphoma Myeloma. 2009;9(1):62-66.
56. Weber DM, Dimopoulos MA, Delasalle K, Rankin K, Gavino M, Alexanian R. 2-Chlorodeoxyadenosine alone and in combination for previously untreated Waldenstrom's macroglobulinemia. Semin Oncol. 2003;30(2):243-247.
57. Treon SP, Branagan AR, Ioakimidis L, et al. Long-term outcomes to fludarabine and rituximab in Waldenström macroglobulinemia. Blood. 2009;113(16):3673-3678.
58. Laszlo D, Andreola G, Rigacci L, et al. Rituximab and subcutaneous 2-chloro-29-deoxyadenosine combination treatment for patients with Waldenstrom macroglobulinemia: clinical and biologic results of a phase II multicenter study. J Clin Oncol. 2010;28(13):2233-2238.
59. Tedeschi A, Benevolo G, Varettoni M, et al. Fludarabine plus cyclophosphamide and rituximab in Waldenstrom macroglobulinemia: an effective but myelosuppressive regimen to be offered to patients with advanced disease. Cancer. 2012;118(2):434-443.
60. th International Workshop on Waldenstrom's Macroglobulinemia; August 14-16, 2014; London, United Kingdom. Abstract W25.
61. Smith TJ, Khatcheressian J, Lyman GH, et al. 2006 update of recommendations for the use of white blood cell growth factors: an evidence-based clinical practice guideline. J Clin Oncol. 2006;24(19):3187-3205.
62. Stone MJ, Bogen SA. Evidence-based focused review of management of hyperviscosity syndrome. Blood. 2012;119(10):2205-2208.
63. Menke MN, Treon SP. Hyperviscosity syndrome. In: Kalaycio B, ed. Clinical Malignant Hematology. Sekeres. New York: McGraw Hill Publishing; 2007:937-941.
64. Renaud S, Gregor M, Fuhr P, et al. Rituximab in the treatment of polyneuropathy associated with anti-MAG antibodies. Muscle Nerve. 2003;27(5):611-615.
65. Léger JM, Viala K, Nicolas G, et al RIMAG Study Group (France and Switzerland). Placebo-controlled trial of rituximab in IgM anti-myelin-associated glycoprotein neuropathy. Neurology. 2013;80(24):2217-2225.
66. Lunn MP, Nobile-Orazio E. Immunotherapy for IgM anti-myelin-associated glycoprotein paraprotein-associated peripheral neuropathies. Cochrane Database Syst Rev. 2012;5:CD002827.
67. Hospital MA, Viala K, Dragomir S, et al. Immunotherapy-based regimen in anti-MAG neuropathy: results in 45 patients. Haematologica. 2013;98(12):e155-e157.
68. Treon SP, Hanzis C, Ioakimidis L, et al. Clinical characteristics and treatment outcome of disease-related peripheral neuropathy in Waldenstrom's macroglobulinemia (WM). J Clin Oncol. 2010;28:15s.
69. Treon SP, Xu L, Hunter ZR. MYD88 mutations and ibrutinib responses in Waldenstrom's macroglobulinemia. N Engl J Med. 2015 (in press).
70. 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(3):237-242.
71. 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;122:1822.
72. Kyriakou C, Canals C, Sibon D, et al. High-dose therapy and autologous stem-cell transplantation in Waldenstrom macroglobulinemia: the Lymphoma Working Party of the European Group for Blood and Marrow Transplantation. J Clin Oncol. 2010;28(13):2227-2232.
73. Gertz MA, Hayman SR, Buadi FK. Transplantation for IgM amyloidosis and IgM myeloma. Clin Lymphoma Myeloma. 2009;9(1):77-79.
74. Kyriakou C, Canals C, Cornelissen JJ, et al. Allogeneic stem-cell transplantation in patients with Waldenström macroglobulinemia: report from the Lymphoma Working Party of the European Group for Blood and Marrow Transplantation. J Clin Oncol. 2010;28(33):4926-4934.
75. Treon SP, Hanzis C, Manning RJ, 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(3):357-362.
76. Rummel MJ, Lerchenmüller C, Greil R, et al. Bendamustine-rituximab induction followed by observation or rituximab maintenance for newly diagnosed patients with Waldenström's macroglobulinemia: results from a prospective, randomized, multicenter study (StiL NHL 7-2008) [abstract]. Blood. 2012;120(21): Abstract 2739.
77. van Oers MH, Klasa R, Marcus RE, et al. Rituximab maintenance improves clinical outcome of relapsed/resistant follicular non-Hodgkin lymphoma in patients both with and without rituximab during induction: results of a prospective randomized phase 3 intergroup trial. Blood. 2006;108(10):3295-3301.
78. Hunter ZR, Manning RJ, Hanzis C, et al. IgA and IgG hypogammaglobulinemia in Waldenström's macroglobulinemia. Haematologica. 2010;95(3):470-475.
79. Owen RG, Kyle RA, Stone MJ, et al. Response Assessment in Waldenstrom Macroglobulinemia. Br J Haematol. 2013;160(2):171-176.
80. Gertz MA, Abonour R, Heffner LT, Greipp PR, Uno H, Rajkumar SV. Clinical value of minor responses after 4 doses of rituximab in Waldenström macroglobulinaemia: a follow-up of the Eastern Cooperative Oncology Group E3A98 trial. Br J Haematol. 2009;147(5):677-680.
81. 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(2):223-228.
82. Tripsas CK, Patterson CJ, Uljon SN, Lindeman NI, Turnbull B, Treon SP. Comparative response assessment by serum immunoglobulin M M-protein and total serum immunoglobulin M after treatment of patients with Waldenström macroglobulinemia. Clin Lymphoma Myeloma Leuk. 2013;13(2):250-252.
83. Uljon SN, Treon SP, Tripsas CK, Lindeman NI. Challenges with serum protein electrophoresis in assessing progression and clinical response in patients with Waldenström macroglobulinemia. Clin Lymphoma Myeloma Leuk. 2013;13(2):247-249.
84. Strauss SJ, Maharaj L, Hoare S, et al. Bortezomib therapy in patients with relapsed or refractory lymphoma: potential correlation of in vitro sensitivity and tumor necrosis factor alpha response with clinical activity. J Clin Oncol. 2006;24(13):2105-2112.
85. Yang G, Liu X, Zhou Y, et al. PI3K/AKT pathway is activated by MYD88 L265P and use of PI3K-delta inhibitors induces robust tumor cell killing in Waldenstrom's Macroglobulinemia [abstract]. Blood. 2013;122(21): Abstract 4255.
86. Gopal AK, Kahl BS, de Vos S, et al. PI3Kd inhibition by idelalisib in patients with relapsed indolent lymphoma. N Engl J Med. 2014;370(11):1008-1018.
87. Brenner L, Arbeit RD, Sullivan T. IMO-8400, an antagonist of Toll-like receptors 7, 8, and 9. Development for genetically defined B-cell lymphomas: safety and activity in phase 1 and phase 2 clinical trials [abstract]. Blood. 2014;122(21): Abstract 3101.
88. 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(7):1222-1232.
89. Lim KH, Romero DL, Chaudhary D, et al. IRAK4 kinase as a novel therapeutic target in the ABC subtype of diffuse large B cell lymphoma [abstract]. Blood. 2012;120(21): Abstract 62.
90. th International Workshop on Waldenstrom's Macroglobulinemia; August 14-16, 2014; London, United Kingdom. Abstract 27.
91. Chng WJ, Schop RF, Price-Troska T, et al. Gene-expression profiling of Waldenstrom macroglobulinemia reveals a phenotype more similar to chronic lymphocytic leukemia than multiple myeloma. Blood. 2006;108(8):2755-2763.
92. Hatjiharissi E, Mitsiades CS, Ciccarelli B, et al. Comprehensive molecular characterization of malignant and microenvironmental cells in Waldenstrom's macroglobulinemia by gene expression profiling [abstract]. Blood. 2007;110(11): Abstract 3174.
93. Cao Y, Yang G, Hunter ZR, et al. The BCL2 antagonist ABT-199 triggers apoptosis, and augments ibrutinib and idelalisib mediated cytotoxicity in CXCR4Wild-type and CXCR4WHIM mutated Waldenstrom macroglobulinaemia cells [published online ahead of print January 12, 2015]. Br J Haematol. doi:10.1111/bjh.13278.
94. Davids MS, Seymour JF, Gerecitano JF, et al. Phase I study of ABT-199 (GDC-0199) in patients with relapsed/refractory non-Hodgkin lymphoma: responses observed in diffuse large B-cell (DLBCL) and follicular lymphoma at higher cohort doses. J Clin Oncol. 2014;32:5s. Abstract 8522.
95. Hurchla MA, Garcia-Gomez A, Hornick MC, et al. The epoxyketone-based proteasome inhibitors carfilzomib and orally bioavailable oprozomib have anti-resorptive and bone-anabolic activity in addition to anti-myeloma effects. Leukemia. 2013;27(2):430-440.
96. 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) [abstract]. Blood. 2014;122(21): Abstract 1785.
97. Chauhan D, Tian Z, Zhou B, et al. In vitro and in vivo selective antitumor activity of a novel orally bioavailable proteasome inhibitor MLN9708 against multiple myeloma cells. Clin Cancer Res. 2011;17(16):5311-5321.
98. Richardson PG, Baz R, Wang M, et al. Phase 1 study of twice-weekly ixazomib, an oral proteasome inhibitor, in relapsed/refractory multiple myeloma patients. Blood. 2014;124(7):1038-1046.
99. Ho AW, Hatjiharissi E, Ciccarelli BT, et al. CD27-CD70 interactions in the pathogenesis of Waldenstrom macroglobulinemia. Blood. 2008;112(12):4683-4689.
100. th International Workshop on Waldenstrom's Macroglobulinemia; August 14-16, 2014; London, United Kingdom. Abstract W38.
101. Cao Y, Hunter ZR, Liu X, et al. The WHIM-like CXCR4S338X 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.
102. Cao Y, Hunter ZR, Liu X, et al. CXCR4 WHIM-like frameshift and nonsense mutations promote ibrutinib resistance but do not supplant MYD88(L265P) -directed survival signalling in Waldenström macroglobulinaemia cells. Br J Haematol. 2015;168(5):701-707.
103. Ghobrial IM, Perez R, Baz R, et al. Phase Ib study of the novel anti-CXCR4 antibody ulocuplumab (BMS-936564) in combination with lenalidomide plus low-dose dexamethasone, or with bortezomib plus dexamethasone in subjects with relapsed or refractory multiple myeloma [abstract]. Blood. 2014;122(21): Abstract 3483.
104. Smith EL, Palomba ML, Park JH, et al. A systemic xenograft model of Waldenström's macroglobulinemia demonstrates the potent anti-tumor effect of second generation CD19 directed chimeric antigen receptor modified T cells in this disease [abstract]. Blood. 2014;122 (21): Abstract 4484.
105. Dhodapkar MV, Jacobson JL, Gertz MA, Rivkin SE, Roodman GD, Tuscano JM, et al. Prognostic factors and response to fludarabine therapy in patients with Waldenström macroglobulinemia: results of United States intergroup trial (Southwest Oncology Group S9003). Blood. 2001;98(1):41-48.
106. Dimopoulos M, Gika D, Zervas K, et al. The international staging system for multiple myeloma is applicable in symptomatic Waldenstrom's macroglobulinemia. Leuk Lymph. 2004;45(9):1809-1813.
107. Morel P, Duhamel A, Gobbi P, et al. International prognostic scoring system for Waldenstrom Macroglobulinemia. Blood. 2009;113(18):4163-4170.
108. Owen RG, Barrans SL, Richards SJ, et al. Waldenström macroglobulinemia. Development of diagnostic criteria and identification of prognostic factors. Am J Clin Pathol. 2001;116(3):420-428.
109. San Miguel JF, Vidriales MB, Ocio E, et al. Immunophenotypic analysis of Waldenstrom's macroglobulinemia. Semin Oncol. 2003;30(2):187-195.
110. Hunter ZR, Branagan AR, Manning R, et al. CD5, CD10, CD23 expression in Waldenstrom's Macroglobulinemia. Clin Lymphoma. 2005;5(4):246-249.
111. Avet-Loiseau H, Garand R, Lode L, Robillard N, Bataille R. 14q32 translocations discriminate IgM multiple myeloma from Waldenstrom's macroglobulinemia. Semin Oncol. 2003;30(2):153-155.
112. Willenbacher W, Willenbacher E, Brunner A, Manzi C. Improved accuracy of discrimination between IgM multiple myeloma and Waldenstrom macroglobulinaemia by testing for MYD88 L265P mutations. Br J Haematol. 2013;161(6):902-904.
113. Schop RF, Kuehl WM, Van Wier SA, et al. Waldenström macroglobulinemia neoplastic cells lack immunoglobulin heavy chain locus translocations but have frequent 6q deletions. Blood. 2002;100(8):2996-3001.
114. Ocio EM, Schop RF, Gonzalez B, et al. 6q deletion in Waldenstrom's macroglobulinemia is associated with features of adverse prognosis. Br J Haematol. 2007;136(1):80-86.
115. Chang H, Qi C, Trieu Y, et al. Analysis of 6q deletion in Waldenstrom's macroglobulinemia. Eur J Haematol. 2007;79(3):244-247.
116. Viala K, Stojkovic T, Maisonobe T, et al. Heterogeneous spectrum of neuropathies in Waldenstrom's macroglobulinemia: A diagnostic strategy to optimize their management. J Peripher Nerv Syst. 2012;17(1):90-101.
117. Menke MN, Feke GT, McMeel JW, Branagan A, Hunter Z, Treon SP. Hyperviscosity-related retinopathy in Waldenstrom's Macroglobulinemia. Arch Opthalmol. 2006;124(11):1601-1606.
118. Stone MJ. Waldenstrom macroglobulinemia: Hyperviscosity syndrome and cryoglobulinemia. Clin Lymphoma Myeloma. 2009;9(1):97-99.
119. Huang H, Li X, Zhu J, et al. Entecavir versus lamivudine for prevention of hepatitis B virus reactivation among patients with untreated diffuse large B-cell lymphoma receiving R-CHOP chemotherapy: A randomized clinical trial. JAMA. 2014;312(23):2521-2530.
120. Poulain S, Boyle EM, Roumier C, et al. MYD88 L265P mutation contributes to the diagnosis of Bing Neel syndrome. Br J Haematol. 2014;167(4):506-513.
121. Nagao T, Oshikawa G, Ishida S, et al. A novel MYD88 mutation, L265RPP, in Waldenström macroglobulinemia activates the NF-κB pathway to upregulate Bcl-xL expression and enhances cell survival. Blood Cancer J. 2015;5:e314.
122. Nobile-Orazio E, Marmiroli P, Baldini L, et al. Peripheral neuropathy in macroglobulinemia: incidence and antigen-specificity of M proteins. Neurology. 1987;37(9):1506-1514.