华氏巨球蛋白血症

Kyle RA, Therneau TM, Rajkumar SV, et al. A long-term study of prognosis in monoclonal gammopathy of undetermined significance. N Engl J Med. 2002;346:564-569.

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McMaster ML. Familial Waldenstrom's macroglobulinemia. Semin Oncol. 2003;30:146-152.

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Gertz MA, Merlini G, Treon SP. Amyloidosis and Waldenström's macroglobulinemia. Hematology Am Soc Hematol Educ Program. 2004:257-282.

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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.

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1.  Cesana C, Miqueleiz S, Bernuzzi P, et al. Smouldering Waldenstrom's macroglobulinemia: factors predicting evolution to symptomatic disease. Semin Oncol. 2003;30:231-235.

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2.  Merlini G. Waldenstrom's macroglobulinemia - clinical manifestations and prognosis. In: Schechter GP, Hoffman R, Schrier SL, eds. Hematology. Washington, DC: American Society of Hematology; 1999:358-369.

3.  Johnson SA, Birchall J, Luckie C, et al; Haemato-Oncology Task Force of the British Committee for Standards in Haematology. Guidelines on the management of Waldenström macroglobulinaemia. Br J Haematol. 2006;132:683-697.

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4.  Groves FD, Travis LB, Devesa SS, et al. Waldenstrom's macroglobulinemia: incidence patterns in the United States, 1988-1994. Cancer. 1998;82:1078-1081.

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5.  Herrinton LJ, Weiss NS. Incidence of Waldenstrom's macroglobulinemia. Blood. 1993;82:3148-3150.

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6.  Phekoo KJ, Jack RH, Davies E, et al. The incidence and survival of Waldenstrom's macroglobulinaemia in South East England. Leuk Res. 2008;32:55-59.

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7.  Kristinsson SY, Koshiol J, Goldin LR, et al. Genetics- and immune-related factors in the pathogenesis of lymphoplasmacytic lymphoma/Waldenstrom's macroglobulinemia. Clin Lymphoma Myeloma. 2009;9:23-26.

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8.  Treon SP, Xu L, Yang G, et al. MYD88 L265P somatic mutation in Waldenström's macroglobulinemia. N Engl J Med. 2012;367:826-833.

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9.  Treon SP, Tripsas CK, Meid K, et al. Ibrutinib in previously treated Waldenström's macroglobulinemia. N Engl J Med. 2015;372:1430-1440.

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10.  Treon SP, Hunter ZR. A new era for Waldenstrom macroglobulinemia: MYD88 L265P. Blood. 2013;121:4434-4436.

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11.  Fulciniti M, Amodio N, Bandi RL, et al. MYD88-independent growth and survival effects of Sp1 transactivation in Waldenstrom macroglobulinemia. Blood. 2014;123:2673-2681.

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12.  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;123:4120-4131.

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13.  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;168:701-707.

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14.  Kyle RA, Therneau TM, Rajkumar SV, et al. A long-term study of prognosis in monoclonal gammopathy of undetermined significance. N Engl J Med. 2002;346:564-569.

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15.  Treon SP, Hunter ZR, Aggarwal A, et al. Characterization of familial Waldenström's macroglobulinemia. Ann Oncol. 2006;17:488-494.

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16.  Sahota SS, Forconi F, Ottensmeier CH, et al. Origins of the malignant clone in typical Waldenstrom's macroglobulinemia. Semin Oncol. 2003;30:136-141.

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17.  Ogmundsdottir HM, Einarsdottir HK, Steingrimsdottir H, et al. Familial predisposition to monoclonal gammopathy of unknown significance, Waldenstrom's macroglobulinemia, and multiple myeloma. Clin Lymphoma Myeloma. 2009;9:27-29.

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18.  McMaster ML. Familial Waldenstrom's macroglobulinemia. Semin Oncol. 2003;30:146-152.

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19.  Giordano TP, Henderson L, Landgren O, et al. Risk of non-Hodgkin lymphoma and lymphoproliferative precursor diseases in US veterans with hepatitis C virus. JAMA. 2007;297:2010-2017.

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20.  Leleu X, O'Connor K, Ho AW, et al. Hepatitis C viral infection is not associated with Waldenström's macroglobulinemia. Am J Hematol. 2007;82:83-84.

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21.  Treon SP. How I treat Waldenström macroglobulinemia. Blood. 2009;114:2375-2385.

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22.  Vijay A, Gertz MA. Waldenström macroglobulinemia. Blood. 2007;109:5096-5103.

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23.  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;110-115.

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24.  Pangalis GA, Kyrtsonis MC, Kontopidou FN, et al. Differential diagnosis of Waldenstrom's macroglobulinemia from other low-grade B-cell lymphoproliferative disorders. Semin Oncol. 2003;30:201-205.

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25.  Leleu X, Moreau AS, Weller E, et al. Serum immunoglobulin free light chain correlates with tumor burden markers in Waldenstrom macroglobulinemia. Leuk Lymphoma. 2008;49:1104-1107.

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26.  Giné E, Rovira J, Martinez-Trillos, et al. Initial features and outcome are similar in patients with lymphoplasmacytic lymphoma regardless they meet or not criteria of Waldenström macroglobulinemia. Blood (ASH Annual Meeting Abstracts). 2011;118:3677.

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27.  Vitolo U, Ferreri AJ, Montoto S. Lymphoplasmacytic lymphoma-Waldenström's macroglobulinemia. Crit Rev Oncol Hematol. 2008;67:172-185.

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28.  Gertz MA, Merlini G, Treon SP. Amyloidosis and Waldenström's macroglobulinemia. Hematology Am Soc Hematol Educ Program. 2004:257-282.

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29.  Banwait R, O'Regan K, Campigotto F, et al. The role of 18F-FDG PET/CT imaging in Waldenstrom macroglobulinemia. Am J Hematol. 2011;86:567-572.

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30.  Berentsen S, Beiske K, Tjønnfjord GE, et al. Primary chronic cold agglutinin disease: an update on pathogenesis, clinical features and therapy. Hematology. 2007;12:361-370.

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31.  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;27:250-255.

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32.  Kyle RA, Greipp PR, Gertz MA, et al. Waldenström's macroglobulinemia: a prospective study comparing daily with intermittent oral chlorambucil. Br J Haematol. 2000;108:737-742.

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33.  Treon S, Hunter Z, Matous J, et al. Multicenter clinical trial of bortezomib in relapsed/refractory Waldenstrom's macroglobulinaemia: results of WMCTG trial 03-248. Clin Cancer Res. 2007;13:3320-3325.

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34.  Dimopoulos MA, Anagnostopoulos A, Kyrtsonis MC, et al. Treatment of relapsed or refractory Waldenström's macroglobulinaemia with bortezomib. Haematologica. 2005;90:1655-1658.

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35.  Chen CI, Kouroukis CT, White D, et al. Bortezomib is active in patients with untreated or relapsed refractory Waldenstrom's macroglobulinemia: a phase II study of the national Cancer Institute of Canada Clinical Trials Group. J Clin Oncol. 2007;25:1570-1575.

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36.  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.

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37.  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;9:62-66.

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38.  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;27:3830-3835.

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39.  Treon SP, Soumerai JD, Hunter ZR, et al. Long-term follow-up of symptomatic patients with lymphoplasmacytic lymphoma/Waldenström macroglobulinemia treated with the anti-CD52 monoclonal antibody alemtuzumab. Blood. 2011;118:276-281.

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40.  Kyriakou C, Canals C, Taghipour G, et al. Autologous stem cell transplantation (ASCT) for patients with Waldenstrom's macroglobulinemia: an analysis of 201 cases from the European Bone Marrow Transplant Registry (EBMT). Haematologica. 2007;92(suppl):PO-1228(abstr).

41.  Kyriakou C, Canals C, Taghipour G, et al. Allogeneic stem cell transplantation (ALLO-SCT) in Waldenstrom macroglobulinemia (WM): an analysis of 106 cases from the European Bone Marrow Registry (EBMT). Haematologica. 2007;92(suppl):WM3.9(abstr).

42.  Kyriakou C, Canals C, Sibon D, et al. High-dose therapy and autologous stem-cell transplantation in Waldenström macroglobulinemia: the Lymphoma Working Party of the European Group for Blood and Marrow Transplantation. J Clin Oncol. 2010;28:2227-2232.

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43.  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.

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44.  Treon SP, Soumerai JD, Branagan AR, et al. Thalidomide and rituximab in Waldenstrom macroglobulinemia. Blood. 2008;112:4452-4457.

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45.  Roccaro AM, Leleu X, Blotta S, et al. Waldenstrom's macroglobulinemia: new therapeutic options. Cancer Therapy. 2008;6:227-238.

46.  Anagnostopoulos A, Giralt S. Stem cell transplantation (SCT) for Waldenstrom's macroglobulinemia (WM). Bone Marrow Transplant. 2002;29:943-947.

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47.  Martino R, Shah A, Romero P, et al. Allogeneic bone marrow transplantation for advanced Waldenstrom's macroglobulinemia. Bone Marrow Transplant. 1999;23:747-749.

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48.  Ueda T, Hatanaka K, Kosugi S, et al. Successful non-myeloablative allogeneic peripheral blood stem cell transplantation (PBSCT) for Waldenström's macroglobulinemia with severe pancytopenia. Bone Marrow Transplant. 2001;28:609-611.

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49.  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.

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50.  Ghobrial IM, Leleu X, Azab AK, et al. Novel therapeutic agents in Waldenstrom's macroglobulinaemia. Clin Lymphoma Myeloma. 2009;9:84-86.

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51.  Banwait R, Weller E, Ropur N, et al. The role of bortezomib-containing regimens in improving response in patients with Waldenstrom macroglobulinemia. Blood. 2011;118:2713.

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52.  Chauhan D, Catley L, Li G, et al. A novel orally active proteosome inhibitor induces apoptosis in multiple myeloma cells with mechanisms distinct from Bortezomib. Cancer Cell. 2005;8:407-419.

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53.  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;154:357-362.

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54.  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.

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55.  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 Waldenström's macroglobulinemia. Clin Cancer Res. 2010;16:1033-1041.

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56.  Rummel MJ, von Gruenhagen U, Niederle N, et al. Bendamustine plus rituximab versus CHOP plus rituximab in the first-line treatment of patients with follicular, indolent and mantle cell lymphomas: results of a randomized phase III study of the Study Group Indolent Lymphomas (STiL). Blood. 2008;112:2596.

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57.  Rummel MJ, Niederle N, Maschmeyer G, et al; Study Group Indolent Lymphomas. 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.

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58.  Ghobrial IM, Banwait R, Poon T, et al. Final results of the phase II trial of single agent panobinostat (LBH589) in relapsed or relapsed/refractory Waldenstrom macroglobulinemia. Blood. 2011;118:2706.

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59.  Furman RR, Eradat H, DiRienzo CG, et al. A phase II trial of ofatumumab in subjects with Waldenstrom's macroglobulinemia. Blood. 2011;118:3701.

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60.  Gopal A, Kahl BS, de Vos S, et al. Mature response data from a phase 2 study of PI3K-delta inhibitor idelalisib in patients with double (rituximab and alkylating agent)-refractory indolent B-cell non-Hodgkin lymphoma (iNHL). Blood (ASH Annual Meeting Abstracts). 2013;122:85.

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61.  US Food and Drug Administration. FDA alerts healthcare professionals about clinical trials with Zydelig (idelalisib) in combination with other cancer medicines. March 2016 http://www.fda.gov/ (last accessed 30 January 2017).

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62.  European Medicines Agency. EMA recommends new safety measures for Zydelig. March 2016. http://www.ema.europa.eu/ (last accessed 30 January 2017).

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63.  Herth I, Hensel M, Rieger M, et al. Pentostatin, cyclophosphamide and rituximab is a safe and effective treatment in patients with Waldenström's macroglobulinemia. Leuk Lymphoma. Leuk Lymphoma. 2015;56:97-102.

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64.  Morel P, Duhamel A, Gobbi P, et al. International prognostic scoring system for Waldenström macroglobulinemia. Blood. 2009;113:4163-4170.

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65.  Kastritis E, Zervas K, Repoussis P, et al. Prognostication in young and old patients with Waldenstrom's macroglobulinemia: importance of the International Prognostic Scoring System and of serum lactate dehydrogenase. Clin Lymphoma Myeloma. 2009;9:50-52.

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66.  Schollkopf C, Kjeldsen L, Bjerrum OW, et al. Rituximab in chronic cold agglutinin disease: a prospective study of 20 patients. Leuk Lymphoma. 2006;47:253-260.

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67.  Chen C, Kouroukis CT, White D, et al. Bortezomib in relapsed or refractory Waldenstrom's macroglobulinemia. Clin Lymphoma Myeloma. 2009;9:74-76.

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68.  Baldini L, Nobile-Orazio E, Guffanti A, et al. Peripheral neuropathy in IgM monoclonal gammopathy and Waldenstrom's macroglobulinemia: a frequent complication in elderly males with low MAG-reactive serum monoclonal component. Am J Hematol. 1994;45:25-31.

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69.  Nobile-Orazio E. Antigenic determinants in IgM paraprotein-related neuropathies. Clin Lymphoma Myeloma. 2009;9:107-109.

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70.  Nobile-Orazio E, Meucci N, Baldini L, et al. Long-term prognosis of neuropathy associated with anti-MAG IgM M-proteins and its relationship with immune therapies. Brain. 2000;123:710-717.

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