Waldenström macroglobulinemia: biology, genetics, and therapy

References

• WaldenstromJIncipient myelomatosis or essential hyperglobulinemia with fibrinogenopenia – a new syndrome?Acta Med Scand194411734216247
   Google Scholar
• CampoESwerdlowSHHarrisNLPileriSSteinHJaffeESThe 2008 WHO classification of lymphoid neoplasms and beyond: evolving concepts and practical applicationsBlood2011117195019503221300984
   PubMed Web of Science ®Google Scholar
• LinPMedeirosLJLymphoplasmacytic lymphoma/Waldenstrom macroglobulinemia: an evolving conceptAdv Anat Pathol200512524625516210920
   PubMed Web of Science ®Google Scholar
• KapoorPPaludoJAnsellSMWaldenstrom macroglobulinemia: familial predisposition and the role of genomics in prognosis and treatment selectionCurr Treat Options Oncol20161731626942591
   PubMed Web of Science ®Google Scholar
• San MiguelJFVidrialesMBOcioEImmunophenotypic analysis of Waldenstrom’s macroglobulinemiaSemin Oncol200330218719512720134
   PubMed Web of Science ®Google Scholar
• OwenRGTreonSPAl-KatibAClinicopathological definition of Waldenstrom’s macroglobulinemia: consensus panel recommendations from the Second International Workshop on Waldenstrom’s MacroglobulinemiaSemin Oncol200330211011512720118
   PubMed Web of Science ®Google Scholar
• KriangkumJTaylorBJTreonSPMantMJBelchARPilarskiLMClonotypic IgM V/D/J sequence analysis in Waldenstrom macroglobulinemia suggests an unusual B-cell origin and an expansion of polyclonal B cells in peripheral bloodBlood200410472134214214764523
   PubMed Web of Science ®Google Scholar
• AnsellSMKyleRAReederCBDiagnosis and management of Waldenstrom macroglobulinemia: Mayo stratification of macroglobu-linemia and risk-adapted therapy (mSMART) guidelinesMayo Clin Proc201085982483320702770
   PubMed Web of Science ®Google Scholar
• WangHChenYLiFTemporal and geographic variations of Waldenstrom macroglobulinemia incidenceCancer2012118153793380022139816
   PubMed Web of Science ®Google Scholar
• WangHChenYLiFTemporal and geographic variations of Waldenstrom macroglobulinemia incidence: a large population-based studyCancer2012118153793380022139816
   PubMed Web of Science ®Google Scholar
• GhobrialIMFonsecaRGertzMAPrognostic model for disease-specific and overall mortality in newly diagnosed symptomatic patients with Waldenstrom macroglobulinaemiaBr J Haematol2006133215816416611306
   PubMed Web of Science ®Google Scholar
• CastilloJJOlszewskiAJKananSMeidKHunterZRTreonSPOverall survival and competing risks of death in patients with Waldenstrom macroglobulinaemia: an analysis of the Surveillance, Epidemiology and End Results databaseBr J Haematol20151691818925521528
   PubMed Web of Science ®Google Scholar
• VallumsetlaNPaludoJAnsellSMOutcomes of young patients with Waldenstrom macroglobulinemia (WM)J Clin Oncol (Meeting Abstracts)20143215_suppl Abstract number 8609
   Google Scholar
• KyleRABensonJTLarsonDRProgression in smoldering Waldenström macroglobulinemia: long-term resultsBlood2012119194462446622451426
   PubMed Web of Science ®Google Scholar
• KapoorPPaludoJVallumsetlaNGreippPRWaldenstrom macroglobulinemia: what a hematologist needs to knowBlood Rev201529530131925882617
   PubMed Web of Science ®Google Scholar
• KyleRATherneauTMRajkumarSVA long-term study of prognosis in monoclonal gammopathy of undetermined significanceN Engl J Med2002346856456911856795
   PubMed Web of Science ®Google Scholar
• VarettoniMArcainiLZibelliniSPrevalence and clinical significance of the MYD88 (L265P) somatic mutation in Waldenstrom’s macroglobulinemia and related lymphoid neoplasmsBlood2013121132522252823355535
   PubMed Web of Science ®Google Scholar
• TreonSPHunterZRAggarwalACharacterization of familial Waldenstrom’s macroglobulinemiaAnn Oncol200617348849416357024
   PubMed Web of Science ®Google Scholar
• HanzisCOjhaRPHunterZAssociated malignancies in patients with Waldenstrom’s macroglobulinemia and their kinClin Lymphoma Myeloma Leuk2011111889221454200
   PubMed Web of Science ®Google Scholar
• McMasterMLGoldinLRBaiYGenomewide linkage screen for Waldenström macroglobulinemia susceptibility loci in high-risk familiesAm J Hum Genet200679469570116960805
   PubMed Web of Science ®Google Scholar
• CastilloJJOlszewskiAJHunterZRKananSMeidKTreonSPIncidence of secondary malignancies among patients with Waldenstrom macroglobulinemia: an analysis of the SEER databaseCancer2015121132230223625757851
   PubMed Web of Science ®Google Scholar
• MorraEVarettoniMTedeschiAAssociated cancers in Waldenstrom macroglobulinemia: clues for common genetic predispositionClin Lymphoma Myeloma Leuk201313670070324070824
   PubMed Web of Science ®Google Scholar
• KristinssonSYLandgrenOWhat causes Waldenstrom’s macroglobulinemia: genetic or immune-related factors, or a combination?Clin Lymphoma Myeloma Leuk2011111858721454199
   PubMed Web of Science ®Google Scholar
• Nguyen-KhacFLambertJChapiroEChromosomal aberrations and their prognostic value in a series of 174 untreated patients with Waldenstrom’s macroglobulinemiaHaematologica201398464965423065509
   PubMed Web of Science ®Google Scholar
• BraggioEPhilipsbornCNovakAHodgeLAnsellSFonsecaRMolecular pathogenesis of Waldenstrom’s macroglobulinemiaHaematologica20129791281129022773606
   PubMed Web of Science ®Google Scholar
• BraggioEFonsecaRGenomic abnormalities of Waldenstrom macroglobulinemia and related low-grade B-cell lymphomasClin Lymphoma Myeloma Leuk201313219820123477936
   PubMed Web of Science ®Google Scholar
• Shapiro-ShelefMCalameKRegulation of plasma-cell developmentNat Rev Immunol20055323024215738953
   PubMed Web of Science ®Google Scholar
• GutierrezNCOcioEMde las RivasJGene expression profiling of B lymphocytes and plasma cells from Waldenstrom’s macroglobulinemia: comparison with expression patterns of the same cell counterparts from chronic lymphocytic leukemia, multiple myeloma and normal individualsLeukemia200721354154917252022
   PubMed Web of Science ®Google Scholar
• TreonSPXuLYangGMYD88 L265P somatic mutation in Waldenstrom’s macroglobulinemiaN Engl J Med2012367982683322931316
   PubMed Web of Science ®Google Scholar
• PertesiMGaliaPNazaretNRare circulating cells in familial Waldenstrom macroglobulinemia displaying the MYD88 L265P mutation are enriched by Epstein-Barr virus immortalizationPLoS ONE2015109e013650526352266
   PubMed Web of Science ®Google Scholar
• XuLHunterZRYangGMYD88 L265P in Waldenstrom macroglobulinemia, immunoglobulin M monoclonal gammopathy, and other B-cell lymphoproliferative disorders using conventional and quantitative allele-specific polymerase chain reactionBlood2013121112051205823321251
   PubMed Web of Science ®Google Scholar
• NgoVNYoungRMSchmitzROncogenically active MYD88 mutations in human lymphomaNature2011470733211511921179087
   PubMed Web of Science ®Google Scholar
• JimenezCSebastianEChillonMCMYD88 L265P is a marker highly characteristic of, but not restricted to, Waldenstrom’s macroglobulinemiaLeukemia20132781722172823446312
   PubMed Web of Science ®Google Scholar
• HunterZRXuLYangGThe genomic landscape of Waldenstrom macroglobulinemia is characterized by highly recurring MYD88 and WHIM-like CXCR4 mutations, and small somatic deletions associated with B-cell lymphomagenesisBlood2014123111637164624366360
   PubMed Web of Science ®Google Scholar
• NgoHTLeleuXLeeJSDF-1/CXCR4 and VLA-4 interaction regulates homing in Waldenstrom macroglobulinemiaBlood2008112115015818448868
   PubMed Web of Science ®Google Scholar
• CaoYHunterZRLiuXCXCR4 WHIM-like frameshift and nonsense mutations promote ibrutinib resistance but do not supplant MYD88(L265P)-directed survival signalling in Waldenstrom macroglobulinaemia cellsBr J Haematol2015168570170725371371
   PubMed Web of Science ®Google Scholar
• RoccaroAMSaccoAJimenezCC1013G/CXCR4 acts as a driver mutation of tumor progression and modulator of drug resistance in lymphoplasmacytic lymphomaBlood2014123264120413124711662
   PubMed Web of Science ®Google Scholar
• TreonSTripsasCMeidKIbrutinib in previously treated patients with Waldenstrom’s macroglobulinemia is highly active, produces durable responses, and is impacted by MYD88 and CXCR4 mutation statusHaematologica2015100311
   Web of Science ®Google Scholar
• VajdicCMLandgrenOMcMasterMLMedical history, lifestyle, family history, and occupational risk factors for lymphoplasmacytic lymphoma/Waldenstrom’s macroglobulinemia: the InterLymph Non-Hodgkin Lymphoma Subtypes ProjectJ Natl Cancer Inst Monogr2014201448879725174029
   PubMedGoogle Scholar
• KyleRATherneauTMRajkumarSVLong-term follow-up of IgM monoclonal gammopathy of undetermined significanceBlood2003102103759376412881316
   PubMed Web of Science ®Google Scholar
• MorelPDuhamelAGobbiPInternational prognostic scoring system for Waldenstrom macroglobulinemiaBlood2009113184163417019196866
   PubMed Web of Science ®Google Scholar
• KastritisEKyrtsonisMCHadjiharissiEValidation of the International Prognostic Scoring System (IPSS) for Waldenstrom’s macroglobulinemia (WM) and the importance of serum lactate dehydrogenase (LDH)Leuk Res201034101340134320447689
   PubMed Web of Science ®Google Scholar
• TreonSPCaoYXuLYangGLiuXHunterZRSomatic mutations in MYD88 and CXCR4 are determinants of clinical presentation and overall survival in Waldenstrom macroglobulinemiaBlood2014123182791279624553177
   PubMed Web of Science ®Google Scholar
• OlszewskiAJFallahJEatonCBTreonSPCastilloJJThe evolution of management and survival outcomes of Waldenström macroglobulinemia (WM) in the United States (US)Blood201512623882882
   Web of Science ®Google Scholar
• GhobrialIMFonsecaRGreippPRInitial immunoglobulin M ‘flare’ after rituximab therapy in patients diagnosed with Waldenstrom macroglobulinemiaCancer2004101112593259815493038
   PubMed Web of Science ®Google Scholar
• GertzMAAbonourRHeffnerLTGreippPRUnoHRajkumarSVClinical value of minor responses after 4 doses of rituximab in Waldenstrom macroglobulinaemia: a follow-up of the Eastern Cooperative Oncology Group E3A98 trialBr J Haematol2009147567768019751237
   PubMed Web of Science ®Google Scholar
• TreonSPEmmanouilidesCKimbyEExtended rituximab therapy in Waldenstrom’s macroglobulinemiaAnn Oncol200516113213815598950
   PubMed Web of Science ®Google Scholar
• GertzMARueMBloodEKaminerLSVesoleDHGreippPRMulticenter phase 2 trial of rituximab for Waldenstrom macroglobulinemia (WM): an Eastern Cooperative Oncology Group Study (E3A98)Leuk Lymphoma200445102047205515370249
   PubMed Web of Science ®Google Scholar
• TreonSPHanzisCManningRJMaintenance rituximab is associated with improved clinical outcome in rituximab naïve patients with Waldenstrom macroglobulinaemia who respond to a rituximab-containing regimenBr J Haematol2011154335736221615385
   PubMed Web of Science ®Google Scholar
• RummelMJAl-BatranSEKimSZBendamustine plus rituximab is effective and has a favorable toxicity profile in the treatment of mantle cell and low-grade non-Hodgkin’s lymphomaJ Clin Oncol200523153383338915908650
   PubMed Web of Science ®Google Scholar
• RummelMJNiederleNMaschmeyerGBendamustine 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 trialLancet201338198731203121023433739
   PubMed Web of Science ®Google Scholar
• DimopoulosMAAnagnostopoulosAKyrtsonisMCPrimary treatment of Waldenstrom macroglobulinemia with dexamethasone, rituximab, and cyclophosphamideJ Clin Oncol200725223344334917577016
   PubMed Web of Science ®Google Scholar
• KastritisEGavriatopoulouMKyrtsonisMCDexamethasone, rituximab, and cyclophosphamide as primary treatment of Waldenstrom macroglobulinemia: final analysis of a phase 2 studyBlood2015126111392139426359434
   PubMed Web of Science ®Google Scholar
• TreonSPHunterZRMatousJMulticenter clinical trial of bortezomib in relapsed/refractory Waldenstrom’s macroglobulinemia: results of WMCTG Trial 03-248Clin Cancer Res200713113320332517545538
   PubMed Web of Science ®Google Scholar
• GhobrialIMXieWPadmanabhanSPhase II trial of weekly bortezomib in combination with rituximab in untreated patients with Waldenstrom macroglobulinemiaAm J Hematol201085967067420652865
   PubMed Web of Science ®Google Scholar
• GhobrialIMHongFPadmanabhanSPhase II trial of weekly bortezomib in combination with rituximab in relapsed or relapsed and refractory Waldenstrom macroglobulinemiaJ Clin Oncol20102881422142820142586
   PubMed Web of Science ®Google Scholar
• DimopoulosMAGarcia-SanzRGavriatopoulouMPrimary 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)Blood2013122193276328224004667
   PubMed Web of Science ®Google Scholar
• TreonSPIoakimidisLSoumeraiJDPrimary therapy of Waldenstrom macroglobulinemia with bortezomib, dexamethasone, and rituximab: WMCTG clinical trial 05-180J Clin Oncol200927233830383519506160
   PubMed Web of Science ®Google Scholar
• GopalAKKahlBSde VosSPI3Kdelta inhibition by idelalisib in patients with relapsed indolent lymphomaN Engl J Med2014370111008101824450858
   PubMed Web of Science ®Google Scholar
• GerecitanoJFRobertsAWSeymourJFA Phase 1 study of venetoclax (ABT-199 / GDC-0199) monotherapy in patients with relapsed/refractory non-Hodgkin lymphomaBlood201512623254
   Web of Science ®Google Scholar
• CaoYYangGHunterZRThe BCL2 antagonist ABT-199 triggers apoptosis, and augments ibrutinib and idelalisib mediated cytotoxicity in CXCR4 wild-type and CXCR4 WHIM mutated Waldenstrom macroglobulinaemia cellsBr J Haematol2015170113413825582069
   PubMed Web of Science ®Google Scholar
• AnagnostopoulosAHariPNPerezWSAutologous or allogeneic stem cell transplantation in patients with Waldenstrom’s macroglobulinemiaBiol Blood Marrow Transplant200612884585416864055
   PubMed Web of Science ®Google Scholar
• GilleeceMHPearceRLinchDCThe outcome of haemopoietic stem cell transplantation in the treatment of lymphoplasmacytic lymphoma in the UK: a British Society Bone Marrow Transplantation studyHematology200813211912718616880
   PubMed Web of Science ®Google Scholar
• KyriakouCCanalsCSibonDHigh-dose therapy and autologous stem-cell transplantation in Waldenstrom macroglobulinemia: the Lymphoma Working Party of the European Group for Blood and Marrow TransplantationJ Clin Oncol201028132227223220368570
   PubMed Web of Science ®Google Scholar
• PaludoJGertzMAnsellSImpact of day-100 response post autologous stem cell transplantation (ASCT) in Waldenstrom macroglobulinemia (WM)Biol Blood Marrow Transplant2016223S130
   Web of Science ®Google Scholar
• KyriakouCCanalsCCornelissenJJAllogeneic stem-cell transplantation in patients with Waldenstrom macroglobulinemia: report from the Lymphoma Working Party of the European Group for Blood and Marrow TransplantationJ Clin Oncol201028334926493420956626
   PubMed Web of Science ®Google Scholar
• BuggyJJEliasLBruton tyrosine kinase (BTK) and its role in B-cell malignancyInt Rev Immunol201231211913222449073
   PubMed Web of Science ®Google Scholar
• CameronFSanfordMIbrutinib: first global approvalDrugs201474226327124464309
   PubMed Web of Science ®Google Scholar
• AdvaniRHBuggyJJSharmanJPBruton tyrosine kinase inhibitor ibrutinib (PCI-32765) has significant activity in patients with relapsed/refractory B-cell malignanciesJ Clin Oncol2013311889423045577
   PubMed Web of Science ®Google Scholar
• TreonSPTripsasCKMeidKIbrutinib in previously treated Waldenstrom’s macroglobulinemiaN Engl J Med2015372151430144025853747
   PubMed Web of Science ®Google Scholar
• TreonSPXuLHunterZMYD88 mutations and response to ibrutinib in Waldenstrom’s macroglobulinemiaN Engl J Med20153736584586
   PubMed Web of Science ®Google Scholar
• QuekLSBolenJWatsonSPA role for Bruton’s tyrosine kinase (Btk) in platelet activation by collagenCurr Biol1998820113711409778529
   PubMed Web of Science ®Google Scholar
• KamelSHortonLYsebaertLIbrutinib inhibits collagen-mediated but not ADP-mediated platelet aggregationLeukemia201529478378725138588
   PubMed Web of Science ®Google Scholar
• YsebaertLLevadeMCedricGElucidation of mild bleeding disorders reported under ibrutinib (Imbruvica(R)) therapy: implications for optimal clinical managementBlood20141242132963296
   Web of Science ®Google Scholar
• DimopoulosMATrotmanJTedeschiAIbrutinib therapy in rituximab-refractory patients with Waldenström’s macroglobulinemia: initial results from an international, multicenter, open-label phase 3 substudy (iNNOVATETM)Blood20151262327452745
   Web of Science ®Google Scholar
• ZhangSQSmithSMZhangSYLynn WangYMechanisms of ibrutinib resistance in chronic lymphocytic leukaemia and non-Hodgkin lymphomaBr J Haematol2015170444545625858358
   PubMed Web of Science ®Google Scholar
• FurmanRRChengSLuPIbrutinib resistance in chronic lymphocytic leukemiaN Engl J Med2014370242352235424869597
   PubMed Web of Science ®Google Scholar
• ChironDDi LibertoMMartinPCell-cycle reprogramming for PI3K inhibition overrides a relapse-specific C481S BTK mutation revealed by longitudinal functional genomics in mantle cell lymphomaCancer Discov2014491022103525082755
   PubMed Web of Science ®Google Scholar
• CaoYHunterZRLiuXThe 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 macroglobulinemiaLeukemia201529116917624912431
   PubMed Web of Science ®Google Scholar
• BeiderKDarash-YahanaMBlaierOCombination of imatinib with CXCR4 antagonist BKT140 overcomes the protective effect of stroma and targets CML in vitro and in vivoMol Cancer Ther20141351155116924502926
   PubMed Web of Science ®Google Scholar
• ScalaSMolecular pathways: targeting the CXCR4-CXCL12 axis-untapped potential in the tumor microenvironmentClin Cancer Res201521194278428526199389
   PubMed Web of Science ®Google Scholar
• CaoYYangGHunterZRThe BCL2 antagonist ABT-199 triggers apoptosis, and augments ibrutinib and idelalisib mediated cytotoxicity in CXCR4 wild-type and CXCR4 WHIM mutated Waldenstrom macroglobulinaemia cellsBr J Haematol2015170113413825582069
   PubMed Web of Science ®Google Scholar
• VakkalankaSNyayapathySViswanadhaSAddition of RP6530, a dual PI3Kδ/γ inhibitor, overcomes ibrutinib resistance in DLBCL cells in vitroBlood20141242144974497
   Web of Science ®Google Scholar
• McDermottDHLiuQVelezDA phase 1 clinical trial of long-term, low-dose treatment of WHIM syndrome with the CXCR4 antagonist plerixaforBlood2014123152308231624523241
   PubMed Web of Science ®Google Scholar