title: Waldenström macroglobulinaemia: presenting features and outcome in a series with 217 cases.
abstract: In this report we analyse the presenting features of a series of patients diagnosed with Waldenström macroglobulinaemia (WM) in Spain over the last 10 years. Criteria for diagnosis required a serum monoclonal IgM protein > or = 30 g/l and > 20% bone marrow lymphocytes. Two hundred and seventeen patients were included in the study, with a median age of 69 years and male/female ratio of 2:1. The most common symptoms at diagnosis were anaemia (38%), hyperviscosity (31%), B symptoms (23%), bleeding (23%) and neurological symptoms (22%). Sixty-one patients (27%) were asymptomatic at diagnosis and, to date, 32 of them have not received chemotherapy. Variables predicting a shorter survival free of therapy were haemoglobin < 12.5 g/dl and high beta2microglobulin (beta2M). The 83% of patients who did receive treatment were distributed as follows: chlorambucil/prednisone (43%), intermittent chlorambucil (11%), continuous chlorambucil (26%), cyclophosphamide/vincristine/prednisone (COP, 13.5%) and other (6.5%). Response to therapy was complete in 2%, partial in 48% and minor in 10%. Finally, 28% and 13% of patients presented stable and progressive disease, respectively, which was more common among patients treated with COP. Progression-free survival was 43% at 5 years, with three independent predictors for shorter progression-free survival (PFS): COP treatment, age > 65 and B symptoms at diagnosis. The 10-year projected overall survival (OS) was 55%. The two most frequent causes of death were development of second malignancies (31%), or infections (19%). The two main variables predicting a poor OS were hyperviscosity and high beta2M. In summary, this study favours the use of chlorambucil-based therapy as the standard treatment for WM, and describes a subset of patients who should be considered as suffering a smouldering form and who therefore do not require treatment for a long period of time.
authors.affiliation: Department of Haematology, University Hospital of Salamanca, Paseo de San Vicente, Salamanca, Spain. ramongarcia@aehh.org
authors.forename: R
authors.initials: R
authors.lastname: García-Sanz
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authors.initials: S
authors.lastname: Montoto
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authors.lastname: Torrequebrada
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authors.lastname: Sureda
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authors.lastname: Rodríguez-García
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authors.lastname: Massó
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authors.lastname: Pérez-Aliaga
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authors.initials: MD
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authors.lastname: Navarro
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authors.lastname: Moreno
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authors.lastname: Toledo
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authors.lastname: Alonso
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authors.lastname: Besses
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authors.lastname: Besalduch
authors.forename: I
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authors.lastname: Jarque
authors.forename: P
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authors.lastname: Salama
authors.forename: J A
authors.initials: JA
authors.lastname: Rivas
authors.forename: B
authors.initials: B
authors.lastname: Navarro
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mesh_terms: D000368:Aged; D000888:Antibodies, Anti-Idiotypic / Q000032:analysis*; D000911:Antibodies, Monoclonal / Q000097:blood; D018906:Antineoplastic Agents, Alkylating / Q000627:therapeutic use; D002699:Chlorambucil / Q000627:therapeutic use; D005260:Female; D006801:Humans; D007075:Immunoglobulin M / Q000276:immunology*; D015994:Incidence; D018655:Lymphocyte Count; D008214:Lymphocytes / Q000097:blood*; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D013030:Spain / Q000453:epidemiology; D015996:Survival Rate; D016896:Treatment Outcome; D008258:Waldenstrom Macroglobulinemia / Q000175:diagnosis* / Q000453:epidemiology / Q000628:therapy*; D001613:beta 2-Microglobulin / Q000032:analysis
chemical_list: D000888:Antibodies, Anti-Idiotypic; D000911:Antibodies, Monoclonal; D018906:Antineoplastic Agents, Alkylating; D007075:Immunoglobulin M; D001613:beta 2-Microglobulin; D002699:Chlorambucil