We retrospectively studied 232 patients with cold agglutinin disease (CAD) at 24 centers in 5 countries. In Norway and a northern region of Italy, the study was close to being population-based. For the first time, we demonstrate fourfold differences between cold and warmer climates regarding prevalence (20 vs 5 cases/million) and incidence (1.9 vs 0.48 cases/million per year). Mean baseline hemoglobin level was 9.3 g/dL, but 27% had hemoglobin <8 g/dL. Identification of typical features of CAD-associated lymphoproliferative disorder in the bone marrow was greatly increased by centralized biopsy assessment. CAD seems to be associated with a slightly increased risk of venous thrombosis. This work includes a follow-up study of therapies, focusing on the long-term outcomes of the rituximab plus bendamustine and rituximab plus fludarabine regimens. Rituximab plus bendamustine therapy resulted in responses in 35 (78%) of 45 patients; 24 (53%) achieved complete response. Interestingly, these rates were still higher than observed in the original (2017) prospective trial, and we also found a shift toward deeper responses with time. This is explained by the prolonged time to response seen in many patients, probably related to long-lived plasma cells. In patients responding to rituximab-bendamustine, median response duration was not reached after 88 months, and estimated 5-year sustained remission was 77%. The regimen appeared safe regarding late-occurring malignancies. Rituximab plus fludarabine therapy seems to carry a higher risk of long-term adverse effects.
Cold agglutinin disease revisited: a multinational, observational study of 232 patients / S. Berentsen, W. Barcellini, S. D'Sa, U. Randen, T.H.A. Tvedt, B. Fattizzo, E. Haukas, M. Kell, R. Brudevold, A.E.A. Dahm, J. Dalgaard, H. Froen, R.F. Hallstensen, P.H. Jaeger, H. Hjorth-Hansen, A. Malecka, M. Oksman, J. Rolke, M. Sekhar, J.H. Sorbo, E. Tjonnfjord, G. Tsykunova, G.E. Tjonnfjord. - In: BLOOD. - ISSN 1528-0020. - 136:4(2020 Aug 01), pp. 480-488.
|Titolo:||Cold agglutinin disease revisited: a multinational, observational study of 232 patients|
|Settore Scientifico Disciplinare:||Settore MED/15 - Malattie del Sangue|
|Data di pubblicazione:||1-ago-2020|
|Digital Object Identifier (DOI):||http://dx.doi.org/10.1182/blood.2020005674|
|Appare nelle tipologie:||01 - Articolo su periodico|