A1 Vertaisarvioitu alkuperäisartikkeli tieteellisessä lehdessä
Cold agglutinin disease revisited: a multinational, observational study of 232 patients
Tekijät: Sigbjørn Berentsen, Wilma Barcellini, Shirley D’Sa, Ulla Randen, Tor Henrik Anderson Tvedt, Bruno Fattizzo, Einar Haukås, Megan Kell, Robert Brudevold, Anders E. A. Dahm, Jakob Dalgaard, Hege Frøen, Randi Fykse Hallstensen, Pernille H. Jæger, Henrik Hjorth-Hansen, Agnieszka Małecka, Markku Oksman, Jürgen Rolke, Mallika Sekhar, Jon Hjalmar Sørbø, Eirik Tjønnfjord, Galina Tsykunova, Geir E. Tjønnfjord
Kustantaja: AMER SOC HEMATOLOGY
Julkaisuvuosi: 2020
Lehti:: Blood
Tietokannassa oleva lehden nimi: BLOOD
Lehden akronyymi: BLOOD
Vuosikerta: 136
Numero: 4
Aloitussivu: 480
Lopetussivu: 488
Sivujen määrä: 9
ISSN: 0006-4971
eISSN: 1528-0020
DOI: https://doi.org/10.1182/blood.2020005674
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. (Blood. 2020;136(4):480-488)
