title: A European collaborative study of cyclophosphamide, bortezomib, and dexamethasone in upfront treatment of systemic AL amyloidosis.
abstract: The combination of cyclophosphamide/bortezomib/dexamethasone (CyBorD) showed early promise of high rates of hematologic responses tempered by studies showing the inability to overcome poor prognosis of advanced cardiac amyloidosis. Large studies are needed to clarify its role in light chain (AL) amyloidosis. We report the outcome of 230 patients treated frontline with CyBorD. Overall hematologic response rate was 60%, and in the 201 patients with measurable disease it was 62%, with 43% achieving at least very good partial response (VGPR). Cardiac response was reached in 17% of patients and renal response in 25% of patients. Advanced cardiac stage III patients (amino-terminal pro-natriuretic peptide type B >8500 ng/L) had lower response rates (42%, ≥ VGPR 23%) and poorer survival (median, 7 months). Nevertheless, hematologic response improved survival in these subjects (67% at 2 years), showing the importance of striving for a good response even in this group.
authors.affiliation: Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, and Department of Molecular Medicine, University of Pavia, Pavia, Italy; and.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Palladini
authors.affiliation: National Amyloidosis Centre, University College London Medical School, Royal Free Hospital Campus, London, United Kingdom.
authors.forename: Sajitha
authors.initials: S
authors.lastname: Sachchithanantham
authors.affiliation: Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, and Department of Molecular Medicine, University of Pavia, Pavia, Italy; and.
authors.forename: Paolo
authors.initials: P
authors.lastname: Milani
authors.affiliation: National Amyloidosis Centre, University College London Medical School, Royal Free Hospital Campus, London, United Kingdom.
authors.forename: Julian
authors.initials: J
authors.lastname: Gillmore
authors.affiliation: Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, and Department of Molecular Medicine, University of Pavia, Pavia, Italy; and.
authors.forename: Andrea
authors.initials: A
authors.lastname: Foli
authors.affiliation: National Amyloidosis Centre, University College London Medical School, Royal Free Hospital Campus, London, United Kingdom.
authors.forename: Helen
authors.initials: H
authors.lastname: Lachmann
authors.affiliation: Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, and Department of Molecular Medicine, University of Pavia, Pavia, Italy; and.
authors.forename: Marco
authors.initials: M
authors.lastname: Basset
authors.affiliation: National Amyloidosis Centre, University College London Medical School, Royal Free Hospital Campus, London, United Kingdom.
authors.forename: Philip
authors.initials: P
authors.lastname: Hawkins
authors.affiliation: Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, and Department of Molecular Medicine, University of Pavia, Pavia, Italy; and.
authors.forename: Giampaolo
authors.initials: G
authors.lastname: Merlini
authors.affiliation: National Amyloidosis Centre, University College London Medical School, Royal Free Hospital Campus, London, United Kingdom.
authors.forename: Ashutosh D
authors.initials: AD
authors.lastname: Wechalekar
mesh_terms: D000682:Amyloid / Q000378:metabolism; D000686:Amyloidosis / Q000188:drug therapy* / Q000378:metabolism; D001897:Boronic Acids / Q000008:administration & dosage*; D000069286:Bortezomib; D003299:Cooperative Behavior; D003520:Cyclophosphamide / Q000008:administration & dosage*; D003907:Dexamethasone / Q000008:administration & dosage*; D004359:Drug Therapy, Combination; D005060:Europe; D006331:Heart Diseases / Q000188:drug therapy / Q000378:metabolism; D006801:Humans; D007674:Kidney Diseases / Q000188:drug therapy / Q000378:metabolism; D011719:Pyrazines / Q000008:administration & dosage*; D016896:Treatment Outcome
chemical_list: D000682:Amyloid; D001897:Boronic Acids; D011719:Pyrazines; C518669:amyloid L, human; D000069286:Bortezomib; D003907:Dexamethasone; D003520:Cyclophosphamide