title: Efficacy of bortezomib, cyclophosphamide and dexamethasone in treatment-naïve patients with high-risk cardiac AL amyloidosis (Mayo Clinic stage III).
abstract: Bortezomib is an active agent in AL amyloidosis and responses to this drug in combination with cyclophosphamide and dexamethasone are both rapid and deep. Here we present an international, multicenter series of 60 patients with Mayo Clinic stage III cardiac amyloidosis to assess the impact of this regimen in improving outcomes in this poor-risk group. The median follow-up for the entire cohort is 11.8 months. The overall response rate was 68%. In a landmark analysis, examining patients who survived more than 3 months, the overall response rate was 86%. A cardiac response was seen in 32% of patients. The estimated 1-year survival rate for the whole cohort was 57% and 24 patients (40%) died while on therapy. Although unable to save the poorest risk patients, the combination of bortezomib, cyclophosphamide and dexamethasone can achieve a high number of hematologic and cardiac responses, likely improving overall survival and justifying a prospective trial.
authors.affiliation: National Amyloidosis Center and Hematology Unit, CHU Limoges, France arnaud.jaccard@chu-limoges.fr.
authors.forename: Arnaud
authors.initials: A
authors.lastname: Jaccard
authors.affiliation: Tufts Medical Center, Boston, MA, USA.
authors.forename: Raymond L
authors.initials: RL
authors.lastname: Comenzo
authors.affiliation: Medical College of Wisconsin, Milwaukee, WI, USA.
authors.forename: Parameswaran
authors.initials: P
authors.lastname: Hari
authors.affiliation: Centre for Amyloidosis and Acute Phase Proteins, University College London Medical School, UK.
authors.forename: Philip N
authors.initials: PN
authors.lastname: Hawkins
authors.affiliation: Hematology Unit, CHU Toulouse, France.
authors.forename: Murielle
authors.initials: M
authors.lastname: Roussel
authors.affiliation: Hematology Unit, CH Lens, France.
authors.forename: Pierre
authors.initials: P
authors.lastname: Morel
authors.affiliation: Hematology Unit, CHU Caen, France.
authors.forename: Margaret
authors.initials: M
authors.lastname: Macro
authors.affiliation: Internal Medicine Units, CHU Bordeaux, France.
authors.forename: Jean-Luc
authors.initials: JL
authors.lastname: Pellegrin
authors.affiliation: Internal Medicine Units, CHU Bordeaux, France.
authors.forename: Estibaliz
authors.initials: E
authors.lastname: Lazaro
authors.affiliation: National Amyloidosis Center and Cardiology Unit, CHU Limoges, France.
authors.forename: Dania
authors.initials: D
authors.lastname: Mohty
authors.affiliation: Internal Medicine Units, CHU Bordeaux, France.
authors.forename: Patrick
authors.initials: P
authors.lastname: Mercie
authors.affiliation: Internal Medicine Unit, CHU Rennes, France.
authors.forename: Olivier
authors.initials: O
authors.lastname: Decaux
authors.affiliation: Centre for Amyloidosis and Acute Phase Proteins, University College London Medical School, UK.
authors.forename: Julian
authors.initials: J
authors.lastname: Gillmore
authors.affiliation: National Amyloidosis Center and Hematology Unit, CHU Limoges, France.
authors.forename: David
authors.initials: D
authors.lastname: Lavergne
authors.affiliation: Nephrology Unit, CHU Poitiers, France.
authors.forename: Frank
authors.initials: F
authors.lastname: Bridoux
authors.affiliation: Centre for Amyloidosis and Acute Phase Proteins, University College London Medical School, UK.
authors.forename: Ashutosh D
authors.initials: AD
authors.lastname: Wechalekar
authors.affiliation: Centre for Amyloidosis and Acute Phase Proteins, University College London Medical School, UK Cross Cancer Institute, University of Alberta, Edmonton, Canada.
authors.forename: Christopher P
authors.initials: CP
authors.lastname: Venner
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000686:Amyloidosis / Q000150:complications / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D001897:Boronic Acids / Q000627:therapeutic use*; D000069286:Bortezomib; D009202:Cardiomyopathies / Q000150:complications / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D003520:Cyclophosphamide / Q000627:therapeutic use*; D003907:Dexamethasone / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000097:blood / Q000737:chemistry; D000075363:Immunoglobulin Light-chain Amyloidosis; D008297:Male; D008875:Middle Aged; D066329:Protein Aggregates; D011719:Pyrazines / Q000627:therapeutic use*; D012720:Severity of Illness Index; D016019:Survival Analysis; D016896:Treatment Outcome
chemical_list: D001897:Boronic Acids; D007147:Immunoglobulin Light Chains; D066329:Protein Aggregates; D011719:Pyrazines; D000069286:Bortezomib; D003907:Dexamethasone; D003520:Cyclophosphamide