Efficacy of bortezomib, cyclophosphamide and dexamethasone in treatment-naïve patients with high-risk cardiac AL amyloidosis (Mayo Clinic stage III)
Arnaud Jaccard,
Raymond L. Comenzo,
Parameswaran Hari,
Philip N. Hawkins,
Murielle Roussel,
Pierre Morel,
Margaret Macro,
Jean-Luc Pellegrin,
Estibaliz Lazaro,
Dania Mohty,
Patrick Mercie,
Olivier Decaux,
Julian Gillmore,
David Lavergne,
Frank Bridoux,
Ashutosh D. Wechalekar,
Christopher P. Venner
Affiliations
Arnaud Jaccard
National Amyloidosis Center and Hematology Unit, CHU Limoges, France
Raymond L. Comenzo
Tufts Medical Center, Boston, MA, USA
Parameswaran Hari
Medical College of Wisconsin, Milwaukee, WI, USA
Philip N. Hawkins
Centre for Amyloidosis and Acute Phase Proteins, University College London Medical School, UK
Murielle Roussel
Hematology Unit, CHU Toulouse, France
Pierre Morel
Hematology Unit, CH Lens, France
Margaret Macro
Hematology Unit, CHU Caen, France
Jean-Luc Pellegrin
Internal Medicine Units, CHU Bordeaux, France
Estibaliz Lazaro
Internal Medicine Units, CHU Bordeaux, France
Dania Mohty
National Amyloidosis Center and Cardiology Unit, CHU Limoges, France
Patrick Mercie
Internal Medicine Units, CHU Bordeaux, France
Olivier Decaux
Internal Medicine Unit, CHU Rennes, France
Julian Gillmore
Centre for Amyloidosis and Acute Phase Proteins, University College London Medical School, UK
David Lavergne
National Amyloidosis Center and Hematology Unit, CHU Limoges, France
Frank Bridoux
Nephrology Unit, CHU Poitiers, France
Ashutosh D. Wechalekar
Centre for Amyloidosis and Acute Phase Proteins, University College London Medical School, UK
Christopher P. Venner
Centre for Amyloidosis and Acute Phase Proteins, University College London Medical School, UK;Cross Cancer Institute, University of Alberta, Edmonton, Canada
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.