Efficacy of bortezomib, cyclophosphamide and dexamethasone in treatment-naïve patients with high-risk cardiac AL amyloidosis (Mayo Clinic stage III). Haematologica 2014 Sep;99(9):1479-85
Date
05/27/2014Pubmed ID
24859879Pubmed Central ID
PMC4562537DOI
10.3324/haematol.2014.104109Scopus ID
2-s2.0-84907321417 (requires institutional sign-in at Scopus site) 108 CitationsAbstract
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.
Author List
Jaccard A, Comenzo RL, Hari P, Hawkins PN, Roussel M, Morel P, Macro M, Pellegrin JL, Lazaro E, Mohty D, Mercie P, Decaux O, Gillmore J, Lavergne D, Bridoux F, Wechalekar AD, Venner CPAuthor
Parameswaran Hari MD Adjunct Professor in the Medicine department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
AdultAged
Aged, 80 and over
Amyloidosis
Boronic Acids
Bortezomib
Cardiomyopathies
Cyclophosphamide
Dexamethasone
Drug Therapy, Combination
Female
Follow-Up Studies
Humans
Immunoglobulin Light Chains
Immunoglobulin Light-chain Amyloidosis
Male
Middle Aged
Protein Aggregates
Pyrazines
Severity of Illness Index
Survival Analysis
Treatment Outcome