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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 PMID: 24859879 PMCID: PMC4562537

Pubmed ID

24859879

DOI

10.3324/haematol.2014.104109

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.

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 CP

Author

Parameswaran Hari MD Chief, Professor in the Medicine department at Medical College of Wisconsin




Scopus

2-s2.0-84907321417   55 Citations

MESH terms used to index this publication - Major topics in bold

Adult
Aged
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
jenkins-FCD Prod-310 bff9d975ec7f2d302586822146c2801dd4449aad