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Multicenter Study
. 2014 Sep;99(9):1479-85.
doi: 10.3324/haematol.2014.104109. Epub 2014 May 23.

Efficacy of Bortezomib, Cyclophosphamide and Dexamethasone in Treatment-Naïve Patients With High-Risk Cardiac AL Amyloidosis (Mayo Clinic Stage III)

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Free PMC article
Multicenter Study

Efficacy of Bortezomib, Cyclophosphamide and Dexamethasone in Treatment-Naïve Patients With High-Risk Cardiac AL Amyloidosis (Mayo Clinic Stage III)

Arnaud Jaccard et al. Haematologica. .
Free PMC article

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.

Figures

Figure 1.
Figure 1.
Overall survival for the whole cohort of patients.
Figure 2.
Figure 2.
Overall survival according to hematologic responses. CR: complete response; VGPR: very good partial response; PR: partial response; NR: no response.
Figure 3.
Figure 3.
Overall survival for patients in the landmark analysis.
Figure 4.
Figure 4.
Overall survival stratified by serum NT-proBNP values (A) and serum NT-proBNP or BNP values (B).

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