Bortezomib-Containing Regimens for the Treatment of Newly Diagnosed and Relapsed Amyloid Light Chain Amyloidosis: A Single-Center Experience

Bortezomib exhibits unprecedented single-agent activity in AL amyloidosis. Here, we performed a review of the use of bortezomib containing regimens to assess the rapidity and quality of response. After a median of 4 cycles, a HR was seen in 49/52 cases (94.2%) demonstrating that bortezomib is a safe and well-tolerated therapy for AL patients showing rapid HR and cardiac responses. Background: The proteasome is an exciting target for the development of novel anticancer therapies. Recent evidence has suggested that bortezomib, a dipeptide boronate proteasome inhibitor, exhibits unprecedented single-agent activity in amyloid light chain (AL) amyloidosis. Patients and Methods: We performed a retrospective review of the use of bortezomib-containing regimens to assess the rapidity and quality of response at our institution. Results: A total of 52 patients with documented newly diagnosed and relapsed AL amyloidosis treated with bortezomib-containing regimens were identified from our institutional database. After a median of 4 cycles (range, 1-22 cycles), a hematologic response was seen in 49 patients (94.2%), including a complete response in 15 (28.8%), a very good partial response in 25 (48.1%), and a partial response in 9 (17.3%). At 6 weeks, 37 patients had already achieved a partial response. An organ response at 6 months was documented in 31 patients (59.6%). With respect to the cardiac response, a > 30% decrease in N-terminal pro-hormone brain natriuretic peptide (NT-proBNP) was observed in 17 of 35 evaluable patients (48.6%; NT-proBNP > 650 ng/L) at a median of 6 months. Overall survival was shorter for the patients with NT-proBNP > 5000 ng/L and for those who achieved less than a very good partial response. Conclusion: Bortezomib is a safe and well-tolerated therapy for patients with AL amyloidosis with a rapid hematologic response and cardiac response, as assessed by the NT-proBNP level. (C) 2016 Elsevier Inc. All rights reserved.