The efficacy and safety of bortezomib-based chemotherapy for immunoglobulin light chain amyloidosis: A systematic review and meta-analysis

Background: The role of bortezomib in the treatment of immunoglobulin light chain (AL) amyloidosis is not well defined. We performed this meta-analysis to evaluate the efficacy and safety of bortezomib-based regimens in patients with AL amyloidosis who are not eligible for or refuse autologous stem cell transplantation.

Methods: A systematic search of Medline, Embase, and the Cochrane Library was conducted to identify related studies.

Results: Twenty-four studies with 1238 patients were included. The pooled overall response rate (ORR) and complete hematological response rate (CHR) were 0.72 (95% CI, 0.67-0.77) and 0.35 (95% CI, 0.30-0.40), respectively. Bortezomib significantly improved the outcome of ORR compared to other regimens (RR 1.28, 95% CI, 1.04-1.57, P = .02). Similar results were observed in CHR (RR 1.90, 95% CI, 1.45-2.50, P < .001) and cardiac response (RR 2.03, 95% CI, 1.31-3.13, P = .002), but not in overall survival (HR 0.82, 95% CI, 0.62-1.09, P = .17). In addition, once-weekly bortezomib was associated with improved overall survival compared with twice-weekly bortezomib (HR 0.52, 95% CI, 0.27-0.99, P = .05). Peripheral neuropathy was the most widely reported adverse event. Incorporation of bortezomib into the standard melphalan + dexamethasone setting showed a trend of increased serious adverse events, though this was not statistically significant (RR 1.29, 95% CI, 0.95-1.75, P = .10).

Conclusions: Current evidence indicates that bortezomib-based regimens might be effective and safe therapies for patients with AL amyloidosis. There is a great need to conduct more well-designed randomized controlled trials to provide high-quality evidence.