Abstract
BackgroundCyclophosphamide, thalidomide, and dexamethasone (CTD) or bortezomib and dexamethasone (BDex) show substantial efficacy in patients with amyloid light-chain (AL) amyloidosis, especially in Chinese patients. Currently, both regimens are recommended as primary treatment options for AL amyloidosis, but no comparative study has been reported. MethodsWe retrospectively evaluated the outcomes of 81 AL patients who received CTD (n = 42) or BDex (n = 39) and used Mayo stage 2012 to match 26 pairs of patients. ResultsIn the whole cohort, the overall hematologic responses were 86% vs 91% in the CTD and BDex groups, including a complete response of 56% vs 71% based on an intention-to-treat (ITT) analysis. One- and 2-year overall survival (OS) was 90.2% and 81.7% with CTD, and 87.6% and 82.7% with BDex. After matching, BDex regimen induced a significantly deeper and more rapid hematologic response over CTD, but no statistically significant difference in OS (ITT analysis, P = 0.24; 6-month landmark analysis, P = 0.48). Cardiac response rates were similar, while there was a trend for higher renal responses in patients treated with BDex (68% vs 44%, P = 0.09). Additionally, BDex was associated with significantly improved survival in patients with advanced disease (Mayo stage III or worse; P = 0.009). Patients treated with BDex reported more episodes of severe hematologic toxicity and diarrhea. ConclusionsCTD and BDex are effective treatments for Chinese patients with AL amyloidosis, but BDex regimen appears superior to CTD in achieving a more rapid and deeper clonal response, and in improving OS in patients with advanced disease.
Highlights
Cyclophosphamide, thalidomide and dexamethasone (CTD) or bortezomib and dexamethasone (BDex) show substantial efficacy in patients with amyloid light-chain (AL) amyloidosis, especially in Chinese patients
CTD and BDex are effective treatments for Chinese patients with AL amyloidosis, but BDex regimen appears superior to CTD in achieving a more rapid and deeper clonal response, and in improving overall survival (OS) in patients with advanced disease
Most of the parameters were comparable between the two treatment groups but variables associated with cardiac involvement, N-terminal pro-B-type natriuretic peptide (NT-proBNP), troponin T (TnT), and intraventricular septum (IVS), and clonal depth measured as the difference between involved and uninvolved free light chain were significantly higher in the BDex group (Table 1)
Summary
Cyclophosphamide, thalidomide and dexamethasone (CTD) or bortezomib and dexamethasone (BDex) show substantial efficacy in patients with amyloid light-chain (AL) amyloidosis, especially in Chinese patients. Both regimens are recommended as primary treatment options for AL amyloidosis, but no comparative study has been reported. AL amyloidosis is the most frequently diagnoses type of systemic amyloid disease in China, and the incidence is increasing, possibly due to a combination of improved diagnostic techniques and an aging population.[8] developing an optimal therapeutic schedule for Chinese AL patients to reduce mortality and improve quality of life is of great importance
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