Bortezomib combined with autologous stem cell transplantation overcomes cytogenetic abnormalities in AL amyloidosis.

Cytogenetic abnormalities are associated with poor prognosis in systemic light chain (AL) amyloidosis, but evidence on treatment options that might overcome this adverse impact remains limited. In this study, we investigated whether the combination of bortezomib and autologous stem cell transplantation (ASCT) could overcome the adverse prognosis of AL amyloidosis with cytogenetic abnormalities. The study included 134 patients. The 52 patients treated with a bortezomib-based regimen and ASCT were defined as Group A, and the 82 patients who received a bortezomib-based regimen without ASCT were defined as Group B. Cytogenetic abnormalities were present in 73 patients, and included IgH rearrangement (71.2%), + 1q21 (46.6%), del13q (37%), and del17p (6.8%). Hematological response and overall survival (OS) were better in Group A than in Group B, especially among patients with cytogenetic abnormalities. Within Group A, there was no difference in hematological response, progression-free survival (PFS), or OS between patients with and without cytogenetic abnormalities. Within Group B, IgH rearrangement was associated with inferior hematological response and OS. In conclusion, our study demonstrated that a bortezomib-based regimen combined with ASCT can overcome the adverse prognosis of AL amyloidosis with cytogenetic abnormalities.