Abstract: Ｏｂｊｅｃｔｉｖｅ：This study aimed to assess the safety and efficacy of ixazomib and dexamethasone (ID) in patients with relapsed or refractory light chain amyloidosis(AL).
Ｍｅｔｈｏｄｏｌｏｇｙ：ID were administered to patients with relapsed or refractory AL amyloidosis. The hematology and organ responses and adverse events (AEs) were observed.
Ｒｅｓｕｌｔｓ：18 patients with relapsed or refractory AL amyloidosis were treated with ID. Overall, 15 patients treated for more than 1 cycle were recruited to assess the efficacy. The hematology response rate was 1667％ in 12 patients. The progression rate was 833％ and 75％ patients kept disease stable. Cardiac responses were seen in 20％ of 10 patients, 60％ patients had cardiac progression and 20％ patients kept cardiac stable. Renal responses were seen in 1818％ of 11 patients, 2727％ patients had renal progression and 5455％ patients kept renal function stable. Among the hematology response assessable patients, the hematology response ratio was 50％(1/2) in patients without bortezomib treatment previously, while 1111％(1/9) in patients with bortezomib treatment. Oneyear progressionfree and overall survival rates were 718％ and 714％ respectively. Most common AEs were gastrointestinal reactions and anemia. 1667％ patients experienced grade 3／4 AEs,such as diarrhea and vomit and herpesvirus infection.
Ｃｏｎｃｌｕｓｉｏｎ：ID is an effective treatment in patients with relapsed and refractory AL amyloidosis with manageable toxicity. Better effects could be achieved in patients without proteasome inhibitor treatment previously.