伊沙佐米联合地塞米松治疗难治复发轻链型淀粉样变性的临床观察

A retrospective study of ixazomib and dexamethasone in patients with relapsed and refractory light chain amyloidosis

目的:观察伊沙佐米联合地塞米松(ID)方案治疗系统性轻链(AL)型淀粉样变性的疗效及安全性。方法:回顾性分析2018年7月至2020年9月间采用ID方案的难治复发的AL型淀粉样变性患者的临床资料,观察其血液学、器官反应及不良反应。结果:18例难治复发的AL型淀粉样变性患者中,15例纳入疗效分析。12例可评估血液学疗效的患者中,16.67%取得血液学缓解,8.33%血液学进展,75%维持稳定。10例心脏受累患者中20%缓解,60%进展,20%维持稳定。11例可评估的肾脏受累患者中18.18%缓解,54.55%维持稳定,27.27%进展。其中,前期治疗中未使用硼替佐米的患者在接受ID治疗后反应率达50%(1/2),而使用过硼替佐米的患者治疗反应率仅11.11%(1/9)。1年总体生存率及无进展生存率分别为71.4%和71.8%。主要不良反应为消化道反应及贫血,3/4级不良反应发生率16.67%,为腹泻、呕吐以及疱疹病毒感染。结论:ID方案可作为复发难治性AL型淀粉样变性的候选治疗方案,在前期未接受蛋白酶体抑制剂的患者中更可能获得治疗反应,在难治复发性AL型淀粉样变性患者中有一定疗效。且ID方案的安全性较好,不良反应可控。

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.