Zhang Yuanyuan, Guo Jinzhou, Chen Wencui, Zhao Liang, Huang Xianghua
Southeast University School of Medicine, Nanjing, 210009, China.
National Clinical Research Center of Kidney Diseases, Jinling Hospital, Nanjing University School of Medicine, Nanjing, 210016, China.
Bone Marrow Transplant. 2024 Mar;59(3):350-358. doi: 10.1038/s41409-023-02185-z. Epub 2023 Dec 26.
Relapse after ASCT is an important factor affecting the long-term prognosis of patients with AL amyloidosis. However, the risk factors of relapse are unknown and there are limited studies on treatment outcomes of these patients. We retrospectively reviewed 170 patients with AL amyloidosis who underwent ASCT between 2010 and 2021. Seventy-six patients confirmed as relapse and the median time from ASCT to relapse was 39 months. On multivariate analysis of variables before and after ASCT, lambda restricted, dFLC >30 mg/L pre ASCT, reduced dose melphalan and dFLC >10 mg/L at 6 months after ASCT were independent risk factors for relapse, and achieving CR after induction therapy and renal response after ASCT were protective factors. Most relapsed patients were treated with bortezomib-based regimens (50%) followed by daratumumab-based regimens (22.2%) and other chemotherapy regimens (13.9%). The overall hematological response in evaluable patients was 68.2% with 56.8% achieving CR/VGPR. The median PFS and OS from post-transplant relapse were 25 months and 81 months, respectively. Patients receiving bortezomib or daratumumab showed a better survival compared to other chemotherapy regimens. In conclusion, this study identified independent risk factors of post-transplant relapse and demonstrated the superiority of bortezomib or daratumumab treatment for these patients. CLINICAL TRIAL REGISTRATION: NCT04210791.
自体干细胞移植(ASCT)后复发是影响AL淀粉样变性患者长期预后的重要因素。然而,复发的危险因素尚不清楚,且针对这些患者治疗结果的研究有限。我们回顾性分析了2010年至2021年间接受ASCT的170例AL淀粉样变性患者。76例患者确诊复发,从ASCT到复发的中位时间为39个月。对ASCT前后的变量进行多因素分析,λ轻链受限、ASCT前dFLC>30mg/L、美法仑剂量减少以及ASCT后6个月dFLC>10mg/L是复发的独立危险因素,诱导治疗后达到完全缓解(CR)以及ASCT后肾脏反应是保护因素。大多数复发患者接受了基于硼替佐米的方案治疗(50%),其次是基于达雷妥尤单抗的方案(22.2%)和其他化疗方案(13.9%)。可评估患者的总体血液学缓解率为68.2%,其中56.8%达到CR/VGPR。移植后复发患者的中位无进展生存期(PFS)和总生存期(OS)分别为25个月和81个月。与其他化疗方案相比,接受硼替佐米或达雷妥尤单抗治疗的患者生存率更高。总之,本研究确定了移植后复发的独立危险因素,并证明了硼替佐米或达雷妥尤单抗治疗这些患者的优越性。临床试验注册号:NCT04210791。