Cornell R F, Zhong X, Arce-Lara C, Atallah E, Blust L, Drobyski W R, Fenske T S, Pasquini M C, Rizzo J D, Saber W, Hari P N
Department of Medicine, Division of Hematology and Oncology, Vanderbilt University Medical Center, Nashville, TN, USA.
Department of Biostatistics, Medical College of Wisconsin, Milwaukee, WI, USA.
Bone Marrow Transplant. 2015 Jul;50(7):914-7. doi: 10.1038/bmt.2015.73. Epub 2015 Apr 27.
Recent studies support the use of bortezomib-based therapies in light chain amyloidosis (AL). We performed a retrospective analysis of the safety, efficacy and long-term survival (median follow-up 3 years) after bortezomib-based treatment in 28 consecutive patients with de novo AL deemed ineligible at initial presentation. The first 14 patients received bortezomib and dexamethasone (VD), and the second 14 patients received cyclophosphamide, bortezomib and dexamethasone (CVD; CyBorD). Both regimens were well tolerated with no treatment-related mortality. The overall hematological response (HR) rate was 93% in both the groups. Median time to response was shorter in the CVD group (39 days vs 96 days in the VD group; P=0.002). Hematological and organ responses induced with bortezomib-based therapy enabled 8 (33%) of initially transplant ineligible patients to undergo autologous hematopoietic stem cell transplantation (AHCT), including 4 patients with cardiac stage III or IV. Seven of the eight patients (88%) who underwent subsequent AHCT achieved sustained HR at a median of 33 months posttransplant. These data suggest that bortezomib-based induction followed by AHCT is a viable therapeutic strategy for transplant-ineligible AL. Larger, multicenter prospective trials are necessary to confirm our findings.
近期研究支持在轻链淀粉样变性（AL）中使用基于硼替佐米的疗法。我们对28例初诊时被认为不符合条件的新发AL患者进行了基于硼替佐米治疗后的安全性、疗效及长期生存情况（中位随访3年）的回顾性分析。前14例患者接受硼替佐米和地塞米松（VD）治疗，后14例患者接受环磷酰胺、硼替佐米和地塞米松（CVD；CyBorD）治疗。两种方案耐受性均良好，无治疗相关死亡。两组的总体血液学缓解（HR）率均为93%。CVD组的中位缓解时间较短（39天，而VD组为96天；P = 0.002）。基于硼替佐米的治疗诱导的血液学和器官缓解使8例（33%）最初不符合移植条件的患者能够接受自体造血干细胞移植（AHCT），其中包括4例心脏功能处于III期或IV期的患者。8例接受后续AHCT的患者中有7例（88%）在移植后中位33个月时实现了持续HR。这些数据表明，基于硼替佐米的诱导治疗后进行AHCT是不符合移植条件的AL患者的一种可行治疗策略。需要更大规模的多中心前瞻性试验来证实我们的发现。
