文章：

达雷妥尤单抗加入免疫球蛋白轻链淀粉样变性患者一线治疗的影响：单中心经验

Impact of the Addition of Daratumumab to the Frontline Treatment of Patients with Immunoglobulin Light-Chain Amyloidosis: A Single-Centre Experience

原文发布日期：25 April 2025

DOI: 10.3390/cancers17091440

类型: Article

开放获取: 是

英文摘要：

Background: Daratumumab-based regimens represent the gold-standard therapy for newly diagnosed AL amyloidosis patients. However, there are few studies about the efficacy of this treatment in real life.Methods: This study included 99 patients: 27 (27.3%) received daratumumab and proteasome inhibitor-based schemes, 46 (46.4%) were treated with proteasome inhibitors and/or immunomodulator-based regimens, and 26 (26.3%) were treated with chemotherapy.Results: Patients receiving daratumumab and proteasome inhibitor-based regimens achieved higher rates of partial haematological responses or better (100.0% vs. 78.3% vs. 58.3%;p= 0.009 andp< 0.001) and complete responses (74.1% vs. 37.0% vs. 12.5%;p= 0.003 andp< 0.001) than the proteasome inhibitors and/or immunomodulators and chemotherapy groups, respectively. Daratumumab and proteasome inhibitor-based schemes resulted in a shorter time to haematological response (1 month to partial response or better and 4 months to complete response). Moreover, in the group treated with daratumumab and proteasome inhibitor-based regimens, there was a trend of obtaining better and faster organ responses. The benefit of daratumumab and proteasome inhibitor-based regimens was that they resulted in an improvement in progression-free survival (not reached) compared to the proteasome inhibitor and chemotherapy groups (18 months;p= 0.022 and 6 months;p= 0.002). In addition, the clinical benefit was consistent in patients with Mayo Clinic stages III–IV.Conclusions: This study supports the efficacy and superiority of adding daratumumab to the frontline treatment over proteasome inhibitor-based regimens and chemotherapy in AL amyloidosis, including in advanced cardiac disease.

摘要翻译： 

背景：以达雷妥尤单抗为基础的方案是新诊断AL型淀粉样变性患者的金标准疗法。然而，关于该疗法在真实世界中的疗效研究较少。 方法：本研究纳入99例患者：27例（27.3%）接受达雷妥尤单抗联合蛋白酶体抑制剂方案治疗，46例（46.4%）接受蛋白酶体抑制剂和/或免疫调节剂方案治疗，26例（26.3%）接受化疗。 结果：与蛋白酶体抑制剂和/或免疫调节剂组及化疗组相比，接受达雷妥尤单抗联合蛋白酶体抑制剂方案治疗的患者获得部分血液学缓解或更佳缓解的比例更高（100.0% vs. 78.3% vs. 58.3%；p=0.009和p<0.001），完全缓解率也更高（74.1% vs. 37.0% vs. 12.5%；p=0.003和p<0.001）。达雷妥尤单抗联合蛋白酶体抑制剂方案能更快获得血液学缓解（至部分缓解或更佳缓解中位时间1个月，至完全缓解中位时间4个月）。此外，在达雷妥尤单抗联合蛋白酶体抑制剂治疗组中，观察到获得更优、更快器官缓解的趋势。该方案的获益还体现在无进展生存期的改善（未达到），优于蛋白酶体抑制剂组（18个月；p=0.022）和化疗组（6个月；p=0.002）。在梅奥分期III–IV期患者中，临床获益保持一致。 结论：本研究证实了在AL型淀粉样变性一线治疗中，达雷妥尤单抗联合蛋白酶体抑制剂方案相较于单纯蛋白酶体抑制剂方案及化疗具有疗效优势和临床优越性，这一结论在晚期心脏受累患者中同样成立。

原文链接：

Impact of the Addition of Daratumumab to the Frontline Treatment of Patients with Immunoglobulin Light-Chain Amyloidosis: A Single-Centre Experience