血清游离轻链水平和非固定周期daratumumab治疗轻链淀粉样变性的策略。

Annals of medicine Pub Date : 2025-12-01 Epub Date: 2024-12-19 DOI:10.1080/07853890.2024.2442075
Zhen Li, Jinzhou Guo, Wencui Chen, Liang Zhao, Guisheng Ren, Xianghua Huang
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引用次数: 0

摘要

背景:近年来,达拉单抗(daratumumab, DARA)在系统性轻链(AL)淀粉样变性的治疗中得到了广泛的应用。在这项研究中,我们评估了基于血清游离轻链(sFLC)水平和非固定周期的DARA治疗策略的有效性和安全性。方法:该研究包括123例在2020年7月至2023年9月期间在我们中心接受DARA治疗的Al淀粉样变性患者。在第一个疗程中,所有患者都接受了标准的DARA治疗(每周16 mg/kg,持续四周)。根据sFLC水平和医生的判断调整后续治疗。结果:结果显示令人印象深刻的总体血液学缓解率(ORR)为94.3%,血液学非常好的部分缓解(VGPR)和完全缓解(CR)率为84.5%。达到最佳血液学反应的中位时间为1个月。心脏和肾脏反应率分别为39.3%和60.3%。30例患者在首次输注后出现1/2级输注相关反应。3/4级不良事件发生率为21%。3级或4级最常见的不良事件是肺部感染(6.5%)、中性粒细胞减少症和淋巴细胞减少症(5.7%)、转氨酶升高(1.6%)、急性肾损伤(1.6%)。中位随访13个月(范围1-38)后,1年OS和PFS估计分别为96.5%和84.4%。结论:基于sFLC水平和非固定周期的DARA策略是新诊断和复发/难治性AL淀粉样变性的有效和安全的治疗策略。
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Serum free light chain level-based and non-fixed cycle daratumumab treatment strategy for patients with light chain amyloidosis.

Background: In recent years, daratumumab (DARA) has gained widespread use in the treatment of systemic light chain (AL) amyloidosis. In this study, we assessed the efficacy and safety of a DARA treatment strategy based on serum free light chain (sFLC) levels and non-fixed cycles.

Methods: The study included 123 patients with Al amyloidosis who received DARA at our center between July 2020 and September 2023. All patients received the standard DARA treatment (16 mg/kg weekly for four weeks) during the first course. Subsequent treatments were adjusted based on sFLC levels and the physician's judgment.

Results: The results demonstrated an impressive overall hematologic response rate (ORR) of 94.3%, with a hematologic very good partial response (VGPR) and complete response (CR) rate of 84.5%. Median time to best hematologic response was 1 months. Cardiac and renal response rates were 39.3% and 60.3%, respectively. Thirty patients experienced grade 1/2 infusion-related reactions after the first infusion. The rate of grade 3/4 adverse events was 21%. The most common adverse events of grade 3 or 4 were pulmonary infection (6.5%), neutropenia and lymphocytopenia (5.7%), elevated transaminases (1.6%), acute kidney injury (1.6%). After a median follow-up of 13 months (range 1-38), The 1-year OS and PFS estimates were 96.5% and 84.4%, respectively.

Conclusion: These findings indicate that the sFLC levels based and non-fixed cycle DARA strategy is an efficacious and safe treatment strategy in both newly diagnosed and relapsed/refractory AL amyloidosis.

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