关键词: 系统性轻链型淀粉样变性, MayoⅢB 期, 达雷妥尤单抗

Abstract: Objective: To observe the efficacy and safety of the daratumumab plus dexamethasone (DD) regimen in patients with Mayo stage ⅢB systemic light⁃chain (AL) amyloidosis. Methods: This retrospective cohort study analyzed patients with Mayo stage ⅢB AL amyloidosis receiving the DD regimen at the National Clinical Research Center for Kidney Diseases (January 2021-November 2024). Hematologic/organ responses, adverse reactions and prognosis were evaluated. Results: Thirty⁃one patients were included (18 males, 58.1%), with a mean age of 69±8 years. All patients had renal involvement. The median baseline NT⁃proBNP and cTnT were 15283 (11067-30281) ng/L and 0.13 (0.08-0.22) μg/L, respectively. Twenty⁃four patients (77.4%) achieved hematologic response, among which 54.8% achieved a very good partial response or better. The median time to the first hematologic response was 4 days. Cardiac and renal response rates were both 54.8%. After a median 21⁃month follow⁃up, the 1⁃year overall survival rate was 67.7%, and the median OS was not reached. The median MOD⁃PFS was 10 months. NYHA class Ⅰ⁃Ⅱ patients had a better prognosis than class Ⅲ⁃Ⅳ. Twenty⁃nine patients were included in safety analysis. Adverse events (AEs) occurred in 17 patients (58.6%), predominantly pulmonary infection (20.7%), infusion⁃related reactions (10.3%), and gastrointestinal symptoms (10.3%), with a grade ≥3 AE rate of 3.4% (1 case). Conclusion: The DD regimen induces high hematologic and organ response rates with a favorable safety profile in Mayo stage ⅢB AL amyloidosis. However, its long⁃term clinical benefits require further validation through large⁃scale, multicenter, prospective studies.