Daratumumab-Based Treatment for Immunoglobulin Light-Chain Amyloidosis
Daratumumab-Based Treatment for Immunoglobulin Light-Chain Amyloidosis
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DOI：
10.1056/nejmoa2028631
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发表时间：
2021-07-01
影响因子：
158.5
通讯作者：
Comenzo, R. L.
中科院分区：
文献类型：
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作者：
Kastritis, E.;Palladini, G.;Comenzo, R. L.
背景系统性免疫球蛋白轻链（AL）淀粉样变性的特征是克隆CD 38+浆细胞产生的轻链淀粉样纤维沉积。Daratumumab是一种人CD 38靶向抗体，可能会改善这种疾病的结局。方法我们将新诊断的AL淀粉样变性患者随机分配接受6个周期的硼替佐米、环磷酰胺和地塞米松单独治疗（对照组）或皮下注射达雷妥尤单抗，随后每4周一次单药达雷妥尤单抗，最多24个周期（达雷妥尤单抗组）。主要终点是血液学完全缓解。结果388例患者接受随机分组。中位随访时间为11.4个月。达雷妥尤单抗组血液学完全缓解的患者百分比显著高于对照组（53.3% vs. 18.1%）（相对风险比，2.9; 95%置信区间[CI]，2.1 - 4.1; P
Background Systemic immunoglobulin light-chain (AL) amyloidosis is characterized by deposition of amyloid fibrils of light chains produced by clonal CD38+ plasma cells. Daratumumab, a human CD38-targeting antibody, may improve outcomes for this disease. Methods We randomly assigned patients with newly diagnosed AL amyloidosis to receive six cycles of bortezomib, cyclophosphamide, and dexamethasone either alone (control group) or with subcutaneous daratumumab followed by single-agent daratumumab every 4 weeks for up to 24 cycles (daratumumab group). The primary end point was a hematologic complete response. Results A total of 388 patients underwent randomization. The median follow-up was 11.4 months. The percentage of patients who had a hematologic complete response was significantly higher in the daratumumab group than in the control group (53.3% vs. 18.1%) (relative risk ratio, 2.9; 95% confidence interval [CI], 2.1 to 4.1; P