The chimeric anti-CD20 monoclonal antibody rituximab is effective in steroid-dependent and calcineurin inhibitor–dependent forms of nephrotic syndrome, but many patients relapse at 1 year. Because ofatumumab, a fully human anti-CD20 monoclonal antibody, has a more extended binding site and higher affinity to CD20 compared with rituximab, it might offer superior efficacy in these patients.

We designed a single-center randomized clinical trial to compare the long-term efficacy of ofatumumab versus rituximab in children and young adults with nephrotic syndrome maintained in remission with prednisone and calcineurin inhibitors. We randomized 140 children and young adults (aged 2–24 years) to receive intravenous ofatumumab (1.50 mg/1.73 m²) or rituximab (375 mg/m²). After infusions, oral drugs were tapered and withdrawn within 60 days. The primary outcome was relapse at 1 year, which was analyzed following the intent-to-treat principle. The secondary endpoint was relapse within 24 months from infusion, on the basis of urine dipstick and confirmed by a urine protein-to-creatinine ratio <200.

At 12 months, 37 of 70 (53%) participants who received ofatumumab experienced relapse versus 36 of 70 (51%) who received rituximab (odds ratio [OR], 1.06; 95% confidence interval [95% CI], 0.55 to 2.06). At 24 months, 53 of 70 (76%) participants who received ofatumumab experienced relapse, versus 46 of 70 (66%) who received rituximab (OR, 1.6; 95% CI, 0.8 to 3.3). The two groups exhibited comparable B cell subpopulation reconstitution and did not differ in adverse events.

A single dose of ofatumumab was not superior to a single dose of rituximab in maintaining remission in children with steroid-dependent and calcineurin inhibitor–dependent nephrotic syndrome.

ClinicalTrials.gov (NCT02394119) and https://www.clinicaltrialsregister.eu/ctr-search/search (2015–000624–28).

嵌合抗 CD20 单克隆抗体利妥昔单抗对类固醇依赖性和神经钙蛋白抑制剂依赖性肾病综合征有效，但许多患者在 1 年时复发。由于 ofatumumab 是一种全人抗 CD20 单克隆抗体，与利妥昔单抗相比，它具有更广泛的结合位点和更高的 CD20 亲和力，因此它可能在这些患者中提供更好的疗效。

我们设计了一项单中心随机临床试验，以比较奥法木单抗与利妥昔单抗对使用强的松和神经钙蛋白抑制剂维持缓解的肾病综合征儿童和青年的长期疗效。我们将 140 名儿童和年轻人（2-24 岁）随机分配接受静脉注射奥法木单抗 (1.50 mg/1.73 m ² ) 或利妥昔单抗 (375 mg/m ² )。输注后，口服药物逐渐减量并在 60 天内停药。主要结果是 1 年时的复发，根据意向治疗原则进行分析。次要终点是在输注后 24 个月内复发，根据尿液试纸和尿蛋白与肌酐比值 <200 确认。

在 12 个月时，接受奥法木单抗治疗的 70 名参与者中有 37 名 (53%) 出现复发，而接受利妥昔单抗治疗的 70 名参与者中有 36 名 (51%) 出现复发（比值比 [OR]，1.06；95% 置信区间 [95% CI]，0.55 至 2.06 ). 在 24 个月时，接受奥法木单抗治疗的 70 名参与者中有 53 名 (76%) 出现复发，而接受利妥昔单抗治疗的 70 名参与者中有 46 名 (66%) 出现复发（OR，1.6；95% CI，0.8 至 3.3）。两组表现出可比的 B 细胞亚群重建，并且在不良事件方面没有差异。

在患有类固醇依赖性和神经钙蛋白抑制剂依赖性肾病综合征的儿童中，单剂量奥法木单抗在维持缓解方面并不优于单剂量利妥昔单抗。

ClinicalTrials.gov (NCT02394119) 和 https://www.clinicaltrialsregister.eu/ctr-search/search (2015–000624–28)。