Département de Néphrologie et Transplantation d'organes, CHU Rangueil, Toulouse, France.
Service de Néphrologie et Transplantation rénale, CHU Hôtel-Dieu, Nantes, France.
Nephrol Dial Transplant. 2014 Nov;29(11):2084-91. doi: 10.1093/ndt/gfu209. Epub 2014 Jun 11.
Minimal-change nephrotic syndrome (MCNS) is a common cause of steroid sensitive nephrotic syndrome (NS) with frequent relapse. Although steroids and calcineurin inhibitors (CNIs) are the cornerstone treatments, the use of rituximab (RTX), a monoclonal antibody targeting B cells, is an efficient and safe alternative in childhood.
Because data from adults remain sparse, we conducted a large retrospective and multicentric study that included 41 adults with MCNS and receiving RTX.
Complete (NS remission and withdrawal of all immunosuppressants) and partial (NS remission and withdrawal of at least one immunosuppressants) clinical responses were obtained for 25 and 7 patients, respectively (overall response 78%), including 3 patients that only received RTX and had a complete clinical response. After a follow-up time of 39 months (6-71), relapses occurred in 18 responder patients [56%, median time 18 months (3-36)]. Seventeen of these received a second course of RTX and then had a complete (n = 13) or partial (n = 4) clinical response. From multivariate analysis, on-going mycophenolate mofetil (MMF) therapy at the time of RTX was the only predictive factor for RTX failure [HR = 0.07 95% CI (0.01-0.04), P = 0.003]. Interestingly, nine patients were still in remission at 14 months (3-36) after B-cell recovery. No significant early or late adverse event occurred after RTX therapy.
RTX is safe and effective in adult patients with MCNS and could be an alternative to steroids or CNIs in patients with a long history of relapsing MCNS.
微小病变肾病综合征(MCNS)是类固醇敏感型肾病综合征(NS)的常见病因,常反复发作。尽管类固醇和钙调磷酸酶抑制剂(CNIs)是基石治疗方法,但利妥昔单抗(RTX),一种针对 B 细胞的单克隆抗体,在儿童中是一种有效且安全的替代方法。
由于成人的数据仍然很少,我们进行了一项大型回顾性多中心研究,该研究纳入了 41 名接受 RTX 治疗的 MCNS 成年患者。
25 名和 7 名患者分别获得完全(NS 缓解和停用所有免疫抑制剂)和部分(NS 缓解和至少停用一种免疫抑制剂)临床反应(总体反应 78%),包括仅接受 RTX 且获得完全临床反应的 3 名患者。在 39 个月(6-71 个月)的随访时间后,18 名应答者患者发生了复发[56%,中位数时间 18 个月(3-36 个月)]。其中 17 名患者接受了第二疗程的 RTX 治疗,随后获得完全(n=13)或部分(n=4)临床反应。多变量分析显示,RTX 时持续使用霉酚酸酯(MMF)治疗是 RTX 失败的唯一预测因素[HR=0.07 95%CI(0.01-0.04),P=0.003]。有趣的是,9 名患者在 B 细胞恢复后 14 个月(3-36 个月)仍处于缓解状态。RTX 治疗后未发生明显的早期或晚期不良事件。
RTX 对 MCNS 成年患者安全有效,对于有反复发作 MCNS 病史的患者,可替代类固醇或 CNIs。