BACKGROUND Long-term outcomes of rituximab-treated adult patients with podocytopathies (either minimal change disease or focal segmental glomerulosclerosis) are largely unknown. METHODS A retrospective study at 30 nephrology departments from 15 countries worldwide included rituximab-treated adults with primary podocytopathies and a minimum clinical follow-up of 36 months. The primary outcome was relapse-free survival at 36 months. RESULTS 183 adult patients (n=64 with focal segmental glomerulosclerosis and n=119 with minimal change disease) with difficult-to-treat nephrotic syndrome (68% steroid-dependent/frequently relapsing, 22% steroid-resistant, 85% previously treated with two or more lines of immunosuppressive therapy) were treated with rituximab as part of a remission induction regimen. Complete or partial remission at 6 months after rituximab treatment was achieved in 82%. Eighty-three of 151 (55%) initial responders achieved long-term relapse-free survival over three years. Maintenance therapy with rituximab was associated with a better relapse-free survival (HR 2.05, 95% CI: 1.07-3.91), irrespective of the dosing regimen. At 36 months, 61% of initial responders receiving maintenance therapy with rituximab achieved long-term relapse-free survival and withdrawal of all concomitant immunosuppressive medication compared to 36% of patients without maintenance treatment (OR 2.69, 95% CI: 1.27-5.73). Relapses per year were reduced from an annual relapse rate of 1.0 (95% CI: 1.0-1.7) before to 0.17 (95% CI: 0.00-0.24) relapses/year after rituximab initiation. Over the 36 months of follow-up, a stable course of estimated glomerular filtration rate (eGFR) was observed in those who initially responded with either complete or partial remission, whereas non-responders experienced a reduction in eGFR reaching -11 (95% CI: -18 to -8) mL/min/1.73m2 . CONCLUSIONS Rituximab facilitated achievement of initial and long-term response in a majority of adult patients with difficult-to-treat podocytopathies. Maintenance treatment with rituximab further associated with long-term relapse-free survival over three years. Non-response to initial rituximab treatment was associated with poor kidney prognosis.

中文翻译：

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利妥昔单抗治疗成年足细胞病患者的长期结果。


背景 利妥昔单抗治疗的成年足细胞病患者（微小病变疾病或局灶节段性肾小球硬化）的长期结局在很大程度上是未知的。方法 对来自全球 30 个国家的 15 个肾脏病科进行的一项回顾性研究纳入了接受利妥昔单抗治疗的原发性足细胞病成人，临床随访时间最短为 36 个月。主要结局是 36 个月时的无复发生存期。结果 183 例患有难治性肾病综合征 （68% 类固醇依赖性/频繁复发，22% 类固醇耐药，85% 既往接受过两种或多种免疫抑制治疗）的成年患者 （n=64 局灶节段性肾小球硬化和 n=119 微小病变肾小球硬化） 接受利妥昔单抗治疗作为缓解诱导方案的一部分。82% 的患者在利妥昔单抗治疗后 6 个月达到完全或部分缓解。151 例初始反应者中有 83 例 （55%） 在三年内实现了长期无复发生存。无论给药方案如何，利妥昔单抗维持治疗与更好的无复发生存率相关 （HR 2.05,95% CI： 1.07-3.91）。在 36 个月时，接受利妥昔单抗维持治疗的初始反应者中有 61% 实现了长期无复发生存期和所有伴随免疫抑制药物的停药，而未接受维持治疗的患者为 36% （OR 2.69,95% CI： 1.27-5.73）。每年复发率从利妥昔单抗治疗前每年 1.0 （95% CI： 1.0-1.7） 的年复发率降低到利妥昔单抗开始后每年的 0.17 （95% CI： 0.00-0.24） 复发率。 在 36 个月的随访中，在最初对完全或部分缓解有反应的患者中观察到估计肾小球滤过率 （eGFR） 的稳定过程，而无反应者的 eGFR 降低至 -11 （95% CI： -18 至 -8） mL/min/1.73m2。结论 利妥昔单抗有助于大多数难治性足细胞病成年患者实现初始和长期反应。利妥昔单抗维持治疗进一步与三年内的长期无复发生存率相关。对初始利妥昔单抗治疗无反应与肾脏预后不良相关。