Tedesco Martina, Mescia Federica, Pisani Isabella, Allinovi Marco, Casazza Giovanni, Del Vecchio Lucia, Santostefano Marisa, Cirillo Luigi, Ferrario Francesca, Esposito Ciro, Esposito Pasquale, Santoro Domenico, Lazzarin Roberta, Rossi Giovanni Maria, Fiaccadori Enrico, Ferrantelli Angelo, Sinico Renato Alberto, Cozzolino Mario, Gallieni Maurizio, Cirami Lino, Scolari Francesco, Vaglio Augusto, Alberici Federico
Department of Medical and Surgical Specialties, Radiological Sciences and Public Health, University of Brescia, Brescia, Italy.
Nephrology Unit, Spedali Civili Hospital, ASST Spedali Civili of Brescia, Brescia, Italy.
Kidney Int Rep. 2022 May 30;7(8):1878-1886. doi: 10.1016/j.ekir.2022.05.024. eCollection 2022 Aug.
Primary focal segmental glomerular sclerosis (FSGS) is a rare, likely immune-mediated disease. Rituximab (RTX) may play a role in management, although data in adults are scanty.
We collected cases of RTX-treated primary FSGS within the Italian Society of Nephrology Immunopathology Working Group and explored response rate (24-hour proteinuria <3.5 g and <50% compared with baseline, stable estimated glomerular filtration rate).
A total of 31 patients were followed for at least 12 months; further follow-up (median 17 months, interquartile range [IQR] 15-33.5) was available for 11. At first RTX administration, median creatinine and 24-hour proteinuria were 1.17 mg/dl (IQR 0.83-1.62) and 5.2 g (IQR 3.3-8.81), respectively. Response rate at 3, 6, and 12 months was 39%, 52%, and 42%, respectively. In the first 12 months, creatinine level remained stable whereas proteinuria and serum albumin level improved, with an increase in the proportion of patients tapering other immunosuppressants. There were 6 patients who were retreated with RTX within 12 months, either for proteinuria increase or refractory disease; only the 2 responders to the first RTX course experienced a further response. At univariate analysis, 6-month response was more frequent in steroid-dependent patients (odds ratio [OR] 7.7 [95% CI 1.16-52.17]) and those with proteinuria <5 g/24 h (OR 8.25 [1.45-46.86]). During long-term follow-up, 4 of 5 responders at 12 months maintained a sustained response, either without further immunosuppression (2 of 4) or with pre-emptive RTX (2 of 4); 1 relapsed and responded to RTX retreatment.
RTX may be an option in primary FSGS, especially in steroid-dependent patients, with 24-hour proteinuria <5 g and previously responders to RTX. Optimal long-term management for responders is unclear, with some patients experiencing sustained remission and others requiring RTX retreatment, either preemptive or after rising proteinuria.
原发性局灶节段性肾小球硬化（FSGS）是一种罕见的、可能由免疫介导的疾病。利妥昔单抗（RTX）可能在其治疗中发挥作用，尽管成人患者的数据较少。
我们收集了意大利肾脏病学会免疫病理学工作组中接受RTX治疗的原发性FSGS病例，并探讨了缓解率（24小时蛋白尿<3.5 g且较基线水平降低<50%，估计肾小球滤过率稳定）。
共有31例患者接受了至少12个月的随访；11例患者有进一步的随访数据（中位随访时间17个月，四分位间距[IQR]为15 - 33.5个月）。首次给予RTX时，肌酐和24小时蛋白尿的中位数分别为1.17 mg/dl（IQR 0.83 - 1.62）和5.2 g（IQR 3.3 - 8.81）。3个月、6个月和12个月时的缓解率分别为39%、52%和42%。在最初的12个月内，肌酐水平保持稳定，而蛋白尿和血清白蛋白水平有所改善，减少其他免疫抑制剂用量的患者比例增加。有6例患者在12个月内因蛋白尿增加或疾病难治而再次接受RTX治疗；只有首次RTX治疗的2例缓解者再次出现缓解。单因素分析显示，依赖类固醇的患者（优势比[OR] 7.7 [95%可信区间1.16 - 52.17]）和24小时蛋白尿<5 g的患者（OR 8.25 [1.45 - 46.86]）6个月时缓解更为常见。在长期随访中，12个月时5例缓解者中的4例维持了持续缓解，其中2例（4例中的）无需进一步免疫抑制，2例（4例中的）采用了预防性RTX治疗；1例复发并对RTX再次治疗有反应。
RTX可能是原发性FSGS的一种治疗选择，尤其是对于依赖类固醇、24小时蛋白尿<5 g且之前对RTX有反应的患者。缓解者的最佳长期管理尚不清楚，一些患者持续缓解，而另一些患者则需要预防性或蛋白尿升高后进行RTX再次治疗。
Zotero 插件