D'Agostino Gennari Juliana, Deveza Giordano B H, Ribeiro Carolina T, Seguro Antonio C, Aikawa Nádia E, Shimizu Maria Heloisa M, Leon Elaine Pires, Guedes Lissiane K N, Kupa Léonard V K, Silva Clovis A A, Bonfa Eloisa, Pasoto Sandra Gofinet
Rheumatology Division, Hospital das Clinicas HCFMUSP, Faculdade de Medicina, Universidade de Sao Paulo, Brazil.
Nephrology Division, and Laboratories of Medical Investigations (LIM/12), Hospital das Clinicas HCFMUSP, Faculdade de Medicina, Universidade de Sao Paulo, Brazil.
Clin Exp Rheumatol. 2024 Dec;42(12):2427-2436. doi: 10.55563/clinexprheumatol/dmd5dv. Epub 2024 Sep 27.
N-acetylcysteine (NAC) is used in Sjögren's disease (SjD) based on limited evidence. The aim of this study was to assess the efficacy of NAC for relieving dryness symptoms in SjD.
In this placebo-controlled double-blind trial, 60 adult SjD females (with low disease activity) were randomised to receive NAC (1,200 mg/day orally) or placebo. At baseline (D0), 30 days (D30) and 90 days (D90), all participants underwent the following evaluations: EULAR Sjögren's Syndrome Patient Reported Index (ESSPRI), Ocular Surface Disease Index (OSDI), Xerostomia Inventory (XI), Leicester Cough Questionnaire (LCQ), unstimulated/stimulated salivary flow, Schirmer's test, and plasma levels of thiobarbituric acid reactive substances (TBARS), glutathione and NAC.
At inclusion, both groups were balanced for age, ethnicity, disease duration, ESSPRI, OSDI, XI, Schirmer's test, salivary flow, ESSDAI and topical/systemic treatments (p>0.05). No significant differences were observed between NAC and placebo groups on D30 and D90 regarding ESSPRI, XI, OSDI, LCQ, Schirmer's test, stimulated salivary flow, ESSDAI and topical/systemic treatments (p>0.05). Unstimulated salivary flow was significantly higher in the placebo group on D90 (p=0.018). NAC blood concentrations were significantly higher in the NAC group on D30 (p=0.018) and D90 (p<0.001), however, no differences were found in TBARS and glutathione. Further analysis showed decrease≥1 in ESSPRI in the NAC compared with placebo group on D30 (p=0.045), a result not found on D90 (p=0.696).
NAC is recommended as a rescue therapy for SjD. However, our well-designed study provides novel evidence demonstrating its inefficacy for improving dryness symptoms or reducing oxidative stress.
gov-NCT04793646.
基于有限的证据，N-乙酰半胱氨酸（NAC）被用于干燥综合征（SjD）的治疗。本研究旨在评估NAC缓解SjD患者干燥症状的疗效。
在这项安慰剂对照双盲试验中，60名成年SjD女性患者（疾病活动度低）被随机分为两组，分别接受NAC（口服1200毫克/天）或安慰剂治疗。在基线期（D0）、30天（D30）和90天（D90）时，所有参与者均接受以下评估：欧洲抗风湿病联盟干燥综合征患者报告指数（ESSPRI）、眼表疾病指数（OSDI）、口干症量表（XI）、莱斯特咳嗽问卷（LCQ）、非刺激性/刺激性唾液流量、泪液分泌试验以及血浆中硫代巴比妥酸反应物质（TBARS）、谷胱甘肽和NAC的水平。
纳入研究时，两组在年龄、种族、病程、ESSPRI、OSDI、XI、泪液分泌试验、唾液流量、欧洲抗风湿病联盟干燥综合征疾病活动指数（ESSDAI）以及局部/全身治疗方面均保持平衡（p>0.05）。在D30和D90时，NAC组与安慰剂组在ESSPRI、XI、OSDI、LCQ、泪液分泌试验、刺激性唾液流量、ESSDAI以及局部/全身治疗方面均未观察到显著差异（p>0.05）。在D90时，安慰剂组的非刺激性唾液流量显著更高（p=0.018）。在D30（p=0.018）和D90（p<0.001）时，NAC组的NAC血药浓度显著更高，然而，在TBARS和谷胱甘肽方面未发现差异。进一步分析显示，在D30时，与安慰剂组相比，NAC组的ESSPRI降低≥1（p=0.045），而在D90时未发现此结果（p=0.696）。
NAC被推荐作为SjD的一种挽救治疗方法。然而，我们精心设计的研究提供了新的证据，证明其在改善干燥症状或降低氧化应激方面无效。
美国国立医学图书馆临床试验注册中心-NCT04793646。
