The Brazilian Cochrane Center and Discipline of Emergency Medicine and Evidence-Based Medicine, Universidade Federal de São Paulo-Escola Paulista de Medicina (UNIFESP-EPM), São Paulo, Brazil.
Orphanet J Rare Dis. 2011 Feb 7;6:5. doi: 10.1186/1750-1172-6-5.
Systemic sclerosis (scleroderma; SSc) is an orphan disease with the highest case-specific mortality of any connective-tissue disease. Excessive collagen deposit in affected tissues is a key for the disease's pathogenesis and comprises most of the clinical manifestations. Lidocaine seems to be an alternative treatment for scleroderma considering that: a) the patient's having excessive collagen deposits in tissues affected by scleroderma; b) the patient's demonstrating increased activity of the enzyme prolyl hydroxylase, an essential enzyme for the biosynthesis of collagen; and c) lidocaine's reducing the activity of prolyl hydroxylase. The aim of this study was to evaluate the efficacy and safety of lidocaine in treating scleroderma.
A randomized double-blind clinical trial included 24 patients with scleroderma randomized to receive lidocaine or placebo intravenously in three cycles of ten days each, with a one-month interval between them.
cutaneous (modified Rodnan skin score), oesophageal (manometry) and microvascular improvement (nailfold capillaroscopy); improvement in subjective self-assessment and in quality of life (HAQ).
There was no statistically significant difference between the groups for any outcome after the treatment and after 6-months follow-up. Improvement in modified Rodnan skin score occurred in 66.7% and 50% of placebo and lidocaine group, respectively (p = 0.408). Both groups showed an improvement in subjective self-assessment, with no difference between them.
Despite the findings of a previous cohort study favouring the use of lidocaine, this study demonstrated that lidocaine at this dosage and means of administration showed a lack of efficacy for treating scleroderma despite the absence of significant adverse effects. However, further similar clinical trials are needed to evaluate the efficacy of lidocaine when administered in different dosages and by other means.
系统性硬化症（硬皮病；SSc）是一种孤儿病，其任何结缔组织疾病的特定死亡率最高。受影响组织中胶原的过度沉积是疾病发病机制的关键，构成了大部分临床表现。考虑到以下几点，利多卡因似乎是硬皮病的一种替代治疗方法：a）患者的组织中存在过多的胶原沉积；b）患者表现出脯氨酰羟化酶活性增加，脯氨酰羟化酶是胶原生物合成所必需的酶；c）利多卡因降低脯氨酰羟化酶的活性。本研究旨在评估利多卡因治疗硬皮病的疗效和安全性。
一项随机双盲临床试验纳入了 24 例硬皮病患者，随机分为三组，每组接受利多卡因或安慰剂静脉注射，每个周期为 10 天，间隔 1 个月。
皮肤（改良罗德里格斯皮肤评分）、食管（测压）和微血管改善（甲襞毛细血管镜）；主观自我评估和生活质量（HAQ）改善。
治疗后和 6 个月随访时，两组间任何结局均无统计学差异。安慰剂组和利多卡因组的改良罗德里格斯皮肤评分分别改善了 66.7%和 50%（p=0.408）。两组的主观自我评估均有改善，但无差异。
尽管之前的队列研究结果支持利多卡因的使用，但本研究表明，尽管没有明显的不良反应，但在这种剂量和给药方式下，利多卡因治疗硬皮病缺乏疗效。然而，需要进一步进行类似的临床试验来评估不同剂量和其他给药方式下利多卡因的疗效。
