Sambo P, Amico D, Giacomelli R, Matucci-Cerinic M, Salsano F, Valentini G, Gabrielli A
Istituto di Clinica Medica Generale, Ematologia ed Immunologia Clinica, University of Ancona, Italy.
J Rheumatol. 2001 Oct;28(10):2257-62.
To assess the efficacy and tolerability of N-acetylcysteine (NAC) in patients with Raynaud's phenomenon (RP) secondary to systemic sclerosis (scleroderma; SSc).
Twenty-two patients with RP secondary to SSc were enrolled in a multicenter, open clinical trial lasting 11 weeks and conducted in winter. Primary outcome measures were frequency and severity of RP attacks, and number of digital ulcers. Secondary outcome measure was improvement in digital cold challenge test assessed by photoelectric plethysmography. Patients received a continuous 5 day intravenous infusion of NAC starting with a 2 h loading dose of 150 mg/kg subsequently adjusted to 15 mg/kg/h.
All 22 patients completed the 5 day infusion and 20 of them the posttreatment followup. Both frequency and severity of RP attacks decreased significantly compared to pretreatment values. Active ulcers were significantly less numerous at all followup visits (25.18% of baseline count on Day 33 from the beginning of infusion). In the cold challenge test mean recovery time fell by 69.56%, 67.70%, 71.42%, and 71.05% on Days 12, 19. 33, and 61 from the beginning of treatment. Side effects were minor, easily controlled, and reversible.
N-acetylcysteine appears to be safe for the treatment of RP secondary to SSc. These preliminary data warrant further controlled studies.
评估N-乙酰半胱氨酸（NAC）对系统性硬化症（硬皮病；SSc）继发雷诺现象（RP）患者的疗效和耐受性。
22例SSc继发RP患者参加了一项为期11周的多中心开放临床试验，试验在冬季进行。主要结局指标为RP发作的频率和严重程度以及指端溃疡的数量。次要结局指标是通过光电体积描记法评估的指端冷激发试验的改善情况。患者接受为期5天的NAC持续静脉输注，起始2小时负荷剂量为150mg/kg，随后调整为15mg/kg/h。
所有22例患者均完成了5天的输注，其中20例完成了治疗后随访。与治疗前相比，RP发作的频率和严重程度均显著降低。在所有随访中，活动性溃疡的数量均显著减少（自输注开始第33天的基线计数的25.18%）。在冷激发试验中，自治疗开始第12、19、33和61天，平均恢复时间分别下降了69.56%、67.70%、71.42%和71.05%。副作用轻微，易于控制且可逆。
N-乙酰半胱氨酸似乎对治疗SSc继发的RP是安全的。这些初步数据值得进一步进行对照研究。
