MQX-503, a Novel Formulation of Nitroglycerin, Improves the Severity of Raynaud's Phenomenon A Randomized, Controlled Trial
MQX-503, a Novel Formulation of Nitroglycerin, Improves the Severity of Raynaud's Phenomenon A Randomized, Controlled Trial
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DOI：
10.1002/art.24351
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发表时间：
2009-03-01
影响因子：
--
通讯作者：
Wigley, Fredrick M.
中科院分区：
文献类型：
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作者：
Chung, Lorinda;Shapiro, Lee;Wigley, Fredrick M.
Objective.雷诺现象（RP）影响3-9%的一般人群和> 90%的系统性硬化症患者。硝酸盐通常用于治疗RP，但目前可用的制剂受到副作用的限制，特别是头痛，头晕和皮肤刺激。本研究的目的是评估一种新的配方的局部硝酸甘油，MQX-503，在治疗RP在门诊设置的耐受性和有效性。我们进行了一项多中心、随机、安慰剂对照研究，包括2周单盲导入期以确定基线严重程度，随后是4周双盲治疗期。219例临床诊断为原发性或继发性RP的成人患者在治疗期间接受了0.9% MQX-503凝胶或匹配的安慰剂。凝胶在RP发作前即刻或开始后5分钟内施用（每天最多施用4次）。终点包括平均雷诺状况评分（RCS; 0-10级）的变化、发作的频率和持续时间以及目标周（治疗期内与导入期在环境温度方面最匹配的一周）的主观评估与基线相比。MQX-503组（0.48 [14.3%]）目标周RCS相对于基线的平均（%）变化显著大于安慰剂组（0.04 [1.3%]; P = 0.04）。两组间RP发作的频率和持续时间以及主观评估的变化无统计学差异。MQX-503的副作用特征与安慰剂相似。MQX-503耐受性良好，治疗RP比安慰剂更有效。
Objective. Raynaud's phenomenon (RP) affects 3-9% of the general population and > 90% of patients with systemic sclerosis. Nitrates are often prescribed for the treatment of RP, but currently available formulations are limited by side effects, particularly headaches, dizziness, and skin irritation. The purpose of this study was to evaluate the tolerability and efficacy of a novel formulation of topical nitroglycerin, MQX-503, in the treatment of RP in an ambulatory setting.Methods. We conducted a multicenter, randomized, placebo-controlled study with a 2-week single-blind run-in period to determine baseline severity, followed by a 4-week double-blind treatment phase. Two hundred nineteen adult patients with a clinical diagnosis of primary or secondary RP received 0.9% MQX-503 gel or matching placebo during the treatment period. Gel was applied immediately before or within 5 minutes of the beginning of an episode of RP (maximum of 4 applications daily). End points included the change in the mean Raynaud's Condition Score (RCS; scale 0-10), the frequency and duration of episodes, and subjective assessments at the target week (the week during the treatment phase that most closely matched the run-in period in terms of ambient temperature) compared with baseline.Results. The mean (%) change in the RCS at the target week compared with baseline was significantly greater in the MQX-503 group (0.48 [14.3%]) than that in the placebo group (0.04 [1.3%]; P = 0.04). Changes in the frequency and duration of RP episodes and subjective assessments were not statistically different between the groups. MQX-503 had a side effect profile similar to that of placebo.Conclusion. MQX-503 is well tolerated and more effective than placebo for the treatment of RP.