Preliminary experience using milnacipran in patients with juvenile fibromyalgia: lessons from a clinical trial program.
Preliminary experience using milnacipran in patients with juvenile fibromyalgia: lessons from a clinical trial program.
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在青少年纤维肌痛患者中使用Milnacipran使用Milnacipran的初步经验：临床试验计划的教训。
DOI：
10.1186/s12969-015-0025-9
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发表时间：
2015-06-26
期刊：
影响因子：
--
通讯作者：
Lin Y
中科院分区：
文献类型：
--
作者：
Arnold LM;Bateman L;Palmer RH;Lin Y
目前还没有批准的药物治疗青少年纤维肌痛(JFM)，这是一种经常被低估的疾病。米那普兰是一种被批准用于治疗成人纤维肌痛(FM)的药物，其疗效在JFM的临床试验计划中进行了评估。符合JFM的尤努斯和马西标准和/或1990年美国风湿病学会FM标准的13-17岁患者参加了一项由应答者充实的随机停药试验。在接受开放标签米那西普兰治疗(8周)后，≥疼痛改善50%的患者接受了双盲随机分组(1：2)，要么服用安慰剂，要么继续服用米那西普兰(8周)。所有患者，包括那些不符合双盲停药随机标准的患者，都被允许进入一项使用开放标签米那西普兰的扩展研究(最多52周)。主要终点是双盲期内的治疗反应丧失(LTR)。其他结果测量包括患者总体严重程度印象(PGIS)、儿科生活质量问卷(PedsQL：通用核心量表、多维度疲劳量表)和儿童多维度焦虑量表(MASC)。安全性评估包括不良事件(AEs)、生命体征、心电图和实验室测试。由于注册人数较少，Milnacpran计划提前终止。由于只有20名患者随机进入双盲停药期，因此没有对LTR终点进行统计分析。然而，116名患者进入了初始研究的开放标签阶段，57名患者参与了开放标签扩展研究。他们的经验提供了有关在JFM患者中使用米那普兰的初步信息。在这两个开放标记期，疼痛严重程度、PGIC、PedsQL和MASC评分均有平均改善。未检测到意外的安全问题。最常见的治疗急症不良反应是恶心、头痛、呕吐和头晕。平均心率和血压的增加被观察到，并与成人FM的声发射谱相一致。这些开放标签的发现提供了初步证据，表明米那西普兰可以改善JFM的症状，其安全性和耐受性与成年FM患者的经验一致。JFM未来的试验设计应该考虑到与成人FM相比，对这种情况的认知度相对较低，以及登记的困难。本文的在线版本(DOI：10.1186/s12969-0150025-9)包含补充材料，可供授权用户使用。
There are no approved medications for juvenile fibromyalgia (JFM), a disorder that is often under-diagnosed. The effects of milnacipran, a drug approved for the management of fibromyalgia (FM) in adults, was assessed in a clinical trial program for JFM. Patients, ages 13–17 years who met the Yunus and Masi criteria for JFM and/or 1990 American College of Rheumatology criteria for FM, were enrolled in a responder-enriched, randomized withdrawal trial. After receiving open-label milnacipran (8 weeks), patients with ≥50 % improvement in pain underwent double-blind randomization (1:2) to either placebo or continuing treatment with milnacipran (8 weeks). All patients, including those who did not meet the randomization criteria for double-blind withdrawal, were allowed to enter an extension study with open-label milnacipran (up to 52 weeks). The primary endpoint was loss of therapeutic response (LTR) during the double-blind period. Additional outcome measures included the Patient Global Impression of Severity (PGIS), Pediatric Quality of Life Inventory (PedsQL: Generic Core Scales, Multidimensional Fatigue Scale), and Multidimensional Anxiety Scale for Children (MASC). Safety assessments included adverse events (AEs), vital signs, electrocardiograms, and laboratory tests. The milnacipran program was terminated early due to low enrollment. Because only 20 patients were randomized into the double-blind withdrawal period, statistical analyses were not conducted for the LTR endpoint. However, 116 patients entered the open-label period of the initial study and 57 participated in the open-label extension study. Their experience provides preliminary information about the use of milnacipran in JFM patients. During both open-label periods, there were mean improvements in pain severity, PGIC, PedsQL, and MASC scores. No unexpected safety issues were detected. The most commonly reported treatment-emergent AEs were nausea, headache, vomiting, and dizziness. Mean increases in heart rate and blood pressure were observed, and were consistent with the AE profile in adults with FM. The open-label findings provide preliminary evidence that milnacipran may improve symptoms of JFM, with a safety and tolerability profile that is consistent with the experience in adult FM patients. Future trial designs for JFM should consider the relatively low recognition of this condition compared to adult FM and the difficulties with enrollment. NCT01328002; NCT01331109 The online version of this article (doi:10.1186/s12969-015-0025-9) contains supplementary material, which is available to authorized users.
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影响因子：
2.3
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通讯作者：
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DOI：
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发表时间：
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期刊：
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