儿童家族性地中海热患者每日一次与每日两次秋水仙碱剂量的疗效比较——一项随机对照非劣效性试验。
Comparison of the efficacy of once- and twice-daily colchicine dosage in pediatric patients with familial Mediterranean fever--a randomized controlled noninferiority trial.
作者信息
Polat Adem, Acikel Cengizhan, Sozeri Betul, Dursun Ismail, Kasapcopur Ozgur, Gulez Nesrin, Simsek Dogan, Saldir Mehmet, Dokurel Ipek, Poyrazoglu Hakan, Bakkaloglu Sevcan, Delibas Ali, Ekinci Zelal, Ayaz Nuray A, Kandur Yasar, Peru Harun, Kurt Yasemin G, Polat Safiye R, Unsal Erbil, Makay Balahan, Gok Faysal, Ozen Seza, Demirkaya Erkan
机构信息
Gulhane Military Medical Academy, Pediatric Rheumatology Unit, Ankara, Turkey.
Gulhane Military Medical Academy, Department of Biostatistics, Ankara, Turkey.
出版信息
Arthritis Res Ther. 2016 Apr 7;18:85. doi: 10.1186/s13075-016-0980-7.
BACKGROUND
In this study, we examined the efficacy and safety of a once-daily dosage schema of colchicine compared with a twice-daily dosage schema in pediatric patients with familial Mediterranean fever (FMF).
METHODS
In this 24-week, multicenter, randomized controlled noninferiority trial, pediatric patients newly diagnosed with FMF carrying a homozygous or compound heterozygous mutation and not receiving any treatment were included. Patients were randomly assigned using a block randomization method to receive treatment with a once- or twice-daily dosage. Clinical and laboratory characteristics and medication side effects were recorded and compared between groups. The study was carried out in compliance with Good Clinical Practice and the Consolidated Standards for Reporting of Trials (CONSORT) statement.
RESULTS
A total of 92 patients were selected, and 79 patients completed the study. There were 42 patients in the once-daily dosage group and 37 in the twice-daily dosage group. The results indicated that the once-daily dosage was not inferior to the twice-daily dosage regarding decrease in attack frequency and duration as well as improvement in clinical findings and Mor severity scores. Alterations in laboratory findings indicating inflammation, such as erythrocyte sedimentation rate, C-reactive protein, and serum amyloid A, were similar in both groups. The rates of drug side effects were similar between the once- and twice-daily dosage groups, implying comparable safety of colchicine, with the exception of diarrhea, which was slightly higher in the once-daily dosage group.
CONCLUSIONS
Using colchicine with either a once- or twice-daily dosage provides similar clinical and laboratory improvements. Considering both efficacy and safety, colchicine can be prescribed with a once-daily dosage.
TRIAL REGISTRATION ID
ClinicalTrials.gov identifier NCT02602028 . Registered 5 November 2015.
背景
在本研究中，我们比较了秋水仙碱每日一次给药方案与每日两次给药方案在家族性地中海热（FMF）儿科患者中的疗效和安全性。
方法
在这项为期24周的多中心随机对照非劣效性试验中，纳入了新诊断为FMF且携带纯合或复合杂合突变且未接受任何治疗的儿科患者。采用区组随机化方法将患者随机分配接受每日一次或两次给药治疗。记录并比较两组的临床和实验室特征以及药物副作用。本研究按照良好临床实践和试验报告统一标准（CONSORT）声明进行。
结果
共入选92例患者，79例患者完成研究。每日一次给药组有42例患者，每日两次给药组有37例患者。结果表明，在发作频率和持续时间的降低以及临床症状和Mor严重程度评分的改善方面，每日一次给药并不劣于每日两次给药。两组中表明炎症的实验室检查结果变化，如红细胞沉降率、C反应蛋白和血清淀粉样蛋白A，相似。每日一次和每日两次给药组的药物副作用发生率相似，这意味着秋水仙碱的安全性相当，但腹泻除外，每日一次给药组的腹泻发生率略高。
结论
每日一次或两次服用秋水仙碱均可带来相似的临床和实验室改善。综合考虑疗效和安全性，秋水仙碱可以每日一次给药。
试验注册号
ClinicalTrials.gov标识符NCT02602028。于2015年11月5日注册。
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