Comparison of the efficacy of once- and twice-daily colchicine dosage in pediatric patients with familial Mediterranean fever - a randomized controlled noninferiority trial

Authors

Adem Polat, Gulhane Military Medical Academy
Cengizhan Acikel, Gulhane Military Medical Academy
Betul Sozeri, Ege Üniversitesi
Ismail Dursun, Erciyes Üniversitesi
Ozgur Kasapcopur, İstanbul University-Cerrahpaşa Cerrahpaşa Faculty of Medicine
Nesrin Gulez, Dr. Behçet Uz Children Research and Training Hospital
Dogan Simsek, Gulhane Military Medical Academy
Mehmet Saldir, Gulhane Military Medical Academy
Ipek Dokurel, İstanbul University-Cerrahpaşa Cerrahpaşa Faculty of Medicine
Hakan Poyrazoglu, Erciyes Üniversitesi
Sevcan Bakkaloglu, Gazi University, Faculty of Medicine
Ali Delibas, Mersin Üniversitesi Tip Fakültesi
Zelal Ekinci, Kocaeli Üniversitesi
Nuray A. Ayaz, Istanbul Kanuni Sultan Suleyman Education and Research Hospital
Yasar Kandur, Gazi University, Faculty of Medicine
Harun Peru, Selçuk Üniversitesi
Yasemin G. Kurt, Gulhane Military Medical Academy
Safiye R. Polat, Gulhane Military Medical Academy
Erbil Unsal, Dokuz Eylül Üniversitesi
Balahan Makay, Dokuz Eylül Üniversitesi
Faysal Gok, Gulhane Military Medical Academy
Seza Ozen, Hacettepe Üniversitesi
Erkan Demirkaya, Gulhane Military Medical AcademyFollow

Document Type

Article

Publication Date

4-7-2016

Journal

Arthritis Research and Therapy

Volume

18

Issue

1

URL with Digital Object Identifier

10.1186/s13075-016-0980-7

Abstract

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.