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

Autor: Polat A; Gulhane Military Medical Academy, Pediatric Rheumatology Unit, Ankara, Turkey., Acikel C; Gulhane Military Medical Academy, Department of Biostatistics, Ankara, Turkey.; Gulhane Military Medical Academy, Institute of Health Sciences, FMF Arthritis Vasculitis and Orphan Disease Research Center (FAVOR), Ankara, Turkey., Sozeri B; Ege University, Pediatric Rheumatology Unit, Izmir, Turkey., Dursun I; Erciyes University, Pediatric Rheumatology Unit, Kayseri, Turkey., Kasapcopur O; İstanbul University Cerrahpasa Medical Faculty, Pediatric Rheumatology Unit, Istanbul, Turkey., Gulez N; Dr. Behçet Uz Children's Training and Research Hospital, Pediatric Allergy and Immunology Department, İzmir, Turkey., Simsek D; Gulhane Military Medical Academy, Pediatric Rheumatology Unit, Ankara, Turkey., Saldir M; Gulhane Military Medical Academy, Pediatric Rheumatology Unit, Ankara, Turkey., Dokurel I; İstanbul University Cerrahpasa Medical Faculty, Pediatric Rheumatology Unit, Istanbul, Turkey., Poyrazoglu H; Erciyes University, Pediatric Rheumatology Unit, Kayseri, Turkey., Bakkaloglu S; Gazi University Medical Faculty, Pediatric Rheumatology Unit, Ankara, Turkey., Delibas A; Mersin University Medical Faculty, Pediatric Rheumatology Unit, Mersin, Turkey., Ekinci Z; Kocaeli University Medical Faculty, Pediatric Rheumatology Unit, Kocaeli, Turkey., Ayaz NA; İstanbul Kanuni Sultan Süleyman Educational and Research Hospital, Pediatric Rheumatology Unit, Istanbul, Turkey., Kandur Y; Gazi University Medical Faculty, Pediatric Rheumatology Unit, Ankara, Turkey., Peru H; Selcuk University Medical Faculty, Pediatric Rheumatology Unit, Konya, Turkey., Kurt YG; Gulhane Military Medical Academy, Department of Biochemistry, Ankara, Turkey., Polat SR; Gulhane Military Medical Academy, Department of Biochemistry, Ankara, Turkey., Unsal E; Dokuz Eylül University, Pediatric Rheumatology Unit, İzmir, Turkey., Makay B; Dokuz Eylül University, Pediatric Rheumatology Unit, İzmir, Turkey., Gok F; Gulhane Military Medical Academy, Pediatric Rheumatology Unit, Ankara, Turkey., Ozen S; Hacettepe University Medical Faculty, Pediatric Rheumatology Unit, Ankara, Turkey., Demirkaya E; Gulhane Military Medical Academy, Pediatric Rheumatology Unit, Ankara, Turkey. erkandemirkaya@yahoo.com.; Gulhane Military Medical Academy, Department of Biostatistics, Ankara, Turkey. erkandemirkaya@yahoo.com.; Gulhane Military Medical Academy, Institute of Health Sciences, FMF Arthritis Vasculitis and Orphan Disease Research Center (FAVOR), Ankara, Turkey. erkandemirkaya@yahoo.com.
Jazyk: angličtina
Zdroj: Arthritis research & therapy [Arthritis Res Ther] 2016 Apr 07; Vol. 18, pp. 85. Date of Electronic Publication: 2016 Apr 07.
DOI: 10.1186/s13075-016-0980-7
Abstrakt: 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.
Databáze: MEDLINE
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