title: Comparison of the efficacy of once- and twice-daily colchicine dosage in pediatric patients with familial Mediterranean fever--a randomized controlled noninferiority trial.
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.
  
  
  BACKGROUND
  ClinicalTrials.gov identifier NCT02602028 . Registered 5 November 2015.
authors.affiliation: Gulhane Military Medical Academy, Pediatric Rheumatology Unit, Ankara, Turkey.
authors.forename: Adem
authors.initials: A
authors.lastname: Polat
authors.affiliation: 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.
authors.forename: Cengizhan
authors.initials: C
authors.lastname: Acikel
authors.affiliation: Ege University, Pediatric Rheumatology Unit, Izmir, Turkey.
authors.forename: Betul
authors.initials: B
authors.lastname: Sozeri
authors.affiliation: Erciyes University, Pediatric Rheumatology Unit, Kayseri, Turkey.
authors.forename: Ismail
authors.initials: I
authors.lastname: Dursun
authors.affiliation: İstanbul University Cerrahpasa Medical Faculty, Pediatric Rheumatology Unit, Istanbul, Turkey.
authors.forename: Ozgur
authors.initials: O
authors.lastname: Kasapcopur
authors.affiliation: Dr. Behçet Uz Children's Training and Research Hospital, Pediatric Allergy and Immunology Department, İzmir, Turkey.
authors.forename: Nesrin
authors.initials: N
authors.lastname: Gulez
authors.affiliation: Gulhane Military Medical Academy, Pediatric Rheumatology Unit, Ankara, Turkey.
authors.forename: Dogan
authors.initials: D
authors.lastname: Simsek
authors.affiliation: Gulhane Military Medical Academy, Pediatric Rheumatology Unit, Ankara, Turkey.
authors.forename: Mehmet
authors.initials: M
authors.lastname: Saldir
authors.affiliation: İstanbul University Cerrahpasa Medical Faculty, Pediatric Rheumatology Unit, Istanbul, Turkey.
authors.forename: Ipek
authors.initials: I
authors.lastname: Dokurel
authors.affiliation: Erciyes University, Pediatric Rheumatology Unit, Kayseri, Turkey.
authors.forename: Hakan
authors.initials: H
authors.lastname: Poyrazoglu
authors.affiliation: Gazi University Medical Faculty, Pediatric Rheumatology Unit, Ankara, Turkey.
authors.forename: Sevcan
authors.initials: S
authors.lastname: Bakkaloglu
authors.affiliation: Mersin University Medical Faculty, Pediatric Rheumatology Unit, Mersin, Turkey.
authors.forename: Ali
authors.initials: A
authors.lastname: Delibas
authors.affiliation: Kocaeli University Medical Faculty, Pediatric Rheumatology Unit, Kocaeli, Turkey.
authors.forename: Zelal
authors.initials: Z
authors.lastname: Ekinci
authors.affiliation: İstanbul Kanuni Sultan Süleyman Educational and Research Hospital, Pediatric Rheumatology Unit, Istanbul, Turkey.
authors.forename: Nuray A
authors.initials: NA
authors.lastname: Ayaz
authors.affiliation: Gazi University Medical Faculty, Pediatric Rheumatology Unit, Ankara, Turkey.
authors.forename: Yasar
authors.initials: Y
authors.lastname: Kandur
authors.affiliation: Selcuk University Medical Faculty, Pediatric Rheumatology Unit, Konya, Turkey.
authors.forename: Harun
authors.initials: H
authors.lastname: Peru
authors.affiliation: Gulhane Military Medical Academy, Department of Biochemistry, Ankara, Turkey.
authors.forename: Yasemin G
authors.initials: YG
authors.lastname: Kurt
authors.affiliation: Gulhane Military Medical Academy, Department of Biochemistry, Ankara, Turkey.
authors.forename: Safiye R
authors.initials: SR
authors.lastname: Polat
authors.affiliation: Dokuz Eylül University, Pediatric Rheumatology Unit, İzmir, Turkey.
authors.forename: Erbil
authors.initials: E
authors.lastname: Unsal
authors.affiliation: Dokuz Eylül University, Pediatric Rheumatology Unit, İzmir, Turkey.
authors.forename: Balahan
authors.initials: B
authors.lastname: Makay
authors.affiliation: Gulhane Military Medical Academy, Pediatric Rheumatology Unit, Ankara, Turkey.
authors.forename: Faysal
authors.initials: F
authors.lastname: Gok
authors.affiliation: Hacettepe University Medical Faculty, Pediatric Rheumatology Unit, Ankara, Turkey.
authors.forename: Seza
authors.initials: S
authors.lastname: Ozen
authors.affiliation: 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.
authors.forename: Erkan
authors.initials: E
authors.lastname: Demirkaya
mesh_terms: D002648:Child; D003078:Colchicine / Q000008:administration & dosage*; D004334:Drug Administration Schedule; D010505:Familial Mediterranean Fever / Q000188:drug therapy*; D005260:Female; D006801:Humans; D008297:Male; D050257:Tubulin Modulators / Q000008:administration & dosage*
chemical_list: D050257:Tubulin Modulators; D003078:Colchicine
keywords: Colchicine; Dosage schema; Familial Mediterranean fever; Pediatric