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

Abstract

BackgroundIn 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).MethodsIn 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.ResultsA 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.ConclusionsUsing 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 IDClinicalTrials.gov identifier NCT02602028. Registered 5 November 2015.

Trombocytopenia related to colchicine: report of two cases

“…FMF is a common disease in Turkey. Colchicine is very effective in preventing FMF attacks and amyloidosis (1,4). It is an anti-mitotic drug that blocks mitotic cells in metaphase.…”

“…Colchicine, derivated from lily, is a drug which is used for the treatment of various rheumatological diseases such as familial Mediterranean fever (FMF), Behcet Disease and gouty arthritis. FMF is the most frequent autoinflammatory disease which can be well controlled with lifelong use of colchicine (1) The disease is frequently observed, especially in the Mediterranean region among Sephardic Jews, Armenians, Turks, and Arabs (2) .Daily colchicine use has been shown to reduce the frequency, severity, and duration of attacks (1) Colchicine also has been found to be effective in decreasing the prevalence of amyloidosis (1). Therapeutic oral dose of colchicine (0.5-2.0 mg/ day) may cause cramping, abdominal pain, hyperperistalsis, diarrhea and vomiting (3,4) Besides, colchicine may rarely cause bone marrow failure, agranulocytosis and thrombocytopenia as well (4).…”

Colchicine

“…We recommend taking the entire colchicine dose at once in order to improve compliance. If the patient develops diarrhea due to ingestion of a relatively high single dose of colchicine the dose should be divided to twice per day without affecting its effectiveness [116].…”

Evidenzbasierte Therapieempfehlungen für das familiäre Mittelmeerfieber

“…In einer französischen Arbeit gaben 40 % der Patienten mit unzureichender Colchicin-Wirkung an, das Medikament regelmäßig einzunehmen [33]. Die täglich einmalige Einnahme von Colchicin stellt gegenüber einer über den Tag verteilten Dosierung hinsichtlich Wirkung und Nebenwirkungen keinen Nachteil dar [57] und kann die Compliance des Patienten erhöhen.…”