Volume 12 Supplement 1
The use of colchicine in PFAPA syndrome, the french experience and literature review
© Dusser et al; licensee BioMed Central Ltd. 2014
Published: 17 September 2014
Background: PFAPA syndrome is the most frequent periodic fever syndrome in non-Mediterranean patients. The cause remains obscure but overexpression of inflammasome-related genes and increase IL-1b during attacks suggest an autoinflammatory mechanism. We wondered whether colchicine could be used as effective prophylactic treatment in PFAPA syndrome.
To compare 2 groups of PFAPA patients distinguished by their response to colchicine prophylaxis, and to identify the predictive factors of response to this treatment.
We performed a retrospective, multicentric, chart review of PFAPA patients under colchicine prophylaxis. We distinguished one responder group, defined as patients, who had no more, or twice fewer crises under colchicine and another one of non-responders. Subgroup analyses were performed using the nonparametric Mann-Whitney test for the quantitative data and calculating the odds ratio and confidence interval for qualitative data. The difference between the two groups was considered significant for p value <0.05 or a confidence interval different from 1.
Twenty children, 65% of boys, were studied. Their mean age at disease onset was 2.3 ± 1.5 years. The mean duration of attacks was 3.2 ± 1.1 days (SD) (1 to 7 days) of strong fever (mean 39.9°C) with chills (30%), pharyngitis (85%), abdominal pain (75%), cervical adenitis (65%), asthenia (60%) and aphtous stomatitis (50%). Half of patients, 57% (8/14) were heterozygous in the MEFV gene. Nine patients were responders to colchicine. No significant differences were found between the two groups (responder and non-responder).
We observed a relatively high rate of response to colchicine; however our study could not sort out the predictive factors of this effect.
Disclosure of interest
This article is published under license to BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.