Background/Purpose: PFAPA syndrome is episodic  disease characterized by periodic episodes of high fever, aphthous stomatitis, pharyngitis, and cervical adenitis, so far no therapy was shown to be effective in preventing the attacks.

The aim of our study was to evaluate the efficacy of colchicine in reducing  attacks of  PFAPA.

Methods:

We carried a randomized control study among patients diagnosed with PFAPA in the pediatric Rheumatology clinic- Rambam Medical center, Israel.

The patients were randomized into two groups followed for 6 months. In months

1-3 both groups were followed without preventive therapy; In months 4-6, group1 continued to be followed in the same manner, patients in group2 received colchicine for three months in a standard dose. During the study patients and physician recorded all PFAPA episodes, in a log book. DNA analysis for the common FMF mutation was done for all patients.

Results:

14 patients 5.8±1.9 years old were evaluated(8 in group1, 6 in group2).

The number of episodes in the first 3 months were 3.5±1.3 in group1 and 4.9±2.45 in group2 (p-0.187), the number of episodes in group2 under colchicine therapy were 1.7±1 compared to 4.9±2.4 in the period without therapy(p-0.01).

Seven patients were found to be carrier of one FMF mutation;(4 in group2 and 3 in group1). Patients with PFAPA who carry one mutation for FMF responded  to colchicine therapy (6.1±2 episodes before and 2±1.4 after therapy p-0.03).

Conclusion: : Colchicine therapy might be effective in increasing intervals between  episodes of PFAPA. Larger studies are needed to confirm these findings.

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« Back to 2012 ACR/ARHP Annual Meeting

ACR Meeting Abstracts - https://acrabstracts.org/abstract/colchicine-as-a-therapeutic-option-in-periodic-fever-aphtous-stomatitis-pharingitis-syndrome-and-cervical-adenitis-pfapa/