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. 2025 Jul 31;184(8):516.
doi: 10.1007/s00431-025-06363-7.

Colchicine efficacy on oral ulcers caused by Behçet's spectrum disorders including idiopathic recurrent aphthous stomatitis, PFAPA, and Behçet's Disease

Zelal Aydin  1 , Feray Kaya  1 , Elif Kucuk  1 , Lutfiye Koru  1 , Eda Nur Dizman  1 , Hatice Kubra Dursun  1 , Merve Ozen Balci  1 , Fatih Haslak  1 , Kubra Ozturk  2
Affiliations

Colchicine efficacy on oral ulcers caused by Behçet's spectrum disorders including idiopathic recurrent aphthous stomatitis, PFAPA, and Behçet's Disease

Zelal Aydin et al. Eur J Pediatr. .

Abstract

Recurrent aphthous stomatitis (RAS) is a common condition in pediatric age. Since there are significant genetic commonalities among idiopathic cases, periodic fever, aphthous stomatitis, pharyngitis, and adenitis syndrome (PFAPA), and Behçet's disease (BD), they were proposed to be considered in one clinical spectrum termed Behçet's Spectrum Disorders (BSDs). This retrospective study aimed to evaluate the efficacy of colchicine on oral ulcers in children with BSD related RAS (BSD-RAS). Secondary aim was to compare the disease groups in terms of clinical and laboratory benefits. Data at admission and one year-after the treatment initiation were compared. A total of 317 patients with RAS were evaluated, and 186 patients classified as BSD-RAS (Female: n = 105, 56%). The median age at symptom onset was 5 (0.5-16.5) years. Colchicine was started to 142 (76%) patients. We observed a significant reduction in the frequency and duration of oral ulcers, as well as in the visual analogue scale (VAS) scores reported by patients/families and physicians, and the erythrocyte sedimentation rate during symptom-free periods across all three groups. In patients with PFAPA, both difference between initial and final recorded values (Δ) patient/families and Δ physician VAS scores were significantly higher than in other groups (p < 0.001). Furthermore, both Δ duration (p = 0.005) and Δ frequency of oral aphthae (p = 0.012) were significantly higher in idiopathic cases than those with PFAPA.

Conclusion: These findings demonstrate a substantial reduction in both clinical symptoms and laboratory markers following colchicine treatment, supporting its effectiveness as a therapeutic option for BSD-RAS.

What is known: •Recurrent aphthous stomatitis, PFAPA, and Behçet's disease may manifest with similar oral ulcers in pediatric patients. •Colchicine is frequently used in this clinical spectrum known as Behçet's Spectrum Disorders, although there is a scarcity of pediatric data.

What is new: •Colchicine treatment significantly improved clinical and laboratory outcomes across all groups. •Idiopathic group had greater reductions in oral ulcer frequency and duration while PFAPA associated cases showed highest improvement in visual analogue scale scores.

Keywords: Behçet’s Spectrum Disorders; Behçet’s disease; Colchicine; PFAPA; Recurrent aphthous stomatitis.

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Conflict of interest statement

Declarations. Competing interests: The authors declare no competing interests. Ethics approval: The study was approved by Institutional Review Board (26/09/2024/911) of Istanbul Medipol University. The study complied with the recommendations of the Declaration of Helsinki for human biomedical research. Informed consent: This study was conducted in compliance with the Helsinki Declaration as well as local laws and regulations. Informed consent was obtained from the patients and their legal caregivers. The Patient and Public Involvement statement: Patients or the public were not involved in the design, or conduct, or reporting, or dissemination plans of our research. Prepublication note: None. Conflict of interest: The authors declare that they have no conflict of interest.

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