Evaluation of clinical responses to probiotics and colchicine in PFAPA patients based on autoinflammatory diseases activity index (AIDAI) scores.
Data on prophylaxis in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) are limited for probiotics. We aimed to evaluate clinical responses of probiotic and colchicine. We included PFAPA patients receiving colchicine or probiotic (Lactobacillus plantarum HEAL9 (5×10 cfu/tablet), Lactobacillus paracasei 8700:2 (5 × 10 cfu/tablet) and a vitamin mixture (0.75 μg/tablet vitamin D3, 0.38 μg/tablet, vitamin B12, 30 μg/tablet folic acid)) for ≥3 months. Disease activity was assessed using Auto-Inflammatory Disease Activity Index (AIDAI). Monthly AIDAI scores from 3-month periods before and after prophylaxis were compared retrospectively. Early clinical response was defined as AIDAI score change from the month before to after prophylaxis. The time to first attack was compared. The study included 130 patients (colchicine: n = 97; probiotics: n = 33). Significant early clinical response occurred in both groups, with median AIDAI scores decreasing from the month before to after prophylaxis (colchicine: p < 0.001; probiotics: p < 0.001). Time to first attack was longer in the colchicine group than probiotic group (p = 0.048). Colchicine and probiotics showed similar short-term improvement in PFAPA activity. However, differences exist in time to first attack and adverse effects and should be discussed with families when selecting prophylaxis. This study provides one of the first evaluations of clinical responses to colchicine and probiotic prophylaxis in children with PFAPA syndrome using objective disease activity scoring. By demonstrating clinical improvements in both groups, the study contributes to the growing body of evidence supporting probiotic use. The identification of gastrointestinal symptoms as a factor affecting colchicine response may contribute to a more personalized treatment approach. These findings contribute valuable data to the limited literature on PFAPA prophylaxis and support shared decision-making in clinical practice.