Tonsillotomy for Periodic Fever Syndrome: A Randomized and Controlled Trial

Objective Tonsillectomy is an effective treatment for periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome. Tonsillotomy has a milder operative risk profile and postoperative morbidity in children than tonsillectomy. We aimed to compare the efficacy of tonsillotomy to observation‐only in children with PFAPA syndrome at a 3‐month follow‐up. Methods This was a randomized multicenter trial with sequential design. Participants were randomized into a tonsillotomy group and a control group that was only observed. The trial started in 1/2017 and was accomplished in 12/2021 with 16 patients (10 boys, six girls, the mean age 4.2 years). The symptoms were monitored with daily symptom diaries. Results After the 3‐month follow‐up, 7/8 patients (87.5%) in the tonsillotomy group and 2/8 (25%) patients in the control group were free from PFAPA symptoms (95% CI 13% to 87%; p = 0.0021). The mean number of days with fever was 2.6 (SD 3.7) in the tonsillotomy group and 8.0 (SD 6.5) days in the control group (n = 8) (p = 0.06). Mean number of fever days compatible with PFAPA syndrome was 0.8 (SD 1.4) in the tonsillotomy group and 6.5 (SD 6.0) in the control group (95%CI −10% to −1%; p = 0.007). Rescue tonsillectomy was needed for all patients in the control group and none of the patients in the tonsillotomy group. Conclusions Tonsillotomy might be an effective treatment option for children with PFAPA syndrome. Further studies are needed to clarify the long‐term efficacy of tonsillotomy for treating PFAPA. Level of Evidence 2 Laryngoscope, 134:968–972, 2024 We randomized 16 PFAPA children in tonsillotomy group and control group (observation only). After 3 months of follow‐up, 88% of the patients in tonsillotomy group and 25% in the control group were free from PFAPA flares (95% CI 13%–87%; p = 0.0021). Tonsillotomy was effective in treating PFAPA in this study.