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Published on: November 6, 2019
Tonsillotomy for Periodic Fever Syndrome: A Randomized and Controlled Trial
Ulla Lantto1,2, Terhi Tapiainen1,3, Tytti Pokka1
1Clinical Medicine Research Unit, Medical Research Center, University of Oulu, Oulu, Finland.
Insights
Tonsillotomy significantly reduced symptoms in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome compared to observation. This procedure offers a promising alternative for managing PFAPA in pediatric patients.
Area of Science:
- Pediatric Otolaryngology
- Pediatric Immunology
- Clinical Trial Research
Background:
- Periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome is a common autoinflammatory disorder in children.
- Tonsillectomy is an established treatment, but tonsillotomy presents a less invasive option with a potentially better safety profile.
- The comparative efficacy of tonsillotomy versus observation for PFAPA syndrome requires further investigation.
Purpose of the Study:
- To evaluate the efficacy of tonsillotomy compared to observation alone in children diagnosed with PFAPA syndrome.
- To assess symptom resolution and fever burden at a 3-month follow-up period.
- To determine the need for subsequent tonsillectomy in both treatment arms.
Main Methods:
- A randomized, multicenter trial with a sequential design was conducted.
- 16 pediatric patients with PFAPA syndrome were randomized into either a tonsillotomy group or an observation-only control group.
- Daily symptom diaries were utilized to monitor disease activity and treatment outcomes.
Main Results:
- A significantly higher proportion of patients in the tonsillotomy group (87.5%) achieved symptom resolution compared to the observation group (25%) (p=0.0021).
- The mean number of fever days compatible with PFAPA syndrome was substantially lower in the tonsillotomy group (0.8 days) versus the control group (6.5 days) (p=0.007).
- All patients in the observation group ultimately required rescue tonsillectomy, while none in the tonsillotomy group did.
Conclusions:
- Tonsillotomy appears to be an effective treatment for children suffering from PFAPA syndrome.
- The procedure demonstrated a significant reduction in PFAPA symptoms and fever episodes.
- Further research is warranted to establish the long-term effectiveness of tonsillotomy in managing PFAPA syndrome.
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.
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