Background/Purpose: Tonsillectomy is considered curative in a majority of patients with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome. Predictors of outcome following tonsillectomy are unknown.

Methods: Patients from Vanderbilt Children’s Hospital and the National Institutes of Health (NIH) who met the diagnostic criteria for PFAPA and underwent tonsillectomy were recruited. Clinical and demographic features of patients who had complete resolution of symptoms and those who had continued symptoms after tonsillectomy were compared with the Chi-squared or Mann-Whitney U test.

Results: Fifty-five patients (38 from Vanderbilt and 17 from NIH) were followed after tonsillectomy for an average of 5.6 years. Following tonsillectomy, 63% of patients from Vanderbilt had complete resolution of episodes, while only 18% of patients from NIH had complete resolution. Of the 29 patients who continued to have episodes following tonsillectomy, 52% had less severe or less frequent episodes, 24% had a period of remission ranging from 9 months to 6 years followed by continued episodes, 17% had no change in episodes, and 3% reported feverless episodes. Patients who did not have complete resolution of episodes following tonsillectomy were more likely to have abdominal pain and limb pain (arthralgia or myalgias) during episodes (Table).  

Conclusion: The majority of patients with PFAPA had improvement in the severity and frequency of episodes following tonsillectomy. Differences in outcome at Vanderbilt and NIH likely stem from referral bias in that NIH is a quaternary referral center and more likely to see patients refractory to conventional therapies. Patients with abdominal pain and limb pain may have lymphoid tissue outside the palatine tonsils that triggers systemic inflammation or may have illnesses distinct from PFAPA.  Further analysis of tonsil immunology and imaging of lymphoid tissue outside of the pharynx in these two groups may help to resolve these questions.