Tonsillotomy or Follow-up in PFAPA Syndrome

Study Overview
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Brief Summary
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Children with periodic fever, aphthous stomatitis, pharyngitis, adenitis (PFAPA) -syndrome diagnosis will be randomised either to tonsillotomy (partial tonsillectomy) or 3 months follow up. At follow up visit 3 months after randomisation the children from either groups with ongoing symptoms will be sent to tonsillectomy.

Detailed Description
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Children with periodic fever, aphthous stomatitis, pharyngitis, adenitis (PFAPA) -syndrome diagnosis will be randomised either to tonsillotomy (partial tonsillectomy) or 3 months follow up. In both groups the symptoms are monitored with symptom diaries. At follow up visit 3 months after randomisation the children from either groups with ongoing symptoms will be sent to tonsillectomy (total remove of palatine tonsil). The design is sequential trial. From the previous trials we know that the effect of tonsillectomy is about 90% and that during follow up for six months as much as 50% of the patients will recover. Because the syndrome is rare and because the rescue operations of the tonsils (tonsillectomy in a person who has gone through tonsillotomy), the sample size has to be as low as possible. That is the reason why we chose sequential design, with assessments after every 8 patient. Randomization is made in blocks of four. In sequential design we use the following assumptions: We are looking for a 40% absolute difference in cure rate between tonsillotomy (90%) and follow up (50%) groups. Two sided Whitehead design Maximum likelihood estimation (MLE) Five assessments (sample size 4+4, 8+8. 12+12, 16+16 and 19+19) type 1 error 5 % and power 80% The sample size will be from 8 to 38 patients depending on when the trial can be stopped along assessments.

Official Title
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Tonsillotomy or Follow-up in PFAPA -Syndrome - Randomised, Controlled Study Using Sequential Design

Conditions
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PFAPA Syndrome

Intervention / Treatment
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* Procedure: Tonsillotomy


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosed PFAPA syndrome: regularly occurring fever episodes for 3-5 days for at least five times or for 6 months Exclusion Criteria: prior tonsil surgery

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Tonsillotomy<br>The patients diagnosed with PFAPA will have tonsillotomy performed in one month from randomisation. | Procedure: Tonsillotomy<br>* About 2/3 of the palatine tonsil tissue is removed with coplator or bipolar knife.<br>|
| No Intervention: Follow up<br>The patients diagnosed with PFAPA will be monitored for 3 months time. If the symptoms still persist, tonsillectomy will be performed | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cure | Proportion of patients who do not have any PFAPA symptoms | 3 months from randomization | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Rescue surgery | Proportion of patients needing rescue surgery (tonsillectomy) | 12 months from randomization | 
| Days with fever | Number of days with fever >38C in symptom diaries | 3 months from randomization | 
| Cure | Proportion of patients who do not have any PFAPA symptoms | 6 months from randomization |