Tonsillotomy or Follow-up in PFAPA Syndrome

Not Applicable

Completed

• Conditions: PFAPA Syndrome
• Interventions: Procedure: Tonsillotomy

• Registration Number: NCT03331497
• Lead Sponsor: University of Oulu

Brief Summary

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

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.

Study Timeline

• Study Start: 2017-10-24
• Study First Submitted: 2017-10-31
• Study First Submitted QC: 2017-10-31
• Study First Posted: 2017-11-06
• Primary Completion: 2021-12-30
• Status Verified: 2022-02
• Study Completion: 2022-02-02
• Last Update Submitted: 2022-02-03
• Last Update Posted: 2022-02-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 22

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Tonsillotomy	Tonsillotomy	The patients diagnosed with PFAPA will have tonsillotomy performed in one month from randomisation.

Primary Outcome Measures

Name	Time	Method
Cure	3 months from randomization	Proportion of patients who do not have any PFAPA symptoms

Secondary Outcome Measures

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

Trial Locations

Locations (3)

Turku University Hospital; 🇫🇮 Turku, Finland

Oulu Unversity Hospital; 🇫🇮 Oulu, Finland

Tampere University Hospital; 🇫🇮 Tampere, Finland