Tonsillotomy or Follow-up in PFAPA Syndrome

PFAPA Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03331497
• Other study ID #: TT 50_2017
• Status: Completed
• Phase: N/A
• Start date: October 24, 2017
• Est. completion date: February 2, 2022

Study information

• Verified date: February 2022
• Source: University of Oulu
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 22
• Est. completion date: February 2, 2022
• Est. primary completion date: December 30, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 12 Years

Eligibility

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

Related Conditions & MeSH terms

• PFAPA Syndrome
• Syndrome

Intervention

Procedure:
• Tonsillotomy
About 2/3 of the palatine tonsil tissue is removed with coplator or bipolar knife.

Locations

Country	Name	City	State
Finland	Oulu Unversity Hospital	Oulu
Finland	Tampere University Hospital	Tampere
Finland	Turku University Hospital	Turku

Sponsors (6)

Lead Sponsor	Collaborator
University of Oulu	Oulu University Hospital, Tampere University, Tampere University Hospital, Turku University Hospital, University of Turku

Country where clinical trial is conducted

Finland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Cure	Proportion of patients who do not have any PFAPA symptoms	3 months from randomization
Secondary	Rescue surgery	Proportion of patients needing rescue surgery (tonsillectomy)	12 months from randomization
Secondary	Days with fever	Number of days with fever >38C in symptom diaries	3 months from randomization
Secondary	Cure	Proportion of patients who do not have any PFAPA symptoms	6 months from randomization