Tonsillotomy or Follow-up in PFAPA Syndrome

University of Oulu

Status: Completed

Conditions: PFAPA Syndrome

Treatments: Procedure: Tonsillotomy

Study type: Interventional

Funder types: Other

Identifiers

NCT03331497

TT 50_2017

Details and patient eligibility

About

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.

Full 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.

Enrollment: 22 patients

Sex: All

Ages: 1 to 12 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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22 participants in 2 patient groups

Tonsillotomy

Experimental group

Description:

The patients diagnosed with PFAPA will have tonsillotomy performed in one month from randomisation.

Treatment:

Procedure: Tonsillotomy

Follow up

No Intervention group

Description:

The patients diagnosed with PFAPA will be monitored for 3 months time. If the symptoms still persist, tonsillectomy will be performed