Tonsillotomy or Follow-up in PFAPA Syndrome

NCT ID: NCT03331497

Last Updated: 2022-02-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

22 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-10-24

Study Completion Date

2022-02-02

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.

Conditions

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PFAPA Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Tonsillotomy

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

Group Type EXPERIMENTAL

Tonsillotomy

Intervention Type PROCEDURE

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

Follow up

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

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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Tonsillotomy

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

Intervention Type PROCEDURE

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
Minimum Eligible Age

1 Year

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Oulu University Hospital

OTHER

Sponsor Role collaborator

Tampere University

OTHER

Sponsor Role collaborator

Tampere University Hospital

OTHER

Sponsor Role collaborator

Turku University Hospital

OTHER_GOV

Sponsor Role collaborator

University of Turku

OTHER

Sponsor Role collaborator

University of Oulu

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Oulu Unversity Hospital

Oulu, , Finland

Site Status

Tampere University Hospital

Tampere, , Finland

Site Status

Turku University Hospital

Turku, , Finland

Site Status

Countries

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Finland

Other Identifiers

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TT 50_2017

Identifier Type: -

Identifier Source: org_study_id

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