Tonsillotomy or Follow-up in PFAPA Syndrome

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

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.

Study Overview

• Status: Completed
• Conditions: PFAPA Syndrome
• Intervention / Treatment: Procedure: Tonsillotomy

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 Type: Interventional
• Enrollment (Actual): 22
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Finland
  • Oulu, Finland
    • Oulu Unversity Hospital
  • Tampere, Finland
    • Tampere University Hospital
  • Turku, Finland
    • Turku University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 months to 10 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Tonsillotomy; The patients diagnosed with PFAPA will have tonsillotomy performed in one month from randomisation.	Procedure: Tonsillotomy; About 2/3 of the palatine tonsil tissue is removed with coplator or bipolar knife.
No Intervention: Follow up; 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?

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

Collaborators and Investigators

• Sponsor: University of Oulu
• Collaborators: Turku University Hospital; University of Turku; Oulu University Hospital; Tampere University; Tampere University Hospital

Study record dates

Study Major Dates

• Study Start (Actual): October 24, 2017
• Primary Completion (Actual): December 30, 2021
• Study Completion (Actual): February 2, 2022

Study Registration Dates

• First Submitted: October 31, 2017
• First Submitted That Met QC Criteria: October 31, 2017
• First Posted (Actual): November 6, 2017

Study Record Updates

• Last Update Posted (Actual): February 21, 2022
• Last Update Submitted That Met QC Criteria: February 3, 2022
• Last Verified: February 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Disease; Syndrome
• Other Study ID Numbers: TT 50_2017

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No