Tonsillotomy or Follow-up in PFAPA Syndrome
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
N/A |
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:
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We are looking for a 40% absolute difference in cure rate between tonsillotomy (90%) and follow up (50%) groups.
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Two sided Whitehead design
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Maximum likelihood estimation (MLE)
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Five assessments (sample size 4+4, 8+8. 12+12, 16+16 and 19+19)
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type 1 error 5 % and power 80%
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The sample size will be from 8 to 38 patients depending on when the trial can be stopped along assessments.
Study Design
Arms and Interventions
| 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 |
Outcome Measures
Primary Outcome Measures
- Cure [3 months from randomization]
Proportion of patients who do not have any PFAPA symptoms
Secondary Outcome Measures
- 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
- Cure [6 months from randomization]
Proportion of patients who do not have any PFAPA symptoms
Eligibility Criteria
Criteria
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Oulu Unversity Hospital | Oulu | Finland | ||
| 2 | Tampere University Hospital | Tampere | Finland | ||
| 3 | Turku University Hospital | Turku | Finland |
Sponsors and Collaborators
- University of Oulu
- Oulu University Hospital
- Tampere University
- Tampere University Hospital
- Turku University Hospital
- University of Turku
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- TT 50_2017