The goal of this clinical trial is to evaluate the efficacy and safety of thalidomide in the treatment of children with Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis (PFAPA) syndrome. The study focuses on children diagnosed with PFAPA syndrome. The main questions it aims to answer are: Can thalidomide significantly reduce the frequency of febrile episodes in children with PFAPA syndrome? What is the safety profile and tolerability of thalidomide in this pediatric population? Researchers will compare the thalidomide group to a colchicine group to see if thalidomide is more effective in controlling recurrent fever and associated symptoms. Participants will: Take the assigned medication (thalidomide or colchicine) daily for a duration of 6 months. Attend follow-up visits every 4 weeks at the clinic. Maintain a diary to record the frequency of fever episodes and any other clinical symptoms. Undergo safety assessments and physical examinations during each scheduled visit.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
106
The starting dose of thalidomide is 1 mg/kg/day, administered orally before bedtime. If febrile episodes persist during treatment, the dosage will be increased starting the day after the next fever (maximum dose not to exceed 2 mg/kg/day, with a maximum total dose of 100 mg/day).
The starting dose of colchicine is 0.5 mg/day administered orally. If febrile episodes persist during treatment, the dosage will be increased starting the day after the next fever (maximum dose not to exceed 1.25 mg/day).
Proportion of Participants Achieving Complete Remission at 6 Months
Complete remission is defined as the total absence of febrile episodes (zero attacks) during the treatment period.
Time frame: 6 months
Complete Remission Rate at Multiple Time Points
Proportion of participants with zero febrile episodes.
Time frame: 3 months
Complete Remission Rate at Multiple Time Points
Proportion of participants with zero febrile episodes.
Time frame: 9 months
Complete Remission Rate at Multiple Time Points
Proportion of participants with zero febrile episodes.
Time frame: 12 months
Partial Remission Rate
Proportion of participants achieving a reduction in the frequency of febrile episodes compared to baseline.
Time frame: 3 months
Recurrence Rate Post-discontinuation
Proportion of participants experiencing a relapse of symptoms after stopping the medication.
Time frame: 6 months post-treatment
Recurrence Rate Post-discontinuation
Proportion of participants experiencing a relapse of symptoms after stopping the medication.
Time frame: 12 months post-treatment
Change in Growth Parameters (Z-scores)
Changes in Height-for-age Z-score (HAZ) and Weight-for-age Z-score (WAZ).
Time frame: 6 months
Change in Growth Parameters (Z-scores)
Changes in Height-for-age Z-score (HAZ) and Weight-for-age Z-score (WAZ).
Time frame: 12 months
Change in Inflammatory Markers
Changes in CRP, ESR, SAA, and cytokine levels.
Time frame: 6 months
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