Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis Syndrome
Conditions
Brief summary
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.
Interventions
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).
DRUGColchicine
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).
Sponsors
Wenjie Zheng
Nanjing Children's Hospital
Children's Hospital of Soochow University
The First Affiliated Hospital of Xiamen University
Tianjin Children's Hospital
Ningbo Women & Children's Hospital
Jinan children's hospital
Jiangxi Children's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
1. Meet the 2019 Eurofever or 2020 CARRA diagnostic criteria for PFAPA syndrome. 2. Aged 3 to 18 years (inclusive) at the time of screening. 3. Have experienced at least 3 febrile episodes within the past six months. 4. History of responsiveness to glucocorticoid treatment during at least 3 previous episodes, but with continued recurrence. (Responsiveness is defined as normalization of body temperature within 24 hours after a maximum dose of 2 mg/kg \[up to 60 mg\] administered as a single or two divided doses).
Exclusion criteria
1.Diagnosis of monogenic or other polygenic periodic fever syndromes. 2.Presence of immunodeficiency or neoplastic diseases. 3.Active bacterial, fungal, or viral infection during the screening period. 4.Prior treatment with immunosuppressive agents. 5.Prior use of thalidomide or colchicine. 6.Laboratory parameters at screening that meet any of the following (based on the most recent test result at the study hospital prior to the first dose): 1. White Blood Cell (WBC) count \< 4 × 10⁹/L, Hemoglobin (HGB) \< 100 g/L, or Platelet (PLT) count \< 100 × 10⁹/L. 2. Serum Alanine Aminotransferase (ALT) \> 2 times the Upper Limit of Normal (ULN). 3. Glomerular Filtration Rate (GFR/CCR) \< 60 mL/min/1.73m².
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of Participants Achieving Complete Remission at 6 Months | 6 months | Complete remission is defined as the total absence of febrile episodes (zero attacks) during the treatment period. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Complete Remission Rate at Multiple Time Points | 3 months | Proportion of participants with zero febrile episodes. |
| Partial Remission Rate | 3 months | Proportion of participants achieving a reduction in the frequency of febrile episodes compared to baseline. |
| Recurrence Rate Post-discontinuation | 6 months post-treatment | Proportion of participants experiencing a relapse of symptoms after stopping the medication. |
| Change in Growth Parameters (Z-scores) | 6 months | Changes in Height-for-age Z-score (HAZ) and Weight-for-age Z-score (WAZ). |
| Change in Inflammatory Markers | 6 months | Changes in CRP, ESR, SAA, and cytokine levels. |
Contacts
CONTACTChang Liu
Outcome results
None listed