From the Research
For PFAPA syndrome with disabling abdominal pain, the most effective treatment approach is to start with a single dose of prednisone (1-2 mg/kg) at the onset of fever, which can abort the episode within hours, as supported by the most recent study 1.
Treatment Approach
When considering treatment options for PFAPA syndrome, it's essential to prioritize morbidity, mortality, and quality of life. The following approaches can be considered:
- Prednisone: A single dose of prednisone (1-2 mg/kg) at the onset of fever can effectively abort the episode within hours 1.
- Colchicine: Daily colchicine (0.5-1.0 mg/day) can be considered for prophylaxis, as it has been shown to decrease flare frequency and intensity in pediatric patients with PFAPA syndrome 2.
- Pain Management: Targeted pain management with acetaminophen (15 mg/kg every 4-6 hours) or ibuprofen (10 mg/kg every 6-8 hours) can help alleviate abdominal pain.
- Tonsillectomy: Tonsillectomy remains a definitive treatment option with 80-90% resolution rates, as reported in a large single-centre cohort study 3.
Additional Considerations
- Regular monitoring of symptoms using a diary can help identify triggers and optimize treatment timing.
- Dietary modifications, including avoiding potential trigger foods during flares, may provide additional relief for some patients.
- For severe cases not responding to these measures, consultation with a rheumatologist about IL-1 inhibitors like anakinra may be warranted. The abdominal pain in PFAPA likely stems from systemic inflammation affecting the gastrointestinal tract, with elevated cytokines like IL-1β playing a key role, as suggested by the clinical characteristics and treatment outcomes in a large single-centre cohort 3.