From the Guidelines
Biological therapy for PFAPA syndrome in pediatric patients includes IL-1 inhibitors such as anakinra, which can be administered at 1-2 mg/kg/day subcutaneously during flares or as daily prophylaxis, and canakinumab, typically given as 2-4 mg/kg subcutaneously every 4-8 weeks, as supported by recent studies 1. The choice of biological therapy for PFAPA syndrome depends on the severity of the disease and the patient's response to conventional therapies.
- IL-1 inhibitors, such as anakinra and canakinumab, have been shown to be effective in reducing fever and other inflammatory symptoms in patients with PFAPA syndrome, as reported in recent studies 1.
- Anakinra can be used at a dose of 1-2 mg/kg/day subcutaneously during flares or as daily prophylaxis, while canakinumab is typically given as 2-4 mg/kg subcutaneously every 4-8 weeks.
- These biologics work by blocking interleukin-1, a key inflammatory cytokine implicated in PFAPA pathogenesis, thereby reducing fever and other inflammatory symptoms.
- They are generally considered second or third-line treatments, reserved for cases that are refractory to conventional therapies like corticosteroids (prednisone or prednisolone), colchicine, or tonsillectomy.
- While these biologics can be highly effective in controlling symptoms and preventing flares, they are expensive and may require insurance approval.
- Side effects include injection site reactions and increased risk of infections.
- Regular monitoring for adverse effects is necessary, and patients should be up-to-date on vaccinations before starting therapy.
- Treatment duration varies based on clinical response, with some patients requiring long-term therapy while others may achieve remission after a period of treatment, as supported by recent studies 1.
From the Research
Biological Therapy for PFAPA in Pediatric Age Group
- The available biological therapies for PFAPA in pediatric age group patients include:
- Anakinra: an interleukin (IL) receptor antagonist that works by blocking the biological activity of IL-1 2, 3
- Canakinumab: an IL-1β blocker that has been effective in preventing febrile attacks in some patients with PFAPA 2
- Rilonacept: an IL-1 receptor antagonist that has been used in the treatment of cryopyrin-associated periodic syndromes, but its effectiveness in PFAPA is not well established 2
Mechanism of Action
- These biological therapies work by targeting the interleukin-1 (IL-1) pathway, which is involved in the pathogenesis of PFAPA 2, 3
- IL-1 production is induced in response to inflammatory stimuli and mediates various physiological mechanisms, including inflammation and immunological reactions 3