A Trial Comparing the Efficacy and Safety of Anakinra Versus Intravenous Immunoglobulin (IVIG) Retreatment, in Patients With Kawasaki Disease Who Failed to Respond to Initial Standard IVIG Treatment

• Status: Not yet recruiting
• Trial end date: 2023-12-01

Summary

Kawasaki disease (KD) is the most frequent vasculitis in younger children <5years, and the first cause of acquired ischemic myocardiopathy in childhood. Exceptionally, KD may cause early death during the acute phase by myocardial infarction, but may compromise the long-term cardiovascular outcome by accelerating atherosclerotic disease.

The incidence of KD is high in far-Eastern countries and Hawaii but KD is relatively rare in other regions (10/100000 children <5years in northern Europe) which makes it difficult to develop research on these rare population.

Early recognition and treatment by intravenous immunoglobulins (IVIG) influences the prognosis positively. IVIG are the standard of care and decrease significantly the risk of coronary aneurysms. However, despite a first infusion of IVIG, 20% of KD patients remain febrile and have high risk of coronary vasculitis. Recent Japanese research group assessed additional cyclosporine treatment in first line KD treatment but failed preventing relapse. To date there is no agreement for a more effective second line treatment.

Based on the auto-inflammatory pattern of KD, the investigators hypothesize that anti IL-1 blocking agents could bring a rapid and sustained effect on systemic and coronary inflammation in patients with KD.

Our hypotheses are:

1. Anakinra treatment may reduce the early and long-term mortality of patients with Kawasaki Disease (KD), by a rapid and sustained effect on vascular inflammation.

2. The safety of anakinra is good, as the drug has a very short half-life, which allows its rapid withdrawal in case of serious adverse event.

The use of anakinra is not associated with the risk of contamination by infectious agents, which remain even minimal, a possibility with the use of IVIG.

• Phase: Phase 3

Details

• Lead Sponsor: Assistance Publique - Hôpitaux de Paris
• Collaborators: Sobi; Swedish Orphan Biovitrum
• Treatments: Antibodies; gamma-Globulins; Immunoglobulins; Immunoglobulins, Intravenous; Interleukin 1 Receptor Antagonist Protein; Rho(D) Immune Globulin