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A stakeholder-driven comparative effectiveness study of treatments to prevent coronary artery damage in patients with resistant Kawasaki disease
Investigator (PI): Burns, Jane; Kim, Katherine K
Performing Organization (PO): (Current): University of California, San Diego, School of Medicine, Department of Pediatrics, Division of Allergy, Immunology, and Rheumatology / (858) 822-5256
Supporting Agency (SA): Patient-Centered Outcomes Research Institute (PCORI)
Initial Year: 2017
Final Year: 2022
Record Source/Award ID: PCORI/CER-1602-34473
Funding: Total Award Amount: $3,109,875
Award Type: Contract
Award Information: PCORI: More information and project results (when completed)
Abstract: Background and significance: Kawasaki disease (KD) is a self-limited illness that affects the heart blood vessels (coronary arteries) of infants and children, and it is now the most common cause of acquired heart disease in children. A mixture of proteins from human blood (intravenous immunoglobulin [IVIG]) is a treatment that reduces the rate of the major complication of the disease: a bulging of the wall of the coronary arteries called an aneurysm. However, 10 percent to 20 percent of children are resistant to this treatment, and the fever returns. These children have the highest rates of aneurysm formation and thus should be treated aggressively. Unfortunately, because the problem has never been adequately studied, there are no guidelines for the best secondary treatment for these resistant patients. Most physicians choose either a second infusion of IVIG or an engineered antibody called infliximab that inactivates a molecule that promotes inflammation. Currently, the American Heart Association KD guidelines assign an evidence level of C (consensus opinion of experts) to retreatment with either second IVIG or infliximab. Specific aims: Specific aim 1 will test the hypothesis that infliximab will be better than a second IVIG infusion for treatment of IVIG resistance. Specific aim 2 will test the hypothesis that infliximab treatment will result in more rapid resolution of inflammation compared with a second IVIG infusion. Specific aim 3 will test the hypothesis that infliximab treatment will result in a greater improvement in the internal diameter of the coronary arteries. Specific aim 4 will collect patient centered outcomes from parent reports during and after hospitalization. Overall study design: We propose a randomized clinical trial of the two leading therapies for IVIG-resistant KD patients at 26 clinical sites across the United States. Main components of the intervention: IVIG-resistant KD patients will be randomized to receive either infliximab (10 mg/kg) or second IVIG infusion (2g/kg). Patients will return for an echocardiogram and laboratory assessment at 2 weeks following discharge. Study population: Eligible subjects will be KD patients ages 4 weeks to 17 years who have return of fever following initial treatment with IVIG. Primary and secondary outcome measures: The primary outcome measure is cessation of fever within 24 hours of initiation of study drug, with no fever recurrence within the next seven days. The secondary outcome measures are 1) change in levels of inflammation markers; 2) change in internal diameter of coronary arteries; 3) total number of fever days from enrollment; 4) duration of hospitalization; and 5) IVIG and infliximab infusion reactions. Analytic methods: Statistical analyses will be performed to compare study outcomes between treatment arms.
MeSH Terms:
  • Adolescent
  • Aneurysm /prevention & control
  • Child
  • Child, Preschool
  • Comparative Effectiveness Research
  • Coronary Artery Disease /*prevention & control
  • Drug Resistance
  • Echocardiography
  • Hospitalization
  • Humans
  • Immunoglobulins, Intravenous /*therapeutic use
  • Infant
  • Infant, Newborn
  • Inflammation
  • Infliximab /therapeutic use
  • Infusions, Intravenous
  • Mucocutaneous Lymph Node Syndrome /*complications
  • /*therapy
  • Outcome Assessment, Health Care
  • Patient Discharge
  • Randomized Controlled Trials as Topic
Country: United States
State: California
Zip Code: 92093
UI: 20181533
CTgovId: NCT03065244
Project Status: Ongoing