MASKd: a Study on Kawasaki Disease (KD) Complicated by Macrophage Activation Syndrome (MAS)
Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age4-17
SponsorMeyer Children's Hospital IRCCS
About this trial
Kawasaki Disease (KD) is one of the most common vasculitides in childhood and represents a leading cause of acquired heart disease in developed countries. Macrophage Activation Syndrome (MAS) is a potentially life threatening hyperinflammatory condition belonging to the spectrum of hemophagocytic lymphohistiocytosis (HLH), and it can complicate various rheumatologic diseases. Awareness of MAS in the context of KD has recently increased, supporting the hypothesis that it is an underdiagnosed complication. The study aims to define the epidemiology, clinical characteristics, management, and therapeutic strategies of MAS in patients with KD, through a multicenter data collection in Europe.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age between 4 weeks and under 18 years at the time of KD diagnosis
Diagnosis of KD made according to the 2024 AHA guidelines
Diagnosis of MAS made by the attending physician within 30 days from the onset of KD.
Disqualifiers
Unconfirmed diagnosis of KD (e.g., mimicking conditions)
Primary (genetic) HLH
Lack of informed consent
MAS diagnosed more than 30 days after or more than 15 days before the onset of KD
Trial population
Since no validated or universally accepted diagnostic criteria currently exist for MAS occurring in the context of KD, the diagnosis of MAS will be based on the clinical judgment of the treating physician. To minimize the risk of misdiagnosis, all cases will be independently and critically reviewed by three experienced pediatric rheumatologists. Control groups will consist of age- and sex-matched patients, divided as follows: * Patients with KD resistant to first-line therapy * Patients with KD responsive to first-line therapy For each patient with KD complicated by MAS included in the study, 2 KD-responsive and 2 KD-resistant patients will be enrolled. The goal is to include at least 30 MAS-KD patients, along with 60 KD-resistant controls and 60 KD-responsive controls.
Trial design
Design model
Case-control
Time perspective
Other
Treatments tested in this trial
Not listed
Trial groups
150 Participants
are grouped into 3 trial groupsGroup A: MAS-KD patients.
Group B: KD-resistant controls
Group C: KD-responsive controls
Trial outcomes
Primary outcomes
1
MAS-KD population
Definition of the proportion of patients with KD who develop MAS in the study population.
Time frame
From the study initiation date onward for 36 months2
Clinical and laboratory features of MAS KD patients
Adjusted Odds ratios of clinical and laboratory risk factors
Time frame
From the study initiation date onward for 36 months3
Applicability and diagnostic performance of currently available MAS classification criteria
Evaluation of diagnostic performance metrics (sensitivity, specificity, positive predictive value, negative predictive value) of existing MAS criteria when applied to KD patients.
Time frame
From the study initiation date onward for 36 monthsSecondary outcomes
1
Heterogeneity of MAS KD population
Differences in clinical course based on e.g., intensive care unit admission (present/absent, days), duration of fever (days), coronary involvement (present/absent,) among three groups of Patients: patients with KD complicated by MAS; patients with KD resistant to first-line therapy; patients with KD responsive to first-line therapy.
Time frame
From the study initiation date onward for 36 months2
Applicability and diagnostic performance of currently available MAS classification criteria
Evaluation of diagnostic performance metrics (sensitivity, specificity, positive predictive value, negative predictive value) of existing MAS criteria when applied to KD patients.
Time frame
From the study initiation date onward for 36 months3
MAS diagnosis
Time from KD onset to MAS diagnosis and its correlation with clinical outcomes;
Time frame
From the study initiation date onward for 36 months4
Treatment and clinical response of MAS KD patients
Description of treatments used for MAS (e.g., corticosteroids, IVIG, biologics) and the corresponding clinical response.
Time frame
From the study initiation date onward for 36 monthsSponsors and contacts
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Source ClinicalTrials.gov
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