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.
KD most frequently affects young children under the age of 5. Its epidemiology varies by geographical location and season. The course of KD can be complicated by the development of MAS. Clinical similarities between KD-especially refractory KD-and MAS, combined with the lack of specific diagnostic criteria, may hinder accurate and timely identification of MAS in KD, complicating treatment decisions and worsening clinical outcomes. Given that MAS is associated with a significant risk of multi-organ failure (MOF), patient prognosis may be severely compromised, with increased morbidity and mortality. Therefore, early recognition of MAS is crucial in order to implement targeted therapeutic strategies as promptly as possible. In this retrospective-prospective, observational, descriptive, international multicenter study, we aim to: * Analyze the clinical features, management, and outcomes of patients with KD complicated by MAS to describe this complication and identify potential risk factors for MAS development; * Evaluate the performance of currently available MAS diagnostic criteria in KD patients and identify specific diagnostic criteria for this condition. The study will include international pediatric rheumatology centers affiliated with the PReS network.
Study Type
OBSERVATIONAL
Enrollment
150
Azienda Ospedaliero-Universitaria IRCCS Meyer
Florence, Italy, Italy
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 months
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 months
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 months
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 months
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 months
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 months
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 months
Heterogeneity of MAS KD population
Differences in treatment strategies 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 months
Heterogeneity of MAS KD population
Differences in outcomes 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 months
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