Kawasaki Disease, Macrophage Activation Syndrome (MAS)
Conditions
Brief summary
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.
Detailed description
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.
Interventions
None listed
Sponsors
Meyer Children's Hospital IRCCS
Study design
Observational model
CASE_CONTROL
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
4 Weeks to 17 Years
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| MAS-KD population | From the study initiation date onward for 36 months | Definition of the proportion of patients with KD who develop MAS in the study population. |
| Clinical and laboratory features of MAS KD patients | From the study initiation date onward for 36 months | Adjusted Odds ratios of clinical and laboratory risk factors |
| Applicability and diagnostic performance of currently available MAS classification criteria | From the study initiation date onward for 36 months | Evaluation of diagnostic performance metrics (sensitivity, specificity, positive predictive value, negative predictive value) of existing MAS criteria when applied to KD patients. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Heterogeneity of MAS KD population | From the study initiation date onward for 36 months | 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. |
| Applicability and diagnostic performance of currently available MAS classification criteria | From the study initiation date onward for 36 months | Evaluation of diagnostic performance metrics (sensitivity, specificity, positive predictive value, negative predictive value) of existing MAS criteria when applied to KD patients. |
| MAS diagnosis | From the study initiation date onward for 36 months | Time from KD onset to MAS diagnosis and its correlation with clinical outcomes; |
| Treatment and clinical response of MAS KD patients | From the study initiation date onward for 36 months | Description of treatments used for MAS (e.g., corticosteroids, IVIG, biologics) and the corresponding clinical response. |
Countries
Italy
Contacts
CONTACTTeresa Giani, MD
Outcome results
None listed