Acute immune-mediated diseases in children and adolescents: • Rheumatologic diseases: Juvenile idiopathic arthritis (polyarticular, systemic), juvenile systemic lupus erythematosus (SLE), non-infectious uveitis, dermatomyositis/connective tissue diseases, autoinflammatory diseases, vasculitides with organ involvement • Neurological diseases: Central nervous system (CNS) vasculitis, acute demyelinating diseases (ADEM, CIS, POMS), myelin oligodendrocyte glycoprotein antibody-associated disease (
Conditions
Interventions
Group 1: Arm 1: 3-day high-dose glucocorticoid pulse therapy
Pediatric patients with acute immune-mediated diseases receiving a 3-day high-dose glucocorticoid pulse therapy as part of routine clinica
Sponsors
Universitätsklinikum Carl Gustav Carus Dresden an der Technischen Universität Dresden
Eligibility
Sex/Gender
All
Age
28 Days to 18 Years
Inclusion criteria
Inclusion criteria: The study population includes children and adolescents from the 29th day of life up to 18 years of age with an acute immune-mediated disease who are receiving high-dose glucocorticoid pulse therapy based on a medical indication. In addition, parents or legal guardians will be invited to participate in the parent proxy assessment. Inclusion criteria Patients are eligible to participate if they meet all of the following criteria: 1. Age: from the 29th day of life up to 18 years; participation in the EMA assessment from 8 years of age. 2. Diagnostic categories (grouped): 2.1. Rheumatologic diseases: juvenile idiopathic arthritis (polyarticular, systemic), juvenile systemic lupus erythematosus (SLE), non-infectious uveitis, dermatomyositis/connective tissue diseases, autoinflammatory diseases, vasculitis with organ involvement. 2.2. Neurological diseases: central nervous system vasculitis, acute demyelinating diseases (ADEM, CIS, POMS), MOGAD, NMOSD, (LE)TM, autoimmune encephalitis (NMDAR antibodies, GABAA antibodies, LGI1 antibodies, CASPR2 antibodies). 2.3. Gastrointestinal diseases: severe flares of inflammatory bowel disease. 2.4. Nephrological diseases: nephrotic syndrome. 2.5. Allergic/systemic reactions. 3. Medical indication for high-dose glucocorticoid pulse therapy according to physician decision. 4. Medication status: no systemic glucocorticoid treatment within the previous 8 weeks. 5. Consent: written informed consent from parents or legal guardians. 6. Availability: availability for follow-up assessments over a period of 3 months.
Exclusion criteria
Exclusion criteria: Patients will be excluded if any of the following criteria apply: 1. Chronic or severe comorbidities: cytostatic drugs/chemotherapy, biologicals associated with marked lymphocyte depletion (e.g. rituximab), primary immunodeficiencies, organ transplantation, malignant diseases. 2. Active infections: bacterial sepsis, meningitis, endocarditis, bacterial pneumonia, osteomyelitis/septic arthritis, varicella infection, herpes simplex virus (HSV) infection without a compelling indication for glucocorticoid treatment. 3. Medication-related contraindications: systemic glucocorticoid therapy within the previous 8 weeks. 4. Other criteria: pregnancy, lack of informed consent, insufficient sample volume for analyses, or inability to participate in follow-up assessments during the 3-month observation period after enrolment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary objective is to identify biomarkers associated with individual treatment response and treatment-related adverse effects following high-dose glucocorticoid pulse therapy. This includes the analysis of genetic, immunological, transcriptomic, proteomic, metabolomic, microbiome, and epigenetic parameters, as well as their integrative evaluation using bioinformatic methods and machine learning approaches. In addition, patient-reported outcomes (PROs) derived from Ecological Momentary Assessment (EMA) and parent proxy assessments will be included to capture individual differences in treatment experience and well-being. Biological markers will be assessed using blood samples, urine samples, and nasal swabs collected at three time points: T1 (prior to treatment initiation), T2 (after 3 days), and T3 (3 months after treatment initiation). Clinical treatment response and treatment-related adverse effects will be assessed using routine clinical data and laboratory parameters collected at T1, T2, and T3. Patient-reported outcomes will be assessed using Ecological Momentary Assessment (EMA), the EQ-5D-Y, EQ-VAS, QuESt, and PROMIS Parent Proxy instruments. EMA will be conducted during two one-week assessment periods: during hospitalization and at the 3-month follow-up. Participants will complete two brief questionnaires per day, one in the morning and one in the evening. Parent proxy questionnaires will be administered at treatment initiation (T1), on day 7, and at the 3-month follow-up (T3). | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary objectives of the study are: a) To compare clinical outcomes and safety parameters between 3-day and 5-day pulse therapy in routine clinical practice. b) To investigate the short- and medium-term systemic effects of glucocorticoid therapy on immune function, metabolism, and epigenetic regulation. c) To analyze the association between genetic variants and interindividual differences in treatment response as well as the occurrence of adverse events. d) To evaluate PROs throughout treatment and during the 3-month follow-up period, and to assess their correlation with clinical and molecular parameters. | — |
Countries
Czechia, Finland, Germany
Contacts
Public ContactLuise Richter
Universitätsklinikum Carl Gustav Carus Dresden an der Technischen Universität Dresden
Outcome results
None listed