Projects / Programmes
SYSTEMIC IMMUNE-MEDIATED DISEASES IN CHILDREN AND ADOLESCENTS
| Code |
Science |
Field |
Subfield |
| 3.01.00 |
Medical sciences |
Microbiology and immunology |
|
| Code |
Science |
Field |
| B660 |
Biomedical sciences |
Pediatrics |
Pediatric antiphospholipid syndrome, Juvenile scleroderma, Vaccination, Mechanisms of immune tolerance, Inherited immune deficiencies
Researchers (17)
Organisations (2)
Abstract
Systemic immune-mediated diseases are one of the most common chronic illnesses of children and an important cause of short- and long-term disability. In the last decade, there have been dramatic advances in understanding the function of immune system, however, morbidity and mortality still remain serious threats for children with several systemic immune-mediated diseases. In addition to the complications that can occur in adults, specific concerns such as those related to growth, development, childhood vaccinations and quality of life characterize the disease in pediatric patients. The proposed research project will focus on the following systemic immune-mediated diseases in children and adolescents:
1. Pediatric antiphospholipid syndrome
Based on literature data and personal experience obtained on treating children with elevated antiphospholipid antibodies (aPL), we believe that there are some important differences in pathophysiology and clinical spectrum of the APS related to the age at onset of the disease. Investigations in the present research proposal are designed to elucidate factors specific to children and to examine the clinical significance and pathogenic mechanisms of aPL in the pediatric population.
2. Juvenile scleroderma
There are no accurate data on the long-term outcome in JLS patients treated with different protocols. In our study, we plan to evaluate the outcome in a large group of patients with JLS to determine impact of treatment on the long term outcome of JLS.
3. Autoimmune responses after vaccination
In the proposed study we will evaluate safety and efficacy of influenza vaccination in a large group of healthy adults and in children with various systemic autoimmune diseases on immunosupressive medications.
4. Mechanisms of immune tolerance and autoimmune responses in pediatric patients on specific immunotherapy
We will compare results of basophil activation test between pediatric patients with good clinical response and those with poor clinical response on treatment with specific immunotherapy. Since there are no accurate data on the induction of autoimmune phenomena during the process of specific immunotherapy, we plan to periodically assess also possible induction of disease-specific autoantibodies.
5. Inherited immune deficiencies
In the proposed study we plan to investigate clinical and genetic characteristics of Central and South-Eastern European patients with chronic granulomatous disease.
Significance for science
Our research project was focused on systemic immune-mediated diseases which are one of the most common chronic disorders in pediatric population. Our studies were clinically focused with two main objectives:
- development of better diagnostic ability for early identification of patients with different systemic immune-mediated diseases
- recognition of patients with systemic-immune mediated disease that have worse long-term outcome and impact of treatment on the long-term outcome
The research project provided data from a large cohort of pediatric patients with antiphospholipid syndrome. We reported original information on clinical presentation, laboratory features, and outcomes of pediatric APS. In addition, we identified some important differences when compared with antiphospholipid syndrome in adult patients.
In a large prospective cohort study evaluating autoimmune responses following annual influenza vaccination we demonstrated that influenza vaccination did not increase the percentage of positive autoantibodies in the general healthy adult population and that newly synthesized autoantibodies after the influenza vaccination had no apparent clinical significance.
Within a network of pediatric immunology centers in eastern and central European countries we performed analyses of clinical and genetic features in large cohorts of patients with rare primary immunodeficiency diseases including chronic granulomatous disease, autoinflammatory diseases, X-linked agammaglobulinemia and Wiskott-Aldrich syndrome.
Significance for the country
The research proposal was clinically focused and was aimed to improvement of health care including development of new clinical and laboratory diagnostic protocols in children with systemic immune-mediated diseases. We identified new clinical, laboratory and genetic features in children with systemic immune-mediated diseases that will contribute to the improved clinical care and will have impact on the long-term outcome of these patients. The results of our research project were presented at several international meetings and published in 16 articles in peer-reviewed journals and 3 book chapters.
Most important scientific results
Annual report
2008,
2009,
final report,
complete report on dLib.si
Most important socioeconomically and culturally relevant results
Annual report
2008,
2009,
final report,
complete report on dLib.si
