Pediatric Endocrinology

Department of Clinical Sciences Malmö

PI: Helena Elding Larsson

Pediatric autoimmune disorders, such as type 1 diabetes, autoimmune thyroid disease and celiac disease may affect as many as 2-3 % of all children. Genetic factors are associated with these diseases, but environmental factors may trigger the autoimmune process in the genetically susceptible individuals. The nature and role of environmental factors in the disease processes are still to be defined. Several autoantibodies are specific markers of the autoimmune disease provess and autoantibodies may be used to predict disease onset.

Type 1 diabertes is one of the most common seriour chronic diseases in children, with an estimated lifetime risk in Sweden of almost 1 %. The disease is caused by immune-mediated destruction of beta cells in the pancreatic islets resulting in a loss of insulin production.
Children developing this autoimmune disease become dependent on a lifelong treatment with insulin, with enormous consequences for both the child and the family and with a high rate of late complications.

Despite intense research during the last decades, the aetiology of type 1 diabetes is still unknown. Genetic factors, such as Human Leucocyte Antigen (HLA) genotypes, confer susceptibility to the disease, but other factors are needed to initiate the autoimmune process that results in beta cell death.

Years to months before the classical clinical symptoms of type 1 diabetes, including weight loss and increased urination and thirst, an autoimmune sub-clinical destruction of the beta cells has been ongoing. This process is marked by the presence of autoantibodies against islet cell autoantigens such as GAD65,
IA-2, ZnT8 and insulin. By combining HLA-genotypes with autoantibodies to islet autoantigens, we are able to predict type 1 diabetes in children.

In our research, we investigate environmental factors that may trigger development of islet autoimmunity and development of autoimmune disease in children. In children who have developed islet autoantibodies and have a high risk for type 1 diabetes, we investigate the predictive value of HLA-genotypes, islet autoantibodies in combination with metabolic profile and growth.

Our ability to predict type 1 diabetes is high and novel treatment strategies to prevent the clinical onset are urgently needed. A phase II clinical trial with alum-GAD65 has been completed to demonstrate protection from beta cell loss. In children who have developed islet autoantibodies and are at high risk of type 1 diabetes we are testing if immune tolerance treatment with ALUM-GAD (Diamyd) may prevent or delay onset of disease in our investigator-initiated clinical trial Diabetes Prevention Immune-Tolerance (DIAPREV-IT).

Last updated: September 19, 2012
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