The coming 5 years research will focus on: 1. the value of specific biomarkers in SCD; 2. evaluation of the development of organ damage in SCD; and 3. performing intervention studies to test new therapies to prevent or treat vaso-occlusive crises. Ad 1.
The biomarker studies will help to determine specific risk groups, but may also be helpful in the understanding of the pathogenesis of SCD. Specific biomarkers of interest are: nucleosomes, zinc and pentaxine-3. In a recent study nucleosomes have been demonstrated to play an important role in the inflammatory response in sickle cell disease crises. Future studies will aim at the source of these nucleosomes and the exact mechanism by which they are related to the inflammatory response and endothelial damage. Ad 2.
Clinical studies will focus on specific complications of SCD. In particular, the prevention of acute chest syndrome (an often fatal complication of sickle cell diseas) by incentive spirometry and the detection of liver fibrosis due to recurrent vaso-occlusion will be the main target for the coming years. The value of spirometry will be tested prospectively and a large cross sectional study with the use of the Fibroscan will be performed to evaluate the increasing frequency of liver failure in relative young sickle cell patients for which no clear explanation could be found. Ad 3.
Different intervention studies to test new therapeutic strategies are planned for the coming years. First, a multicentre study will start this year to study the effect of the antioxidant n-acetylcysteine on the prevention of sickle cell crises and daily pain. A smaller phase 2 intervention study will be performed to evaluate the effect of vitamin D suppletion on daily pain which may be caused by osteomalacia due to vitamin D deficiency which is very prevalent in SCD. Within a recently started European collaboration a large randomized controlled trial is currently being designed to assess the effect of a selective p-selectine inhibitor in the treatment of patients with a sickle cell crisis. The AMC will be the leading centre in this European collaboration.
Effective start/end date01/01/2012 → …

ID: 172739