Hereditary blooddisorder Sicklecell anaemia
Conditions
Interventions
Once daily deferasirox in a maximum dose of 360 mg for 6 weeks. For
particapants with effect, there can be following periods of treatment for 6
weeks with a lower dose (180mg and 90mg).
Sponsors
Academisch Medisch Centrum
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Sickle cell disease diagnosis: HbSS, or HbSβ0-thalassemia genotype 2. Age 18-65 years 3. Willing and able to provide written informed consent
Exclusion criteria
Exclusion criteria: 1. Blood transfusion in the preceding four months 2. Already using iron chelation due to iron overload 3. Ferritin levels of
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The main endpoints of this study are safety and efficacy. -We will evaluate safety by analysis of adverse events, medication use and physical and laboratory examinations. -The primary efficacy endpoint will be the effect of deferasirox on sickling of red blood cells, measured as changes in Point of Sickling (PoS), as quantified by Oxygenscan. | — |
Secondary
| Measure | Time frame |
|---|---|
| - To evaluate RBC degradation as expressed by phosphatidylserine (PS) exposure on the outer surface of RBC membrane and markers of hemolysis (cell-free heme, lactate dehydrogenase (LDH), bilirubin, reticulocytes and hemoglobin - To evaluate the effect of deferasirox on RBC HbS percentage - Effect of deferasirox on levels of non-transferrin bound iron (NTBI) and labile plasma iron (LPI) - To evaluate the effect of deferasirox on oxidative stress as expressed by intracellular metabolomics and by plasma levels of AGEs - To evaluate the effect of deferasirox on clinical characteristics such as fatigue and pain | — |
Countries
Netherlands
Outcome results
None listed