Sickle cell disease (SCD) Haematological Disorders Sickle-cell disorders
Conditions
Interventions
The study duration was 52 weeks. The initial 24 patients enroled were randomised to receive deferasirox 10 mg/kg or deferoxamine at recommended doses of 20 - 60 mg/kg based on initial liver iron conce
Sponsors
Novartis Pharmaceuticals Corporation (USA)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patients with SCD requiring chronic blood transfusions to prevent complications (stroke, chest syndrome) and thus developing transfusional iron overload requiring chronic chelation therapy.
Exclusion criteria
Exclusion criteria: 1. Serum creatinine above the upper limit of normal (ULN) 2. Significant proteinuria (as indicated by a urinary protein:creatinine ratio of greater than or equal to 0.5 confirmed at two visits) 3. Active hepatitis B or C: 3.1. Active hepatitis B defined as liver function tests above the normal range, together with a positive antigen (hepatitis B e antigen, hepatitis B surface antigen) test or positive immunoglobulin M (IgM) core antibody test in conjunction with a negative hepatitis B surface antibody test 3.2. Active hepatitis C defined as liver function tests above the normal range in the presence of a positive hepatitis C antibody test and detectable hepatitis C ribonucleic acid (RNA) levels 4. Second and third atrioventricular block 5. QT interval prolongation 6. Therapy with digoxin or similar medications (treatment with ß-blockers or angiotensin-converting enzyme inhibitors was permitted) 7. Chelation therapy-associated ocular toxicity
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety assessments: 1. Laboratory assessments: performed monthly and included complete blood counts with differential counts: 1.1. Biochemistry testing (electrolytes, glucose, liver function tests, gamma-glutaryl-transferase, lactate dehydrogenase, cholesterol, triglycerides, uric acid, total protein, C-reactive protein, copper and zinc level) 1.2. Iron parameters (total iron, transferrin, transferrin saturation and ferritin) 1.3. Urinary testing performed on random collections (determination of creatinine, total protein and albumin) 2. Physical examinations (electrocardiograms [ECG], audiometry and ophthalmological tests) were performed at baseline, 12, 24, 36 and 52 weeks 3. In patients less than 16 years of age, additional assessments included growth velocity and pubertal stage | — |
Secondary
| Measure | Time frame |
|---|---|
| Efficacy assessments: 1. Liver iron concentration: determined by superconducting quantum interference device (SQUID) biosusceptometry at baseline, 24 and 52 weeks 2. Serum ferritin: assessed monthly during the study and the change was determined using the baseline and final ferritin level Compliance: 1. For deferasirox, compliance was assessed by counting the number of tablets returned in bottles at each visit 2. For deferoxamine, the numbers of vials returned at each visit were counted | — |
Countries
Canada, France, Italy, United Kingdom, United States of America
Outcome results
None listed