1.
Design of the silent cerebral infarct transfusion (SIT) trial
Casella JF, King AA, Barton B, White DA, Noetzel MJ, Ichord RN, Terrill C, Hirtz D, McKinstry RC, Strouse JJ, et al
Pediatric Hematology and Oncology. 2010;27((2):):69-89.
Abstract
BACKGROUND Silent cerebral infarct (SCI) is the most common cause of serious neurological disease in sickle cell anemia (SCA), affecting approximately 22% of children. The goal of this trial is to determine whether blood transfusion therapy will reduce further neurological morbidity in children with SCI, and if so, the magnitude of this benefit. PROCEDURE The Silent Cerebral Infarct Transfusion (SIT) Trial includes 29 clinical sites and 3 subsites, a Clinical Coordinating Center, and a Statistical and Data Coordinating Center, to test the following hypothesis: prophylactic blood transfusion therapy in children with SCI will result in at least an 86% reduction in the rate of subsequent overt strokes or new or progressive cerebral infarcts as defined by magnetic resonance imaging (MRI) of the brain. The intervention is blood transfusion versus observation. Two hundred and four participants (102 in each treatment assignment) will ensure 85% power to detect the effect necessary to recommend transfusion therapy (86% reduction), after accounting for 10% drop out and 19% crossover rates. MRI examination of the brain is done at screening, immediately before randomization and study exit. Each randomly assigned participant receives a cognitive test battery at study entry, 12-18 months later, and study exit and an annual neurological examination. Blood is obtained from all screened participants for a biologic repository containing serum and a renewable source of DNA. CONCLUSION The SIT Trial could lead to a change in standard care practices for children affected with SCA and SCI, with a consequent reduction in neurological morbidity.
2.
Long-term safety and efficacy of deferasirox (Exjade®) in transfused patients with sickle cell disease treated for up to 5 years
Vichinsky E, Bernaudin F, Forni GL, Gardner R, Hassell K, Heeney MM, Inusa B, Kutlar A, Lane PA, Mathias L, et al
Blood. 2010;116((21):): Abstract No. 845.
3.
Cost effectiveness of once-daily oral chelation therapy with deferasirox versus infusional deferoxamine in transfusion-dependent thalassaemia patients: US healthcare system perspective
Delea TE, Sofrygin O, Thomas SK, Baladi JF, Phatak PD, Coates TD
Pharmacoeconomics. 2007;25((4):):329-42.
4.
Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review
Delea TE, Edelsberg J, Sofrygin O, Thomas SK, Baladi JF, Phatak PD, Coates TD
Transfusion. 2007;47((10):):1919-1929.
5.
Chelation therapy with deferasirox versus deferoxamine in transfusion-dependent sickle-cell disease: a cost-effectiveness analysis from the US perspective
Delea T, Sofrygin O, Baladi J, Thomas SK, Coates TD
Haematologica. 2006;91((Suppl 1):): Abstract No. 0003.
6.
Sensitivity analysis of the cost-effectiveness of chelation therapy with deferasirox or deferoxamine in transfusion-dependent thalassemia patients based on European costs
Delea T, Sofrygin O, Baladi J, Thomas SK, Phatak PD, Coates TD
Haematologica. 2006;91((Suppl 1):): Abstract No. 0008.