The deferiprone and deferasirox combination is efficacious in iron overloaded patients with -thalassemia major: A prospective, single center, open-label study

BackgroundThe high cost, coupled with the need for continuous infusion, renders Desferrioxamine (DFO), a non-feasible option for iron-chelation in a large majority of patients with -thalassemia major in developing countries. Monotherapy with deferiprone (DFP) or deferasirox (DFX) may not always attain optimal control, particularly in heavily iron-loaded patients. Combination of DFP and DFX is a potential alternative. ProcedureA prospective, single-center, open-label, uncontrolled study was conducted to evaluate the safety and efficacy of the combination in patients with -thalassemia major. Patients who had received either DFP or DFX for >1 year and a serum ferritin >2,000g/L were enrolled. Blood counts, liver/renal functions, and serum ferritin were monitored during the 1-year study period. Facilities for cardiac T2*-MRI were unavailable. ResultsThirty-six patients with a mean age of 13 6.9 years (range: 4-29) and a ferritin of 6,768 +/- 4,145g/L formed the study cohort. Eight (22%) patients had transient gastrointestinal adverse effects. DFX was discontinued in one patient for persistent abdominal pain/diarrhea. Eight (22%) had joint symptoms; DFP was discontinued in two. Four (11%) patients had elevation in AST/ALT levels, managed with temporary interruption of DFX. Nine (25%) had an inconsistent elevation of creatinine to >33% of baseline; no intervention was done. One had transient proteinuria. None had neutropenia. At the end of 1 year, the serum ferritin reduced by a mean value of 3,275.3 +/- 618.2g/L (P<0.001). ConclusionsThe oral combination was found to be safe, efficacious, and a feasible option in patients with suboptimal response to monotherapy. Pediatr Blood Cancer 2015;62:1592-1596. (c) 2015 Wiley Periodicals, Inc.