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


PROCEDURE
A prospective, single-center, open-label, uncontrolled study was conducted to evaluate the safety and efficacy of the combination in patients with β-thalassemia major. Patients who had received either DFP or DFX for >1 year and a serum ferritin >2,000 μg/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.


RESULTS
Thirty-six patients with a mean age of 13 ± 6.9 years (range: 4-29) and a ferritin of 6,768 ± 4,145 μg/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 ± 618.2 μg/L (P < 0.001).


CONCLUSIONS
The oral combination was found to be safe, efficacious, and a feasible option in patients with suboptimal response to monotherapy.
['Totadri|Sidharth|S|', 'Bansal|Deepak|D|', 'Bhatia|Prateek|P|', 'Attri|Savita V|SV|', 'Trehan|Amita|A|', 'Marwaha|R K|RK|']
D000293:Adolescent D000328:Adult D001565:Benzoates D015913:Chelation Therapy D056486:Chemical and Drug Induced Liver Injury D002648:Child D002675:Child, Preschool D000077588:Deferasirox D000077543:Deferiprone D003875:Drug Eruptions D004359:Drug Therapy, Combination D005260:Female D005293:Ferritins D005767:Gastrointestinal Diseases D006801:Humans D007501:Iron D007502:Iron Chelating Agents D019190:Iron Overload D007592:Joint Diseases D008297:Male D011446:Prospective Studies D011507:Proteinuria D011728:Pyridones D065227:Transfusion Reaction D016896:Treatment Outcome D014230:Triazoles D055815:Young Adult D017086:beta-Thalassemia
India arthropathy chelation desferal exjade