The deferiprone and deferasirox combination is efficacious in iron overloaded patients with ß-thalassemia major: A prospective, single center, open-label study.
Pediatr Blood Cancer
; 62(9): 1592-6, 2015 Sep.
Article
em En
| MEDLINE
| ID: mdl-25820920
ABSTRACT
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 ß-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 ß-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.Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Piridonas
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Triazóis
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Benzoatos
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Terapia por Quelação
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Quelantes de Ferro
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Talassemia beta
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Sobrecarga de Ferro
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Ferro
Tipo de estudo:
Etiology_studies
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Observational_studies
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Risk_factors_studies
Idioma:
En
Ano de publicação:
2015
Tipo de documento:
Article