Efficacy of Deferasirox for the treatment of iron overload in Chinese thalassaemia major patients: Results from a prospective, open-label, multicentre clinical trial

Transfusion Medicine

Volumn 23, Issue 6, 2013, Pages 389-396

  Lai, Y R ^a   Liu, R R ^a   Li, C F ^b   Huang, S L ^c   Li, Q ^d   Habr, D ^e   Martin, N ^e   Shen, Z X ^f  

^a FIRST AFFILIATED HOSPITAL OF GUANGXI MEDICAL UNIVERSITY   (China)

^b SOUTHERN MEDICAL UNIVERSITY   (China)

^c SUN YAT SEN UNIVERSITY   (China)

^d SECOND PEOPLE S HOSPITAL OF SHENZHEN   (China)

^e NOVARTIS PHARMA AG   (Switzerland)

^f SHANGHAI JIAO TONG UNIVERSITY   (China)

Author keywords

Deferasirox; Iron chelation; Iron overload; Serum ferritin; Thalassaemia major

Indexed keywords

ALANINE AMINOTRANSFERASE; ASPARTATE AMINOTRANSFERASE; BILIRUBIN; CHELATING AGENT; CREATININE; DEFERASIROX; DEFERIPRONE; FERRITIN; TRIACYLGLYCEROL; UREA;

ABDOMINAL PAIN; ADOLESCENT; ADULT; AGED; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ARTICLE; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; BILIRUBIN BLOOD LEVEL; BLOOD TRANSFUSION; CHELATION; CHILD; CHINESE; CHOLESTASIS; CREATININE BLOOD LEVEL; CREATININE CLEARANCE; DIARRHEA; DOSE RESPONSE; DRUG DOSE ESCALATION; DRUG DOSE TITRATION; DRUG EFFICACY; DRUG SAFETY; DRUG WITHDRAWAL; FEMALE; FERRITIN BLOOD LEVEL; HUMAN; IRON BALANCE; IRON CHELATION; IRON OVERLOAD; KIDNEY FUNCTION TEST; LIVER FUNCTION TEST; LYMPHADENITIS; MAJOR CLINICAL STUDY; MALE; MONOTHERAPY; MULTICENTER STUDY; PATIENT COMPLIANCE; PROSPECTIVE STUDY; RASH; SIDE EFFECT; THALASSEMIA MAJOR; TREATMENT OUTCOME; TRIACYLGLYCEROL BLOOD LEVEL; UREA BLOOD LEVEL;

DEFERASIROX; IRON CHELATION; IRON OVERLOAD; SERUM FERRITIN; THALASSAEMIA MAJOR;

ADOLESCENT; ADULT; AGE FACTORS; AGED; ASIAN CONTINENTAL ANCESTRY GROUP; BENZOATES; CHILD; CHILD, PRESCHOOL; CHINA; FEMALE; HUMANS; INFANT; IRON; IRON CHELATING AGENTS; IRON OVERLOAD; MALE; MIDDLE AGED; PROSPECTIVE STUDIES; THALASSEMIA; TRIAZOLES;

EID: 84888347683     PISSN: 09587578     EISSN: 13653148     Source Type: Journal    
DOI: 10.1111/tme.12077     Document Type: Article

References (23)

1. Angastiniotis, M. & Modell, B. (1998) Global epidemiology of globin disorders. Annals of the New York Academy of Sciences, 850, 251-269.
2. Cappellini, M.D., Cohen, A., Piga, A., Bejaoui, M., Perrotta, S. & Agaogluet, L. (2006) A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia. Blood, 107, 3455-3462.
3. Cappellini, M.D., Porter, J., El-Beshlawy, A., Li, C.-K., Seymour, J.F. & Elalfy, M. (2010) Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of 1744 patients with transfusion-dependent anemias. Haematologica, 95, 557-566.
4. Cappellini, M.D., Bejaoui, M., Agaoglu, L., Canatan, D., Capra, M. & Cohen, A. (2011) Iron chelation with deferasirox in adult and pediatric patients with thalassemia major; efficacy and safety during 5years' follow-up. Blood, 118, 884-893.
5. Chan, J.C., Chim, C.S., Ooi, C.G., Cheung, B., Liang, R. & Chan, T.K. (2006) Use of the oral chelator deferiprone in the treatment of iron overload in patients with HbH disease. British Journal of Haematology, 133, 198-205.
6. Chong, Y.M., Tan, J.A.M.A., Zubaidah, Z., Rahimah, A., Kuldip, K. & George, E. (2006) Screening of concurrent alpha-thalassaemia 1 in beta-thalassaemia carriers. The Medical Journal of Malaysia, 61, 217-220.
7. Cohen, A.R., Glimm, E. & Porter, J.B. (2008) Effect of transfusional iron intake on response to chelation therapy in beta-thalassemia major. Blood, 111, 583-587.
8. Exjade US prescribing information. (2013). http://www.pharma.us.novartis.com/product/pi/pdf/exjade.pdf
9. Galanello, R., Piga, A., Alberti, D., Rouan, M.-C., Bigler, H. & Séchaud, R. (2003) Safety, tolerability, and pharmacokinetics of ICL670, a new orally active iron chelating agent in patients with transfusion-dependent iron overload due to beta-thalassemia. Journal of Clinical Pharmacology, 43, 565-572.
10. Galanello, R., Piga, A., Forni, G.L., Bertrand, Y., Foschini, M.L. & Bordone, E. (2006) Phase II clinical evaluation of deferasirox, a once-daily oral chelating agent, in pediatric patients with beta-thalassemia major. Haematology, 91, 1343-1351.
11. Ladis, V., Chouliaras, G., Berdoukas, V., Chatziliami, A., Fragodimitri, C. & Karabatsos, F. (2011) Survival in a large cohort of Greek patients with transfusion-dependent beta thalassaemia and mortality ratios compared to the general population. European Journal of Haematology, 86, 332-338.
12. Li, C.G., Li, C.F., Li, Q. & Li, M. (2009) Thalassemia incidence and treatment in China with special reference to Shenzhen City and Guangdong province. Hemoglobin, 33, 296-303.
13. Liu, J.Z., Gao, Q.S., Jiang, Z. et al. (1989) Studies of beta-thalassemia mutations in families living in three provinces in southern China. Hemoglobin, 13, 585-595.
14. Modell, B., Khan, M. & Darlison, M. (2000) Survival in β-thalassaemia major in the UK: data from the UK Thalassaemia Register. Lancet, 355, 2051-2052.
15. Olivieri, N.F., Nathan, D.G., MacMillan, J.H., Wayne, A.S., Liu, P.P. & McGee, A. (1994) Survival in medically treated patients with homozygous B-thalassemia. New England Journal of Medicine, 331, 574-578.
16. Piga, A., Galanello, R., Forni, G.L., Cappellini, M.D., Origa, R. & Zappu, A. (2006) Randomized phase II trial of deferasirox (Exjade, ICL670), a once-daily, orally-administered iron chelator, in comparison to deferoxamine in thalassemia patients with transfusional iron overload. Haematologica, 91, 873-880.
17. Porter, J., Galanello, R., Saglio, G., Neufeld, E.J., Vichinsky, E. & Cappellini, M.D. (2008) Relative response of patients with myelodysplastic syndromes and other transfusion-dependent anaemias to deferasirox (ICL670): a 1-yr prospective study. European Journal of Haematology, 80, 168-176.
18. Taher, A., Elalfy, M.S., Al Zir, K., Daar, S., Al Jefri, A. & Habr, D. (2011) Importance of optimal dosing ≥30mg/kg/d during deferasirox treatment: 2.7-yr follow-up from the ESCALATOR study in patients with β-thalassemia. European Journal of Haematology, 87, 355-365.
19. Thalassaemia International Federation (2008) Guidelines for the Clinical Management of Thalassaemia (2 edn). Thalassaemia International Federation, Nicosia.
20. Vichinsky, E., Onyekwere, O., Porter, J. et al. (2007) A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease. British Journal of Haematology, 136, 501-508.
21. Yang, Y., He, M., Cui, H., Bian, L. & Wang, Z. (2000) The prevalence of lactase deficiency and lactose intolerance in Chinese children of different ages. Chinese Medical Journal, 113, 1129-1132.
22. Zeng, Y.T. & Huang, S.Z. (1987) Disorders of haemoglobin in China. Journal of Medical Genetics, 24, 578-583.
23. Zheng, J.J. & Rosenberg, I.H. (1984) Lactose malabsorption in healthy Chinese adults. Ecology of Food and Nutrition, 15, 1-6.