Oral iron chelation and the treatment of iron overload in a pediatric hematology center

Pediatric Blood and Cancer

Volumn 52, Issue 5, 2009, Pages 616-620

  Raphael, Jean L ^a,c   Bernhardt, M Brooke ^b   Mahoney, Donald H ^a   Mueller, Brigitta U ^a  

^a BAYLOR COLLEGE OF MEDICINE   (United States)

^b TEXAS CHILDREN S HOSPITAL   (United States)

^c Texas Children's Hospital   (United States)

Author keywords

Anemia; Chelation; Iron overload

Indexed keywords

CREATININE; DEFERASIROX; FERRITIN; IRON CHELATING AGENT; BENZOIC ACID DERIVATIVE; TRIAZOLE DERIVATIVE;

ABDOMINAL PAIN; ARTICLE; BLOOD TRANSFUSION; CHILD; CONSTIPATION; CONTROLLED STUDY; DEMOGRAPHY; DIARRHEA; DOSE RESPONSE; DRUG ERUPTION; DRUG SAFETY; DRUG WITHDRAWAL; FEMALE; HUMAN; INFANT; INFORMATION PROCESSING; IRON OVERLOAD; LIVER FUNCTION TEST; LONG TERM CARE; MAJOR CLINICAL STUDY; MALE; MEDICAL DOCUMENTATION; NAUSEA; NEWBORN; PATIENT CARE; PRESCHOOL CHILD; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; SCHOOL CHILD; SICKLE CELL ANEMIA; TREATMENT DURATION; VOMITING; BLOOD; HEMATOLOGY; HOSPITAL; ORAL DRUG ADMINISTRATION; PEDIATRICS;

ADMINISTRATION, ORAL; BENZOIC ACIDS; CHILD; CHILD, PRESCHOOL; FEMALE; FERRITINS; HEMATOLOGY; HOSPITALS; HUMANS; INFANT; INFANT, NEWBORN; IRON CHELATING AGENTS; IRON OVERLOAD; MALE; PEDIATRICS; TRIAZOLES;

EID: 63649136756     PISSN: 15455009     EISSN: 15455017     Source Type: Journal    
DOI: 10.1002/pbc.21929     Document Type: Article

References (32)

1. Adams RJ, McKie VC, Hsu L, et al. Prevention of a first stroke by transfusions in children with sickle cell anemia and abnormal results on transcranial Doppler ultrasonography. N Engl J Med 1998;339:5 -11.
2. Ballas SK. Iron overload is a determinant of morbidity and mortality in adult patients with sickle cell disease. Semin Hematol 2001;38:30- 36.
3. Manci EA, Culberson DE, Yang YM, et al. Causes of death in sickle cell disease: An autopsy study. Br J Haematol 2003;123:359-365.
4. McLaren CE, Barton JC, Adams PC, et al. Hemochromatosis and iron overload screening (HEIRS) study design for an evaluation of 100,000 primary care-based adults. Am J Med Sci 2003;325:53-62.
5. Porter JB. Practical management of iron overload. Br J Haematol 2001;115:239-252.
6. Nick H, Acklin P, Lattmann R, et al. Development of tridentate iron chelators: From desferrithiocin to ICL670. Curr Med Chem 2003;10:1065 -1076.
7. De Sanctis V, Roos M, Gasser T, et al. Impact of long-term iron chelation therapy on growth and endocrine functions in thalassae- mia. J Pediatr Endocrinol Metab 2006;19:471 -480.
8. Olivieri NF. The beta-thalassemias. N Engl J Med 1999;341:99- 109.
9. Britton RS, Leicester KL, Bacon BR. Iron toxicity and chelation therapy. Int J Hematol 2002;76:219-228.
10. NHLBI., The management of sickle cell disease, In.: Washington, DC: National Institutes of Health; 2002.
11. Olivieri NF, Brittenham GM. Iron-chelating therapy and the treatment of thalassemia. Blood 1997;89:739-761.
12. Cohen AR, Martin MB. Iron chelation therapy in sickle cell disease. Semin Hematol 2001;38:69-72.
13. National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology- v.1.2007: Myelodysplastic Syndromes. 2007. (Accessed at http://www.nccn.org/professionas/physician-gls/pdf/mds.pdf).
14. Neufeld EJ. Oral chelators deferasirox and deferiprone for transfusional iron overload in thalassemia major: New data, new questions. Blood 2006;107:3436-3441.
15. Gabutti V, Piga A. Results of long-term iron-chelating therapy. Acta Haematol 1996;95:26-36.
16. Treadwell MJ, Law AW, Sung J, et al. Barriers to adherence of deferoxamine usage in sickle cell disease. Pediatr Blood Cancer 2005;44:500-507.
17. Nisbet-Brown E, Olivieri NF, Giardina PJ, et al. Effectiveness and safety of ICL670 in iron-loaded patients with thalassaemia: A randomised, double-blind, placebo-controlled, dose-escalation trial. Lancet 2003;361:1597-1602.
18. Hershko CM, Link GM, Konijn AM, et al. Iron chelation therapy. Curr Hematol Rep 2005;4:110-116.
19. Vichinsky E, Onyekwere O, Porter J, et al. A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease. Br J Haematol 2007;136:501 -508.
20. Cappellini MD, Cohen A, Piga A, et al. A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia. Blood 2006;107:3455-3462.
21. Cappellini MD, Bejaoui M, Agaoglu L, et al. Prospective evaluation of patient-reported outcomes during treatment with deferasirox or deferoxamine for iron overload in patients with beta-thalassemia. Clin Ther 2007;29:909-917.
22. Delea TE, Sofrygin O, Thomas SK, et al. Cost effectiveness of once-daily oral chelation therapy with deferasirox versus infusional deferoxamine in transfusion-dependent thalassaemia patients: US healthcare system perspective. Pharmacoeconomics 2007;25:329-342.
23. Piga A, Galanello R, Forni GL, et al. 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 2006;91:873-880.
24. Exjade (Accessed at http://www.pharma.us.novartis.com/product/pi/pdf/ exjade.pdf.).
25. Walsh JC, Mandalia S, Gazzard BG. Responses to a 1-month self- report on adherence to antiretroviral therapy are consistent with electronic data and virological treatment outcome. AIDS 2002;16:269-277.
26. Haynes RB, Taylor DW, Sackett DL, et al. Can simple clinical measurements detect patient noncompliance? Hypertension 1980; 2:757-764.
27. Galanello R, Piga A, Forni GL, et al. Phase II clinical evaluation of deferasirox, a once-daily oral chelating agent, in pediatric patients with beta-thalassemia major. Haematologica 2006;91:1343 -1351.
28. Osterberg L, Blaschke T. Adherence to medication. N Engl J Med 2005;353:487 -497.
29. Jackevicius CA, Mamdani M, Tu JV. Adherence with statin therapy in elderly patients with and without acute coronary syndromes. JAMA 2002;288:462-467.
30. Cramer J, Rosenheck R, Kirk G, et al. Medication compliance feedback and monitoring in a clinical trial: Predictors and outcomes. Value Health 2003;6:566-573.
31. Haynes RB, McDonald HP, Garg AX. Helping patients follow prescribed treatment: Clinical applications. JAMA 2002;288:2880-2883.
32. Arboretti R, Tognoni G, Alberti D. Pharmacosurveillance and quality of care of thalassaemic patients. A large scale epidemiological survey. Eur J Clin Pharmacol 2001;56:915 -922.