EFFICACY OF INTERRUPTED AND MODIFIED DEFERASIROX DOSE AMONG PAEDIATRIC PATIENTS WITH Β- THALASSEMIA MAJOR AND HIGH ALANINE AMINOTRANSFERASE LEVEL

Nashwan M AL Hafidh

Abstract


Background: Abnormal liver function tests lead to interruptions of Deferasirox therapy. The aim of this study is to assess the efficacy of deferasirox dose 30 mg /kg /day in maintaining cardiac protective level of serum ferritin of <2500 ng/ml among patients who received interrupted and modified doses. Methods: A retrospective cohort study was conducted in Ibn Al Atheer paediatric hospital in Mosul city, Iraq, utilizing the monthly reading of serum ferritin level during the period started in February 2013 to march 2014 using documented patients' records. Group A, patients included thirty-five patients with β- thalassemia major whose Deferasirox dose of 30 mg/kg/day was interrupted and modified due to ≥ 5-fold raise in alanine aminotransferase during any month of the study period. Compared group B patients included 40 children who received constant median deferasirox dose 30 mg/kg/day throughout one year of study period. Serum ferritin and alanine aminotransferase levels were routinely analysed every month among those patients. Results: Interrupted and modified Deferasirox dose of 30 mg/kg/day significantly (p=0.000) increase the frequency of having mean serum ferritin >2500 ng/ml, and was associated with 55 times relative risk of having mean serum ferritin >2500 ng/ml compared to group B with steady median deferasirox dose. Conclusions: Interrupted and modified deferasirox dose of 30 mg/kg/day has a significant adverse effect on cardiac protective level of serum ferritin.

Keywords: Deferasirox dose, β- thalassemia major, Alanine aminotransferase, Serum ferritin


Full Text:

PDF

References


Taher A, Elalfy MS, Al Zir K, Daar S, Al Jefri A, Habr D, et al. Achieving treatment goals of reducing or maintaining body iron burden with deferasirox in patients with b -thalassaemia: results from the ESCALATOR study. Eur J Haematol 2011;87(4):349–54.

Cappellini MD, Bejaoui M, Agaoglu L, Canatan D, Capra M, Cohen A, et al. Iron chelation with deferasirox in adult and pediatric patients with thalassemia major: efficacy and safety during 5 years' follow-up. Blood 2011;118(4):884–93.

Viprakasit V, Ibrahim H, Ha SY, Ho PJ, Li CK, Chan LL, et al. Clinical efficacy and safety evaluation of tailoring iron chelation practice in thalassaemia patients from Asia-Pacific: a subanalysis of the EPIC study of deferasirox. Int J Hematol 2011;93(3):319–28.

Taher A, Cappellini MD, Vichinsky E, Galanello R, Piga A, Lawniczek T, et al. Efficacy and safety of deferasirox doses of >30 mg/kg per d in patients with transfusion-dependent anaemia and iron overload. Br J Hematol 2009;147(5):752–9.

Dubey AP, Sudha S, Parakh A. Deferasirox: The New Oral Iron Chelator. Indian Pediatr 2007;44(8):603–7.

Taher A, Cappellini MD. Update on the use of deferasirox in the management of iron overload. Ther Clin Risk Manag 2009;5:857–68. Ref no 6&15 are same

Cappellini MD, Cohen A, Piga A, Bejaoui M, Perrotta S, Agaoglu L, et al. A phase 3 study of deferasirox (ICL670), aonce-daily oral iron chelator, in patients with beta-thalassemia. Blood 2006;107(9):3455–62.

Taher A, Zir KA, Elalfy MS, Daar S, Jefri AA, Habr D, et al. Importance of optimal dosing >30 mg/kg/d during deferasirox treatment: 2.7-yr follow-up from the ESCALATOR study in patients with β- thalassemia. Eur J Hematol 2011;87(4):355–65. Ref no 8&10 are same

Porter, J. Deferasirox: an effective once daily orally active iron chelator. Drugs Today (Barc) 2006;42(10):623–37.

Wood JC, Kang BP, Thompson A, Giardina P, Harmatz P, Glynos T, et al. The effect of deferasirox on cardiac iron in thalassemia major: impact of total body iron stores. Blood 2010;116(4):537–43.

Pennell DJ, Udelson JE, Arai AE, Bozkurt B, Cohen AR, Galanello R, et al. Cardiovascular function and treatment in β-thalassemia major: a consensus statement from the American Heart Association. Circulation 2013;128(3):281–308.

Pathare A, Taher A, Daar S. Deferasirox (Exjade) significantly improves cardiac T2* in heavily iron-overloaded patients with beta-thalassemia major. Ann Hematol 2010;89(4):405–9.

Chaudhary P, Pullarkat V. Deferasirox: appraisal of safety and efficacy in long-term therapy. J Blood Med 2013;4:101–10.

Borgna-Pignatti C, Rugolotto S, De Stefano P, Zhao H, Cappellini MD, Del Vecchio GC, et al. Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine. Haematologica 2004;89(10):1187–93.

Modell B, Khan M, Darlison M. Survival in beta-thalassaemia major in the UK: data from the UK Thalassaemia Register. Lancet 2000;355(9220):2051–2.


Refbacks

  • There are currently no refbacks.


Contact Number: +92-992-382571

email: [jamc] [@] [ayubmed.edu.pk]