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Category: Volume 06 Issue 03 March 2018
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Title: Defrasirox versus defriprone in patients with Thalassaemia major: A study to compare the efficacy, safety & economy of oral iron chelators

Authors: Dr Mamta Gupta, Dr Pankaj Gupta, Dr Prem Nyati, Dr Swati P. Raipurkar, Dr Suraj Tripathi, Dr Sharad Thora

 DOI:  https://dx.doi.org/10.18535/jmscr/v6i3.169

Abstract

Background: Thalassemia is an inherited impairment of hemoglobin production, in which there is partial or complete failure to synthesize a specific type of globin chain. It is characterized by ineffective erythropoesis and hemolysis. It requires frequent blood transfusion and so becomes the most common chronic iron overloading disorder. Excess iron deposits may be in various tissues of the body, particularly the liver, heart, and endocrine organs.

Materials and Methods: Frequent serum ferritin values at the time of enrolment in the study and after 1, 3, and 6 months were recorded. Serum ferritin was by using Partial Chemiluminescent Axim Abet System method after clinically ruling out any active infection. In this study a total of 73 patients were included, 39 were in Deferasirox group and 34 were in Deferiprone group. Out of these 4 from Deferasirox group and 1 from Deferiprone group were excluded in view of their non-compliance for the regular follow-up. Remaining 68 patients were studied in two groups; 35 in Deferasirox and 33 in Deferiprone group. All these patients were the diagnosed cases of Thalassemia major with more than fifteen blood transfusions in a year.

Results: Base line mean ± SD Hemoglobin at the time of enrollment was 7.32 ± 1.5 in Deferasirox group and 7.74 ± 1.07 in Deferiprone group. After 6 months of regular follow up mean hemoglobin was raised significantly to 8.77 ± 0.84 and 8.69 ± 0.9 respectively in both the groups. There were no significant changes in total White blood cells count, creatinine and SGPT level with the six month of study. About 19(28%) patients reported side effects, 14.71 % with Deferasirox and 13.23% with Deferiprone but none of them required interruption of therapy due to them. The adverse events like G.I. upsets including abdominal pain, nausea, vomiting and diarrhea were observed to the extent of 17.14 % with Deferasirox and 12.12% with Deferiprone. Arthralgia was reported by 2.85% patient in Deferasirox group and 12.12 % in Deferiprone group. Skin rashes were observed in three patients (8.57%) in Deferasirox group. For a patient of 20 Kg, per month average acquisition cost of Deferasirox is Rs.1200/ and for Deferiprone is Rs. 620.

Conclusion: Thus we conclude that Deferasirox and Deferiprone are well tolerated, have few adverse effects and almost has a comparable effect in lowering of the patient's serum ferritin level. Deferiprone is more cost effective but needs a strict control on compliance owing to requirement in three divided doses per day. However in view of limitations to our study further well designed, randomized controlled trials with better sophisticated parameters of iron load are required to put a final remark on the most appropriate oral iron chelator suitable for the Indian population.

Keywords: Thalassemia major, Oral iron chelators, Deferasirox, Deferiprone, Hemoglobin, Ferritin, Efficacy, Safety, Pharmacoeconomy.

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Corresponding Author

Dr Pankaj Gupta

Post Graduate Trainee, Department of Radiodiagnosis, Index Medical College Hospital & Research Centre,

Index City, Nemawar Road, NH-59A, Indore, Madhya Pradesh 452016

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