Abstract
Thalassemia is a group of complex haemoglobin disorders common in the Mediterranean countries, the Middle East and South East Asia1. Thalassemia is common in the United Arab Emirates (UAE) affecting indigenous population and the expatriates. The exact prevalence of thalassemia is not known.
The management of thalassemia has improved significantly due to improvement in transfusion support and the management it's complications. This has led to the improvement of the overall survival and most patients reaching their adulthood2. Despite this some die at young age mainly due to poor access to health care2, proper management and lack of compliance to chelation therapy which leads to the development of otherwise preventable complications2.
Blood transfusion is the corner stone of the management of patient with thalassemia major and intermediate3. Blood transfusion improves the anemia and its symptoms but also suppress the resultant ineffective erythropoiesis. This is usually started once the patient has signs and symptoms of anemia and unable to compensate for the low haemoglobin3. The aim of blood transfusion is to maintain good quality of life and to prevent the skeletal abnormalities2. Although blood transfusion is essential in the management of patients with thalassemia, it will ultimately lead to iron overload. In addition there is excess of gastrointestinal iron absorption in these patients, secondary to the ineffective erythropoiesis4. To reduce the iron absorption the target is to maintain the pre-transfusion hemoglobin level between 9 and 10 g/dl. Iron overload is major cause of mortality and morbidity in patient's thalassemia major and intermedia5.
In this retrospective analysis we reviewed the ferritin levels over 1 year in 25 patients with thalassemia major or intermedia attending the adult hematology department at Tawam hospital The patients received regular blood transfusion once every three weeks. Of the 25 patients 16 were males and 9 were females. The age range was 15 years to 47 years. Nineteen patients had their chelation therapy changed during the year from Exjade (Deferasirox tablets for oral suspension) to Jadenu (Deferasirox tablets) which improved our patient's compliance. The remaining 6 patients continued on Exjade. In addition to this 17 patients received additional Desferral infusion in connection with their blood transfusion. None of the patient was on subcutaneous desferral infusion. During our observation two patients were lost to follow up and one patient died.
The medium ferritin level for our evaluable patients was at start of the review 2488 mcg/l, with a range between 609 mcg/l to 5147 mcg/l. The medium ferritin level at the end was 1691 mcg/l with a range between 470 mcg/l to 3731 mcg/l. Figure 1 one shows the ferritin value a start and end of the review for each patient. Our patients with the highest ferritin levels are despite repeated counselling not compliant with their medications.
Despite significant improvement in the management of thalassemia patients, iron overload remain a challenge. The main problems to achieving full control over the iron overload are lack of compliance. Hopefully the newest oral formulation will increase the compliance and at least we have seen a decrease in the ferritin levels over the last year in most patients.
References.
1 De Dreuzy E, Bhukhai K, Leboulch P, Payen E Current and future alternative therapies for
Beta Thalassemia major. Biomedical Journal 39, 124-38
2 Modell, B., Khan, M., and Darlison, M. Survival in beta thalassaemia major in the UK:
Data from the UK Thalassaemia Register. Lancet 355 [2000]: 2051-2052.
3 Rebulla P, Modell B. Transfusion requirements and effects in patients with thalassaemia
major. Cooleycare Programme. Lancet 1991;337:277-80
4 Gardenghi S, Marongiu MF, Ramos P, et al. - Ineffective erythropoiesis in beta-
thalassemia is characterized by increased iron absorption mediated by down-
regulation of hepcidin and up-regulation of ferroportin. Blood 2007; 109:5027-35
5 Porter, J.B., and Davis, B.A. Monitoring chelation therapy to achieve optimal
outcome in the treatment of thalassaemia. Best Practice & Research: Clinical
Haematology 15 [2002]: 329-368
6 Ayidinok Y, Kattamis A, Cappellini MD, et al. Deferasirox- Deferoxamine
combination therapy reduces cardiac iron with rapid liver iron removal after 24
months in patients with severe transfusional iron overload (abstract).
Haematologica 2014;99:229.
No relevant conflicts of interest to declare.
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