Abstract
BackgroundThalassaemia is a chronic disease without an effective cure in a majority. The clinical management has improved considerably during recent years; however, minimal attempts are made to up lift the quality of life among patients, especially in developing countries. Here we aim to describe and compare and to determine factors associated with health related quality of life among patients with transfusion dependent β-thalassaemia major and haemoglobin E β-thalassemia in Sri Lanka.MethodsA case control study was conducted in the three largest thalassaemia centres of Sri Lanka. All patients with transfusion dependent β-thalassaemia (β-thalassaemia major and haemoglobin E β-thalassaemia) aged 5–18 years were recruited as cases whilst a randomly selected group of children without chronic diseases were recruited as controls. Socio-demographic and clinical data were collected using an interviewer-administered questionnaire and health related quality of life was measured using the validated Paediatric Quality of Life Inventory Version 4.0.ResultsTwo hundred and seventy one patients with transfusion dependent β-thalassaemia (male-49.1%; mean age- 10.9 ± 3.6 years) and 254 controls (male-47.2%; mean age- 10.4 ± 3.5 years) were recruited. Mean health-related quality of life scores were significantly lower in patients compared to controls (72.9 vs. 91.5, p < 0.001). Of the patients, 224 (84%) had β-thalassaemia major and 43 (16%) had haemoglobin E β-thalassaemia. Quality of life scores in psychological health (p < 0.05), emotional functioning (p < 0.05) and social functioning (p < 0.05) were significantly lower in patients with haemoglobin E β-thalassaemia compared to β-thalassaemia major. Splenectomy (p < 0.05), short stature (p < 0.05), under nutrition (p < 0.05) and longer hospital stays (p < 0.05) were significantly associated with lower quality of life scores.ConclusionsDespite improvements in management, the quality of life among patients with β-thalassaemia still remains low. This is more pronounced in the subset of patients with haemoglobin E β-thalassaemia. Splenectomy, short stature, undernutrition and longer hospital stays were significantly associated with poor quality of life. It is timely, even in developing countries, to direct emphasis and to take appropriate steps to improve standards of living and quality of life of patients with β-thalassaemia.
Highlights
Introduction βThalassaemia is one of the most common monogenic diseases in the world [1, 2]
In this study we aim to describe and compare and to determine factors associated with health related quality of life (HRQoL) among patients with transfusion dependent βthalassaemia major and haemoglobin E β-thalassemia in Sri Lanka and to compare it with non-thalassaemia controls
Health-related quality of life among patients with βthalassaemia major and haemoglobin E β-thalassaemia we evaluated the HRQoL scores among patients with β-thalassaemia major and Haemoglobin E (HbE) β-thalassaemia (Fig. 3)
Summary
Introduction βThalassaemia is one of the most common monogenic diseases in the world [1, 2]. A majority of patients with transfusion dependent β-thalassaemia (TDBT) are managed medically with regular blood transfusions and iron chelation therapy for life [9]. Uniform guidelines which are published and frequently updated by the Thalassaemia International Federation (TIF) are available to guide treatment of these patients [11] These guidelines advocate 2–5 weekly blood transfusions to maintain a pre-transfusion haemoglobin level between 9.0–10.5 g/dl and regular iron chelation therapy to achieve a serum ferritin value below 1000 ng/dL. Through these management strategies, patients with TDBT live longer - commonly into the fifth decade and have lesser rates of medical complications [12].
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