Author: Mok, Sio-peng
Title: Use of oral chelator (L1, 1,2-dimethy1-3-hydroxypyrid-4-ones) and subcutaneous desferal for reversing poor cardiac function and vascular stiffness of transfusion-dependent thalassaemic patients
Degree: M.Sc.
Year: 2008
Subject: Hong Kong Polytechnic University -- Dissertations.
Thalassemia -- Treatment.
Iron chelates.
Department: School of Nursing
Pages: 77 leaves ; 30 cm.
Language: English
Abstract: Cardiac failure due to myocardial iron deposition remains the major cause of death in thalassaemia major (TM) patients in many countries as well as in Hong Kong (HK). This project was a single arm controlled study which aimed to examine the efficacy of combined use of daily oral deferiprone (LI) with subcutaneous desferrioxamine (DFO) 3 to 5 days per week in reducing cardiac iron and improving cardiac function in Hong Kong patients with thalassaemia major. Nineteen patients (age range: 14 to 33.3 years) in the Department of Paediatrics and Adolescent Medicine in Queen Mary Hospital of Hong Kong were recruited to receive the combined treatment for 12 months. For the drug efficacy evaluation, various approaches were taken including: (1) T2* magnetic resonance imaging (MRI) for myocardial and liver iron and MRI for left ventricle ejection fraction (LVEF); (2) echocardiography (Echo) for left ventricle shortening fraction (SF) and left ventricle ejection fraction (LVEF), (3) Multiple uptake gated acquisition (MUGA) for left ventricle ejection fraction (LVEF); and (4) carotid arterial stiffness for calculating stiffness index (SI). These measurements were performed pre and post combined treatment as the primary functional outcome parameters. In addition, (5) serum ferritin was measured bi-monthly to predict iron load and toxicity in the body. For safety evaluation; regular laboratory blood testing was done. Reduction in iron level and improvement in cardiac function were demonstrated. T2* MRI of myocardial and liver iron were significantly reduced (myocardial T2*: from 15.52+-6.32 m/sec to 17.99+-6.57 m/sec, p=0.000 and liver T2*: from 3.06+-2.32 m/sec to 5.09+-5.00 m/sec, p=0.000). Serum ferritin levels had also declined (from 6690+-3116 pmol/L to 4930+-2685 pmol/L; p=0.004). Significant increases were seen in the LVEF by MRI, Echo and MUGA (MRI: from 59.78+-5.82% to 63.36+-6.43%, p=0.004; Echo: from 62.95+-9.56% to 63.57+-6.13%, p=0.043 and MUGA: from 58.39+-6.78% to 62.30+-7.03%, p=0.001) which had indicated the improvement in cardiac function. SF by Echo had slightly increased (from 33.95+-6.54% to 34.35+-4.45%, p=0.126), a sign of cardiac function improvement, and carotid arterial stiffness by SI was found to be mildly decreased (from 5.26+-1.70 to 4.90+-1.45, p=0.484) which had indicated an improvement in vascular rigidity, albeit both statistically not significant. The combined treatment was found to be safe. Neutropenia (5%), agranulocytosis (5%), transient and mild gastrointestinal upset (21%), arthropathy (21%) and fatigue (11%), were observed as the common side-effects of LI. This is the first evaluation on the combined therapy of oral deferiprone (LI) with subcutaneous desferrioxamine (DFO) on improving cardiac function and vascular stiffness for TM patients in Hong Kong. It has proved that the combined treatment is effective in reducing body and cardiac iron with concomitant improvement in cardiac function in these patients. It has implications for health care professionals in clinical management to TM patients.
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