Long-term administration of desferrioxamine in thalassaemia major

Abstract

(1974). Archives of Disease in Childhood, 49,621. Long-term administration of desferrioxamine in thalassaemia major. Studies of desferrioxamine (DFA) as a specific iron-chelating agent in the treatment of haemosiderosis have been reported in various conditions, but there has been only limited evaluation of its long-term use in thalassaemia major. In the present study 1 g DFA was given intramuscularly daily 6 days per week in 3 patients for 5 to 8 years, and in another 7 patients for 10 to 22 months. In addition, 500 mg DFA was added to each unit of donor blood at the time of transfusions. Urinary iron excretion after DFA was estimated using atomic absorption spectrophotometry both before and periodically during DFA treatment. During follow-up, cardiac and liver functions were assessed by ECG, chest x-ray, and liver function tests.The results show that DFA therapy increased urinary iron excretion, and that this was associated with a significant improvement in the clinical symptoms and signs of haemosiderosis and with return towards normal in the ECG and liver function tests.

Total management of thalassaemia major.

“…The only useful ironchelating drug at present commercially available is desferrioxamine, which is expensive and has to be given parenterally (Sephton-Smith, 1962). Because it offers the only hope of an improved long-term prognosis it has been used regularly in many British and Australian patients in doses of from 0 * 5-1 g daily with or without vitamin C for up to 9 years (Barry et al, 1974;Seshadri et al, 1974;Modell and Beck, 1974).…”

“…Although this policy is bound to produce decreasing returns as patients grow, it nevertheless protects the liver against iron toxicity (Barry et al, 1974;Risdon et al, 1975) and appears to improve long-term survival: this last conclusion will be subjected to more rigorous examination in the near future. This makes it likely that a properly weight-related dose will more effectively protect against iron toxicity, and indeed reversal of established cardiac and hepatic pathology has been observed clinically in older patients on appropriately high desferrioxamine doses (Seshadri et al, 1974).…”

“…Rigorous treatment may even reverse established pathology Seshadri et al, 1974) so treatment should not be withheld from older patients on the supposition that they are beyond recovery. Social aspects.…”

Inhibition of autoimmune neuropathological process by treatment with an iron-chelating agent.

“…There is thus some reason to think that, both from the aspect of removing iron from the brain and of reducing lymphocyte stimulation, DFOM might merit a trial as a therapeutic agent in multiple sclerosis. The drug is relatively nontoxic, having been used for many years to treat both iron overload and iron poisoning (30,31) and, more recently, the tissue damage of long-term hemodialysis (32). Our in vitro results also suggest that drugs that remove free iron may be of general value as mild immunosuppressive agents to inhibit autoreactive, or rejection processes involving lymphocytes of the DTH/helper subset.…”

Electrocardiographic Presentation, Cardiac Arrhythmias, and Their Management in β‐Thalassemia Major Patients

“…[24][25][26][27][28][29] New-onset electrocardiographic abnormalities are usually evident in b-TM with HF and may include electrocardiographic findings that suggest left-sided heart (Q1S3 pattern and extreme left-axis deviation) or right-sided heart involvement (S1Q3 pattern and right-axis deviation), new-onset T wave inversion beyond lead V 1 , P wave prolongation or abnormalities, and a consistent decrease in QRS amplitude. [24][25][26][27][28][29] New-onset electrocardiographic abnormalities are usually evident in b-TM with HF and may include electrocardiographic findings that suggest left-sided heart (Q1S3 pattern and extreme left-axis deviation) or right-sided heart involvement (S1Q3 pattern and right-axis deviation), new-onset T wave inversion beyond lead V 1 , P wave prolongation or abnormalities, and a consistent decrease in QRS amplitude.…”