Desferrioxamine in thalassaemia.

Diwany, M.; Gabr, Mamdouh; Hefni, A. el; Mokhtar, Nihad

doi:10.1136/adc.43.229.340

Cited by 8 publications

(7 citation statements)

References 11 publications

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“…Of these two agents, DFA is easier to administer as it can be given intramuscularly and it is well tolerated. Most reports on the use of DFA in thalassaemia were confined to short-term trials (Smith, 1962; Hwang and Brown, 1964;McDonald, 1966;Diwany et al, 1968;Markum et al, 1969). Moreover, measurement of urinary iron excretion was the only criterion of response, and this does not provide evaluation of its clinical benefits.…”

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confidence: 99%

Long-term administration of desferrioxamine in thalassaemia major

Seshadri¹,

Colebatch²,

Gordon³

et al. 1974

Archives of Disease in Childhood

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(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.

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confidence: 99%

Long-term administration of desferrioxamine in thalassaemia major

Seshadri¹,

Colebatch²,

Gordon³

et al. 1974

Archives of Disease in Childhood

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“…For these reasons wide variations may occur in the amount of iron excreted after DFA in thalassaemia. Thus, previous reports have mentioned DFA-induced urinary iron excretion in thalassaemia major ranging from 0 * 84 to 24 -1 mg in 24 hours per g/DFA (Smith, 1962;Hwang iand Brown, 1964;McDonald, 1966;Diwany et al, 1968;Markum et al, 1969).…”

Section: Discussionmentioning

confidence: 95%

Urinary iron excretion in thalassaemia after desferrioxamine administration

Seshadri¹,

Colebatch²,

Fisher³

1974

Archives of Disease in Childhood

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Seshadri, R., Colebatch, J. H., and Fisher, R. (1974). Archives of Disease in Childhood, 49, 195. Urinary iron excretion in thalassaemia after desferrioxamine administration. Urinary iron excretion induced with desferrioxamine (DFA) was estimated in 26 children with thalassaemia major. Four separate 24-hour urine collections were made-during a baseline period, after intramuscular injection of DFA 1 g, during blood transfusion with DFA 0 5 g/unit transfused blood, and during the post-transfusion period. Urinary iron, plasma iron, and total iron-binding capacity were estimated by atomic absorption spectrophotometry. Urinary iron excretion in the 24 hours after 1 g DFA ranged from 1 1 to 23 mg/M2 surface area compared with 0 * 1 to 1 * 6 mg/M2 during the baseline period. A positive correlation was obtained between DFA-induced urinary iron excretion and the amount of blood transfused, plasma iron level, and size of the spleen. Splenectomized patients excreted less iron after DFA than those who were not splenectomized. DFA-induced urinary iron was measured before and after splenectomy in 6 patients. In 5 of the 6 patients a drop in iron excretion was observed and the analysis of the 6 pairs of results indicated that splenectomy produced a decrease in DFA-induced urinary iron excretion.These findings indicate that the enlarged spleen in thalassaemia is a major source of iron chelated by DFA. It is suggested that treatment with DFA in the presence of a large spleen may be ineffective in removing excess iron from the myocardium and liver.The survival of children with thalassaemia major depends on repeated blood transfusions. Marked iron deposition develops in various vital organs, and if this is untreated it leads to a severe degree of morbidity and finally death, commonly during the second decade of life. Hence, assessment of the degree of iron overload is important in the prognosis and management of children with thalassaemia major. In recent years desferrioxamine (DFA), a specific and effective chelator of iron, has been used for the assessment of iron stores (Ploem et al., 1966;Hedenberg, 1969 size. They also suggest that splenectomy diminishes urinary iron excretion, which indicates that the spleen plays a major role in iron chelation by DFA in thalassaemia major. PatientsIn 1972-73, 26 patients with thalassaemia major in the Royal Children's Hospital, Melbourne, were studied. There were 13 boys and 13 girls, their ages at the time of the study ranging from 2 to 21 years with a median of 10 years. The 3 oldest patients, aged 15, 21, and 21 *ears, will be discussed separately because in addition to splenectomy they had been receiving DFA daily for 5 to 8 years before the present study was carried out. This prolonged chelation therapy may have altered significantly the degree of iron overload compared with the remaining 23 patients. Those 23 patients, except for the ones who had received less than 50 units of blood, had clinical evidence of haemosiderosis such as skin pigmentation, cardiomegaly, and liver enlargeme#t...

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“…When Dr. Nathan decided to start regular transfusions, the increasing iron overload contributed to the possibility of heart failure making the treatment even more challenging. Fortunately, iron chelation was entering clinical protocols for thalassemia major, even if intramuscular administration of the drug allowed insufficient urinary excretion of the metal[ 31 , 32 ]. Great advances were achieved with continuous intravenous administration, and in 1977 subcutaneous delivery of the medication was implemented.…”

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confidence: 99%

Innovation for rare diseases and bioethical concerns: A thin thread between medical progress and suffering

Tommasini

Magnolato

Bruno

2018

WJCP

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With the development of precision medicines based on small molecules, antibodies, RNAs and gene therapy, technological innovation is providing some exciting possibilities to treat the most severe genetic diseases. However, these treatments do not always lead to a cure for the disease, and there are several factors that may hinder their overall success. Patients living during a period of great medical change and innovation may benefit from these technological advances but may also just face failures, both in terms of frustrated hopes as well as suffering. In this article, we are telling the stories of three children with rare and severe disorders, who live in an age of significant medical changes, bearing the burden of difficult scientific and ethical choices. The first two cases that are suffering respectively from severe immunodeficiency and beta thalassemia have already been described in scientific journals, as well as in popular magazines. Although similar when considering the medical challenges, the two cases had opposite outcomes, which resulted in distinct ethical implications. The third case is a baby with spinal muscular atrophy, living at a time of continued innovation in the treatment of the disease. With these cases, we discuss the challenges of providing correct information and proper counseling to families and patients that are making the bumpy journey on the road of medical innovation.

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Desferrioxamine in thalassaemia.

Cited by 8 publications

References 11 publications

Long-term administration of desferrioxamine in thalassaemia major

Long-term administration of desferrioxamine in thalassaemia major

Urinary iron excretion in thalassaemia after desferrioxamine administration

Innovation for rare diseases and bioethical concerns: A thin thread between medical progress and suffering

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