Bone marrow transplantation in patients with thalassemia

Article Abstract:

Beta-thalassemia is a genetic disorder in which the beta-globin pieces of the hemoglobin molecule are defective or completely absent. This results in severe anemia and many complications, including expansion of the bone marrow, which may be kept under control by transfusions of normal red blood cells. At present, the only treatment which may allow the patient to live a disease-free life is bone marrow transplantation (BMT). To analyze the effectiveness of BMT, 222 patients who underwent bone marrow transplantation were studied to determine the success rate of the operation and to identify the different factors which might determine success or failure. The longest-surviving patient is disease-free almost six years after the transplantation procedure; about 75 percent of the bone-marrow recipients are disease-free after a year. Risk factors for failure of BMT included enlargement of the liver (hepatomegaly) and portal fibrosis, or the abnormal spread of fibrous tissue in a major liver vein. It could not be concluded whether absence of hepatomegaly or the drug which had been given to prevent it had the positive effect on the outcome. Until this is learned, it is recommended that thalassemia patients with acceptable bone marrow donors receive transplantation as quickly as possible. (Consumer Summary produced by Reliance Medical Information, Inc.)

Care and treatment, Research, Bone marrow, Bone marrow transplantation, Transplantation of organs, tissues, etc., Organ transplantation, Tissue transplantation, Transplantation

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Long-term safety and effectiveness of iron-chelation therapy with deferiprone for thalassemia major

Article Abstract:

The drug deferiprone does not appear to effectively control blood iron levels and may increase the risk of liver damage. This drug is used to reduce iron levels in patients with thalassemia major, who must receive regular blood transfusions. Researchers compared iron levels in liver samples and signs of liver damage in liver biopsies from 14 patients treated with long-term deferiprone and 12 patients treated with deferoxamine. Those taking deferiprone had high levels of iron in their liver samples and were more likely to develop progressive liver damage compared to those taking deferoxamine.

Evaluation, Complications and side effects, Chelation therapy, Deferoxamine

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Hepatic iron concentration and total body iron stores in thalassemia major

Article Abstract:

Measuring the amount of iron in a liver biopsy specimen may be a good way to determine total body iron in patients with thalassemia. Thalassemia is a blood disease that can be treated with a bone marrow transplant. However, one side effect of the treatment is excessive amounts of iron in the body. Blood-letting can reduce the amount of iron in the body but doctors need some accurate way of measuring the remaining iron. Researchers determined that the amount of total body iron stores is about 10 times the level in the liver.

Measurement, Physiological aspects, Liver, Liver biopsy

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