Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0039730 (thalassemia)
 10,305 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

The therapeutic role of xanthinol nicotinate, with its potent action on the peripheral circulation, in promoting healing of leg ulcers when added to conservative measures of ulcer treatment in adult beta-thalassaemia major and sickle cell thalassaemia, was evaluated in a double-blind crossover trial in 16 patients suffering from multiple leg ulcers. Conservative measures of ulcer treatment were leg raising in horizontal position for 14 hours, bed-rest, local antiseptic dressings and antibiotics. Xanthinol nicotinate or placebo was administered in a daily dose of 8 tablets (2400 mg) for 10 weeks. Comparison of the treatment results of conservative measures plus xanthinol nicotinate and those of conservative measures plus placebo revealed a statistically significant higher rate of complete ulcer healing during xanthinol nicotinate therapy (p less than 0.01). Apart from a low incidence of generalized itching and flushing at the start of the trial, xanthinol nicotinate was well tolerated in the prescribed dose.
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PMID:Xanthinol nicotinate in the management of leg ulcers associated with haemoglobinopathies. 54 May 21

Since the obtained results from the pilot study indicated that dilazep which was a membrane stabilizer would be benefit to treatment and prevention of anemia and chronic leg ulcer in beta-thalassemia/hemoglobin E (beta-thal/HbE) patients, the authors had continued the study in a second phase, ie a double blind placebo control trial. Twenty-seven beta-thal/HbE patients were recruited in the study. Eight patients who suffered from chronic leg ulcer were given dilazep. The rest of patients were given dilazep or placebo according to a randomized table. Hence, 16 patients received dilazep and 11 received placebo. When we compared the number of unit of blood transfusion, hemoglobin level, 2-3 DPG and P50 value between the dilazep and placebo groups using unpaired t-test, we found that there were no statistical differences in any of the parameters. However, when we compared the data within the group using paired t-test, there was statistical decrease in blood requirement after treatment in the dilazep group (p < 0.05). Concerning with the treatment of chronic leg ulcer, 3 in 8 patients were completely healed within 3 months, 4 in 8 patients were improved and 1 in 8 patients was not improved. There were complaints of skin itching and mild epigastric pain in placebo group but the liver function tests, kidney function tests and cardiac enzyme did not significantly change during the medication.
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PMID:A double-blind placebo control trial of dilazep in beta-thalassemia/hemoglobin E patients. 964 Jun 22

Thalassaemia major is a severe chronic hemolytic disease, resulted with iron overload mainly due to regular blood transfusions. Iron overload may lead to serious organ toxicity and even fatal complications, if no iron excretion is achieved by a chelating agent. First introduced in 1976 as s.c. treatment for thalassaemia major, desferrioxamine (DFO) has substantially improved the life expectancy in the disease. While DFO can cause local allergic reactions including redness, itching, pain and lumps, on rare occasion anaphylactic reactions can occur. The mechanism of anaphylaxis like reactions is not well understood. In this case report, we presented a 10 years-old girl with thalassaemia major who had to stop DFO therapy after appearing of systemic allergic reactions with hypotension, tachycardia, pruritus and urticaria against this drug. Serum IgE level was normal, specific IgE and skin prick tests were negative. Intradermal test was resulted with positive reaction to DFO. The patient was hospitalized and desensitization protocol was initiated with rapid s.c. infusions per 15 min. The protocol was stopped at the 17th cycle because of local reaction reappeared. After that, DFO was further diluted and was restarted with lower dosage and longer infusion period. Then, DFO dosage was increased and the dilutions and infusion times were decreased gradually. By this desensitization programme, the patient would continue to use DFO chelation safely for 10 months.
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PMID:Successful desensitization of a case with desferrioxamine hypersensitivity. 1709 79