Serum erythropoietin (EPO) levels were determined by radioimmunoassay in 37 beta-thalassemia patients, the phenotype being thalassemia major (TM) in 30 and thalassemia intermedia (TI) in 7. The control group consisted of 37 healthy children. The mean serum EPO levels were significantly higher in patients with both TM (215.1 +/- 144.5) and TI (53.8 +/- 40.2) compared with the control group (9.3 +/- 4.6). Although the mean hemoglobin (Hb) concentrations in the patients with TM and TI were similar (8.6 +/- 0.9 and 8.7 +/- 1.1, respectively), the mean serum EPO level was significantly higher in TM patients than the patients with TI (P < .01). This finding may indicate that some other factors contributing to the metabolic adaptation to low oxygen concentration or improvement of the tissue oxygenation are as effective as the Hb concentration in EPO production. It is also suggestive of the fact that some amount of tissue hypoxia cannot be prevented in spite of polytransfusion regimens in TM patients. Serum EPO levels of TM patients were not found to be age related or correlated with the mean pretransfusional Hb levels. In the TM patients, the serum EPO concentration was not consistently correlated with clinical signs of erythropoietic activity. This may be indicative of personal differences with respect to the sensitivities of erythroid precursors to the increasing EPO levels in TM patients.
Augmentation of γ-gene synthesis by using recombinant human erythropoietin (r-Hu-EPO) represents a new approach to the therapy of β-thalassemia. A prospective study was conducted in 26 transfusion-dependent β-thalassemia major patients. r-Hu-EPO (Eprex/Cilag, Switzerland) was given to the patients at an initial dose of 500 IU/kg s.c. 3 times a week for at least 2 months during which no transfusion was applied. A sustained hemoglobin (Hb) level greater than 8 g/dl was considered as a response to EPO treatment. In the patients whose Hb levels remained under 8 g/dl or did not increase in comparison to pretreatment levels within 4 weeks, the dose of r-Hu-EPO was increased to 1,000 IU/kg 3 times a week and applied for another 4 weeks. Only 16 cases also received oral iron supplementation. The whole blood and reticulocyte counts, the biochemical tests including BUN, creatinine, AST, ALT, alkaline phosphatase and ferritin were done and the percentages of HbF and F cells were analyzed regularly. At the end of the 2nd month, 6 cases qualified to continue with the trial. At the end of the 6th month, r-Hu-EPO therapy was ceased in 3 cases of the 6 since their Hb levels had decreased below 7 g/dl. Only 3 cases (11.5%) continued with the r-Hu-EPO therapy without transfusion for up to 12 months. In conclusion, r-Hu-EPO may be useful in some selected transfusion-dependent patients with β-thalassemia major. Selection criteria should include a mild β-genotype or coinheritance of α-thalassemia, splenectomy and pretreatment reticulocyte response of the patients as well as the patients’ compliance.
It has been shown that high doses of human recombinant erythropoietin (r epo) increase haemoglobin levels by augmentation of F-cells, and Hb-F production in animal models and in human trials. In this study, r epo was used in patients with beta thalassemia intermedia. Our purpose was to improve haemoglobin levels by at least 2 g and maintain an average level between 10 and 12 g/dl. Ten patients aged 6-29 years (mean 14 +/- 7.6 years) with thalassemia intermedia were treated with r epo. It was given subcutaneously in rising doses from 500 to 1000 U/kg three times weekly for 3 months. During r epo therapy eight cases (80 per cent) showed an increase in haemoglobin, haematocrit, and reticulocyte levels, and an increase of at least 2 g of haemoglobin was obtained. Blood transfusion was not needed during the study except in one case. Five cases (50 per cent) improved life quality with therapy. Hb levels of all patients returned to baseline values over 1 or 2 months after r epo was discontinued. There was no significant change in absolute Hb-F, F-cells, and ferritin levels during treatment. Generally, the drug was well tolerated. No patient had hypertension. Recombinant erythropoietin seems to be an effective treatment for anaemia of beta-thalassemia intermedia, but longer term randomized trials are needed especially in patients with beta thalassemia major.
Prophylaxis has been practiced for many years in Europe and is gaining acceptance worldwide with current viral inactivation procedures. Unfortunately, the high cost of prophylaxis is currently the major obstacle to its implementation in developing countries such as Turkey. The aim of this controlled preliminary study is to evaluate the efficacy, safety, and feasibility of prophylaxis. Seven boys aged 1.5-7 years (5.0 +/- 1.8), who had severe hemophilia (six A, one B) received 20-50 IU/kg factor twice weekly and were followed up for 6-24 months (14.5 +/- 6.6). Intermediate concentrates have been used in hemophilia A and ultrapure product for hemophilia B. The data obtained for the same group of patients before prophylaxis were used as a control group. Another control group was selected in another group of 10 hemophiliacs, mean age 12.5, and received treatment on demand. During prophylactic treatment, the episodes of bleeding were decreased (from 10.5 +/- 3.2 to 4.5 +/- 3.6). Orthopedic and radiologic joint scores were stable (from 0 to 1 and from 1.1 +/- 1.2 to 1.0 +/- 1.5). The patients spent significantly fewer days in the hospital (from 18 +/- 12 to 0.7 +/- 0.6). None of the patients was infected with hepatitis A, hepatitis B, or human immunodeficiency virus. One patient was seroconverted with anti-hepatitis C virus in the third month of prophylaxis. Mean consumption of concentrates for prophylaxis was 3489 +/- 960 IU/kg per year compared with 2073 +/- 1302 in conventional therapy. Prophylaxis was superior to treatment on demand even when given in a twice-weekly period with intermediate concentrates. In Third World countries, prophylaxis should be tried at least in selected severely hemophilic children in order to prevent disabilities.
Although the beta (beta) thalassaemia carrier frequency in Turkey was stated to be 2 per cent, the prevalence rate varies widely in different regions and there is limited data confirming the disorder in Aegean region. This prevalence study was planned to determine frequency of beta thalassaemia trait in the Aegean region among 1124 high school students, between 13 and 18 years old, who were selected as target population. Sensitivity of mean corpuscular volume (MCV) and mean corpuscular haemoglobin (MCH) in prediction of beta thalassaemia trait were evaluated. Venous blood samples were obtained for haemoglobin electrophoresis, HbA2 and HbF, serum iron and total iron binding capacity from students in whom the levels of haemoglobin (Hb), haemotocrite (Hct), MCV, or MCH, were low compared to normal values. The prevalence of beta thalassaemia trait in Aegean region was 3 per cent. Sensitivity of MCV and MCH for determining beta thalassaemia trait were 100 and 96 per cent, respectively.
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