Thirty-seven patients with thalassemina major (TM) were studied to determine the extent and rate of endocrine complications. Mean haemoglobin and ferritin concentrations were 8.8 +/- 0.6 and 3,597 +/- 1,931, respectively. Provocation tests for growth hormone secretion were applied in patients with standing heights below the third centile and/or growth velocities below the 10th centile. Sexual maturation was assessed by using the criteria of Tanner. Glucose metabolism was assessed by fasting plasma glucose and glucose tolerance test. Basal thyroid function was measured and thyrotropin-releasing hormone tolerance test was carried out. Growth retardation was found in 40 per cent of patients and growth hormone deficiency was a prominent cause of growth retardation. Gonadal dysfunction was detected in 47 per cent of patients. Hypothyroidism was observed in 16 per cent and impaired glucose metabolism in 10.8 per cent patients. The high rate of endocrine disturbances indicates the importance of regular follow-up of thalassemia major patients with regard to endocrine complications of the disease.
The effectiveness of the sequential use of deferiprone and desferrioxamine (DFO) in children with thalassaemia major was examined. Seven thalassaemic children in whom urinary iron induced by deferiprone was sufficient to maintain a negative iron balance were enrolled in the long-term trial. Deferiprone at a dose of 75 mg/kd/day in 3 divided doses was given for 4 school days a week. The group was given DFO at a dose of 40–50 mg/kg/day s.c. over 8–12 h with a battery-operated pump for 2 days at the weekend. In addition to the safety variables, they were monitored for serum ferritin levels at 2-month intervals and hepatic iron concentrations in liver tissues were determined at the beginning and the 6th month of therapy. The severity of hepatic damage was graded according to the Knodell hepatic activity index and the fibrosis was quantified. None of the patients suffered adverse effects of the therapy but a transient increase in serum ALT levels was noted. A nonsignificant decline in serum ferritin was observed (p = 0.08), a significant reduction in hepatic iron concentration was also determined (p = 0.03). The hepatic activity index in liver tissues of the patients at the 6th month of the sequential therapy significantly decreased (p = 0.03) whereas fibrosis scores did not differ significantly (p = 0.25).
Fibrin glue is a useful treatment for circumcision in patients with haemophilia; it lessens the need for factor substitution after circumcision and thus reduces the high cost of treatment.
Social and cultural integration of hemophilic boys into society is one of the most important cornerstones of modern hemophilia therapy. Circumcision, a traditional procedure, is an important ritual for Muslims and Jews and an important social problem for the hemophiliac patient and his family. The aim of this study was to evaluate the psychosocial dimension of circumcision and the opinions of parents and children. A total of 105 hemophiliac patients and parents were interviewed and surveyed. Of these, 94% of the parents of uncircumcised patients wanted circumcision for their children. Most parents saw circumcision as a mandatory procedure. Hemophilic boys (60%) and their parents (82%) have an inferiority complex because the boys are unable to be circumcised. Bleeding risk is the primary reason of anxiety (70%). The parents of all the circumcised patients were happy after circumcision. In conclusion, circumcision is an important social problem of hemophilic patients that needs to be solved.
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.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
hi@scite.ai
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.