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)

Gonadotropin (Gn) replacement therapy using HCG plus HU-FSH was administered to 24 patients affected by beta-thalassaemia major with hypogonadotropic hypogonadism aged 18-40 years (25.2 +/- 5.4 yr, m +/- SEM). The age range at the start of treatment was 14.5-24.5 years (16.7 +/- 2.6 yr); the mean duration of Gn treatment was 8.6 +/- 3.9 years (range 1-15.2 yr). Gn therapy was begun with HCG alone, the dosage being initially 500 IU twice a week and then increased to a maximum of 3000 IU twice a week, according to the individual serum testosterone levels obtained. HU-FSH (75 IU twice a week) was added to initiate spermatogenesis in all cases when the HCG-induced testosterone serum levels normalized. The duration of HU-FSH treatment ranged from 1-2 years and then therapy was continued with HCG alone. In nine patients Gn therapy was discontinued after 6-14 years and was replaced by testosterone depot therapy, 75-100 mg i.m. twice a month, for a period ranging from 1-1.5 years. Using Gn therapy, the testosterone levels normalized. The compliant patients obtained good virilization and normal sexual function; testicular volume increased within the normal adult range and spermatogenesis was achieved. When Gn therapy was replaced by testosterone-depot therapy, a marked decrease in testicular volume and sperm count was observed, but the patients complied better and showed a slight increase in coarse hair. In conclusion gonadotropins are an effective replacement therapy for male hypogonadism in thalassaemic patients. If we consider the advantages and disadvantages of this therapy, the former seem to outweigh the latter. Finally, it should be emphasized that physicians caring for these patients must foster compliance during frequent check-ups and examinations.
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PMID:Hormonal replacement therapy with HCG and HU-FSH in thalassaemic patients affected by hypogonadotropic hypogonadism. 1009 Nov 62

beta-Thalassemia major is associated with a high prevalence of hypogonadotropic hypogonadism affecting adolescents and young men with this disease. The pharmacokinetics of Androderm, a non-scrotal permeation-enhanced testosterone transdermal system, was previously studied in this population using three application regimens designed to mimic the nocturnal secretion and circadian patterns of testosterone production characteristics of puberty and young adulthood. In regimen I, designed for prepubertal 14 to 16 year-olds, a single Androderm patch (2.5 mg/day nominal delivery rate) is applied at night and removed 12 hours later in the morning. In regimen II, designed for partially virilized 17 to 19 year-olds, a single Androderm patch is applied nightly for 24 hours. In regimen III, intended for virilized men aged 20 years and older, two Androderm patches (total dose of 5 mg/day) are applied nightly for 24 hours. This report presents the results of a 12-month open label study using these three Androderm regimens to treat nine hypogonadal males with beta-thalassemia (ages 16.8 to 31.8 yr). Our data show that Androderm produced physiologically appropriate testosterone levels, lowered SHBG levels, promoted growth and virilization, increased bone mineral density, and was generally well tolerated in this population of hypogonadal adolescents and young men with beta-thalassemia.
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PMID:Clinical experience using the Androderm testosterone transdermal system in hypogonadal adolescents and young men with beta-thalassemia major. 1009 Nov 63