Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0002874 (aplastic anemia)
 5,905 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

In 1967 the first patient in Northern Ireland commenced growth hormone treatment for short stature. By the end of December 1988 a total of 89 patients had been treated. Thirty-two had idiopathic isolated growth hormone deficiency, an incidence of 1.5 new cases per year (in a population of 1.5 million with approximately 30,000 births per year). Since 1967 the mean age at starting treatment has fallen from 18 years to 10 years and the height standard deviation score has fallen from -4.7 +/- 0.6 to -3.4 +/- 0.3. The group with classical growth hormone deficiency (maximum GH less than 7 mU/l during insulin-induced hypoglycaemia) had a greater increase in height velocity over the first year of treatment, 3.8 +/- 0.4 cm, than those with a partial deficiency (maximum growth hormone 7.1 - 20 mU/l), 1.9 +/- 0.4 cm. All pre-pubertal children responded with a rise in the height velocity standard deviation score from -1.8 +/- 0.3 before treatment to +3.5 +/- 0.4 over the first year of treatment. 58% of the adult males and 25% of adult females have attained an adult height within the normal range (3rd centile or above). There have been three deaths, one each from Fanconi's aplastic anaemia which predated growth hormone treatment, an accidental fire injury and a relapsing craniopharyngioma. There have been no deaths from Creutzfeldt-Jakob disease. Growth hormone therapy is safe and effective, but continues to be commenced late in terms both of age and height standard deviation score.
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PMID:The Northern Ireland experience of growth hormone therapy for short stature. 260 65

The characteristic of transmissible spongiform encephalopathies (TSE) is an accumulation of partially protease resistant (PrP(res)) abnormal prion protein (PrP(sc)). This pathological prion protein is very resistant to conventional inactivation methods. The risk of transmission of TSE, such as Creutzfeldt-Jakob disease (CJD), by biopharmaceutical products prepared from human cells must be taken into account. The nanofiltration process has been proved to be effective in removing viruses and scrapie agent. The major advantages of this technique are flexibility and efficacy in removing infectious particles without altering biopharmaceutical characteristics and properties. This study focused on the removal of human PrP(sc) by means of a nanofiltration method after spiking a Lymphoglobuline solution with a CJD brain homogenate. Lymphoglobuline equine anti-human thymocyte immunoglobulin is a selective immunosuppressive agent acting mainly on human T lymphocytes. The therapeutic indications are: immunosuppression for transplantation: prevention and treatment of graft rejection; treatment of aplastic anemia. In our study, CJD homogenate was spiked at three different dilutions (low, moderate and high) in the Lymphoglobuline product. The nanofiltration process was performed on each sample. Using the western blot technique, the PrP(res) signal detected in nanofiltrates was compared to that obtained with a reference scale (dilution series of CJD brain homogenate in Lymphoglobuline detected by western blot and elaborated on 3.3 log). After nanofiltration, the PrP(res) western blot signal was detected with a significant reduction in the less dilute sample, whereas the signal was undetectable in the two other samples. These are the first data in CJD demonstrating a clearance between 1.6 and 3.3 log with a Lymphoglobuline recovery of over 93%. The nanofiltration process confirms its relative efficacy in removing human CJD PrP(sc).
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PMID:CJD PrPsc removal by nanofiltration process: application to a therapeutic immunoglobulin solution (Lymphoglobuline). 1649 Mar 61