Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0026986 (myelodysplastic syndrome)
 14,926 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Recombinant human erythropoietin (rhEPO) has now been in clinical trials for over three years. It has been shown to be nearly uniformly effective in correcting the anaemia of patients on haemodialysis or patients with progressive chronic renal failure not yet on dialysis. Preliminary results indicate that rhEPO is effective in increasing the ability of individuals to donate blood for self-use and early trials have shown the drug to increase the haematocrit in patients with rheumatoid arthritis. Trials in patients with anaemia associated with cancer or myelodysplastic syndromes are warranted. rhEPO will have a major impact as a therapeutic, particularly in patients with renal disease.
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PMID:The use of recombinant human erythropoietin in humans. 218 Jun 46

Recombinant human erythropoietin (rHuEpo) has now been in clinical trials for over four years. rHuEpo has been shown to be nearly uniformly effective in correcting the anaemia of patients on haemodialysis or patients with progressive chronic renal failure not yet on dialysis. rHuEpo has been shown to be effective in increasing the ability of individuals to donate blood for self-use and to increase the haematocrit in patients with rheumatoid arthritis. Preliminary results indicate that rHuEpo will decrease transfusion requirements of patients with the acquired immune deficiency syndrome who are anaemic. Trials in patients with anaemia associated with cancer or myelodysplastic syndromes are in early stages. rHuEpo will have a major impact as a therapeutic agent, particularly in patients with renal disease.
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PMID:The use of recombinant human erythropoietin (rHuEpo) in humans. 227 12

Recombinant DNA technology has allowed for the production of large quantities of several hematopoietic growth factors as cloned gene products. Three of these factors--recombinant human erythropoietin (r-HuEPO), granulocyte macrophage colony-stimulating factor (GM-CSF), and granulocyte colony-stimulating factor (G-CSF)--are currently undergoing clinical trials and appear to offer considerable promise for the treatment of a variety of hematopoietic abnormalities. Therapy with r-HuEPO can raise the hematocrit levels of patients with end-stage renal disease. Therapy with GM-CSF in patients undergoing marrow transplantation results in acceleration of granulocyte and platelet recovery by 1 to 2 weeks, leading to fewer infections and earlier discharge from the hospital. Other demonstrated uses of GM-CSF include treatment for aplastic anemia, myelodysplastic syndromes, chemotherapy-induced neutropenia, and neutropenia associated with the acquired immunodeficiency syndrome (AIDS). Similar beneficial effects have been reported with G-CSF. Other hematopoietic growth factors, including the interleukins (IL)-1, -3, and -6, will soon be entering clinical trials. For the first time, the availability of a large number of hematopoietic growth factors may allow physicians to regulate closely the entire hematopoietic system of their patients.
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PMID:The clinical use of hematopoietic growth factors. 247 5

The differentiation and maturation of hematopoietic progenitor cells are regulated by certain growth factors. Several of these glycoproteins have been characterized, and their amino acid sequences have been delineated. Modern DNA technology provides sufficient quantities of these hormones for testing in clinical trials. Erythropoietin (EPO) has been shown to increase the hemoglobin level and hematocrit in patients with end-stage renal disease. Granulocyte colony-stimulating factor (G-CSF) and granulocyte-macrophage CSF (GM-CSF) can increase the numbers of neutrophils and monocytes, in a dose-dependent fashion. The function of granulocytes and monocytes is also enhanced. Clinical studies of the toxicity and activity of G-CSF and GM-CSF have been conducted in patients with acquired immune deficiency syndrome, aplastic anemia, myelodysplastic syndromes, and neutropenia due to cancer and chemotherapy. In almost all patients the neutrophil count increased within 24 hours after the start of treatment. Side effects of G-CSF and GM-CSF are infrequent and usually mild. Combinations of CSFs may be even more effective.
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PMID:Clinical applications of recombinant human colony-stimulating factors. 264 25

Optimum urologic management of children with myelodysplasia includes periodic assessment of upper tract anatomy and renal growth. To investigate the perception that many children with spina bifida but without a history of intrinsic renal disease have small kidneys when compared with age-matched standard renal growth charts, 212 children with spina bifida were studied retrospectively, measuring renal length on excretory urograms. Patients with known vesicoureteric reflux of grade 2 or greater, congenital renal anomalies, hydronephrosis, renal scarring, or urinary diversion were excluded. For 95 patients aged 2 days to 19 days, 249 urograms were available for evaluation. Renal lengths were measured on the supine 5-min urogram. Mean values and standard deviations were calculated for each age group. Mean renal length for each age group fell below the mean of the standard curves devised by Hodson and Currarino, with increasing deviation from the mean with age.
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PMID:Normal renal growth in children with myelodysplasia. 348 82

In a study of 94 patients with myelodysplastic syndrome (MDS) associated vasculitis was observed in 5, which preceded hematologic diagnosis in 4. Leukocytoclastic vasculitis was observed in 5 cases being associated to lobular panniculitis in one. Three patients had refractory anemia, one sideroblastic anemia (SA) and another refractory anemia with excess blasts (RAEB). In the latter case lymphomatoid papulosis was also observed which, to date, has not been previously described in association with MDS. Another case presented seronegative polyarthritis and renal disease coinciding with vasculitis. Polyarteritis nodosa was diagnosed in a third patient in agreement with the criteria of the American College of Rheumatology, the association of which with MDS is exceptional. In the same case medullary cytogenetic study showed 46, XY,t (12;20), an abnormally which has not been described to date in cases of MDS. All the cases were treated with glucocorticoids in addition to cyclophosphamide in the patient with polyarteritis nodosa, with an improvement in the vasculitis being observed in all the patients. Two patients died, one (SA) due to pneumonia and the other (RAEB) due to subdural hematoma following transformation to acute myeloblastic leukemia. With 5% of the MDS studied presenting vasculitis, this syndrome should be included in the differential diagnosis of vasculitis observed in patients with cytopenia.
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PMID:[Vasculitis associated with a myelodysplastic syndrome: a report of 5 cases]. 779 67

Among 825 cases of de novo myelodysplastic syndromes (MDS) diagnosed over a period of 13 years in our center, 4 had clinically significant glomerulopathy. All 4 fulfilled diagnostic criteria of chronic myelomonocytic leukemia (CMML), and could be classified in the low or intermediate risk groups according to two scoring systems. Presenting symptoms of renal involvement were edema in 3 cases and acute renal failure in the remaining patient. Three patients had the nephrotic syndrome. Renal biopsy (performed in 2 cases but considered as contraindicated in the other cases) showed AL amyloidosis on one case and extracapillary glomerulonephritis in the other case. The 4 patients were treated with V16 or hydroxyurea and two had renal improvement. Only one previous case of MDS associated with glomerulopathy has been reported before and also very probably had CMML. This, and the response of renal disease to chemotherapy in 2 of our patients suggests a possible relationship between the two disorders. More systematic investigation of glomerular function, in CMML, could possibly disclose a higher incidence of cases of glomerular injury in this type of MDS.
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PMID:Glomerular injury in chronic myelomonocytic leukemia. 852 56

A consecutive series of 90 children with anorectal malformations was reviewed for urologic problems related to lower urinary tract dysfunction. Neurogenic bladder-sphincter dysfunction was seen in 22 patients (24%), all but one of whom had sacral agenesis. Vesicoureteral reflux was present in 60% of the patients with dysfunction, and 32% had reflux nephropathy. Urinary incontinence was present in 91% of the patients who had bladder-sphincter dysfunction. However, the management of bladder-sphincter dysfunction and urinary incontinence was not as straightforward as for patients with myelodysplasia because the parents were more reluctant to accept the therapeutic measures. It was particularly difficult to introduce clean intermittent catheterization (CIC), especially in older patients, because genital and urethral sensation often was undisturbed. Only if CIC had been started in the neonatal period or early infancy were there no problems with acceptance and parental compliance. The authors emphasize the importance of urodynamic testing of neonates and infants who have an anorectal malformation and associated sacral agenesis in identifying those who have neurogenic bladder-sphincter dysfunction. Consequently, patients with lower urinary tract dysfunction should receive prompt treatment, including CIC if necessary, to prevent or reduce secondary urologic morbidity, especially loss of renal function.
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PMID:Urologic problems in anorectal malformations. Part 2: functional urologic sequelae. 886 70

The purpose of this review is to give an update of the recent progress in research on erythropoietin (Epo), the hormone that regulates red blood cell production. Epo is a glycoprotein with a molecular mass of approx 30 kDa, which circulates in plasma of the human with 165 amino acids with three N-linked and one O-linked acidic oligosaccharide side chains in the molecule. Both the alpha (39% CHO) and beta (24% CHO) forms are available for clinical use, and there does not appear to be any difference in the pharmacokinetics of these two forms of Epo. Radioimmunoassays and enzyme-linked immunoabsorbant (ELISA) assays are available in a kit form. Serum levels of Epo in normal human subjects range between 1 and 27 mmu/ml or approx 5 pmol/l. It seems clear that the cells in the adult mammalian kidney which produce Epo are the interstitial cells in the peritubular capillary bed and the perivenous hepatocytes in the liver. Expression of the human Epo gene sequences that direct expression in the kidney are located 6-14 kilobases 5' to the gene; whereas the sequences that control hepatocyte-specific expression are located within 0.7 KS to the 3'-flanking region and 0.5 KS to the 5'-flanking region. The signal transduction pathways postulated to be involved in the expression of Epo are: kinases A, G and C; both a constitutive factor and a second hypoxia-inducible factor-1 (HIF-1) located in the 5' end of an hypoxia inducible enhancer region of the Epo gene; and reactive oxygen species. The primary target cell in the bone marrow acted on by Epo is the colony-forming unit erythroid (CFU-E) which has the highest number of Epo receptors. It has been postulated that Epo decreases the rate which Epo-dependent progenitor cells undergo programed cell death (apoptosis). There are two major signal transduction pathways activated by the Epo receptor: the JAK2-STAT5 pathway and the ras pathway. Both pathways involve tyrosine phosphorylation. The approved clinical uses of Epo are the anemias associated with end-stage renal disease, cancer chemotherapeutic agents, and patients with HIV infection receiving AZT. Other anemias reported to respond to Epo therapy are anemia of prematurity, rheumatoid arthritis, and myelodysplasia. Other uses of Epo under investigation are in perioperative surgery and preoperative autologous blood donation.
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PMID:Erythropoietin: physiologic and pharmacologic aspects. 940 40

Of the uncommon anemias, "common" types include the anemia of renal disease, thalassemia, myelodysplastic syndrome and the anemia of chronic disease. These conditions may be suggested by the clinical presentation, laboratory test values and peripheral blood smear, or by failure of the anemia to respond to iron supplements or nutrient replacement. The principal cause of the anemia of renal disease is a decreased production of red blood cells related to a relative deficiency of erythropoietin. When treatment is required, erythropoietin is administered, often with iron supplementation. In the anemia of chronic disease, impaired iron transport decreases red blood cell production. Treatment is predominantly directed at the underlying condition. Since iron stores are usually normal, iron administration is not beneficial. Thalassemia minor results from a congenital abnormality of hemoglobin synthesis. The disorder may masquerade as mild iron deficiency anemia, but iron therapy and transfusions are often not indicated. In the myelodysplastic syndrome, blood cell components fail to mature, and the condition may progress to acute nonlymphocytic leukemia. The rate of progression depends on the subtype of myelodysplasia, but the leukemia is usually resistant to therapy.
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PMID:'Common' uncommon anemias. 1006 9


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