Research and reports in endocrine disorders最新文献

{"title":"Simvastatin/fenofibrate combination in the treatment of dyslipidemia: current evidence","authors":"José G. Jiménez-Montero, G. Haft","doi":"10.2147/RRED.S50952","DOIUrl":"https://doi.org/10.2147/RRED.S50952","url":null,"abstract":"(unported, v3.0) License. The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. Permissions beyond the scope of the License are administered by Dove Medical Press Limited. Information on how to request permission may be found at: http://www.dovepress.com/permissions.php Research and Reports in Endocrine Disorders 2015:5 1–13 Research and Reports in Endocrine Disorders Dovepress","PeriodicalId":90317,"journal":{"name":"Research and reports in endocrine disorders","volume":"5 1","pages":"1-13"},"PeriodicalIF":0.0,"publicationDate":"2014-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/RRED.S50952","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68477390","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical potential of eliglustat tartrate in the treatment of type 1 Gaucher disease","authors":"P. Kaplan","doi":"10.2147/RRED.S36669","DOIUrl":"https://doi.org/10.2147/RRED.S36669","url":null,"abstract":"Nonneuropathic type 1 Gaucher disease is an autosomal recessive inherited disease caused by the deficiency or absence of beta glucocerebrosidase (beta glucosidase). The highest prevalence of type 1 is in Ashkenazi Jews, but it affects all ethnic groups. It manifests at any age but is seen predominantly in the first two decades. The phenotype is characterized by painless splenomegaly and secondary hypersplenism (low hemoglobin concentration and low platelet and white blood cell counts). Symptoms and signs include splenomegaly; chronic fatigue, frequent nose bleeds, prolonged bleeding, and/or bruising; hepatomegaly; bone pain, bone destruction and low bone density; and poor growth in childhood and delayed pubertal development. Current treatment with intravenous enzyme replacement has been generally successful. However, oral treatments have been developed because enzyme replacement is time-consuming and invasive, and intravenous infusions are not universally available for patients who live far from medical centers or home infusion nurses. Furthermore, it may become difficult to access veins after repeated infusions. Orally administered substrate reduction is a newer treatment approach. The aim is to limit the synthesis of the substrate, glucosylceramide. The residual intrinsic enzyme, acting alone or with recombinant enzyme, can then completely catabolize the smaller amounts of glucosylceramide that are transported into lysosomes. Eliglustat tartrate is a new specific inhibitor of glucosylceramide synthase. Phase III trials in humans have been completed. Eli- glustat tartrate has been shown to be efficacious and safe in adult humans. The results are as good or better compared with intravenous replacement with regard to reductions in spleen and liver enlargement and improvements in hemoglobin concentrations, platelet counts, and bone density, as well as decreases in biomarkers of Gaucher disease activity. Few adverse events, none of which was serious, have been reported. Eliglustat tartrate has the clinical potential to enable a larger number of patients with type 1 Gaucher disease to be treated successfully.","PeriodicalId":90317,"journal":{"name":"Research and reports in endocrine disorders","volume":"4 1","pages":"1-8"},"PeriodicalIF":0.0,"publicationDate":"2014-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/RRED.S36669","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68477398","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"New options in the treatment of Cushing’s disease: a focus on pasireotide","authors":"Anne-Gaelle Poullot, N. Chevalier","doi":"10.2147/RRED.S30972","DOIUrl":"https://doi.org/10.2147/RRED.S30972","url":null,"abstract":"Cushing's disease is caused by a corticotroph pituitary adenoma secreting adreno- corticotropin and can be fatal in the absence of adequate treatment. Transsphenoidal surgery remains the treatment of choice in almost all cases. However, remission rates are relatively low, and recurrence is usual and can be diagnosed up to decades after the initial diagnosis. Repeat surgery or radiation can be useful in these cases, although both have clear limitations with respect to efficacy and/or side effects. Hence, there is a clear unmet need for an effective medical treatment in patients with recurrent or persistent Cushing's disease. Pasireotide is a novel multireceptor-targeted somatostatin analog with a high affinity for somatostatin receptor (sstr)-1, sstr-2, sstr-3, and sstr-5. Compared with octreotide, pasireotide has an in vitro binding affinity 40-fold higher for sstr-5, which is the major receptor subtype expressed by corticotroph pituitary adenoma. Recent studies have suggested a role for this new multireceptor somatosta- tin analog in Cushing's disease. We review in this article the current data available regarding pharmacokinetics, clinical efficiency, and tolerance of pasireotide in patients with de novo, persistent, or recurrent Cushing's disease, with a special focus on the disturbances of glucose metabolism induced by such a treatment.","PeriodicalId":90317,"journal":{"name":"Research and reports in endocrine disorders","volume":"3 1","pages":"31-38"},"PeriodicalIF":0.0,"publicationDate":"2013-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/RRED.S30972","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68476627","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Visceral adiposity and cardiometabolic risks: epidemic of abdominal obesity in North America","authors":"S. Wimalawansa","doi":"10.2147/RRED.S32041","DOIUrl":"https://doi.org/10.2147/RRED.S32041","url":null,"abstract":"Correspondence: Sunil J Wimalawansa Robert Wood Johnson Medical School, University of Medicine and Dentistry of New Jersey, 1 Robert Wood Johnson Place, New Brunswick, NJ 08903, USA Tel +1 73 2235 9584 Fax +1 73 2235 8892 Email wimalawansa1@hotmail.com Abstract: Over the past 40 years, the prevalence of obesity has more than doubled in the United States. Approximately 67% of American adults older than 20 years of age are either obese or overweight. Obesity has now become a critically important issue facing more than 40% of Americans and has become a major burden on the American health care system. Today, obesity cannot be considered a simple lifestyle issue; it is a disease with major public health and economic consequences that requires serious attention by all stakeholders. Each individual has different causes and risk factors that lead to obesity and its associated complications. In addition to preventing becoming overweight, focusing on identifying the causes of obesity and then individualizing care and treatment plans to targeting weight loss, particularly intra-abdominal fat, could potentially generate huge cost savings. Excess intra-abdominal fat (visceral adiposity) is linked to excess morbidity and mortality, and positively correlates with the risks of insulin resistance, type 2 diabetes, cardiovascular diseases, certain cancers, and premature death. Therefore, overweight and obese patients should be offered healthy lifestyle changes including education about causes leading to excess weight, weight-reducing diets, physical activity regimens, and monitoring for progress. Medications and bariatric surgery are effective but are the last options and should be complementary to lifestyle and behavioral changes. The costs associated with managing obesity-related disorders and their complications are astounding; unless we intervene now, these are likely to triple over the next 2 decades. Thus, policymakers must pay serious attention to this progressive, insidious epidemic and determine the right paths for tackling obesity, which requires a paradigm shift in thinking and combined approaches. The increasing prevalence of obesity is a major health hazard in all health sectors, in both low and high income societies and countries. Thus, comprehensive programs are needed to minimize the effects of the epidemic.","PeriodicalId":90317,"journal":{"name":"Research and reports in endocrine disorders","volume":"3 1","pages":"17-30"},"PeriodicalIF":0.0,"publicationDate":"2013-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/RRED.S32041","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68476682","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Graves’ thyrotoxicosis following Hashimoto’s thyroiditis","authors":"A. Troisi, P. Novati, L. Sali, M. Colzani, G. Monti, C. Cardillo, M. Tesauro","doi":"10.2147/RRED.S38053","DOIUrl":"https://doi.org/10.2147/RRED.S38053","url":null,"abstract":"Autoimmune thyroid disease traditionally includes chronic thyroiditis, autoimmune hyperthyroidism (Graves' disease), and primary nongoitrous myxedema, and these diseases have a common syndrome-sharing pathophysiology. Here we report a rare case of simultane- ous occurrence of thyrotoxicosis linked to Graves' disease and chronic hypothyroidism due to","PeriodicalId":90317,"journal":{"name":"Research and reports in endocrine disorders","volume":"3 1","pages":"13-15"},"PeriodicalIF":0.0,"publicationDate":"2013-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/RRED.S38053","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68476990","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An update on the treatment of acromegaly","authors":"Kari L Edling, A. Heaney","doi":"10.2147/RRED.S24231","DOIUrl":"https://doi.org/10.2147/RRED.S24231","url":null,"abstract":"Correspondence: Anthony P Heaney Department of Medicine and Neurosurgery, David Geffen School of Medicine at UCLA, 200 Medical Building Suite 530, Los Angeles, CA 90095, USA Tel +1 310 825 7922 Fax +1 310 267 1899 Email aheaney@mednet.ucla.edu Abstract: Acromegaly is caused by pituitary somatotroph hypersecretion of growth hormone leading to elevated hepatic-derived and local levels of insulin-like growth factor-1. It is associated with increased morbidity and mortality due primarily to cardiovascular disease and diabetes mellitus. Normalization of growth hormone and insulin-like growth factor-1 levels has been associated with decreased morbidity from metabolic and cardiovascular effects, as well as reduced overall mortality in epidemiologic studies. Many patients experience a delay in obtaining a diagnosis, have pituitary macroadenomas at presentation, and accordingly, a significant number will not be cured by tumor surgical resection alone. Adjunctive radiation therapy cannot always offer biochemical and clinical disease control and carries a 40% risk of partial or total pituitary failure in the medium term. Several monotherapies or combination medical therapies are currently available for both primary and adjuvant acromegaly treatment, and include long-acting somatostatin analogs, the growth hormone receptor antagonist pegvisomant, and dopamine agonists. Next generation somatostatin analogs and new drug delivery methods of existing agents are in ongoing clinical studies. This paper will review current and novel therapies under development for acromegaly.","PeriodicalId":90317,"journal":{"name":"Research and reports in endocrine disorders","volume":"3 1","pages":"1-11"},"PeriodicalIF":0.0,"publicationDate":"2013-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/RRED.S24231","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68476480","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sustained biochemical control in patients with acromegaly treated with lanreotide depot 120 mg administered every 4 weeks, or an extended dosing interval of 6 or 8 weeks: a pharmacokinetic approach","authors":"E. Gomez-Panzani, Stephen Chang, J. Ramis, M. M. Landolfi, B. Bakker","doi":"10.2147/RRED.S38149","DOIUrl":"https://doi.org/10.2147/RRED.S38149","url":null,"abstract":"Correspondence: Edda Gomez-Panzani 106 Allen Road, 3rd Floor, Basking Ridge, NJ 07920, USA Tel +1 650 238 1627 Fax +1 650 243 5153 Email edda.gomez-panzani@ipsen.com Objective: Lanreotide depot is a long-acting somatostatin receptor ligand injected deep subcutaneously every 4 weeks for the treatment of acromegaly. The aim of the presented studies was to establish whether lanreotide depot, administered to patients with acromegaly at an extended dosing interval of 6 or 8 weeks, is effective in maintaining appropriate serum growth hormone (GH) and insulin-like growth factor-1 (IGF-1) levels, with acceptable tolerability. Methods: Two studies were conducted. Study B1 compared lanreotide depot 120 mg (every 4, 6, or 8 weeks) with lanreotide microparticle formulation 30 mg (every 7, 10, or 14 days) in 98 patients who had a GH level of #2.5 ng/mL and normalized IGF-1. Study B2 evaluated lanreotide depot 120 mg administered to 64 patients every 8 weeks, after which the dosing interval was adjusted based on GH levels. Results: Mean lanreotide trough serum concentrations at steady state for all dosing intervals were .1.13 ng/mL, shown to achieve a GH level of #2.5 ng/mL. In Study B1, following treatment with lanreotide depot given every 6 or 8 weeks, 87.5% and 93.9% of patients, respectively, had normalized GH, whereas 83.3% and 88.5% of patients, respectively, had both normalized GH and IGF-1. In Study B2, 88.9% had normalized GH and 42.9% of patients had normalized GH and IGF-1 following lanreotide depot every 8 weeks. Gastrointestinal disorders, generally mild/moderate in severity, were the most common adverse events. Conclusion: In the studies presented, lanreotide depot 120 mg every 4, 6, or 8 weeks provided effective hormonal control with acceptable safety. An extended dosing interval is a feasible approach for patients adequately controlled with lanreotide depot 60 or 90 mg every 4 weeks.","PeriodicalId":90317,"journal":{"name":"Research and reports in endocrine disorders","volume":"2 1","pages":"79-84"},"PeriodicalIF":0.0,"publicationDate":"2012-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/RRED.S38149","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68477300","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Current and emerging treatments and surgical interventions for Morquio A syndrome: a review.","authors":"Shunji Tomatsu, William G Mackenzie, Mary C Theroux, Robert W Mason, Mihir M Thacker, Thomas H Shaffer, Adriana M Montaño, Daniel Rowan, William Sly, Carlos J Alméciga-Díaz, Luis A Barrera, Yasutsugu Chinen, Eriko Yasuda, Kristen Ruhnke, Yasuyuki Suzuki, Tadao Orii","doi":"10.2147/RRED.S37278","DOIUrl":"10.2147/RRED.S37278","url":null,"abstract":"<p><p>Patients with mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome) have accumulation of the glycosaminoglycans, keratan sulfate, and chondroitin-6-sulfate, in bone and cartilage, causing systemic spondyloepiphyseal dysplasia. Features include lumbar gibbus, pectus carinatum, faring of the rib cage, marked short stature, cervical instability and stenosis, kyphoscoliosis, genu valgum, and laxity of joints. Generally, MPS IVA patients are wheelchair-bound as teenagers and do not survive beyond the second or third decade of life as a result of severe bone dysplasia, causing restrictive lung disease and airway narrowing, increasing potential for pneumonia and apnea; stenosis and instability of the upper cervical region; high risk during anesthesia administration due to narrowed airway as well as thoracoabdominal dysfunction; and surgical complications. Patients often need multiple surgical procedures, including cervical decompression and fusion, hip reconstruction and replacement, and femoral or tibial osteotomy, throughout their lifetime. Current measures to intervene in disease progression are largely palliative, and improved therapies are urgently needed. A clinical trial for enzyme replacement therapy (ERT) and an investigational trial for hematopoietic stem cell transplantation (HSCT) are underway. Whether sufficient enzyme will be delivered effectively to bone, especially cartilage (avascular region) to prevent the devastating skeletal dysplasias remains unclear. This review provides an overview of historical aspects of studies on MPS IVA, including clinical manifestations and pathogenesis of MPS IVA, orthopedic surgical interventions, and anesthetic care. It also describes perspectives on potential ERT, HSCT, and gene therapy.</p>","PeriodicalId":90317,"journal":{"name":"Research and reports in endocrine disorders","volume":"2012 2","pages":"65-77"},"PeriodicalIF":0.0,"publicationDate":"2012-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4020877/pdf/nihms432831.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"32352379","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Emerging treatment options for the mucopolysaccharidoses","authors":"R. Giugliani, A. Federhen, André Anjos Silva, C. Bittar, C. Souza, C. Netto, F. Q. Mayer, G. Baldo, U. Matte","doi":"10.2147/RRED.S24769","DOIUrl":"https://doi.org/10.2147/RRED.S24769","url":null,"abstract":"Roberto Giugliani1–6 Andressa Federhen1,4 Andre Anjos Silva1,5 Camila Matzenbacher Bittar1,5 Carolina Fischinger Moura de Souza1 Cristina Brinckmann Oliveira Netto1 Fabiana Quoos Mayer2,7 Guilherme Baldo1,2,8 Ursula Matte1–5 1Medical Genetics Service, 2Gene Therapy Center, Hospital de Clinicas (HCPA), Porto Alegre, RS, Brazil; 3Department of Genetics, 4Postgraduate Program in Child and Adolescent Health, 5Postgraduate Program in Genetics and Molecular Biology, Universidade Federal do Rio Grande do Sul (UFRGS), Porto Alegre, RS, Brazil; 6National Institute of Population Medical Genetics (INAGEMP), Porto Alegre, RS, Brazil; 7Fundacao Estadual de Pesquisa Agropecuaria (FEPAGRO), Eldorado do Sul, RS, Brazil; 8Department of Biophysics, Universidade Federal do Rio Grande do Sul (UFRGS), Porto Alegre, RS, Brazil","PeriodicalId":90317,"journal":{"name":"Research and reports in endocrine disorders","volume":"2 1","pages":"53-64"},"PeriodicalIF":0.0,"publicationDate":"2012-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/RRED.S24769","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68476820","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Optimizing glycemic control: clinical utility of exenatide prolonged release injection","authors":"G. Derosa, P. Maffioli","doi":"10.2147/RRED.S24237","DOIUrl":"https://doi.org/10.2147/RRED.S24237","url":null,"abstract":"Despite the large variety of antidiabetic drugs currently available, reaching an adequate glycemic control is still difficult. Recently, a new exenatide long acting release (LAR) formulation, which can be administered once a week, has been released. We conducted a review analyzing the clinical utility of this new formulation and its place in antidiabetic therapy, and included the most important studies about exenatide LAR in the latest 10 years. A systematic search strategy was developed to identify randomized controlled trials in both MEDLINE and the Cochrane Register of Controlled Trials. The terms \"exenatide,\" \"exenatide long active release,\" \"GLP-1 agonists,\" \"incretins,\" and \"glycemic control\" were incorporated into an electronic search strategy that included the Dickersin filter for randomized controlled trials. We concluded that exenatide LAR can be a valid option for the treatment of type 2 diabetes mellitus because it showed to be effective in reducing HbA 1c , and because of its pleiotropic effects, such as the reduction of blood pressure, the improvement of the patient's lipid profile, and the positive effects on body weight and β-cell function. Moreover, exenatide LAR has demonstrated a favorable cost/effectiveness ratio, and its once weekly administration may help to increase patient compliance.","PeriodicalId":90317,"journal":{"name":"Research and reports in endocrine disorders","volume":"2 1","pages":"41-51"},"PeriodicalIF":0.0,"publicationDate":"2012-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/RRED.S24237","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68476612","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}