发布求助
文献互助 智能选刊 最新文献

Kliničeskaâ farmakologiâ i terapiâ最新文献

筛选
英文 中文
The value of registries in the study of rare diseases: Fabry Outcome Survey 登记在罕见病研究中的价值:法布里结果调查
Kliničeskaâ farmakologiâ i terapiâ Pub Date : 2023-09-02 DOI: 10.32756/0869-5490-2023-3-56-62
E. Tao, A. Moiseev, N. Bulanov, S. Moiseev
{"title":"The value of registries in the study of rare diseases: Fabry Outcome Survey","authors":"E. Tao, A. Moiseev, N. Bulanov, S. Moiseev","doi":"10.32756/0869-5490-2023-3-56-62","DOIUrl":"https://doi.org/10.32756/0869-5490-2023-3-56-62","url":null,"abstract":"Randomized controlled clinical trials are frequently hardly feasible in patients with orphan disorders given the limited number of potential participants and variablity of clinical manifestations and the course of disease. Registry data, particularly for rare diseases, provide additional information regarding long-term treatment effectiveness and safety. The year 2021 marked the 20th anniversary of the Fabry Outcome Survey (FOS), an international, multicenter, observational registry (NCT03289065). The aim of FOS was to study Fabry disease, an X-linked lysosomal storage disorder caused by mutations in the galactosidase alpha (GLA) gene that result in reduced or absent a-galactosidase A activity and sphyngolipids accumulation in the various tissues. As of January 2021, 4484 patients with Fabry disease have been enrolled in FOS from 144 centers across 26 countries. Analyses of FOS data has confirmed the efficacy and safety of enzyme replacement therapy with agalsidase alfa and its favorable impact on the course of Fabry disease and mortality. FOS data have also shown the advantages of prompt treatment that was more effective in prevention of cardiovascular and renal events regardless of type of disease.","PeriodicalId":474663,"journal":{"name":"Kliničeskaâ farmakologiâ i terapiâ","volume":"77 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134971243","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}
引用次数: 0
Late-onset Pompe disease in adults: clinical manifestations, diagnosis and treatment 成人迟发性庞贝病:临床表现、诊断与治疗
Kliničeskaâ farmakologiâ i terapiâ Pub Date : 2023-09-02 DOI: 10.32756/0869-5490-2023-3-63-71
Z. Muruzheva, V. Larionova, P. Novikov, S. Moiseev
{"title":"Late-onset Pompe disease in adults: clinical manifestations, diagnosis and treatment","authors":"Z. Muruzheva, V. Larionova, P. Novikov, S. Moiseev","doi":"10.32756/0869-5490-2023-3-63-71","DOIUrl":"https://doi.org/10.32756/0869-5490-2023-3-63-71","url":null,"abstract":"Pompe disease is a rare inherited lisosomal storage disease characterized by accumulation of glycogen in muscular cells due to a deficient activity of acid a-glucosidase (GAA), an enzyme which degrades lysosomal glycogen. Late-onset Pompe disease can have onset at any age and sometimes mani fests in the elderly. It presents with a predominant proximal muscle weakness (limb girdle phenotype), moderately elevated serum creatine kinase and other muscle enzymes and respiratory dysfunction mainly due to a diaphragmatic weakness. Diagnosis requires low GAA activity in dried blood spots and genetic testing. The authors present the case of late-onset Pompe disease diagnosed in 45-year old male patient and discuss the efficacy of enzyme replacement therapy with recombinant GAA.","PeriodicalId":474663,"journal":{"name":"Kliničeskaâ farmakologiâ i terapiâ","volume":"341 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134971245","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}
引用次数: 0
Eculizumab for pregnancy associated atypical hemolytic uremic syndrome: a retrospective study in 85 patients Eculizumab用于妊娠相关非典型溶血性尿毒症综合征:85例患者的回顾性研究
Kliničeskaâ farmakologiâ i terapiâ Pub Date : 2023-09-02 DOI: 10.32756/0869-5490-2023-3-36-43
Y.V. Korotchaeva, N.L. Kozlovskaya, E.M. Shifman, S. Moiseev
{"title":"Eculizumab for pregnancy associated atypical hemolytic uremic syndrome: a retrospective study in 85 patients","authors":"Y.V. Korotchaeva, N.L. Kozlovskaya, E.M. Shifman, S. Moiseev","doi":"10.32756/0869-5490-2023-3-36-43","DOIUrl":"https://doi.org/10.32756/0869-5490-2023-3-36-43","url":null,"abstract":"To evaluate the efficacy of prompt and delayed complement inhibition therapy with eculizumab in patients with pregnancy associated atypical hemolytic uremic syndrome (aHUS).","PeriodicalId":474663,"journal":{"name":"Kliničeskaâ farmakologiâ i terapiâ","volume":"15 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134971249","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}
引用次数: 0
Treatment of asthenic syndrome in young and middleaged patients with subjective cognitive impairment: observational non-interventional program 中青年主观认知障碍患者衰弱综合征的治疗:观察性非介入方案
Kliničeskaâ farmakologiâ i terapiâ Pub Date : 2023-09-02 DOI: 10.32756/0869-5490-2023-3-24-29
Ya.D. Belousova, V.V. Rafalsky, A.A. Tsapkova, A.M. Tinterova, O.V. Pilyugin, A.A. Badarin, Yu.A. Filimonkina
{"title":"Treatment of asthenic syndrome in young and middleaged patients with subjective cognitive impairment: observational non-interventional program","authors":"Ya.D. Belousova, V.V. Rafalsky, A.A. Tsapkova, A.M. Tinterova, O.V. Pilyugin, A.A. Badarin, Yu.A. Filimonkina","doi":"10.32756/0869-5490-2023-3-24-29","DOIUrl":"https://doi.org/10.32756/0869-5490-2023-3-24-29","url":null,"abstract":"To evaluate the efficacy and safety of Prospekta in the treatment of asthenic syndrome in young and middle-aged patients with subjective cognitive impairment.","PeriodicalId":474663,"journal":{"name":"Kliničeskaâ farmakologiâ i terapiâ","volume":"23 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134971246","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}
引用次数: 0
Prevalence and risk factors of chronic kidney disease in Takayasu arteritis 高须动脉炎患者慢性肾脏疾病的患病率及危险因素
Kliničeskaâ farmakologiâ i terapiâ Pub Date : 2023-09-02 DOI: 10.32756/0869-5490-2023-3-19-23
N. Bulanov, V. Logina, A.A. Efimova, N. Vlasova, K. Makarova, A. Skvortsov, A. Suvorov, P. Novikov, S. Moiseev
{"title":"Prevalence and risk factors of chronic kidney disease in Takayasu arteritis","authors":"N. Bulanov, V. Logina, A.A. Efimova, N. Vlasova, K. Makarova, A. Skvortsov, A. Suvorov, P. Novikov, S. Moiseev","doi":"10.32756/0869-5490-2023-3-19-23","DOIUrl":"https://doi.org/10.32756/0869-5490-2023-3-19-23","url":null,"abstract":"Takayasu arteritis (TA) is a rare systemic vasculitis affecting aorta and its branches. Glomerulonephritis and tubulointerstitial nephritis are not typical for TA. However progressive kidney dysfunction can result from renal artery involvement or refractory hypertension.","PeriodicalId":474663,"journal":{"name":"Kliničeskaâ farmakologiâ i terapiâ","volume":"163 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134971244","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}
引用次数: 0
Rare inherited diseases with kidney involvement: approaches to diagnosis and treatment 罕见遗传性疾病伴肾脏受累:诊断和治疗方法
Kliničeskaâ farmakologiâ i terapiâ Pub Date : 2023-09-02 DOI: 10.32756/0869-5490-2023-3-6-18
S. Moiseev, N. Chebotareva, N. Bulanov, E.M. Shilov
{"title":"Rare inherited diseases with kidney involvement: approaches to diagnosis and treatment","authors":"S. Moiseev, N. Chebotareva, N. Bulanov, E.M. Shilov","doi":"10.32756/0869-5490-2023-3-6-18","DOIUrl":"https://doi.org/10.32756/0869-5490-2023-3-6-18","url":null,"abstract":"Many rare inherited disorders can be associated with the various types of kidney involvement, including glomerular disease, tubulopathies, congenital anomalies of the kidneys and urinary tract, urolithiasis, multiple cysts, malignant and benign tumors. Hereditary nephropathy should be always considered in children, adolescents and young patients with the kdineys or urinary tract disorders and/or patients with positive family anamnesis although certain genetic diseases can manifest in adult or even in the elderly whereas proband family members frequently show no signs of the disease. Extrarenal manifestations can be a valuable clue for diagnosis of various genetic disorders, e.g. neurosensory deafness in Alport syndrome, cornea verticillata and angiokeratoma in Fabry disease, photofobia in nephropathic cystinosis. Genetic tests are essential for verification of monogenic inherited diseases. In the nearest future, whole-exome or genome sequencing at constantly decreasing cost may replace targeted sequencing of the known causal gene(s) or gene panels, particularly when various inherited disorders present with the similar clinical phenotypes.","PeriodicalId":474663,"journal":{"name":"Kliničeskaâ farmakologiâ i terapiâ","volume":"17 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134971247","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}
引用次数: 0
Comparison of classification criteria for giant cell arteritis 巨细胞动脉炎分类标准的比较
Kliničeskaâ farmakologiâ i terapiâ Pub Date : 2023-09-02 DOI: 10.32756/0869-5490-2023-3-44-49
E. Filatova, N. Bulanov, O.O. Borodin, A.D. Meshkov, I.O. Smitienko, E. Chachilo, A. Nartov, A. Suvorov, A. Filatova, A.V. Naumov, L.V. Iwanitsky, P. Novikov, S. Moiseev
{"title":"Comparison of classification criteria for giant cell arteritis","authors":"E. Filatova, N. Bulanov, O.O. Borodin, A.D. Meshkov, I.O. Smitienko, E. Chachilo, A. Nartov, A. Suvorov, A. Filatova, A.V. Naumov, L.V. Iwanitsky, P. Novikov, S. Moiseev","doi":"10.32756/0869-5490-2023-3-44-49","DOIUrl":"https://doi.org/10.32756/0869-5490-2023-3-44-49","url":null,"abstract":"To compare the diagnostic value of classification criteria for giant cell arteritis (GCA).","PeriodicalId":474663,"journal":{"name":"Kliničeskaâ farmakologiâ i terapiâ","volume":"57 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134971248","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}
引用次数: 0
Pharmacokinetics and bioequivalence study of Duonica® Duonica®的药代动力学及生物等效性研究
Kliničeskaâ farmakologiâ i terapiâ Pub Date : 2023-09-02 DOI: 10.32756/0869-5490-2023-3-50-55
A.S. Goncharov, V. Drobot, A. Globenko, I.S. Goncharov, K.A. Muratov, A. Sazonov, D. Guranda, A. Kapashin, O. Kovchan, A.I. Bashkatova, M. Pasko
{"title":"Pharmacokinetics and bioequivalence study of Duonica®","authors":"A.S. Goncharov, V. Drobot, A. Globenko, I.S. Goncharov, K.A. Muratov, A. Sazonov, D. Guranda, A. Kapashin, O. Kovchan, A.I. Bashkatova, M. Pasko","doi":"10.32756/0869-5490-2023-3-50-55","DOIUrl":"https://doi.org/10.32756/0869-5490-2023-3-50-55","url":null,"abstract":"To evaluate the pharmacokinetics, safety and bioequivalence of the test drug product Duonica® (INN: doxylamine + pyridoxine), enteric-coated tablets, 10 mg + 10 mg (Valenta Pharm JSC, Russia), and the reference drug product Diclectin®, delayed release tablets, 10 mg + 10 mg (Duchesnay Inc., Canada).","PeriodicalId":474663,"journal":{"name":"Kliničeskaâ farmakologiâ i terapiâ","volume":"68 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134971241","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}
引用次数: 0
Rituximab for treatment of endocrine ophthalmopathy 利妥昔单抗治疗内分泌性眼病
Kliničeskaâ farmakologiâ i terapiâ Pub Date : 2023-09-02 DOI: 10.32756/0869-5490-2023-3-72-80
M. Khachatrian, K. Popova, K. Nabiev, D. Gilmanova, D. Fedoryakina, A. Pautova, J. Asharapova, E. Kazak, A. Ponomarenko, P. Magomedova, A. Novikova, S. Rodionova, E. Stepanov
{"title":"Rituximab for treatment of endocrine ophthalmopathy","authors":"M. Khachatrian, K. Popova, K. Nabiev, D. Gilmanova, D. Fedoryakina, A. Pautova, J. Asharapova, E. Kazak, A. Ponomarenko, P. Magomedova, A. Novikova, S. Rodionova, E. Stepanov","doi":"10.32756/0869-5490-2023-3-72-80","DOIUrl":"https://doi.org/10.32756/0869-5490-2023-3-72-80","url":null,"abstract":"Endocrine ophthalmopathy (EOP) is a vision-threatening and disfiguring extrathyroid manifestation of Graves' disease, which often impairs the quality of life of patients and causes severe social and psychological consequences. Currently, intravenous glucocorticosteroids are the first line treatment for EOP. However, their effectiveness in these patients is frequently unsatisfactory, whereas the recurrence rate of EOP is high. Rituximab (RТX) is a chimeric monoclonal antibody directed against CD20, a surface antigen present on B cells. Numerous clinical studies of RТX in patients with EOP have shown promising results. RТX is currently considered an acceptable second-line option in patients who do not respond to previous treatments or present with relapse of the disease. This review summarizes the available data on the efficacy and safety of RTX in EOP patients, including the results of two randomized controlled trials. The potential benefits as well as limitations of RТX therapy are discussed.","PeriodicalId":474663,"journal":{"name":"Kliničeskaâ farmakologiâ i terapiâ","volume":"14 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134971242","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}
引用次数: 0
Characteristics of patients with atrial fibrillation and chronic heart failure treated with direct oral anticoagulants: single-center registry 直接口服抗凝剂治疗心房颤动和慢性心力衰竭患者的特点:单中心登记
Kliničeskaâ farmakologiâ i terapiâ Pub Date : 2023-09-02 DOI: 10.32756/0869-5490-2023-3-30-35
Y. Stavtseva, M. Davletova, S. Galochkin, Zh.D. Kobalava
{"title":"Characteristics of patients with atrial fibrillation and chronic heart failure treated with direct oral anticoagulants: single-center registry","authors":"Y. Stavtseva, M. Davletova, S. Galochkin, Zh.D. Kobalava","doi":"10.32756/0869-5490-2023-3-30-35","DOIUrl":"https://doi.org/10.32756/0869-5490-2023-3-30-35","url":null,"abstract":"To evaluatie the clinical and demographic characteristics, comorbidities, risks of thromboembolic events and bleeding, physicians preferences and dosing errors for direct oral anticoagulants (DOAC) in patients with atrial fibrillation (AF) and chronic heart failure (CHF).","PeriodicalId":474663,"journal":{"name":"Kliničeskaâ farmakologiâ i terapiâ","volume":"18 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134971240","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}
引用次数: 0
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整，为了账户安全，请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
请完成安全验证×
相关产品
×
本文献相关产品
联系我们：info@booksci.cn Book学术提供免费学术资源搜索服务，方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号：481959085
Book学术官方微信