V. M. Kozlova, Ekaterina E. Zelenova, T. T. Valiev, V. V. Semenova, T. V. Nasedkina, S. Mikhailova
Hematooncological diseases head the list in the structure of malignant neoplasms of childhood. Somatic mutations in tumor clone cells have been well studied, included in modern classifications, and are used to stratify patients into prognostic risk groups and select a therapy program. At the same time, more than 50 hereditary syndromes associated with the development of hemoblastoses have been described. Some of them (Down’s syndrome, Klinefelter’s syndrome, microdeletion syndromes et al.) are caused by chromosomal pathology, while others describe alterations of one or more genes with different types of inheritance and age of manifestation of hematooncological diseases. Genes of predisposition to hematooncological diseases are involved in the processes of DNA repair, regulation of the cell cycle, immune response and bone marrow function. This article presents current data on genetic syndromes associated with the development of hemoblastosis with a description of their own clinical observations.
在儿童恶性肿瘤结构中，血液肿瘤疾病居首位。肿瘤克隆细胞中的体细胞突变已被充分研究，并被纳入现代分类中，用于将患者分为预后风险组和选择治疗方案。与此同时，50 多种与血母细胞瘤发病相关的遗传综合征已被描述。其中一些（唐氏综合征、克雷菲尔特综合征、微缺失综合征等）是由染色体病变引起的，而另一些则描述了一个或多个基因的改变，其遗传类型和血液肿瘤疾病的表现年龄各不相同。易患血液肿瘤疾病的基因涉及 DNA 修复过程、细胞周期调节、免疫反应和骨髓功能。本文介绍了与血细胞增多症发病有关的遗传综合征的最新数据，并描述了自己的临床观察结果。
{"title":"Hereditary syndromes in pediatric hematooncology","authors":"V. M. Kozlova, Ekaterina E. Zelenova, T. T. Valiev, V. V. Semenova, T. V. Nasedkina, S. Mikhailova","doi":"10.15690/pf.v20i6.2665","DOIUrl":"https://doi.org/10.15690/pf.v20i6.2665","url":null,"abstract":"Hematooncological diseases head the list in the structure of malignant neoplasms of childhood. Somatic mutations in tumor clone cells have been well studied, included in modern classifications, and are used to stratify patients into prognostic risk groups and select a therapy program. At the same time, more than 50 hereditary syndromes associated with the development of hemoblastoses have been described. Some of them (Down’s syndrome, Klinefelter’s syndrome, microdeletion syndromes et al.) are caused by chromosomal pathology, while others describe alterations of one or more genes with different types of inheritance and age of manifestation of hematooncological diseases. Genes of predisposition to hematooncological diseases are involved in the processes of DNA repair, regulation of the cell cycle, immune response and bone marrow function. This article presents current data on genetic syndromes associated with the development of hemoblastosis with a description of their own clinical observations.","PeriodicalId":19997,"journal":{"name":"Pediatric pharmacology","volume":"11 6","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-01-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139630759","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":"The Nobel Prize for Medicine was Awarded to Scientists from Hungary and The USA for the Study of RNA","authors":"A. Editorial","doi":"10.15690/pf.v20i5.2630","DOIUrl":"https://doi.org/10.15690/pf.v20i5.2630","url":null,"abstract":"<jats:p>.</jats:p>","PeriodicalId":19997,"journal":{"name":"Pediatric pharmacology","volume":"223 5","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139261048","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":"Victories of Russian Pediatricians in the International Arena","authors":"A. Editorial","doi":"10.15690/pf.v20i5.2637","DOIUrl":"https://doi.org/10.15690/pf.v20i5.2637","url":null,"abstract":"<jats:p>.</jats:p>","PeriodicalId":19997,"journal":{"name":"Pediatric pharmacology","volume":"143 4","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139261359","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}
A. A. Baranov, L. S. Namazova-Baranova, N. I. Il'ina, A. Kubanov, Elena R. Araviyskaya, N. Astafieva, Vitaly Bazaev, E. Borzova, Elena V. Vishneva, Yulia A. Gallyamova, I. Danilycheva, O. Elisyutina, Ludmila F. Znamenskaya, V. Kalugina, A. Karamova, Yulia G. Levina, R. Y. Meshkova, O. Y. Olisova, G. Novik, A. V. Samtsov, L. Selimzyanova, Evgeniy V. Sokolovsky, E. Fedenko, O. Fedorova, D. Fomina, V. Khayrutdinov, V. Chikin, A. Shulzhenko
The Union of Pediatricians of Russia together with the Russian Association of Allergologists and Clinical Immunologists and the Russian Society of Dermatovenerologists and Cosmetologists have developed new clinical guidelines for the urticaria in adults and children. Urticaria is a common disease; its various clinical variants are diagnosed in 15–25% of people in the global population, and a quarter of all cases belongs to chronic urticaria. The prevalence of acute urticaria is 20%, and 2.1–6.7% in child population, whereas acute urticaria is more common in children than in adults. The prevalence of chronic urticaria in adults in the general population is 0.7 and 1.4%, and 1.1% in children under 15 years of age, according to the systematic review and meta-analysis, respectively. This article covers features of epidemiology, etiology, and pathogenesis of the disease with particular focus on differential diagnostic search. Guidelines on treatment and step-by-step therapy scheme (both based on principles of evidencebased medicine) for pediatric patients were presented. Clarification on the analysis of the therapy efficacy and the degree of disease activity was given.
俄罗斯儿科医生联盟（Union of Pediatricians of Russia）与俄罗斯过敏学家和临床免疫学家协会（Russian Association of Allergologists and Clinical Immunologists）以及俄罗斯皮肤病学家和美容学家协会（Russian Society of Dermatovenerologists and Cosmetologists）共同制定了新的成人和儿童荨麻疹临床指南。荨麻疹是一种常见疾病；全球有 15-25% 的人被诊断出患有荨麻疹的各种临床变异，其中四分之一的病例属于慢性荨麻疹。急性荨麻疹的发病率为 20%，在儿童中的发病率为 2.1-6.7%，而急性荨麻疹在儿童中的发病率高于成人。根据系统综述和荟萃分析，成人慢性荨麻疹的发病率在普通人群中分别为 0.7% 和 1.4%，在 15 岁以下儿童中为 1.1%。本文介绍了该病的流行病学、病因学和发病机理，尤其侧重于鉴别诊断搜索。介绍了儿科患者的治疗指南和分步治疗方案（均基于循证医学原则）。对疗效分析和疾病活动程度进行了说明。
{"title":"Modern Approaches to the Management of Patients with Urticaria","authors":"A. A. Baranov, L. S. Namazova-Baranova, N. I. Il'ina, A. Kubanov, Elena R. Araviyskaya, N. Astafieva, Vitaly Bazaev, E. Borzova, Elena V. Vishneva, Yulia A. Gallyamova, I. Danilycheva, O. Elisyutina, Ludmila F. Znamenskaya, V. Kalugina, A. Karamova, Yulia G. Levina, R. Y. Meshkova, O. Y. Olisova, G. Novik, A. V. Samtsov, L. Selimzyanova, Evgeniy V. Sokolovsky, E. Fedenko, O. Fedorova, D. Fomina, V. Khayrutdinov, V. Chikin, A. Shulzhenko","doi":"10.15690/pf.v20i5.2629","DOIUrl":"https://doi.org/10.15690/pf.v20i5.2629","url":null,"abstract":"The Union of Pediatricians of Russia together with the Russian Association of Allergologists and Clinical Immunologists and the Russian Society of Dermatovenerologists and Cosmetologists have developed new clinical guidelines for the urticaria in adults and children. Urticaria is a common disease; its various clinical variants are diagnosed in 15–25% of people in the global population, and a quarter of all cases belongs to chronic urticaria. The prevalence of acute urticaria is 20%, and 2.1–6.7% in child population, whereas acute urticaria is more common in children than in adults. The prevalence of chronic urticaria in adults in the general population is 0.7 and 1.4%, and 1.1% in children under 15 years of age, according to the systematic review and meta-analysis, respectively. This article covers features of epidemiology, etiology, and pathogenesis of the disease with particular focus on differential diagnostic search. Guidelines on treatment and step-by-step therapy scheme (both based on principles of evidencebased medicine) for pediatric patients were presented. Clarification on the analysis of the therapy efficacy and the degree of disease activity was given.","PeriodicalId":19997,"journal":{"name":"Pediatric pharmacology","volume":"29 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139264831","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}
Andrej N. Surkov, A. A. Baranov, L. S. Namazova-Baranova, Anna L. Arakelyan, E. E. Bessonov, N. V. Zhurkova
Glycogen storage disease type Ib (GSD Ib) — is a disease from the group of hereditary metabolic diseases caused by insufficiency of the glucose-6-phosphate transporter (G6PT, SLC37A4), which leads to a violation of both glycogenolysis and gluconeogenesis and, as a consequence, to excessive accumulation of glycogen and fat in the liver, kidneys and intestinal mucosa. The main clinical manifestations and laboratory data include growth retardation, hepatomegaly, hypoglycemia, lactic acidosis, hyperuricemia and hyperlipidemia. Complications of this disease are hepatocellular adenoma with a possible risk of malignancy, nephropathy and osteoporosis. A specific sign of GSD Ib is neutropenia with impaired neutrophil function, which creates prerequisites for recurrent infections and the development of inflammatory bowel disease. Until the present, enzyme replacement therapy of GSD Ib has not been developed, therefore, the main methods of treatment are a specialized diet with the addition of raw corn starch (for relief of hypoglycemia) and the use of granulocyte colony stimulating factor (for relief of neutropenia). However, the recent establishment of the role of 1,5-anhydroglucitol in the pathogenesis of neutrophil dysfunction in GSD Ib has led to a reprofiling of indications for the use of empagliflozin, a type 2 renal sodium—glucose cotransporter inhibitor (SGLT2). In the modern literature, it is reported about a minor, but very successful experience of its use in patients with GSD Ib (outside the framework of official indications for use) and a beneficial effect on neutrophil dysfunction and its clinical consequences. Oddly enough, this hypoglycemic drug improved not only metabolic, but also glycemic control in patients with GSD Ib, despite the fact that the pathology is based on chronic hypoglycemia. More and more evidence points to the role of empagliflozin in the regulation of cellular homeostasis (for example, fatty acid metabolism, glucose, cholesterol, apoptosis and cell proliferation, in particular in the liver) by influencing the activity of sirtuin 1 (SIRT1), AMP-activated protein kinase (AMPK) and signal molecules such as -serine/threonine protein kinase (Akt) and a mechanical target of rapamycin (mTOR), which leads to an improvement in the structure and function of mitochondria, stimulation of autophagy, reducing oxidative stress and suppressing inflammation. Modulation of these pathways shifts oxidative metabolism from carbohydrates to lipids and leads to a key decrease in insulin levels, resistance to it, glucose and lipotoxicity. This review presents current data on the pathogenesis of neutropenia and the possibility of using empagliflozin for its relief in patients with GSD Ib.
{"title":"Glycogen storage disease type Ib: modern understanding of the pathogenesis of neutropenia and prospects for its treatment with empagliflozin","authors":"Andrej N. Surkov, A. A. Baranov, L. S. Namazova-Baranova, Anna L. Arakelyan, E. E. Bessonov, N. V. Zhurkova","doi":"10.15690/pf.v20i5.2646","DOIUrl":"https://doi.org/10.15690/pf.v20i5.2646","url":null,"abstract":"Glycogen storage disease type Ib (GSD Ib) — is a disease from the group of hereditary metabolic diseases caused by insufficiency of the glucose-6-phosphate transporter (G6PT, SLC37A4), which leads to a violation of both glycogenolysis and gluconeogenesis and, as a consequence, to excessive accumulation of glycogen and fat in the liver, kidneys and intestinal mucosa. The main clinical manifestations and laboratory data include growth retardation, hepatomegaly, hypoglycemia, lactic acidosis, hyperuricemia and hyperlipidemia. Complications of this disease are hepatocellular adenoma with a possible risk of malignancy, nephropathy and osteoporosis. A specific sign of GSD Ib is neutropenia with impaired neutrophil function, which creates prerequisites for recurrent infections and the development of inflammatory bowel disease. Until the present, enzyme replacement therapy of GSD Ib has not been developed, therefore, the main methods of treatment are a specialized diet with the addition of raw corn starch (for relief of hypoglycemia) and the use of granulocyte colony stimulating factor (for relief of neutropenia). However, the recent establishment of the role of 1,5-anhydroglucitol in the pathogenesis of neutrophil dysfunction in GSD Ib has led to a reprofiling of indications for the use of empagliflozin, a type 2 renal sodium—glucose cotransporter inhibitor (SGLT2). In the modern literature, it is reported about a minor, but very successful experience of its use in patients with GSD Ib (outside the framework of official indications for use) and a beneficial effect on neutrophil dysfunction and its clinical consequences. Oddly enough, this hypoglycemic drug improved not only metabolic, but also glycemic control in patients with GSD Ib, despite the fact that the pathology is based on chronic hypoglycemia. More and more evidence points to the role of empagliflozin in the regulation of cellular homeostasis (for example, fatty acid metabolism, glucose, cholesterol, apoptosis and cell proliferation, in particular in the liver) by influencing the activity of sirtuin 1 (SIRT1), AMP-activated protein kinase (AMPK) and signal molecules such as -serine/threonine protein kinase (Akt) and a mechanical target of rapamycin (mTOR), which leads to an improvement in the structure and function of mitochondria, stimulation of autophagy, reducing oxidative stress and suppressing inflammation. Modulation of these pathways shifts oxidative metabolism from carbohydrates to lipids and leads to a key decrease in insulin levels, resistance to it, glucose and lipotoxicity. This review presents current data on the pathogenesis of neutropenia and the possibility of using empagliflozin for its relief in patients with GSD Ib.","PeriodicalId":19997,"journal":{"name":"Pediatric pharmacology","volume":"5 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139263295","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}
This article presents practical data, topical for pediatricians, on the child’s body provision with the essential trace element — iron; and on iron deficiency conditions development and staging in children. Clinical and laboratory criteria for the identification of such conditions are defined; data on their prevalence in tender-age infants is outlined. The results of modern studies showing the correlations between iron deficiency and delayed developmental conditions in children (including cognitive ones) are presented. Alimental factors (associated with body provision with iron) and nutritional strategies (associated with supplemental feeding timely administration, adequacy, and diversity) are described in detail. They are focused on effective and safe prevention of latent iron deficiency.
{"title":"Latent Iron Deficiency in Tender-Age Infants: Modern Preventive Measures","authors":"I. Belyaeva, E. Bombardirova, T. Turti","doi":"10.15690/pf.v20i5.2634","DOIUrl":"https://doi.org/10.15690/pf.v20i5.2634","url":null,"abstract":"This article presents practical data, topical for pediatricians, on the child’s body provision with the essential trace element — iron; and on iron deficiency conditions development and staging in children. Clinical and laboratory criteria for the identification of such conditions are defined; data on their prevalence in tender-age infants is outlined. The results of modern studies showing the correlations between iron deficiency and delayed developmental conditions in children (including cognitive ones) are presented. Alimental factors (associated with body provision with iron) and nutritional strategies (associated with supplemental feeding timely administration, adequacy, and diversity) are described in detail. They are focused on effective and safe prevention of latent iron deficiency.","PeriodicalId":19997,"journal":{"name":"Pediatric pharmacology","volume":"75 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139264589","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}
Elena A. Degtyareva, B. Mwela, Andrey P. Prodeus, Dmitry Yu. Ovsyannikov, M. Kantemirova, O.V. Alekseeva, D. Kudlay, Alexey I. Kim, Inessa E. Nefedova, Tatiana V. Rogova, M. Tumanyan, I. A. Korsunskiy
The study data of the last two decades on primary and secondary immunodeficiency in congenital heart defects (CHD) as a cause of frequent infectious complications before and after cardiac surgery are presented. Based on screenings of various levels, data are provided on the greater severity of immunological disorders in critical and cyanotic CHD in conotruncal defects compared with those in septal defects and stenotic defects. Violations were more often related to T-cell function and immunoglobulin deficiency (especially the IgG and IgG4 subgroups). Various types of primary immunodeficiency were found in 13 genetic syndromes in combination with CHD. The review discusses the possibility of using the technique of quantitative determination of DNA TREC and KREC — by-products of maturation of T- and B-cell receptors, which allows us to judge the defects of the T- and B-cell links of the immune system to predict infectious complications in children with CHD. The data of our own study of 200 infants with CHD (in 5% of cases with syndromic forms of CHD) are presented, where a decrease in TREC was found in 23.5% of cases, including all infants with syndromic forms, more often with cyanotic and conotruncal CHD and in children admitted in critical conditions. In children with reduced TREC values, infectious complications in the postoperative period were observed significantly more often than in children with normal indicators (36 and 3.6%, respectively). The analysis of publications confirmed the importance of TREC and KREC screening for targeted preoperative preparation in order to reduce postoperative complications and reduce the risk of mortality in CHD.
本文介绍了近二十年来关于先天性心脏缺损（CHD）原发性和继发性免疫缺陷作为心脏手术前后频繁出现感染并发症的原因的研究数据。根据不同程度的筛查，数据显示与房间隔缺损和狭窄缺损相比，先天性心脏病中的危重症和紫绀型先天性心脏病的免疫功能紊乱更为严重。免疫紊乱多与 T 细胞功能和免疫球蛋白缺乏有关（尤其是 IgG 和 IgG4 亚组）。在 13 种合并有先天性心脏病的遗传综合征中发现了各种类型的原发性免疫缺陷。综述讨论了使用 DNA TREC 和 KREC（T 细胞和 B 细胞受体成熟的副产品）定量测定技术的可能性，该技术可让我们判断免疫系统中 T 细胞和 B 细胞环节的缺陷，从而预测先天性心脏病患儿的感染性并发症。我们介绍了自己对 200 名患有先天性心脏病的婴儿（其中 5%患有先天性心脏病综合征）进行研究的数据，结果发现 23.5% 的病例中 TREC 值下降，其中包括所有患有综合征的婴儿，更常见于发绀型和圆锥型先天性心脏病以及病情危重的患儿。在TREC值降低的患儿中，术后出现感染性并发症的比例明显高于指标正常的患儿（分别为36%和3.6%）。对出版物的分析证实了TREC和KREC筛查对于有针对性地做好术前准备以减少术后并发症和降低先天性心脏病死亡风险的重要性。
{"title":"Immunodeficiency Disorders in Congenital Heart Diseases (Review)","authors":"Elena A. Degtyareva, B. Mwela, Andrey P. Prodeus, Dmitry Yu. Ovsyannikov, M. Kantemirova, O.V. Alekseeva, D. Kudlay, Alexey I. Kim, Inessa E. Nefedova, Tatiana V. Rogova, M. Tumanyan, I. A. Korsunskiy","doi":"10.15690/pf.v20i5.2647","DOIUrl":"https://doi.org/10.15690/pf.v20i5.2647","url":null,"abstract":"The study data of the last two decades on primary and secondary immunodeficiency in congenital heart defects (CHD) as a cause of frequent infectious complications before and after cardiac surgery are presented. Based on screenings of various levels, data are provided on the greater severity of immunological disorders in critical and cyanotic CHD in conotruncal defects compared with those in septal defects and stenotic defects. Violations were more often related to T-cell function and immunoglobulin deficiency (especially the IgG and IgG4 subgroups). Various types of primary immunodeficiency were found in 13 genetic syndromes in combination with CHD. The review discusses the possibility of using the technique of quantitative determination of DNA TREC and KREC — by-products of maturation of T- and B-cell receptors, which allows us to judge the defects of the T- and B-cell links of the immune system to predict infectious complications in children with CHD. The data of our own study of 200 infants with CHD (in 5% of cases with syndromic forms of CHD) are presented, where a decrease in TREC was found in 23.5% of cases, including all infants with syndromic forms, more often with cyanotic and conotruncal CHD and in children admitted in critical conditions. In children with reduced TREC values, infectious complications in the postoperative period were observed significantly more often than in children with normal indicators (36 and 3.6%, respectively). The analysis of publications confirmed the importance of TREC and KREC screening for targeted preoperative preparation in order to reduce postoperative complications and reduce the risk of mortality in CHD.","PeriodicalId":19997,"journal":{"name":"Pediatric pharmacology","volume":"99 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139266021","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}
E. I. Kleshchenko, E. Shimchenko, Aleksander F. Komarov, Valeria E. Kharchenko
Cystic fibrosis is a severe hereditary disease with polysystemic manifestations and progressive course. Malnutrition in cystic fibrosis occurs as a result of exocrine insufficiency of the pancreas, an increase in energy losses in chronic inflammation in the bronchopulmonary system, manifested by increased stress on the respiratory system. The presented literature review highlights the modern principles of prevention and correction of malnutrition in children with cystic fibrosis, identifies the most promising methods for further development that correct nutritional status disorders. The review has shown that an active approach to nutrition at any age, the use of aggressive methods of nutritional support against the background of enzyme replacement therapy, timely and adequate therapy of respiratory tract pathology lead to an improvement in the indicators of nutritional status in cystic fibrosis. The most promising is the further development of targeted therapy, which allows, as a result of exposure to the etiopathogenetic mechanisms of the disease, to reduce the frequency and severity of bronchopulmonary exacerbations, partially restore the exocrine function of the pancreas, which is manifested in patients with cystic fibrosis by an increase in body weight and mass-growth index.
{"title":"The Risk of Developing of Malnutrition and the Principles of Correction of Nutritional Status Disorders in Children with Cystic Fibrosis","authors":"E. I. Kleshchenko, E. Shimchenko, Aleksander F. Komarov, Valeria E. Kharchenko","doi":"10.15690/pf.v20i5.2632","DOIUrl":"https://doi.org/10.15690/pf.v20i5.2632","url":null,"abstract":"Cystic fibrosis is a severe hereditary disease with polysystemic manifestations and progressive course. Malnutrition in cystic fibrosis occurs as a result of exocrine insufficiency of the pancreas, an increase in energy losses in chronic inflammation in the bronchopulmonary system, manifested by increased stress on the respiratory system. The presented literature review highlights the modern principles of prevention and correction of malnutrition in children with cystic fibrosis, identifies the most promising methods for further development that correct nutritional status disorders. The review has shown that an active approach to nutrition at any age, the use of aggressive methods of nutritional support against the background of enzyme replacement therapy, timely and adequate therapy of respiratory tract pathology lead to an improvement in the indicators of nutritional status in cystic fibrosis. The most promising is the further development of targeted therapy, which allows, as a result of exposure to the etiopathogenetic mechanisms of the disease, to reduce the frequency and severity of bronchopulmonary exacerbations, partially restore the exocrine function of the pancreas, which is manifested in patients with cystic fibrosis by an increase in body weight and mass-growth index.","PeriodicalId":19997,"journal":{"name":"Pediatric pharmacology","volume":"29 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139263477","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}
N. A. Nikolaev, Y. Skirdenko, K. Andreev, A. V. Gorbenko, E. B. Pavlinova, G. Usov, M. M. Fedorin
The aim of the study is assess the reliability, internal stability and effectiveness of adaptation of individual questions of the QAA-25 questionnaire to assess the potential adherence to treatment of persons aged 12–14 years, in comparison with a similar QAA-25 questionnaire for persons aged 15–17 years. Material and Methods. In a descriptive observational study, including 314 students of general schools in Omsk the level of treatment adherence was determined using the QAA-25 questionnaire, using traditional and alternative formulations of individual questions. Results. For adolescents aged 12–14 years a good diagnostic value was shown higher individual testing quality (absence of blank answers to questions) of the tested version of the questionnaire compared to the alternative one. 96.8% of respondents indicated that the wording of the specialized questionnaire was “more accurate” and “more correct”. The tested version of the questionnaire demonstrated good reliability (Cronbach’s alpha total 0.901, based on standardized items αst 0.914) with high internal consistency (consistent elimination of scale items maintains questionnaire validity in the 0.886–0.909 range), with near perfect expert agreement (Cohen’s kappa 0.908). Conclusion. The questionnaire is characterized by good validity and has a high internal stability, and the content and wording of all questions of the questionnaire are adequately perceived by the audience for which the questionnaire is designed. This makes it advisable to use it to assess the potential adherence to treatment of adolescents aged 12–14 years.
{"title":"Specialized Questionnaire of the QAA-25 Questionnaire System for Assessing the Potential Adherence to Treatment of Adolescents Aged 12–14 Years: Reliability and Internal Stability","authors":"N. A. Nikolaev, Y. Skirdenko, K. Andreev, A. V. Gorbenko, E. B. Pavlinova, G. Usov, M. M. Fedorin","doi":"10.15690/pf.v20i5.2616","DOIUrl":"https://doi.org/10.15690/pf.v20i5.2616","url":null,"abstract":"The aim of the study is assess the reliability, internal stability and effectiveness of adaptation of individual questions of the QAA-25 questionnaire to assess the potential adherence to treatment of persons aged 12–14 years, in comparison with a similar QAA-25 questionnaire for persons aged 15–17 years. Material and Methods. In a descriptive observational study, including 314 students of general schools in Omsk the level of treatment adherence was determined using the QAA-25 questionnaire, using traditional and alternative formulations of individual questions. Results. For adolescents aged 12–14 years a good diagnostic value was shown higher individual testing quality (absence of blank answers to questions) of the tested version of the questionnaire compared to the alternative one. 96.8% of respondents indicated that the wording of the specialized questionnaire was “more accurate” and “more correct”. The tested version of the questionnaire demonstrated good reliability (Cronbach’s alpha total 0.901, based on standardized items αst 0.914) with high internal consistency (consistent elimination of scale items maintains questionnaire validity in the 0.886–0.909 range), with near perfect expert agreement (Cohen’s kappa 0.908). Conclusion. The questionnaire is characterized by good validity and has a high internal stability, and the content and wording of all questions of the questionnaire are adequately perceived by the audience for which the questionnaire is designed. This makes it advisable to use it to assess the potential adherence to treatment of adolescents aged 12–14 years.","PeriodicalId":19997,"journal":{"name":"Pediatric pharmacology","volume":"27 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139265046","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}
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