As a result of the use of blood culture vials for seeding joint and bone exudates, and the development of nucleic acid amplification methods, Kingella kingae is being increasingly recognized as an emerging invasive pathogen and the most common etiology of septic arthritis in children aged 6–36 months. K. kingae is carried asymptomatically in the pharynx, and is transmitted from child-to-child by close contact between family members and playmates. K. kingae organisms enter the bloodstream through breaches in the respiratory mucosa and disseminate to remote sites. Skeletal system infections are the most common presentations of K. kingae disease, followed by bacteremia, pneumonia and endocarditis. Children with invasive K. kingae infections frequently show a mild clinical picture and normal acute-phase reactants, requiring a high index of suspicion. The organism is usually susceptible to antibiotics and, with the exception of endocarditis cases, most patients promptly respond to adequate antimicrobial therap...
{"title":"Kingella kingae: from asymptomatic colonization to invasive pediatric infections","authors":"Inbal Weiss-Salz, P. Yagupsky","doi":"10.2217/PHE.10.28","DOIUrl":"https://doi.org/10.2217/PHE.10.28","url":null,"abstract":"As a result of the use of blood culture vials for seeding joint and bone exudates, and the development of nucleic acid amplification methods, Kingella kingae is being increasingly recognized as an emerging invasive pathogen and the most common etiology of septic arthritis in children aged 6–36 months. K. kingae is carried asymptomatically in the pharynx, and is transmitted from child-to-child by close contact between family members and playmates. K. kingae organisms enter the bloodstream through breaches in the respiratory mucosa and disseminate to remote sites. Skeletal system infections are the most common presentations of K. kingae disease, followed by bacteremia, pneumonia and endocarditis. Children with invasive K. kingae infections frequently show a mild clinical picture and normal acute-phase reactants, requiring a high index of suspicion. The organism is usually susceptible to antibiotics and, with the exception of endocarditis cases, most patients promptly respond to adequate antimicrobial therap...","PeriodicalId":88627,"journal":{"name":"Pediatric health","volume":"63 1","pages":"311-320"},"PeriodicalIF":0.0,"publicationDate":"2010-06-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2217/PHE.10.28","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68242593","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}
Asthma guidelines recommend the use of long-acting β2-agonists (LABAs) as the preferred add-on therapy for adults and children over 5 years of age when asthma is inadequately controlled by inhaled corticosteroids alone. It has been suggested that LABA use may be associated with an increased risk of morbidity and mortality; however, this view is controversial since study findings have been inconsistent. While the safety profile of LABA monotherapy has been questioned, the value of concomitant inhaled corticosteroids to eliminate possible risks remains unproven. There is a paucity of efficacy and safety data for LABA use in children, and existing evidence is not sufficiently convincing to demonstrate a clear position for LABAs in the management of childhood asthma. The main aims of this article are to place LABAs in context in the management of childhood asthma and evaluate the current evidence for safety and efficacy.
{"title":"Use and safety of long-acting β2-agonists for pediatric asthma","authors":"H. Elkout, J. McLay, Colin Simpson, P. Helms","doi":"10.2217/PHE.10.31","DOIUrl":"https://doi.org/10.2217/PHE.10.31","url":null,"abstract":"Asthma guidelines recommend the use of long-acting β2-agonists (LABAs) as the preferred add-on therapy for adults and children over 5 years of age when asthma is inadequately controlled by inhaled corticosteroids alone. It has been suggested that LABA use may be associated with an increased risk of morbidity and mortality; however, this view is controversial since study findings have been inconsistent. While the safety profile of LABA monotherapy has been questioned, the value of concomitant inhaled corticosteroids to eliminate possible risks remains unproven. There is a paucity of efficacy and safety data for LABA use in children, and existing evidence is not sufficiently convincing to demonstrate a clear position for LABAs in the management of childhood asthma. The main aims of this article are to place LABAs in context in the management of childhood asthma and evaluate the current evidence for safety and efficacy.","PeriodicalId":88627,"journal":{"name":"Pediatric health","volume":"4 1","pages":"295-310"},"PeriodicalIF":0.0,"publicationDate":"2010-06-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68242227","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}
Prevalence The epidemiology of Type 2 diabetes mellitus (T2DM) in adolescents in the USA and world wide has been reviewed multiple times [1–3]. These reviews confirm the rise in the global inci dence of T2DM in children and adolescents over the last two decades and suggest a close relation ship between an increase in obesity in the general population, an increase in the rate of T2DM in adults and the later appearance of the disorder in adolescents [2]. There have been few recent population based studies of Type 2 diabetes in adolescents since these reviews were published. The SEARCH for Diabetes in Youth Study is a six center population based ascertainment of physician diagnosed diabetes in adolescents of less than 20 years of age in the USA. Among older adolescents, the proportion of T2DM ranged from 6% of new cases of diabetes (0.19 cases per 1000 adolescents for non Hispanic white adoles cents) to 76% (1.74 cases per 1000 adolescents for American–Indian adolescents) [4].
{"title":"Type 2 diabetes in children: recognition and complications","authors":"P. Zeitler","doi":"10.2217/PHE.10.10","DOIUrl":"https://doi.org/10.2217/PHE.10.10","url":null,"abstract":"Prevalence The epidemiology of Type 2 diabetes mellitus (T2DM) in adolescents in the USA and world wide has been reviewed multiple times [1–3]. These reviews confirm the rise in the global inci dence of T2DM in children and adolescents over the last two decades and suggest a close relation ship between an increase in obesity in the general population, an increase in the rate of T2DM in adults and the later appearance of the disorder in adolescents [2]. There have been few recent population based studies of Type 2 diabetes in adolescents since these reviews were published. The SEARCH for Diabetes in Youth Study is a six center population based ascertainment of physician diagnosed diabetes in adolescents of less than 20 years of age in the USA. Among older adolescents, the proportion of T2DM ranged from 6% of new cases of diabetes (0.19 cases per 1000 adolescents for non Hispanic white adoles cents) to 76% (1.74 cases per 1000 adolescents for American–Indian adolescents) [4].","PeriodicalId":88627,"journal":{"name":"Pediatric health","volume":"4 1","pages":"123-127"},"PeriodicalIF":0.0,"publicationDate":"2010-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2217/PHE.10.10","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68241813","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}
Anemia is one of the most common medical complications encountered in critically ill children. Based on the results of clinical trials, transfusion practices across the world have generally become more restrictive. The decision process leading to the conduction of a red blood cell transfusion should be based on available evidence as much as possible. The risks and benefits of the transfusion as well as the risks attributable to anemia must be taken into account. This review summarizes the current practice approach to the critically ill pediatric patient.
{"title":"Transfusions in the critically ill pediatric patient","authors":"J. Roganović","doi":"10.2217/PHE.10.12","DOIUrl":"https://doi.org/10.2217/PHE.10.12","url":null,"abstract":"Anemia is one of the most common medical complications encountered in critically ill children. Based on the results of clinical trials, transfusion practices across the world have generally become more restrictive. The decision process leading to the conduction of a red blood cell transfusion should be based on available evidence as much as possible. The risks and benefits of the transfusion as well as the risks attributable to anemia must be taken into account. This review summarizes the current practice approach to the critically ill pediatric patient.","PeriodicalId":88627,"journal":{"name":"Pediatric health","volume":"4 1","pages":"201-208"},"PeriodicalIF":0.0,"publicationDate":"2010-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2217/PHE.10.12","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68241849","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}
Valganciclovir has been used extensively for the prevention and treatment of cytomegalovirus in adults for many years. In August 2009, valganciclovir was approved by the US FDA for use in children at the same time as a valganciclovir oral solution. This oral solution will facilitate dosing in patients who are unable to swallow tablets or who require intermediate doses. This paper reviews the current status of the general use of valganciclovir in organ transplant patients with a particular focus on children. Valganciclovir appears to be safe and effective in children. The dose of valganciclovir that appears appropriate for children is based on both renal function and body surface area – specifically, dose (in mg) = seven-times the body surface area multiplied by the creatinine clearance (calculated using a modified Schwartz formula).
{"title":"Valganciclovir in pediatric organ transplantation","authors":"M. Pescovitz","doi":"10.2217/PHE.10.16","DOIUrl":"https://doi.org/10.2217/PHE.10.16","url":null,"abstract":"Valganciclovir has been used extensively for the prevention and treatment of cytomegalovirus in adults for many years. In August 2009, valganciclovir was approved by the US FDA for use in children at the same time as a valganciclovir oral solution. This oral solution will facilitate dosing in patients who are unable to swallow tablets or who require intermediate doses. This paper reviews the current status of the general use of valganciclovir in organ transplant patients with a particular focus on children. Valganciclovir appears to be safe and effective in children. The dose of valganciclovir that appears appropriate for children is based on both renal function and body surface area – specifically, dose (in mg) = seven-times the body surface area multiplied by the creatinine clearance (calculated using a modified Schwartz formula).","PeriodicalId":88627,"journal":{"name":"Pediatric health","volume":"4 1","pages":"147-156"},"PeriodicalIF":0.0,"publicationDate":"2010-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68241971","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. Coppola, M. Franchini, M. D. Palo, Emiliana Marrone, C. D. Perna, A. Tagliaferri
Hemophilia A and B, the congenital deficiencies of coagulation factors VIII and IX, are characterized by recurrent joint and muscle bleeding episodes and progressive musculoskeletal damage (hemophilic arthropathy). Primary prophylaxis – that is, the regular infusion of factor concentrates after the first hemarthrosis and/or before 2 years of age – is now recognized as the first-choice treatment for children with severe hemophilia. Preventing bleeding from an early age enables avoidance of the clinical impact of hemophilic arthropathy and the consequences regarding psychosocial development and quality of life for these children. Interestingly, recent data suggest a role for early prophylaxis in also preventing inhibitor development, the most serious complication of treatment in hemophilia. Secondary prophylaxis, initiated after 2 years of age or after two or more joint bleeds, aims to avoid (or delay) the progression of arthropathy. In addition, better outcomes and better quality of life have been reported...
{"title":"Enabling normal psychophysical development in children with hemophilia: the choice for prophylaxis","authors":"A. Coppola, M. Franchini, M. D. Palo, Emiliana Marrone, C. D. Perna, A. Tagliaferri","doi":"10.2217/PHE.10.9","DOIUrl":"https://doi.org/10.2217/PHE.10.9","url":null,"abstract":"Hemophilia A and B, the congenital deficiencies of coagulation factors VIII and IX, are characterized by recurrent joint and muscle bleeding episodes and progressive musculoskeletal damage (hemophilic arthropathy). Primary prophylaxis – that is, the regular infusion of factor concentrates after the first hemarthrosis and/or before 2 years of age – is now recognized as the first-choice treatment for children with severe hemophilia. Preventing bleeding from an early age enables avoidance of the clinical impact of hemophilic arthropathy and the consequences regarding psychosocial development and quality of life for these children. Interestingly, recent data suggest a role for early prophylaxis in also preventing inhibitor development, the most serious complication of treatment in hemophilia. Secondary prophylaxis, initiated after 2 years of age or after two or more joint bleeds, aims to avoid (or delay) the progression of arthropathy. In addition, better outcomes and better quality of life have been reported...","PeriodicalId":88627,"journal":{"name":"Pediatric health","volume":"4 1","pages":"183-199"},"PeriodicalIF":0.0,"publicationDate":"2010-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2217/PHE.10.9","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68243270","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}
As one of the common childhood infections, urinary tract infections (UTIs) take up an important part of a pediatrician’s daily routine practice. Not only can UTIs lead to life-threatining complications in infants, but also to renal scarring and related complications in all age groups. In order to avoid morbidity, it is suggested that treatment should be started as soon as a UTI is recognized, but because urine cultures (the gold-standard for the diagnosis of UTI) take 24–48 h to process, initial treatment is started empirically. At this point, knowledge of the pathogens causing the UTI and local antibiotic sensitivity patterns are needed to ensure appropriate treatment. With this purpose, studies were conducted all over the world and high levels of resistance to ampicillin, cephalexin and trimethoprim-sulphametoxazole were commonly reported. The prevalence of antibiotic resistance of uropathogens in complicated and recurrent UTIs was found to be even higher than that in first, uncomplicated UTIs. Multidru...
{"title":"Antimicrobial resistance of childhood uropathogens","authors":"I. O. Ipek, A. Bozaykut","doi":"10.2217/PHE.10.8","DOIUrl":"https://doi.org/10.2217/PHE.10.8","url":null,"abstract":"As one of the common childhood infections, urinary tract infections (UTIs) take up an important part of a pediatrician’s daily routine practice. Not only can UTIs lead to life-threatining complications in infants, but also to renal scarring and related complications in all age groups. In order to avoid morbidity, it is suggested that treatment should be started as soon as a UTI is recognized, but because urine cultures (the gold-standard for the diagnosis of UTI) take 24–48 h to process, initial treatment is started empirically. At this point, knowledge of the pathogens causing the UTI and local antibiotic sensitivity patterns are needed to ensure appropriate treatment. With this purpose, studies were conducted all over the world and high levels of resistance to ampicillin, cephalexin and trimethoprim-sulphametoxazole were commonly reported. The prevalence of antibiotic resistance of uropathogens in complicated and recurrent UTIs was found to be even higher than that in first, uncomplicated UTIs. Multidru...","PeriodicalId":88627,"journal":{"name":"Pediatric health","volume":"4 1","pages":"219-225"},"PeriodicalIF":0.0,"publicationDate":"2010-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2217/PHE.10.8","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68243690","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}
The aim of this study was to provide a qualitative perspective of young people’s experiences of long-term illness using an innovative multimedia research methodology. Three young individuals recorded video diaries and were interviewed about their experiences of living with a long-term illness; the resulting footage was edited into a documentary film and showed to local healthcare professionals, commissioners and policy makers. The original unedited interview transcripts were then analyzed by thematic analysis. Four main themes were identified, representing common shared experiences among participants. These were related to coping with their illness, the impact of illness on various aspects of their life, their experiences of healthcare and transitions from pediatric to adult services. While significant efforts are being made to increase the knowledge and understanding of the experiences of long-term illness within the child and adolescent population, there is still much to be learned, as is evident from t...
{"title":"Consulting with young people about healthcare. Part 2: experience of long-term health conditions","authors":"A. McCormack, S. Norrish, L. Parker, I. Frampton","doi":"10.2217/PHE.10.13","DOIUrl":"https://doi.org/10.2217/PHE.10.13","url":null,"abstract":"The aim of this study was to provide a qualitative perspective of young people’s experiences of long-term illness using an innovative multimedia research methodology. Three young individuals recorded video diaries and were interviewed about their experiences of living with a long-term illness; the resulting footage was edited into a documentary film and showed to local healthcare professionals, commissioners and policy makers. The original unedited interview transcripts were then analyzed by thematic analysis. Four main themes were identified, representing common shared experiences among participants. These were related to coping with their illness, the impact of illness on various aspects of their life, their experiences of healthcare and transitions from pediatric to adult services. While significant efforts are being made to increase the knowledge and understanding of the experiences of long-term illness within the child and adolescent population, there is still much to be learned, as is evident from t...","PeriodicalId":88627,"journal":{"name":"Pediatric health","volume":"4 1","pages":"167-175"},"PeriodicalIF":0.0,"publicationDate":"2010-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2217/PHE.10.13","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68241907","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}
Although less common than hemophilia or von Willebrand disease, inherited rare bleeding disorders, comprising afibrinogenemia and deficiencies in factors II, V, VII, X, XI, XIII or V plus VIII combined, or in vitamin K-dependent coagulation factors, may lead to severe bleeding episodes such as recurrent hemarthroses and neonatal intracranial or gastrointestinal hemorrhage. Consanguinity significantly increases the risk of the occurrence of all rare bleeding disorders that are associated with an autosomal recessive pattern of inheritance. Each of the disorders is characterized by a wide interindividual variation in clinical phenotype and a large mutational spectrum with no clear correlation between the phenotype and genotype. Replacement therapy relies on specific molecules or concentrates (afibrinogenemia, factor VII, XI and XIII deficiencies), on a mixture of different concentrates that are otherwise known as a prothrombin complex, which contains factors II, VII, IX and X, or on fresh frozen plasma. Inte...
{"title":"Comprehensive pediatric care of rare bleeding disorders","authors":"M. Giansily-Blaizot, J. Schved","doi":"10.2217/PHE.10.15","DOIUrl":"https://doi.org/10.2217/PHE.10.15","url":null,"abstract":"Although less common than hemophilia or von Willebrand disease, inherited rare bleeding disorders, comprising afibrinogenemia and deficiencies in factors II, V, VII, X, XI, XIII or V plus VIII combined, or in vitamin K-dependent coagulation factors, may lead to severe bleeding episodes such as recurrent hemarthroses and neonatal intracranial or gastrointestinal hemorrhage. Consanguinity significantly increases the risk of the occurrence of all rare bleeding disorders that are associated with an autosomal recessive pattern of inheritance. Each of the disorders is characterized by a wide interindividual variation in clinical phenotype and a large mutational spectrum with no clear correlation between the phenotype and genotype. Replacement therapy relies on specific molecules or concentrates (afibrinogenemia, factor VII, XI and XIII deficiencies), on a mixture of different concentrates that are otherwise known as a prothrombin complex, which contains factors II, VII, IX and X, or on fresh frozen plasma. Inte...","PeriodicalId":88627,"journal":{"name":"Pediatric health","volume":"4 1","pages":"209-217"},"PeriodicalIF":0.0,"publicationDate":"2010-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2217/PHE.10.15","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68241931","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}
R. Giugliani, A. Federhen, C. Carvalho, O. Artigalás
Mucopolysaccharidosis type I (MPS I) is a storage disorder caused by the deficiency of the lysosomal enzyme α-L-iduronidase. MPS I has a chronic progressive evolution with multisystemic symptomatology and wide clinical variability, with its main manifestations in the skeletal, respiratory, cardiac and neurological systems. Until recently, therapeutic options for MPS I were limited to hematopoietic stem cell transplantation for severe cases, and palliative care. Laronidase (Aldurazyme®, BioMarin/Genzyme, CA, USA), a synthetic variant of the human α-L-iduronidase, is a specific treatment for MPS I. Enzyme replacement therapy with intravenous infusion of laronidase aims to reduce and/or prevent the accumulation of glycosaminoglycans (dermatan and heparan sulfate), which is probably the most important, although possibly not the only, cause of the clinical manifestations of MPS I. This article reviews the data published to date on the clinical indications and user experience of laronidase in patients with MPS I.
粘多糖病I型(MPS I)是由溶酶体酶α- l -伊杜糖醛酸酶缺乏引起的一种储存障碍。MPS I具有慢性进行性发展，多系统症状和广泛的临床变异性，主要表现在骨骼、呼吸、心脏和神经系统。直到最近，MPS I的治疗选择仅限于严重病例的造血干细胞移植和姑息治疗。Laronidase (Aldurazyme®，BioMarin/Genzyme, CA, USA)是人α-L-iduronidase的合成变体，是MPS i的特异性治疗方法。静脉输注Laronidase的酶替代疗法旨在减少和/或防止糖胺聚糖(皮聚糖和硫酸肝素)的积累，这可能是最重要的，尽管可能不是唯一的。本文综述了迄今为止发表的关于多磺酸粘多糖I患者的临床适应症和使用laronidase的数据。
{"title":"Enzyme replacement therapy for mucopolysaccharidosis I: laronidase","authors":"R. Giugliani, A. Federhen, C. Carvalho, O. Artigalás","doi":"10.2217/PHE.10.5","DOIUrl":"https://doi.org/10.2217/PHE.10.5","url":null,"abstract":"Mucopolysaccharidosis type I (MPS I) is a storage disorder caused by the deficiency of the lysosomal enzyme α-L-iduronidase. MPS I has a chronic progressive evolution with multisystemic symptomatology and wide clinical variability, with its main manifestations in the skeletal, respiratory, cardiac and neurological systems. Until recently, therapeutic options for MPS I were limited to hematopoietic stem cell transplantation for severe cases, and palliative care. Laronidase (Aldurazyme®, BioMarin/Genzyme, CA, USA), a synthetic variant of the human α-L-iduronidase, is a specific treatment for MPS I. Enzyme replacement therapy with intravenous infusion of laronidase aims to reduce and/or prevent the accumulation of glycosaminoglycans (dermatan and heparan sulfate), which is probably the most important, although possibly not the only, cause of the clinical manifestations of MPS I. This article reviews the data published to date on the clinical indications and user experience of laronidase in patients with MPS I.","PeriodicalId":88627,"journal":{"name":"Pediatric health","volume":"4 1","pages":"133-145"},"PeriodicalIF":0.0,"publicationDate":"2010-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2217/PHE.10.5","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"68242691","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}
京公网安备 11010802042870号
京ICP备2023020795号-1
扫码关注我们
求助内容：
应助结果提醒方式：