Abstract:
PURPOSE OF REVIEW:Disorders of hemostasis such as hemophilia, von Willebrand disease (VWD), and other clotting protein deficiencies lead to significant morbidity in the pediatric population. Because of the limitations of current treatment options, novel therapies are being developed, many of which are reviewed here. RECENT FINDINGS:Several new observations about the nature of clotting protein physiology have been made recently, creating novel perspectives on the treatment options. This review will mostly focus on the current therapy as well as new progress in hemophilia care (particularly strategies to prolong half-life of clotting factor replacements, the management of inhibitors, gene therapy, and novel therapeutic approaches), and briefly mention some progress in VWD and fibrinogen deficiency therapies. SUMMARY:New therapeutic developments have the potential to dramatically decrease morbidity and improve the quality of life of children with bleeding disorders.
journal_name
Curr Opin Pediatrjournal_title
Current opinion in pediatricsauthors
Branchford BR,Monahan PE,Di Paola Jdoi
10.1097/MOP.0b013e32835bf77esubject
Has Abstractpub_date
2013-02-01 00:00:00pages
23-30issue
1eissn
1040-8703issn
1531-698Xjournal_volume
25pub_type
杂志文章,评审abstract:PURPOSE OF REVIEW:To review recent advances in severe congenital neutropenia (SCN) syndromes. RECENT FINDINGS:The majority of patients with SCN bear monoallelic mutations in the neutrophil elastase (ELANE) gene. Biallelic mutations in the antiapoptotic gene HAX1 were identified in patients with autosomal recessive SCN...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0b013e32834262f8
更新日期:2011-02-01 00:00:00
abstract::Now known as a Garrodian inborn error caused by the homozygous state of many different autosomal recessive mutations of the 7-dehydrocholesterol reductase gene leading to deficient conversion of 7-dehydrocholesterol to cholesterol, the RSH (so-called Smith-Lemli-Opitz) syndrome has become a paradigmatic metabolic malf...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/00008480-199908000-00015
更新日期:1999-08-01 00:00:00
abstract:PURPOSE OF REVIEW:This study reviews the history of neonatal drug development, recent legislative efforts designed to facilitate the study of therapeutic agents in neonates, and future steps necessary to advance drug development. RECENT FINDINGS:Although many federal regulations have been introduced over the past 15 y...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0000000000000463
更新日期:2017-04-01 00:00:00
abstract:PURPOSE OF REVIEW:Extremely low gestational age newborns (ELGANs), born at less than 28 weeks' estimated gestational age, suffer the greatest consequences of prematurity. There have been significant advances in their care over the last several decades, but the prospects for major advances within traditional treatment m...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0000000000000083
更新日期:2014-06-01 00:00:00
abstract:PURPOSE OF REVIEW:To highlight studies that have contributed significantly to our current knowledge of inhaled glucocorticoids in childhood asthma. RECENT FINDINGS:In 2006, three important studies were published that investigated whether inhaled glucocorticoid therapy, if started soon after the onset of asthma symptom...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0b013e3280d35893
更新日期:2007-06-01 00:00:00
abstract:PURPOSE OF REVIEW:Describe developments in the etiological understanding of Tourette syndrome. RECENT FINDINGS:Tourette syndrome is a complex heterogenous clinical syndrome, which is not a unitary entity. Pathophysiological models describe gamma-aminobutyric acid-ergic-associated disinhibition of cortico-basal ganglia...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0000000000000546
更新日期:2017-12-01 00:00:00
abstract:PURPOSE OF REVIEW:Osteoid osteoma is a benign painful bone tumor usually found in the lower extremities of children and young adults. It has been traditionally treated by surgical excision. Despite the small size of the lesion, the operative procedure for its removal can be extensive, but still sometimes remains incomp...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/01.mop.0000193277.47119.15
更新日期:2006-02-01 00:00:00
abstract::Allergen avoidance should be considered as a first line of defense for all types of allergic disease. For some allergens, such as the common pollens, significant avoidance may be difficult or even impossible to accomplish without major alterations in lifestyle. For others, however, particularly the common indoor aller...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/00008480-199312000-00009
更新日期:1993-12-01 00:00:00
abstract:PURPOSE OF REVIEW:Children with chronic kidney disease (CKD) have impaired growth that leads to short stature in adulthood. The problem persists even with successful transplantation and steroid withdrawal protocols. The aim of this review is to provide an overview of the pressing issues related to growth failure in chi...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0000000000000070
更新日期:2014-04-01 00:00:00
abstract:PURPOSE OF REVIEW:Patients seen at pediatric rheumatology are at increased risk of immediate and long-term consequences from sun exposure. The objective of this review is to build awareness of the need of sun protection in pediatric rheumatology patients. RECENT FINDINGS:Sun exposure can lead to disease exacerbations ...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0000000000000798
更新日期:2019-08-01 00:00:00
abstract:PURPOSE OF REVIEW:This review will focus on therapeutic considerations and recent advances in treatment of caustic ingestion injuries. RECENT FINDINGS:A retrospective study suggests that it may be safe to advance the endoscope beyond the first circumferential burn to allow for a more complete assessment of extent of i...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0000000000000257
更新日期:2015-10-01 00:00:00
abstract:PURPOSE OF REVIEW:Rotavirus infection is the most common cause of severe diarrhea disease in infants and young children worldwide and has a major global impact on childhood morbidity and mortality. Vaccination is the only control measure likely to have a significant impact on the incidence of severe dehydrating rotavir...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/01.mop.0000147907.30720.04
更新日期:2005-02-01 00:00:00
abstract::The biology of human breast milk, and the policy of encouraging breastfeeding, continue to be the object of much scientific inquiry. The past year has seen several advances in these fields of nutrition, and this article reviews some of the most interesting and pertinent studies. Four general themes have been apparent ...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/00008480-199906000-00020
更新日期:1999-06-01 00:00:00
abstract::This paper reviews recent studies of attention deficit-hyperactivity disorder, focusing on research with implications for adolescents. Only 10 of the 31 recent investigations discussed employed an exclusively adolescent sample, necessitating considerable extrapolation from studies of children or adults or mixed sample...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/00008480-199608000-00003
更新日期:1996-08-01 00:00:00
abstract:PURPOSE OF REVIEW:This review examines neonatal bacterial skin infections with respect to host immunity, bacterial pathogens, patterns of infection, and new therapeutic approaches. RECENT FINDINGS:Advances have been made in our understanding of innate host defense and the emerging role of cutaneous antimicrobial pepti...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/01.mop.0000171321.68806.bd
更新日期:2005-08-01 00:00:00
abstract:PURPOSE OF REVIEW:Joint hypermobility is a common, although largely ignored physical sign. Joint hypermobility is often asymptomatic but may be a feature of an underlying genetic disorder with systemic manifestations. The present article presents a comprehensive approach to considering joint hypermobility and clinicall...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0000000000000541
更新日期:2017-12-01 00:00:00
abstract:PURPOSE OF REVIEW:To highlight recent advances in management of peanut allergy. RECENT FINDINGS:Peanut allergy presents during early childhood. The prevalence of peanut allergy in children in developed countries appears to be increasing. Several factors, such as peanut-specific or environmental, are hypothesized as co...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0b013e32833d95cb
更新日期:2010-10-01 00:00:00
abstract::Herbal medicaments are in common use. In general, the judicious use of carefully selected and prepared herbal medications seems to cause few adverse effects and may be beneficial. However, toxic effects of these products have been reported with increasing frequency. Infants and children may be even more susceptible to...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/00008480-200104000-00014
更新日期:2001-04-01 00:00:00
abstract:PURPOSE OF REVIEW:To emphasize an exploration of mechanisms of kidney stone disease based on a molecular understanding of excess urinary excretions of calcium, oxalate, cystine, and uric acid. RECENT FINDINGS:Hypercalciuria is discussed relative to mutations in the renal chloride genes CLCN5 and CLCNKB, WNK kinases, A...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/00008480-200404000-00013
更新日期:2004-04-01 00:00:00
abstract:PURPOSE OF REVIEW:Diaper dermatitis is the most common cutaneous diagnosis in infants. In this review, common causes of diaper dermatitis and similarly presenting conditions will be covered, as well as updates on treatments of common diaper dermatitides. RECENT FINDINGS:There have been recent advancements in the treat...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0b013e32835585f2
更新日期:2012-08-01 00:00:00
abstract::Cerebral palsy is caused by a static lesion to the cerebral motor cortex that is acquired before, at, or within 5 years of birth. Multiple causes for the condition exist and include cerebral anoxia, cerebral hemorrhage, infection, and genetic syndromes. Cerebral palsy is commonly classified according to the type of mo...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/00008480-199702000-00017
更新日期:1997-02-01 00:00:00
abstract:PURPOSE OF REVIEW:Adolescents, in particular those that are most disenfranchised, are increasingly at risk of acquiring HIV and, when acquiring HIV, have worse outcomes than adults. This article reviews the recent approaches to combination prevention aiming to optimize the HIV prevention and HIV treatment continua. RE...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0000000000000580
更新日期:2018-02-01 00:00:00
abstract:PURPOSE OF REVIEW:Intrauterine growth restriction (IUGR) is associated with an increased propensity to develop adult-onset disease and is described by the developmental origins of adult disease hypothesis. Sequelae of fetal growth restriction include metabolic disease as well as nonmetabolic disorders. Although it has ...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/mop.0b013e328326773b
更新日期:2009-04-01 00:00:00
abstract::A number of growth factors have been identified that participate in lung growth and repair. The early stages of the repair cascade are important in preventing the later development of fibrosis. Both transforming growth factor-beta and basic fibroblast growth factor can have beneficial effects when given early in some ...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/00008480-199506000-00002
更新日期:1995-06-01 00:00:00
abstract::In the past year, significant progress has been achieved in the research on aquaporins (AQPs), a family of structurally related molecular water channels. Three novel AQPs were identified, giving a total of ten mammalian AQPs. An important step forward in identifying the aqueous pore in AQP molecules was the determinat...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/00008480-199808000-00018
更新日期:1998-08-01 00:00:00
abstract::This review highlights the most significant progress in treatment of constipation occurring in the last year, underlying the advances in pathophysiology, clinical presentation, diagnosis, and treatment of both organic and functional constipation. Recent developments in the field of the pathophysiology of idiopathic ch...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/00008480-199810000-00011
更新日期:1998-10-01 00:00:00
abstract::The use of extracorporeal techniques for the treatment of pediatric diseases has expanded dramatically in the past decade. During this time, experience with both plasmapheresis and renal replacement therapy has become commonplace in ICUs and nephrology programs throughout the world. The indications for these therapies...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/00008480-200206000-00005
更新日期:2002-06-01 00:00:00
abstract:PURPOSE OF REVIEW:We review recommendations from recent publications on the management of fever with antipyretics, the classification and diagnosis of fevers of unknown origin (FUO), and the evaluation of fever in infants under 90 days of age. RECENT FINDINGS:Anxiety about fever persists in the population, while the t...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0b013e32835333e3
更新日期:2012-06-01 00:00:00
abstract:PURPOSE OF REVIEW:Increasing numbers of children are now traveling to high-altitude destinations, and pediatricians often see these children prior to and immediately following their travels. Thus, pediatricians have the opportunity to provide guidance for the prevention of altitude illness and must treat high-altitude ...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0000000000000519
更新日期:2017-08-01 00:00:00
abstract:PURPOSE OF REVIEW:A second-generation 13-valent pneumococcal conjugate vaccine (PCV13) was licensed and recommended for universal immunization of children through age 5 years in 2010. Its introduction is intended to address the residual burden of pneumococcal diseases that persists a decade after the introduction of PC...
journal_title:Current opinion in pediatrics
pub_type: 杂志文章,评审
doi:10.1097/MOP.0b013e328341d1f5
更新日期:2011-02-01 00:00:00