3,758 publications found
Sort by
Bone health in children undergoing solid organ transplantation.

Pediatric solid organ transplant recipients are a unique and growing patient population who are at risk for metabolic bone disease both before and after transplantation. The odds of sustaining a fracture in adulthood are significantly higher if an individual has sustained at least one childhood fracture, therefore, close monitoring before and after transplant is essential. Emerging data in patients with chronic kidney disease mineral and bone disorder (CKD-MBD) and hepatic osteodystrophy highlights the role of fibroblast growth factor 23 in the pathogenesis of metabolic bone disease in these conditions. While dual X-ray absorptiometry (DXA) is the most widely used imaging modality for assessment of bone mass in children, quantitative computer tomography (QCT) is an emerging modality, especially for patients with glucocorticoid-induced osteoporosis. Solid organ transplantation improves organ function and quality of life; however, bone mineral density can decline following transplantation, particularly during the first three to six months. Immunosuppressive medications, including glucocorticoids, are a major contributing factor. Following transplant, treatment should be tailored to achieve mineral homeostasis, correct nutritional deficiencies, and improve physical conditioning. In summary, early identification and treatment of metabolic bone disease can improve the bone health status of pediatric transplant recipients as they enter adulthood. http://links.lww.com/MOP/A71.

A practical approach to caring for atopic dermatitis in children.

Atopic dermatitis is a chronic, systemic disease with primary cutaneous clinical manifestations and is commonly attributed to an exaggerated Th2 inflammatory response. Recent research regarding risk factors, prevention, clinical features, and management of atopic dermatitis will be reviewed. In the last decade, advances have been made in identifying the factors that either confer increased risk for or protection from atopic dermatitis and associated atopy. Progress has also been made in the clinical management of this disease. Promising biomarkers and therapeutically informative characteristics of this disease have been identified in young children with and without the presence of eczema, but much has yet to be elucidated. Progress has also been made in clarifying the advantages and disadvantages of respective medical managements, including but not limited to topical corticosteroids, topical calcineurin inhibitors, phototherapy, systemic immunosuppressants, and targeted immunotherapy. Given that medical management may show variable efficacy in a child, an optimized skin care regimen is of utmost importance as well. Atopic dermatitis is a challenging, chronic systemic disease that incurs significant morbidity in affected children. Although management options have been somewhat disappointing in years past, promising results have been observed in recent advances in targeted immunotherapy.

Epidemiology, causes, and morbidities of stroke in the young.

The purpose is to describe the latest research on epidemiology, causes, and morbidities of stroke in neonates and children. The global incidence of childhood stroke is approximately 2 per 100 000 person-years, which is significantly lower compared to neonates (20-40 per 100 000 live births) and adults (80-90 per 100 000 person-years). Placental abnormalities are a risk factor for perinatal stroke, although cause is usually multifactorial. In children, nonatherosclerotic arteriopathies and arteriovenous malformations are major causes of ischemic and hemorrhagic strokes, respectively. The perinatal period confers a high risk of stroke and can lead to long-term disability, including motor delay, cognitive or speech impairment, and epilepsy. Recent studies suggest that at least 50% of survivors of perinatal stroke have abnormal neurodevelopmental scores in long-term follow up. Childhood stroke is associated with significant morbidity, including epilepsy, motor impairments, and behavioral disability. Recent studies have also identified an association between pediatric stroke and behavioral disorders, such as attention deficit hyperactivity disorder and autism. Perinatal and childhood strokes are important causes of neurological morbidity. Given the low incidence of childhood stroke, prospective research studies on epidemiology, causes, and outcomes remain limited, highlighting the need for continued multisite collaborations.

Progress in the field of hematopoietic stem cell-based therapies for inborn errors of immunity.

Hematopoietic stem cell-based therapies, including allogeneic hematopoietic cell transplantation (HCT) and autologous gene therapy (GT), have been used as curative therapy for many inborn errors of immunity (IEI). As the number of genetically defined IEI and the use of HCT and GT increase, valuable data on outcomes and approaches for specific disorders are available. We review recent progress in HCT and GT for IEI in this article. Novel approaches to prevention of allogeneic complications and experience in adolescents and young adults have expanded the use of HCT. Universal newborn screening for severe combined immunodeficiency (SCID) has led to improved outcome after HCT. Analysis of outcomes of HCT and GT for SCID, Wiskott-Aldrich syndrome (WAS) and chronic granulomatous disease (CGD) reveal risk factors for survival, the impact of specific conditioning regimens, and vector- or disease-specific impacts on efficacy and safety. Preclinical studies of GT and gene editing show potential for translation to the clinic. Emerging data on outcome after HCT for specific IEI support early evaluation and treatment, before development of co-morbidities. Data in large cooperative retrospective databases continues to yield valuable insights clinicians can use in patient selection and choice of therapy.