Infectious Prophylaxis in Paediatric Oncology and Stem Cell Transplantation
Abstract
The advances in the survival of children with cancer and following stem cell transplant have, in part, been due to more effective infection prevention strategies. Morbidity and mortality following bacterial, viral and fungal infection remain significant necessitating further refinement of existing prophylactic strategies. Novel antiviral agents, newer and better tolerated antifungal drugs, antibiotic bacterial prophylaxis and adoptive immunotherapy in high-risk groups are potential approaches for which supportive evidence is emerging. Questions remain as to the optimal approach to infectious prop...
Source: Current Pediatrics Reports - March 14, 2015 Category: Pediatrics Source Type: research
Acute Kidney Injury (AKI): Current Thoughts and Controversies in Pediatrics
Abstract
AKI is a common syndrome that is independently associated with increased mortality. This review hopes to address recent advances in scoring systems as they have advanced and become paediatric specific. Causes of AKI are widespread but specifically sepsis with relevance of molecular mechanisms will be reviewed. Prevention of AKI is important and needs attention to nephrotoxic drugs specifically. Fluid management in AKI following resuscitation can be specifically challenging requiring removal if fluid overloaded and use of continuous renal replacement therapy if available. Biomarkers remain very to...
Source: Current Pediatrics Reports - February 19, 2015 Category: Pediatrics Source Type: research
The Clinical Trials Gap for Adolescents and Young Adults with Cancer: Recent Progress and Conceptual Framework for Continued Research
Abstract
Over the past 30 years, adolescents and young adults (AYA, 15–39 years of age) with cancer have shown significantly less improvement in survival than younger and older patients. Because evidence suggests this may be related to their low participation in cancer clinical trials, increasing accrual to these trials has become a priority for closing this “AYA gap.” This paper reviews data documenting low AYA enrollment, presents a conceptual framework for research and intervention (Clinical Trials Pathway to Enrollment) and summarizes recent developments in the United States National Cancer I...
Source: Current Pediatrics Reports - February 18, 2015 Category: Pediatrics Source Type: research
Survival and Quality of Life Following Treatment for Sarcoma
Abstract
Advances in multimodality therapy have improved the survival rates of pediatric sarcomas over the last 40 years, with current rates over 65 %. However, the advances have not been equal, and patients with metastatic and relapsed disease continue to have a poor prognosis. This review focuses on the epidemiology, prognostic factors, treatment, and survival of the three most common pediatric sarcomas: osteosarcoma, Ewing sarcoma, and rhabdomyosarcoma. It highlights a new collaboration to study osteosarcoma, recent advances in Ewing sarcoma treatment with increased intensity of chemotherapy timing, ...
Source: Current Pediatrics Reports - February 11, 2015 Category: Pediatrics Source Type: research
Improving the Transition to Adult Care for Young People with Chronic Kidney Disease
Abstract
The transition from pediatric- to adult-oriented care typically occurs during emerging adulthood, a developmental stage during which the risks of poor treatment adherence and adverse outcomes are elevated. Inadequate support during this change in care environment may augment the risks. With a focus on chronic kidney disease, we will briefly review the evidence linking transition from pediatric- to adult-oriented care to adverse medical, social, and quality of life outcomes, and outline potential reasons for the associations between transition and adverse outcomes. Then we will summarize the recen...
Source: Current Pediatrics Reports - January 17, 2015 Category: Pediatrics Source Type: research
Hematopoietic Stem Cell Transplantation for Severe Combined Immunodeficiency
Abstract
Hematopoietic stem cell transplantation is an effective approach for the treatment of severe combined immunodeficiency (SCID). However, SCID is not a homogeneous disease, and the treatment required for successful transplantation varies significantly between SCID subtypes and the degree of HLA mismatch between the best available donor and the patient. Recent studies are beginning to more clearly define this heterogeneity and how outcomes may vary. With a more detailed understanding of SCID, new approaches can be developed to maximize immune reconstitution, while minimizing acute and long-term toxi...
Source: Current Pediatrics Reports - January 13, 2015 Category: Pediatrics Source Type: research
The Genetic and Molecular Basis of Severe Combined Immunodeficiency
Abstract
Severe combined immunodeficiency (SCID) is a life-threatening disease caused by a heterogeneous group of genetic defects. It is characterized by profound defects of T-cell development, also affecting B and NK cells in some cases. Since the first molecular identification of a causal gene for SCID in 1985, 14 more molecular causes have been identified in patients with a classical SCID phenotype, with no T cells. Some genetic defects specifically block lymphocyte ontogeny, whereas others affect T-cell function and a few cause extra-hematopoietic alterations with a rare complex phenotype. Over...
Source: Current Pediatrics Reports - December 24, 2014 Category: Pediatrics Source Type: research
Newborn Screening for Severe Combined Immunodeficiency
Abstract
Newborn screening (NBS) for severe combined immunodeficiency (SCID) utilizing the T-cell receptor excision circle assay is a sensitive and specific method to detect T-cell lymphopenia in early infancy. Starting in 2008, several programs have implemented SCID NBS with successful detection of SCID-affected infants; currently over two thirds of U.S. infants receive SCID NBS. Population-based, unbiased screening has established an incidence of SCID to be 1 in 58,000 births, and has revealed a distribution of SCID genotypes different from prior reports from specific SCID treatment centers. Detecting S...
Source: Current Pediatrics Reports - December 24, 2014 Category: Pediatrics Source Type: research
Gene Therapy for SCID
Abstract
Severe combined immunodeficiencies (SCIDs) are a group of rare genetic diseases characterized by profound abnormalities of cellular and humoral immunity. They have been identified as ideal candidates for treatment by gene therapy and have led the development of gene therapy for other bone marrow disorders. SCID-X1 and adenosine deaminase SCID have been successfully treated with hematopoietic stem cell gene therapy (HSC-GT) and patients show impressive levels of immune reconstitution. Initial clinical trials employed γ-retroviral vectors, but leukaemia arose in five SCID-X1 patients due to insert...
Source: Current Pediatrics Reports - December 21, 2014 Category: Pediatrics Source Type: research
Measures of GFR in Health and Disease
Abstract
Glomerular filtration rate (GFR) is the most important measure of kidney function used for chronic kidney disease screening, dosing medications, and following kidney disease progression. Gold standard GFR measurement is inulin clearance; however, GFR is most commonly measured by plasma disappearance of exogenously administered substances. Recent improvements in GFR measurement have been made, and understanding the limitations of different methods is important. In clinical practice, GFR is typically estimated using equations. Most equations are based on serum creatinine, but other markers, in part...
Source: Current Pediatrics Reports - December 12, 2014 Category: Pediatrics Source Type: research
Nephrotic Syndrome: State of the Art
This article overviews the pathogenesis and management of idiopathic nephrotic syndrome, a common childhood glomerulopathy. While initial evidence supported an imbalance of T helper responses in patients with nephrotic syndrome, recent studies suggest alterations in both innate and adaptive immune responses, including evidence for impaired T regulatory function. The central role of the podocyte in causing proteinuria is confirmed by the observation of mutations in key podocyte proteins in steroid-resistant nephrotic syndrome and experimental evidence of altered podocyte signaling and cytoskeletal organization, which might ...
Source: Current Pediatrics Reports - December 6, 2014 Category: Pediatrics Source Type: research
Rituximab Treatment for Nephrotic Syndrome in Children
Abstract
In the past 10 years, many reports have suggested that rituximab, a chimeric anti-CD20 monoclonal antibody, is effective for children with complicated, frequently relapsing or steroid-dependent nephrotic syndrome (FRNS/SDNS). However, those reports were case reports, case series, retrospective surveys, and single-arm or short-term trials. Therefore, well-designed controlled trials are required to establish the value of rituximab in this condition. To evaluate the efficacy and safety of rituximab in childhood-onset, complicated FRNS/SDNS, a multicenter, double-blind, randomized, placebo-controlle...
Source: Current Pediatrics Reports - December 6, 2014 Category: Pediatrics Source Type: research
Genes in FSGS: Diagnostic and Management Strategies in Children
Abstract
FSGS is a molecularly heterogeneous disease, and can be classified into primary or secondary disease. Primary FSGS can be due to immune or genetic causes. To date, more than 20 genes have been implicated in FSGS. The discovery of so many FSGS genes, together with the advent of next-generation sequencing, has led to a paradigm shift in the genetic diagnosis and management of this disease. However, genetic screening in FSGS may not be so straightforward because of the difficulty in selection and prioritization of genes. It is challenging to appreciate the prevalence of mutations in the different ge...
Source: Current Pediatrics Reports - December 3, 2014 Category: Pediatrics Source Type: research
Donor Human Milk: No Longer A Place For Formula in the Neonatal Intensive Care Unit?
This report discusses the evidence for donor human milk in the nutritional support of the preterm infant. (Source: Current Pediatrics Reports)
Source: Current Pediatrics Reports - October 10, 2014 Category: Pediatrics Source Type: research
Necrotizing Enterocolitis in Preterm Infants is Related to Enteral Feeding, But the Mechanisms Remain Uncertain and Have Changed Over Time
In this report, the impact of feeding on NEC will be reviewed, and it is suggested that enteral feedings can have a protective role in the disease, while conversely in other situations, enteral feedings might initiate changes contributing to this devastating condition. It is concluded that our understanding of enteral feeding and NEC has changed over time, and new insights continue to provide clues to the pathogenesis of this challenging condition. (Source: Current Pediatrics Reports)
Source: Current Pediatrics Reports - October 2, 2014 Category: Pediatrics Source Type: research
