Current opinion in pediatrics最新文献

Purpose of review: Allogeneic hematopoietic cell transplantation (HCT) is a curative option for many for inborn errors of immunity (IEI). This review highlights recent progress in the field of HCT for IEI.

Recent findings: Alternative donor transplantation continues to expand donor options for patients with IEI. Reduced intensity and reduced toxicity conditioning approaches are being investigated and optimized. Immunomodulatory bridging therapies are yielding impressive progress in outcomes for primary immune regulatory disorders (PIRD) but require further study in prospective trials. Single-institution, multicenter and consortium studies have improved our understanding of factors that affect overall outcomes in IEI and outcomes in Wiskott-Aldrich syndrome (WAS), chronic granulomatous disease (CGD) and PIRD in particular. Data show that second HCT offers a viable chance of cure to some IEI patients. Late effects in IEI HCT survivors are being better characterized. Preclinical studies of chemo(radiation)-free HCT strategies hold promise for decreasing HCT toxicity.

Summary: Improvements in our understanding of HCT donor choice, conditioning regimen, immunomodulatory bridging therapies, diagnostic and post-HCT surveillance testing and late effects continue to yield advancements in the field of HCT for IEI.

Purpose of review: The growth of rich electronic health record (EHR) data and large health databases has introduced new opportunities to link individuals together into households and relational networks. These 'linked relational networks' hold promise for providing family-level care and studying intergenerational epidemiology and clinical outcomes. However, as linked relational networks become more commonly available in EHRs and research databases, it is critical to understand their challenges and limitations.

Recent findings: Matching algorithms are being used to create linked relational networks in EHR and health databases. Clinically, these algorithms have been most useful to provide dyadic maternal-newborn care. In research, studies using these algorithms investigate topics ranging from the pharmacoepidemiology of parental drug exposure on childhood health outcomes, to heritability of chronic conditions, to associations between parental and child healthcare access and service delivery. However, ethical and technical challenges continue to limit use of these algorithms. There is also a critical research gap in the external validity of these matching algorithms.

Summary: Linked relational networks are in widespread use in pediatric clinical care and research. More research is needed to understand the scope, limitations, and biases inherent in existing matching strategies.

Purpose of review: To summarize the impact of financial hardship on children whose parents have been incarcerated, describe both existing cash transfer and guaranteed income programs, and highlight their impact on child and family well being.

Recent findings: Emerging data on guaranteed income programs for formerly incarcerated adults indicates that the funds improve recipient health and legal system outcomes and allow participants to spend funds on stabilizing themselves and their families. Guaranteed income programs in the broader population similarly highlight the use of funds to support families' basic needs and improved parent-child relationships, but more data are needed to understand the impact on child health and well being among families impacted by the criminal legal system.

Summary: Over 5 million children experience parental incarceration, which directly contributes to financial hardship and subsequent negative health outcomes. Cash transfers may assist these families, but policies and administrative burdens limit the ability of existing programs to fully address financial hardship. Guaranteed income programs may therefore serve a unique role in supporting the financial needs of families impacted by incarceration. More longitudinal data focusing on child health outcomes are necessary to fully understand the impact of guaranteed income of children and families impacted by parental incarceration.

Purpose of review: New social risk screening standards and quality metrics reward or penalize healthcare delivery organizations for social risk screening. After summarizing the recent literature on social risk screening in pediatric healthcare settings we consider how this evidence - and persistent evidence gaps - might inform future standards development.

Recent findings: Reported social risk screening rates, measures, and modality differ greatly across recent work. Although many caregivers report acceptability of screening, experiences and expectations around effective follow-up vary. Likewise, although most frontline clinical providers find screening acceptable, they report significant implementation challenges related to time constraints, insufficient workforce, and availability of social services. Qualitative findings suggest opportunities to improve screening implementation. Literature examining the impacts of screening continues to focus on immediate posited impacts of assistance programs; few studies assess health outcomes.

Summary: The existing literature does not clearly indicate whether, when, how, or for whom social risk screening standards focused on screening quantity will contribute to child health or health equity. Informed by studies on patient experience, quality measures focused on screening quantity (e.g. how many individuals are screened) should be paired with efforts to improve screening quality (i.e. patient/caregiver screening experience) as well as social and health outcomes.

Purpose of review: To highlight the clinical presentation, diagnostic approach, and management of acquired inflammatory demyelinating syndromes in children.

Recent findings: The identification of myelin oligodendrocyte glycoprotein antibody-associated disease in 2017 and evolving evidence regarding best practices for management has had a significant impact on pediatric neuroimmunology, as has the shift in treatment of pediatric-onset multiple sclerosis, with the use of high-efficacy disease-modifying therapies early in the disease course.

Summary: With expanding awareness and growing interest in pediatric onset neuroinflammatory conditions, the number of children diagnosed with acquired demyelinating syndromes is rising. It is critical to refine our understanding of the underlying pathophysiological mechanisms in these disorders to provide the most effective care. Much of our practice continues to be modeled on adult care, and further large-scale pediatric studies are necessary to explore the natural history and assess the safety and efficacy of immunotherapies in childhood-onset demyelinating diseases.

Purpose of review: Patients with a functionally single ventricle (SV) are palliated with a series of procedures leading to a Fontan circulation. Over the life span, a substantial proportion of SV patients develop heart failure that can arise from circulatory or ventricular failure. Diastolic dysfunction (DD) is an important determinant of adverse outcomes in SV patients. However, assessment and categorization of DD in the SV remains elusive. We review recent literature and developments in assessment of DD in the SV and its relation to clinical outcomes.

Recent findings: DD is prevalent in the SV and associated with worse outcomes. Occult DD can be exposed with provocative testing by exercise or preload challenge during catheterization. Likewise, sensitivity to detect DD may be increased via assessment of atrial function and strain imaging. Recent studies revisiting previous concepts such as incoordinate diastolic wall motion show that these are associated with SV end-diastolic pressures and post-Fontan recovery, yielding accessible DD assessment. Emerging technologies such as ultrafast ultrasound (UFUS) can provide noninvasive assessment of myocardial stiffness, inefficient diastolic flow patterns and intraventricular pressure gradients, thereby yielding new tools and insights into diastolic myocardial and hemodynamic properties.

Summary: Characterizing DD in the SV continues to have substantial limitations, necessitating synthesis of multiple parameters into an overall assessment, accounting for their change over time, and in the context of the patient's clinical status. New and emerging techniques may help advance DD assessment and the ability to track response to treatment of new targets.

Purpose of review: To provide an updated review of novel and complementary treatment approaches for children and adolescents with attention-deficit/hyperactivity disorder.

Recent findings: The evidence for complementary attention-deficit/hyperactivity disorder treatments is often promising, but limited to small, unblinded studies. Recent evidence from larger, more rigorous studies reveals that most of these treatments have modest efficacy. Omega-3 polyunsaturated fatty acids, saffron, broad spectrum micronutrients, and physical exercise have potential benefits that seem to outweigh known risks. However, neurofeedback, cognitive training, and trigeminal nerve stimulation need further research to determine whether specific sub-groups of children/adolescents with attention-deficit/hyperactivity disorder would benefit long-term with their associated tolerable risks.

Summary: There is not sufficient evidence for complementary treatments to be recommended as substitutes for first-line pharmacological and psychosocial treatment options. Nonetheless, some adjuvant therapies to currently recommended attention-deficit/hyperactivity disorder treatments can be safe. Physicians should be familiar with existing and emerging complementary treatments to help guide families.

Purpose of review: Pulmonary hypertension (PH) is commonly observed in premature infants with bronchopulmonary dysplasia (BPD) and is associated with poor outcomes and increased mortality. This review explores the management of this intricate condition of the pulmonary vasculature, which exhibits heterogeneous effects and may involve both arterial and postcapillary components.

Recent findings: Current management of BPD-PH should focus on optimizing ventilatory support, which involves treatment of underlying lung disease, transitioning to a chronic phase ventilation strategy and evaluation of the airway. Data on management is limited to observational studies. Diuretics are considered a part of the initial management, particularly in infants with right ventricular dilation. In many cases, pulmonary vasodilator therapy is required to induce pulmonary arterial vasodilation, reduce right ventricular strain, and prevent coronary ischemia and heart failure. Echocardiography plays a pivotal role in guiding treatment decisions and monitoring disease progression.

Summary: BPD-PH confers a heightened risk of mortality and long-term cardio-respiratory adverse outcomes. Echocardiography has been advocated for screening, while catheterization allows for confirmation in select more complex cases. Successful management of BPD-PH requires a multidisciplinary approach, focusing on optimizing BPD treatment and addressing underlying pathologies.