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Risk and surrogate benefit for pediatric Phase I trials in oncology: A systematic review with meta-analysis.
Waligora, M, Bala, MM, Koperny, M, Wasylewski, MT, Strzebonska, K, Jaeschke, RR, Wozniak, A, Piasecki, J, Sliwka, A, Mitus, JW, et al
PLoS medicine. 2018;(2):e1002505
Abstract
BACKGROUND Pediatric Phase I cancer trials are critical for establishing the safety and dosing of anti-cancer treatments in children. Their implementation, however, must contend with the rarity of many pediatric cancers and limits on allowable risk in minors. The aim of this study is to describe the risk and benefit for pediatric cancer Phase I trials. METHODS AND FINDINGS Our protocol was prospectively registered in PROSPERO (CRD42015015961). We systematically searched Embase and PubMed for solid and hematological malignancy Phase I pediatric trials published between 1 January 2004 and 1 March 2015. We included pediatric cancer Phase I studies, defined as "small sample size, non‑randomized, dose escalation studies that defined the recommended dose for subsequent study of a new drug in each schedule tested." We measured risk using grade 3, 4, and 5 (fatal) drug-related adverse events (AEs) and benefit using objective response rates. When possible, data were meta-analyzed. We identified 170 studies meeting our eligibility criteria, accounting for 4,604 patients. The pooled overall objective response rate was 10.29% (95% CI 8.33% to 12.25%), and was lower in solid tumors, 3.17% (95% CI 2.62% to 3.72%), compared with hematological malignancies, 27.90% (95% CI 20.53% to 35.27%); p < 0.001. The overall fatal (grade 5) AE rate was 2.09% (95% CI 1.45% to 2.72%). Across the 4,604 evaluated patients, there were 4,675 grade 3 and 4 drug-related AEs, with an average grade 3/4 AE rate per person equal to 1.32. Our study had the following limitations: trials included in our review were heterogeneous (to minimize heterogeneity, we separated types of therapy and cancer types), and we relied on published data only and encountered challenges with the quality of reporting. CONCLUSIONS Our meta-analysis suggests that, on the whole, AE and response rates in pediatric Phase I trials are similar to those in adult Phase I trials. Our findings provide an empirical basis for the refinement and review of pediatric Phase I trials, and for communication about their risk and benefit.
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Integrating lipid screening with ideal cardiovascular health assessment in pediatric settings.
Blackett, P, George, M, Wilson, DP
Journal of clinical lipidology. 2018;(6):1346-1357
Abstract
Pediatric lipid screening and management with the aim of reducing and preventing adult disease is an internationally accepted concept, and guidelines have been published in several countries. However, implementation by the practicing pediatric community in the United States has been less than expected and delays have been attributed to uncertainty among providers. Reduced screening rates have also been reported for conditions contributing to arterial wall pathology such as obesity, hypertension, and prediabetes despite accumulating evidence that detection and intervention can lead to risk reversal. Consistent with graded and evidence-based national guidelines for comprehensive cardiovascular risk assessment and management, we present how the American Heart Association ideal cardiovascular health (ICVH) model can be integrated with lipid screening, and how it can be compatible with comprehensive pediatric lipidology practice and enhanced familial hypercholesterolemia detection. Since being introduced and retrospectively validated in adults and children in cross-sectional studies, ICVH evaluates thresholds for seven ideal health metrics representing measurements of obesity, dyslipidemia, diabetes risk, and blood pressure, and includes exercise, diet, and smoking behaviors. When each metric is valued as a point, the maximum health score is 7, but national surveys have shown unacceptable low scores in adolescence. Inverse correlation of scores with arterial structural change supports use of ICVH as a collection of treatable targets forming a cardiovascular prevention construct including and supporting lipid screening in pediatric settings, but implementation in clinical practice requires more expertise and administrative support than lipid screening alone.
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Preoperative fasting guidelines in pediatric anesthesia: are we ready for a change?
Andersson, H, Schmitz, A, Frykholm, P
Current opinion in anaesthesiology. 2018;(3):342-348
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Abstract
PURPOSE OF REVIEW Study after study shows that prolonged fasting before anesthesia is common in children. Pediatric anesthesiologists around the world are concerned that the current guidelines may be part of the problem. This review focuses on what can be done about it. RECENT FINDINGS We discuss new insights into the physiology of gastric emptying of different categories of food and drink. The evidence for negative effects of prolonged fasting occurring in spite of implementation of the current guidelines is examined. We also critically appraise the concept of a strict association between fasting time and the risk of aspiration and discuss recent studies in which children have been allowed clear fluids less than 2 h before anesthesia induction. SUMMARY Accumulating evidence indicates that changes of the current guidelines for preoperative fasting should be considered for children undergoing elective procedures. VIDEO ABSTRACT.
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Dietary and Nutrition Recommendations in Pediatric Primary Care: A Call to Action.
Harrison, SE, Greenhouse, D
Southern medical journal. 2018;(1):12-17
Abstract
Rapid increases in childhood obesity reflect widespread changes in diet and physical activity, which are of significant concern because obesity increases a child's risk for negative health outcomes and frequently creates a pathway to adult obesity. Diet and nutrition play a key role in maintaining energy balance and preventing weight gain; therefore, they are obvious targets for obesity prevention efforts. Primary care providers are in an optimal position to convey messages about healthy eating to children and families, and specific guidelines exist for how to incorporate diet and nutrition prevention into primary care. Providers should be aware of the scientific evidence supporting these preventive practices. This review provides a summary of recommendations for integrating diet and nutrition into pediatric primary care and presents a call to action to make diet/nutrition assessment, counseling, and intervention routine aspects of the preventive care visit.
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An overview of the clinical management of cyclic vomiting syndrome in childhood.
Romano, C, Dipasquale, V, Rybak, A, Comito, D, Borrelli, O
Current medical research and opinion. 2018;(10):1785-1791
Abstract
This narrative review provides an update on cyclic vomiting syndrome pathogenesis, diagnosis and management, based upon studies published after the 2008 North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) official recommendations. The review began with a comprehensive PubMed/Medline search for "cyclic vomiting syndrome", "periodic syndromes" and "pediatrics" from 2000 up to October 2017. Additional papers were identified by reviewing the re-ference lists of retrieved publications. Cyclic vomiting syndrome is a severe, debilitating disorder of the brain-gut axis with unclear pathogenesis, that significantly affects long-term quality of life of affected children and their families. The 2008 NASPGHAN recommendations defined the major clinical, diagnostic and therapeutic peculiarities. Over the last 10 years, advancements in pathogenesis and diagnostic criteria have been made, and new prophylactic and therapeutic strategies have been proposed. These aspects are discussed in this manuscript. For the pediatrician, the major aim is to have early clinical suspicion to avoid diagnostic delay and to start adequate, phase-related, symptom-tailored management.
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Magnesium sulfate in pediatric anesthesia: the Super Adjuvant.
Eizaga Rebollar, R, García Palacios, MV, Morales Guerrero, J, Torres, LM
Paediatric anaesthesia. 2017;(5):480-489
Abstract
Magnesium is an essential chemical element in all organisms, intervening in most cellular enzymatic reactions; thus, its importance in homeostasis and as a therapeutic tool in highly challenging patients such as pediatrics. The primary purpose of this paper was to review the role of magnesium sulfate as an adjuvant drug in pediatric anesthesia. This compound already has the scientific backing in certain aspects such as analgesia or muscle relaxation, but only theoretical or empirical backing in others such as organ protection or inflammation, where it seems to be promising. The multitude of potential applications in pediatric anesthesia, its high safety, and low cost make magnesium sulfate could be considered a Super Adjuvant.
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Recommended strategies for the oral administration of paediatric medicines with food and drinks in the context of their biopharmaceutical properties: a review.
Martir, J, Flanagan, T, Mann, J, Fotaki, N
The Journal of pharmacy and pharmacology. 2017;(4):384-397
Abstract
OBJECTIVES This review focuses on the recommended strategies for the oral administration of paediatric medicines with food in the context of their biopharmaceutical properties. KEY FINDINGS Acceptability of oral medicines in young patients is more challenging than in adult patients. Mixing oral dosage forms with foods and drinks is sometimes suggested to administer a specific dose and enhance compliance in the paediatric population. In this review, the strategies for the co-administration of paediatric medicines with food and drinks are discussed. Current administration practices as reported by healthcare professionals and parents/carers are compared with the relevant guidelines. Differences in the type of vehicles recommended to be used and actually used in current practice were identified. Correlations of the type of food recommended, the type of formulation and the drug's biopharmaceutical classification system (BCS) class were performed and revealed that recommendations should be made on a case-by-case basis. SUMMARY The propensity for physiochemical or bioavailability changes that may occur from the co-administration of medicines with food and drinks in the paediatric population should be considered, and harmonisation of the recommended administration strategies is needed.
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Role of Contrast-Enhanced Ultrasound (CEUS) in Paediatric Practice: An EFSUMB Position Statement.
Sidhu, PS, Cantisani, V, Deganello, A, Dietrich, CF, Duran, C, Franke, D, Harkanyi, Z, Kosiak, W, Miele, V, Ntoulia, A, et al
Ultraschall in der Medizin (Stuttgart, Germany : 1980). 2017;(1):33-43
Abstract
The use of contrast-enhanced ultrasound (CEUS) in adults is well established in many different areas, with a number of current applications deemed "off-label", but the use supported by clinical experience and evidence. Paediatric CEUS is also an "off-label" application until recently with approval specifically for assessment of focal liver lesions. Nevertheless there is mounting evidence of the usefulness of CEUS in children in many areas, primarily as an imaging technique that reduces exposure to radiation, iodinated contrast medium and the "patient-friendly" circumstances of ultrasonography. This position statement of the European Federation of Societies in Ultrasound and Medicine (EFSUMB) assesses the current status of CEUS applications in children and makes suggestions for further development of this technique.
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[Requirements for pediatric parenteral nutrition].
Martínez Costa, C, Pedrón Giner, C
Nutricion hospitalaria. 2017;(Suppl 3):14-23
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10.
Knowns and unknowns in the care of pediatric familial hypercholesterolemia.
Martin, AC, Gidding, SS, Wiegman, A, Watts, GF
Journal of lipid research. 2017;(9):1765-1776
Abstract
Familial hypercholesterolemia (FH) is a common genetic disorder that causes elevated LDL cholesterol levels from birth. Untreated FH accelerates atherosclerosis and predisposes individuals to premature coronary artery disease (CAD) in adulthood. Mendelian randomization studies have demonstrated that LDL cholesterol has both a causal and cumulative effect on the risk of CAD. This supports clinical recommendations that children with FH commence pharmacological treatment from the age of 8 to 10 years, to reduce the burden of hypercholesterolemia. Worldwide, the majority of children with FH remain undiagnosed. Recent evidence suggests that the frequency of FH is at least 1 in 250 and this constitutes a public health issue. We review and identify the knowns and unknowns concerning the detection and management of pediatric FH that impact on the developing model of care for this condition.