Standard Of Care For Children Research Articles

Effectiveness of a nurse-led one-to-one education programme in addition to standard care in children with atopic dermatitis: a multicentre randomized control trial.

Therapeutic patient education (TPE) is recommended for children with atopic dermatitis (AD), but no consensus has been reached on the optimal tailoring of delivery. While repeated multidisciplinary group education sessions have shown effectiveness, the benefits of one-on-one educational interventions led by nurses for children with AD have not yet been assessed. To assess the benefits of additional, well-structured, 1-h nurse-led individual TPE interventions in children with AD and their families compared with standard care alone. Children with moderate-to-severe AD and their parents were randomized to receive a 1-h nurse-led education session in addition to standard care vs. standard care alone. The primary outcome was the area under the curve (AUC) of the SCORing of Atopic Dermatitis index (SCORAD) from baseline to week 24 (lower AUC values represent better long-term control of the disease). In our study, 176 patients were randomized across 11 centres, and 153 were included in the full analysis set. The mean (SD) age was 4.47 (4.57) years. By week 24, there were no significant differences in the AUCs of the SCORAD between the two groups (P = 0.3). Secondary outcomes including patient-reported severity and quality of life [AUCs of the patient-oriented SCORAD (PO-SCORAD) and Infants' Dermatitis Quality of Life Index (IDLQI), Children's Dermatitis Quality of Life Index (CDLQI) and Family Dermatitis Quality of Life Index (FDLQI)] were not significantly different between the two groups. The only significant change observed in the intervention group, when compared with the one receiving standard care, was a decrease in topical steroid phobia, as assessed by the topical corticosteroid phobia (TOPICOP) score. Prespecified subgroup analyses showed that disease severity in the intervention group was significantly lower throughout the study, compared with the standard-care group when participants had moderate AD at baseline (n = 47); while participants with severe AD at baseline (n = 106) did not show benefit from the intervention. Participants showed no additional benefit from the intervention regardless of age group. This study did not show any additional effectiveness, in long-term severity control, of a 1-h nurse-led TPE intervention in children with AD treated with standard care, compared with those treated with standard care alone. However, it should be noted that the intervention reduced the fear of using topical steroids and may be beneficial for patients in the subgroup with moderate AD.

Intensive Multidisciplinary Feeding Day Programs in the United States: A Report Regarding the Treatment Landscape

Intensive multidisciplinary intervention is increasingly recognized as the standard of care for children with complex feeding problems. Much, however, remains unknown about this treatment model. This current qualitative, prospective study sought to identify intensive multidisciplinary day hospital programs operating in the US, describe the treatment approach, and summarize current capacity.

Emergency Department Sepsis Triage Scoring Tool Elements Associated With Hypotension Within 24 Hours in Children With Fever and Tachycardia.

Pediatric sepsis screening is becoming the standard of care for children presenting to the emergency department (ED) and has been shown to improve recognition of severe sepsis, but it is unknown if these screening tools can predict progression of disease. The objective of this study was to determine if any elements of a sepsis triage trigger tool were predictive of progression to hypotensive shock in children presenting to the ED with fever and tachycardia. This study is a retrospective case-control study of children ≤18 years presenting to an ED with fever and tachycardia, comparing those who went on to develop hypotensive shock in the subsequent 24 hours (case) to those who did not (control). Primary outcome was the proportion of encounters where the patient had specific abnormal vital signs or clinical signs as components of the sepsis triage score. The secondary outcomes were the proportion of encounters where the patient had a sepsis risk factor. During the study period, there were 94 patients who met case criteria and 186 controls selected. In the adjusted multivariable model, the 2 components of the sepsis triage score that were more common in case patients were the presence of severe cerebral palsy (adjusted odds ratio, 9.4 [3.7, 23.9]) and abnormal capillary refill at triage (adjusted odds ratio, 3.1 [1.4, 6.9]). Among children who present to a pediatric ED with fever and tachycardia, those with prolonged capillary refill at triage or severe cerebral palsy were more likely to progress to decompensated septic shock, despite routine ED care.

Impact of gastrointestinal and urological symptoms in children with myotonic dystrophy type 1

Gastrointestinal and urological symptoms are frequently reported by people with myotonic dystrophy type 1 (DM1) but have remained understudied. In a cross-sectional study, frequency, nature, treatment and impact of gastrointestinal and urological symptoms in children with DM1 aged 5–18 years were assessed. We included 58 children (30 males, 28 females) with a mean age of 13 years; 74.1 % reported at least one gastrointestinal symptom. Abdominal pain was the most frequently reported symptom (51.7 %), followed by dysphagia (41.8 %), diarrhoea (36.2 %), encopresis (36.0 %), constipation (32.7 %), bloating and flatulence (both 25.9 %). The most frequently reported urological symptoms were difficulty with toilet training (59.3 %), urinary incontinence (22.0 %), enuresis nocturna (10.3 %) and voiding (23.5 % hesitancy, 4.8 % intermittency and 13.8 % dysuria). The majority considered urological and gastrointestinal symptoms to have a negative influence on their daily life; 22.4 % of parents reported severe influence on daily family life (shame, social restrictions, school absence and concerns for their children's future). Considering the high prevalence of urological and gastrointestinal symptoms in children with DM1 and their influence on daily life it is key to correctly recognize, diagnose and treat these symptoms. We recommend screening for gastrointestinal and urological symptoms in the standard of care for children with DM1.

Socioeconomic efficiency of neonatal screening for spinal muscular atrophy in the Russian Federation

Background. Spinal muscular atrophy (SMA) is a severe rare disease that has been widely discussed in recent years. Achievements in etiopathogenetic therapy and the social significance of the disease (child population, high mortality), the cost of treatment attracted the attention of the public and the goverment, which, among other things, led to the formation of a separate area with the creation of a fund to finance the treatment of patients with orphan diseases.Aim. To conduct an analysis of the socio-economic efficiency of mass neonatal screening for SMA in the Russian Federation.Materials and methods. A survey of patients (their parents) and doctors was conducted. The current clinical guidelines and the standard of medical care for children with SMA were studied. The cost of medicines is taken from the State Register of Maximum Selling Prices. If the drug is not included in the List of Vital Essential and Necessary Drugs, cost information from the procurement data is used.Results. The socioeconomic burden of SMA on identified patients was about 3,994,289,548 rubles per year prior to screening. The very introduction of mass neonatal screening will amount to about 679,224,000 rubles in year. At the same time, a disease detected in a timely manner due to neonatal screening and timely prescribed effective treatment can lead to a reduction in the cost of specialized and palliative care by 54,073,271 rubles, direct non-medical costs by 88,137,423 rubles, and indirect costs by 154,197,900 rubles per year, which together is more than 7 % of the calculated burden of SMA.Conclusion. The introduction of mass screening will lead to the fact that the number of annually detected patients will increase from current values to the actual value of the prevalence when registering patients with milder forms of SMA. The need for drug supply with drugs and medical care in general will increase. At the same time, children diagnosed with SMA will not die in the early years of life, their survival rate, life expectancy will increase, the quality of life will improve, infant mortality will decrease, which is the main task of neonatal screening and one of the goals of the National Healthcare Project.

Tisagenlecleucel vs. historical standard of care in children and young adult patients with relapsed/refractory B-cell precursor acute lymphoblastic leukemia

In the absence of randomized controlled trials comparing tisagenlecleucel vs. standard of care (SOC) in pediatric and young adult patients with relapsed or refractory acute lymphoblastic leukemia (r/r ALL), the objective was to compare the efficacy of tisagenlecleucel with historical controls from multiple disease registries using patient-level adjustment of the historical controls. The analysis is based on patient-level data of three tisagenlecleucel studies (ELIANA, ENSIGN and CCTL019B2001X) vs. three registries in Germany/Austria. Statistical analyses were fully pre-specified and propensity score weighting of the historical controls by fine stratification weights was used to adjust for relevant confounders identified by systematic literature review. Results showed high comparability of cohorts after adjustment with absolute SMD ≤ 0.1 for all pre-specified confounders and favorable outcomes for tisagenlecleucel compared to SOC for all examined endpoints. Hazard ratios for OS(Intention to treat)ITT,adjusted, EFS(Full analysis set)FAS,naïve and RFSFAS,naïve were 0.54 (95% CI: 0.41–0.71, p < 0.001), 0.67 (0.52–0.86, p = 0.001) and 0.77 (0.51–1.18, p = 0.233). The OSITT, adjusted, EFSFAS,naïve and RFSFAS,naive survival probability at 2 years was 59.49% for tisagenlecleucel vs. 36.16% for SOC population, 42.31% vs. 30.23% and 59.60% vs. 54.57%, respectively. Odds ratio for ORRITT,adjusted was 1.99 (1.33–2.97, p < 0.001). Results for OS and ORR were statistically significant after adjustment for confounders and provide evidence supporting a superiority of tisagenlecleucel in r/r ALL given the good comparability of cohorts after adjustment for confounders.

M-Follow up for zinc adherence by caretakers of children with acute watery diarrhoea: A randomized controlled trial.

The standard of care for children with acute watery diarrhea (AWD) with no dehydration comprises oral rehydration solution, zinc, and feeding advice. Adherence to zinc therapy may be an issue in the management of acute watery diarrhoea. Mobile phones are used by over 90% of the population in Tanzania, thus good means to improve adherence to prescribed medication and/or attendance to follow-up visits. The objective of this study was to see whether m-follow-up improves adherence rate to zinc therapy, possible reasons for non-adherence, in children with diarrhea.: A randomized controlled trial was carried out in a suburban municipality in Dar-es-Salaam. Block randomization of participants was carried out with a block size of 4 and a 1:1 ratio of intervention: control. The intervention group comprised participants who were to be followed up using text messages and voice calls; the control group was to be followed up in outpatient. The outcome of interest was adherence to the full course of 10 days' oral zinc, reasons for nonadherence and acceptability. Chi-square was used to compare the categorical variables. δ, the targeted difference in adherence between arms, was pre-set at 20%. The total number of participants were 196, of which 98 participants were enrolled in each arm. Full adherence to the 10-day course of zinc sulphate in children with AWD and no dehydration was 84.1% in the control arm and 89.7% in the m-follow up group (P = 0.33). m-follow up significantly improved physical attendance at 14-day clinic visit compared to control group (39.8% vs. 60.2%; P = 0.006). Commonest reasons for non-adherence in both groups were related to vomiting (67%). Vomiting at enrolment due to gastroenteritis was significantly associated with vomiting zinc sulphate with RR 2.17 (95% CI 1.24-3.79, P = 0.007). The acceptability of m-follow-up was high (99%). In conclusion the idea of m-follow-up was well received by participants who considered it acceptable. In this study, the adherence to Zinc dosing was not significantly different between the intervention and control group, and we consider that for zinc in AWD, counselling alone was good enough to achieve high adherence. The trial was registered with the Pan-African Clinical Trial Registry. Trial number: PACTR201711002737120.

The Role of Genetic Testing in Pediatric Renal Diseases: Diagnostic, Prognostic, and Social Implications.

Pediatric renal diseases vary widely and are linked to high morbidity and mortality; hence, early diagnosis is vital. Presently, genetic testing is being incorporated into the standard of care for children and their families with kidney disease, primarily as a diagnostic tool. In the present review, we aim to collect all potential evidence from relevant studies that reported the role of genetic testing in pediatric renal disease diagnostic, prognostic, and social implications. We have conducted both electronic and manual searches within PubMed, the Cochrane Library, Web of Science, and Scopus to find relevant studies. Studies from the years 2013-2023 were included. Case reports with limited sample sizes and no descriptive statistics, along with review papers and meta-analyses, were excluded from this review. Quality assessment for all included studies was performed. The pooled diagnostic yields were calculated using the common effect and random effect models utilizing the R program (R Foundation for Statistical Computing, Vienna, Austria). The pooled result for the diagnostic yield as per the common effect model is a pooled proportion of 0.42 (42%) 95% confidence interval (CI): [0.39,0.44], while with the random effects model the pooled proportion is 0.43 (43%) 95% CI: [0.31,0.57]. The diagnostic yield for the included studies ranged from 78.10% to 16.8%. The spectrum of kidney diseases included nephrolithiasis/nephrocalcinosis, glomerular diseases, cystic kidney disease, ciliopathies, tubulopathies, chronic kidney disease, and congenital anomalies of the kidneys and urinary tracts (CAKUT), while hematuria and proteinuria were reported by two studies and autosomal recessive and autosomal dominant idiopathic kidney disease was reported by only one study. Genetic testing validates clinical diagnosis and aids in tailoring management strategies; hence, a more precise treatment plan is developed and unnecessary investigations are avoided, which is crucial in the case of children during routine nephrology clinic visits. Genetic counselling is of the utmost importance, so all ethical and social concerns related to genetic testing are addressed in addition to patient satisfaction.

Femininity, class & status: the societal devaluation of the female early years workforce

ABSTRACT This thesis responds to contemporary debates about early childhood education and the gendered nature of the workforce, drawing on feminist theory, interpretive methodologies and practitioner accounts. This project amplifies the female voice of practitioners in the workforce, using data drawn from semi- structured interviews with 11 early years practitioners. Drawing on intersectional feminist theories surrounding women’s liberation from misogyny, and Foucauldian conceptualisations of power, this project will highlight the role of femininity on predominantly working-class early childhood practitioners, and how it distorts their identity and their relationships with colleagues under the current neoliberal regulation of the sector. Through analysing the qualitative data, this thesis will explore the impact of the assumption that women are the traditional caregivers on practitioners themselves; how the patriarchy reproduces this norm by perpetuating misogyny and marginalisation; and how this notion affects women’s oppression and the consequent movements towards liberation. This thesis offers two conceptual and empirical contributions to the field of early childhood education research. Firstly, the theoretical underpinnings of Foucauldian and feminist theory with interpretive methodologies to explore women’s oppression in the early childhood workforce. Secondly, in exploring the role of gendered language and gossip in interactions between early childhood professionals, this thesis analyses the extent to which patriarchal language surrounding gossip is used to trivialise and devalue the female voice and probes the extent to which gossip is a form of power for women. This study contributes important perspectives on how current drives towards the ‘educationalization’ of early years settings can have harmful consequences for the professional development of early childhood educators, rather than raising the standards of childcare in the UK.

Universal assessment and tiered intervention: A model of care to meet the pediatric oncology psychosocial standard of care for school support.

The feasibility and acceptability of a tiered intervention model of school intervention services was investigated in response to the publication of evidence-based Psychosocial Standards of Care for Children with Cancer and their Families. Children with newly diagnosed malignancy or transitioning to long-term survivorship care were eligible. Families received universal school needs assessment and intervention targeted at the level of risk identified. Academic risk and resource utilization data collected over 1year were used to evaluate model feasibility and surveys evaluated family and medical provider satisfaction with the model. One hundred and eight children were enrolled and had academic risk assessed at enrollment as high (25%), moderate (44.4%) or low (30.6%) risk. School liaison staff time spent providing intervention was related to the level of risk. 54.6% of patients reported a decrease in risk status at the end of 1year of intervention. Parents (94%) and medical providers (100%) chose responses of "agree" or "strongly agree" when asked if they were satisfied with the intervention received over the year. The tiered model of care identifies the students with the highest need for intervention, allows for more targeted use of resources, and successfully meets the Psychosocial Standard of Care for academic needs.

Children's Oncology Group's 2023 blueprint for research: Diversity and health disparities.

The Children's Oncology Group (COG) Diversity and Health Disparities Committee's (DHDC's) mission is to guarantee the highest standard of care for children and adolescents and young adults (AYA) with cancer regardless of ethnic, racial, gender, or socioeconomic background. We strive to identify and address issues of disparity within the existing scientific structure of COG and to support research across COG to improve survival by ensuring equitable access to COG-sponsored clinical trials. We are committed to advance COG-led research identifying mechanistic drivers of disparities and, concurrently, evaluating interventions to alleviate disparities in the COG trial setting. As trials identify the most promising therapies, diverse representation is critical to ensure that findings are relevant to everyone. Factors impacting clinical trial participation among vulnerable populations are complex, consisting of barriers at societal, systems, and individual levels. Recent efforts by investigators within DHDC demonstrated that trial-embedded collection of family-reported sociodemographic data and social determinants of health (SDoH) is feasible and acceptable in the context of COG. Diversity in the pediatric oncology workforce is essential and one potential approach to improving representation on clinical trials. To support and retain diverse oncology providers and researchers, a Minority Young Investigator Award (MYIA) was created to facilitate opportunities for graduating trainees and YIs with an interest in childhood cancer disparities research within COG. Although there are challenges to achieve the DHDC's priorities, only through collaboration and support for this work we will be able to elucidate mechanisms underlying inferior survival outcomes for historically marginalized children and AYA, and more importantly, implement interventional investigation to improve outcomes.

Parent coaching via telerehabilitation for young children with autism spectrum disorder (ASD): study protocol for a randomised controlled trial

BackgroundEarly parent-implemented intervention enhances parent-child interaction and improves language skills in children with autism spectrum disorders (ASD). Parent coaching is often delivered as standard care for children with ASD, where parents are taught to apply strategies in their child’s play activities and daily routines to achieve the prior stated goals. However, the ability to conduct parent coaching in physical in-clinic sessions is limited by resource constraints such as clinic space and therapist manpower. Furthermore, parents may experience difficulties with the generalisation of intervention strategies taught in the clinic to their natural home environments. In this study, telerehabilitation is evaluated as an alternative platform to deliver parent coaching for parent-implemented interventions to children with ASD in their homes.MethodsThis parallel-group, randomised, controlled, non-inferiority trial aims to evaluate the effectiveness of parent coaching delivered through video conferencing (telerehabilitation) versus in-clinic (standard care) delivery. Children aged 15 to 48 months (n = 200) who meet the cut-off score for ASD on the Autism Diagnostic Observation Schedule-2 are eligible. Parent-child dyads are randomly assigned to receive parent coaching either through weekly telerehabilitation or standard care. The primary outcome is the child’s development as measured by the subscale and composite scores of a standardised developmental assessment. Primary analysis will determine if the lower boundary of the 95% confidence interval for the mean difference in pre-post change between groups exceeds −5 (the non-inferiority margin). Secondary outcomes are the child’s adaptive behaviour, parent-child interaction, parental stress, and family quality of life. Outcomes will be measured pre-intervention, midterm, and post-intervention. Secondary analysis will determine if there is any between-group difference for the pre-post change in scores at the 5% significance level using two-sample t-test or Mann-Whitney U test.DiscussionAs a randomised controlled trial of a moderately large scale, this study will contribute to the limited existing literature on the effectiveness of parent coaching via telerehabilitation for early parent-implemented intervention for children with ASD. The results of this study will provide insights on whether telerehabilitation is comparable to conventional in-clinic parent coaching in enhancing parent-child interaction and improving language skills.Trial registrationClinicalTrials.gov NCT05792449. Registered (retrospectively) on 31 March 2023.

Effect of Video Games or Virtual Reality in Reducing Symptoms of Cognitive Deficits in Children and Adolescents with ADHD: A Systematic Review

Introduction: Attention-deficit hyperactivity disorder (ADHD) is one of the most frequent neurodevelopmental diseases affecting children and adolescents worldwide. Standard treatments often include a combination of pharmacotherapy and cognitive interventions. In the last decade, the effects of video game- and virtual reality (VR)-based treatments have been investigated as viable additional strategies to tackle ADHD symptoms. Methods: A systematic literature review was conducted to identify studies that compared video game or VR-based interventions with standard care in children and adolescents affected by ADHD, evaluating cognitive, motor, and behavioral outcomes. The RoB-2 and MINORS tools were utilized to assess the risk of bias in included studies. Results: Five studies published between 2001 and 2021 were included (437 patients, mean age: 10.74). Four studies investigated a video game-based intervention, and the remaining study employed VR. Collectively, the studies showed little to no effects on investigated outcomes. However, most of them were also affected by severe methodological issues carrying a moderate-to-high risk of bias. Discussion: ADHD is a complex disorder often needing a multi-dimensional, individualized therapeutical approach. Video games and VR carry substantial advantages, such as higher patient involvement, cost-effectiveness, and subtle improvements in cognitive and behavioral outcomes. However, additional well-designed clinical trials are needed to prove their efficacy in pediatric patients with ADHD.

Parents' and adolescents' perspectives and understanding of information about childhood cancer precision medicine.

Precision medicine is projected to become integral to childhood cancer care. As such, it is essential to support families to understand what precision medicine entails. A total of 182 parents and 23 adolescent patients participating in Precision Medicine for Children with Cancer (PRISM), an Australian precision medicine clinical trial for high-risk childhood cancer, completed questionnaires after study enrollment (time 0 [T0]). Of the parents, 108 completed a questionnaire and 45 completed an interview following return of precision medicine results (time 1 [T1]). We analyzed the mixed-methods data comprising measures exploring families' perceptions and understanding of PRISM's participant information sheet and consent form (PISCF), and factors associated with understanding. Most parents were satisfied with the PISCF, rating it as at least "somewhat" clearly presented (n=160/175; 91%) and informative (n=158/175; 90%). Many suggested improvements including the use of clearer language and a more visually engaging format. Parents' actual understanding of precision medicine was low on average, but scores improved between T0 and T1 (55.8/100-60.0/100; p=.012). Parents from culturally and/or linguistically diverse backgrounds (n=42/177; 25%) had lower actual understanding scores than those from a Western/European background whose first language was English (p=.010). There was little correlation between parents' perceived and actual understanding scores (p=.794; Pearson correlation -0.020; 95% CI, -0.169 to 0.116). Most adolescent patients read the PISCF either "briefly" or "not at all" (70%) and had a perceived understanding score of 63.6/100 on average. Our study revealed gaps in families' understanding of childhood cancer precision medicine. We highlighted areas for potential intervention such as through targeted information resources. Precision medicine is projected to become part of the standard of care for children with cancer. Precision medicine aims to give the right treatment to the right patient and involves several complex techniques, many of which may be challenging to understand. Our study analyzed questionnaire and interview data from parents and adolescent patients enrolled in an Australian precision medicine trial. Findings revealed gaps in families' understanding of childhood cancer precision medicine. Drawing on parents' suggestions and the literature, we make brief recommendations about improving information provision to families, such as through targeted information resources.

Effectiveness of acupuncture therapy for the prevention of emergence agitation in children: A systematic review and meta-analysis with trial sequential analysis.

This study aimed to evaluate the effectiveness of acupuncture therapy in preventing emergence agitation (EA) in children. A systematic review and meta-analysis were conducted across multiple locations according to the articles searched. Seven databases, including trial registration sites, were searched. A total of six trials were included involving 489 patients; of them, 244 received acupuncture therapy. Randomized clinical trials (RCTs) evaluating the incidence of EA compared with placebo/sham or standard care in children were included. The primary outcome was the incidence of EA, as evaluated using a specific assessment tool. Data about the incidence rate of EA, heterogeneity, quality of trials and evidence, and adverse events were collected. Additionally, data about patient demographic characteristics, type of anesthesia, duration and onset of acupuncture therapy, EA and pain score, time taken for extubation, and post-anesthesia care unit length of stay were collected. The results indicated that the overall incidence of EA in the acupuncture therapy group and the control group was 23.4% and 39.5%, respectively, with no significant difference (risk ratio, 0.62; 95% confidence interval, 0.26-1.48; I2 = 63%). Subgroup analysis showed a significant difference in the overall incidence of EA in the acupuncture therapy and control groups according to surgery type (high-risk vs. low-risk surgery), suggesting that acupuncture therapy may be effective in reducing EA for patients undergoing high-risk surgery. The quality of evidence was downgraded to "very low" due to the study designs, inconsistency, and possible publication bias. In conclusion, this meta-analysis shows that the currently available RCTs are insufficient to determine the effectiveness of acupuncture therapy in preventing EA in children undergoing general anesthesia.

Short-course, high-dose primaquine regimens for the treatment of liver-stage vivax malaria in children

ObjectivesTo assess the pharmacokinetics, safety, and tolerability of two high-dose, short-course primaquine (PQ) regimens compared with standard care in children with Plasmodium vivax infections. MethodsWe performed an open-label pediatric dose-escalation study in Madang, Papua New Guinea (Clinicaltrials.gov NCT02364583). Children aged 5-10 years with confirmed blood-stage vivax malaria and normal glucose-6-phosphate dehydrogenase activity were allocated to one of three PQ treatment regimens in a stepwise design (group A: 0.5 mg/kg once daily for 14 days, group B: 1 mg/kg once daily for 7 days, and group C: 1 mg/kg twice daily for 3.5-days). The study assessments were completed at each treatment time point and fortnightly for 2 months after PQ administration. ResultsBetween August 2013 and May 2018, 707 children were screened and 73 met the eligibility criteria (15, 40, and 16 allocated to groups A, B, and C, respectively). All children completed the study procedures. The three regimens were safe and generally well tolerated. The pharmacokinetic analysis indicated that an additional weight adjustment of the conventionally recommended milligram per kilogram PQ doses is not necessary to ensure the therapeutic plasma concentrations in pediatric patients. ConclusionsA novel, ultra-short 3.5-day PQ regimen has potential benefits for improving the treatment outcomes in children with vivax malaria that warrants further investigation in a large-scale clinical trial.

Assessment of the risk of stroke in children with sickle cell anemia using transcranial doppler ultrasound with imaging in Northwestern Nigeria

Background: Stroke is a major cause of morbidity and mortality affecting about 10% of patients with sickle cell anemia (SCA). Transcranial Doppler (TCD) ultrasonography helps to identify children with SCA who are at an increased risk for stroke. This study aimed to determine the risk of stroke in children with SCA in steady state using Transcranial Doppler with Imaging (TCDI) scan abnormalities and the prevalence of TCDI scan abnormalities among children with SCA in northern Nigeria.&#x0D; Materials and Methods: We conducted acomparative study among 240 persons with SCA who attended a pediatric sickle cell clinic in northern Nigeria and were in steady state condition. We performed a transcranial ultrasound examination and collected blood samples to perform full blood counts and hemoglobin electrophoresis.&#x0D; Results: Abnormal velocities were obtained in 11 (9.3%) children with SCA; while 16 (13.6%) had conditional velocities and 89 (76.7%) of those had normal velocities in one or more of the four vessels studied. Most of the children in the control group 116 (96.7%) had normal velocities and the difference between the groups was not statistically significant (χ 2 = 0.59, p &gt; 0.05).&#x0D; Conclusion: The use of TCDI sonography to predict the risk of stroke should be included in the standard of care in children with SCA in Nigeria and should be included in routine evaluation of disease severity in children with SCA

From the infant to the geriatric patient-Strategies for inhalation therapy in asthma and chronic obstructive pulmonary disease.

Inhalation therapy represents the standard of care in children, adolescents as well as in young, middle-aged and geriatric adults with asthma or chronic obstructive pulmonary disease. However, there are only few recommendations for the choice of inhalation devices, which consider both, age-specific limitations in young and geriatric patients. Transition concepts are lacking. In this narrative review, the available device technologies and the evidence for age-specific problems are discussed. Pressurized metered-dose inhalers may be favoured in patients who fulfill all cognitive, coordinative and manual power requirements. Breath-actuated metered-dose inhalers, soft-mist inhalers or the use of add-on devices such as spacers, face masks and valved holding chambers may be suitable for patients with mild to moderate impairments of these variables. In these cases, available resources of personal assistance by educated family members or caregivers should be used to allow metered-dose inhaler therapy. Dry powder inhalers may be reserved for patients with a sufficient peak inspiratory flow and good cognitive and manual abilities. Nebulizers may be indicated in persons who are either unwilling or unable to use handheld inhaler devices. After initiation of a specific inhalation therapy, close monitoring is essential to reduce handling mistakes. An algorithm is developed that considers age and relevant comorbidities to support the decision-making process for the choice of an inhaler device.

Hydrocortisone to reduce dexamethasone-induced neurobehavioral side-effects in children with acute lymphoblastic leukaemia—results of a double-blind, randomised controlled trial with cross-over design

BackgroundDexamethasone is a cornerstone of paediatric acute lymphoblastic leukaemia (ALL) treatment, although it can induce serious side-effects. Our previous study suggests that children who suffer most from neurobehavioural side-effects might benefit from physiological hydrocortisone in addition to dexamethasone treatment. This study aimed to validate this finding. MethodsOur phase three, double-blind, randomised controlled trial with cross-over design included ALL patients (3–18 years) during medium-risk maintenance therapy in a national tertiary hospital between 17th May 2018 and 5th August 2020. A baseline measurement before and after a 5-day dexamethasone course was performed, whereafter 52 patients with clinically relevant neurobehavioural problems were randomised to receive an intervention during four subsequent dexamethasone courses. The intervention consisted of two courses hydrocortisone (physiological dose 10 mg/m2/d in circadian rhythm), followed by two courses placebo, or vice versa. Neurobehavioural problems were assessed before and after each course using the parent-reported Strengths and Difficulties Questionnaire (SDQ) as primary end-point. Secondary end-points were sleep problems, health-related quality of life (HRQoL), hunger feeling, and parental stress, measured with questionnaires and actigraphy. A generalised mixed model was estimated to study the intervention effect. ResultsThe median age was 5.5 years (range 3.0–18.8) and 61.5% were boys. The SDQ filled in by 51 primary caregivers showed no difference between hydrocortisone and placebo in reducing dexamethasone-induced neurobehavioral problems (estimated effect -2.05 (95% confidence interval (CI) -6.00–1.90). Also, no benefit from hydrocortisone compared to placebo was found for reducing sleep problems, hunger, parental stress or improving HRQoL. ConclusionsHydrocortisone, when compared to placebo, had no additional effect in reducing clinically relevant dexamethasone-induced neurobehavioural problems. Therefore, hydrocortisone is not advised as standard of care for children with ALL who experience dexamethasone-induced neurobehavioural problems. Trial registrationNetherlands Trial Register NTR6695/NL6507 (https://trialsearch.who.int/) and EudraCT 2017–002738–22 (https://eudract.ema.europa.eu/).

Changing Agendas on Sleep, Treatment and Learning in Epilepsy (CASTLE) Sleep-E: a protocol for a randomised controlled trial comparing an online behavioural sleep intervention with standard care in children with Rolandic epilepsy

IntroductionSleep and epilepsy have an established bidirectional relationship yet only one randomised controlled clinical trial has assessed the effectiveness of behavioural sleep interventions for children with epilepsy. The intervention was...