Whimsical metaphor? Not intentionally. In the context of this (or any other month’s issue), the role of the column in guiding a reader to a station of disembarkation/paper is very important. This month’s issue is very strong on global health, endocrinology and infectious disease. None of the excellent papers I describe are difficult conceptually, all are practical and each rewarded reading. I know you will enjoy them too.

Last month after both extensive and intensive consultations, WHO released a new set of standards aimed at improving child and adolescent health. These are centred on eight key areas, and though clinical aspects form one spoke, they take us a step beyond standard practice guidance in that central tenets are child and parent appropriateness, quality improvement and the psychological and environmental aspects of illness. Like the Millennium and Sustainable...

There has been a large increase in the number of children and adolescents who question conventional gender expectations and seek recognition and acceptance of their gender diversity, wishing to develop a body that is congruent with their gender feelings.1 Professionals may be unsure how best to provide supportive care, how to access the national Gender Identity Development Service (GIDS) for children and adolescents, or how to deal with a transgender young person presenting with another clinical problem unrelated to their gender transition. Faced with very distressed young people, they may feel under pressure to initiate physical intervention without consultation with psychosocial colleagues. It is important that all professionals are aware of the care pathway for transgender children that may be of relevance in a range of paediatric settings. The purpose of this practice review is to present an up-to-date perspective on the care of transgender children...

Thyrotoxicosis is both rarer and more severe in children than in adults, rendering management difficult and often unsatisfactory.

To ascertain outcome in a geographically defined area of Scotland between 1989 and 2014.

Retrospective case note review with follow-up questionnaire to family doctors for patients with Graves’ disease and Hashimoto’s thyroiditis.

Sixty-six patients (58 females:8 males) comprising 53 with Graves’ disease and 13 with Hashimoto’s thyroiditis were diagnosed at median 10.4 (2.9–15.8) years and followed up for 11.8 (2.6–30.2) years. Antithyroid drug (ATD) therapy was stopped electively in 35 patients after 4.5 (1.5–8.6) years, resulting in remission in 10/13 Hashimoto’s thyroiditis and 10/22 Graves’ disease. Side effects occurred in 12 patients receiving carbimazole, six of whom changed to propylthiouracil; no adverse events occurred in the latter patients.

Second-line therapy was given to 37 patients (34 with Graves’ disease), comprising radioiodine (22) at 15.6 (9.3–24.4) years for relapse (6), poor control/adherence (14) or electively (2); and surgery (16) at 12 (6.4–21.3) years for relapse (4), poor control/adherence (5) and electively (7). Adherence problems with thyroxine replacement were reported in 10/33 patients in adulthood.

Hashimoto’s thyroiditis should be distinguished from Graves’ disease at diagnosis since the prognosis for remission is better. Remission rates for Graves’ disease are low (10/53 patients), time to remission variable and adherence with both ATD and thyroxine replacement often problematic. We recommend (a) the giving of long-term ATD rather than a fixed course of treatment in GD and (b) meticulous and realistic counselling of families from the time of diagnosis onwards.

To describe the clinical presentation, risk factors, serotype distribution and outcomes of invasive pneumococcal disease (IPD) in children with sickle cell disease (SCD) following the introduction of the 13-valent pneumococcal conjugate vaccine (PCV13) in the UK.

Prospective national newborn screening for SCD and enhanced national IPD surveillance.

Children with SCD born in England between 1 September 2010 and 31 August 2014 who developed laboratory-confirmed IPD by 31 December 2015.

Risk of IPD in children with SCD compared with children without SCD during the surveillance period.

Eleven children homozygote for haemoglobin S (HbSS) and one double heterozygote for haemoglobin S and C (HbSC) developed IPD. Septicaemia (n=7) and lower respiratory tract infection (n=4) were the main clinical presentations, and serogroup 15 (not present in PCV13) was responsible for 73% (8/11) of cases. Three children with HbSS (27%) died compared with <5% nationally. Children with HbSS had a 49-fold (95% CI 27 to 89, P<0.001) higher risk of IPD compared with their peers without SCD.

Children with SCD remain at increased risk of IPD despite national newborn screening, early penicillin prophylaxis and high pneumococcal vaccine uptake. They are also more likely to die of their infection compared with their peers without SCD. Most IPD cases are now due to serotypes not covered by PCV13. Healthcare professionals need to work more closely with families with SCD and local communities to emphasise the importance of penicillin prophylaxis, explore barriers, allay misguided beliefs and facilitate rapid access to healthcare.

To evaluate England’s NHS newborn sickle cell screening programme performance in children up to the age of 5 years.

Cohort of resident infants with sickle cell disease (SCD) born between 1 September 2010 and 31 August 2015 and followed until August 2016.

1317 infants with SCD were notified to the study from all centres in England and 1313 (99%) were followed up.

Early enrolment in clinical follow-up, parental education and routine penicillin prophylaxis.

Age seen by a specialist clinician, age at prescription of penicillin prophylaxis and mortality.

All but two resident cases of SCD were identified through screening; one baby was enrolled in care after prenatal diagnosis; one baby whose parents refused newborn screening presented symptomatically. There were 1054/1313 (80.3%, 95% CI 78% to 82.4%) SCD cases seen by a specialist by 3 months of age and 1273/1313 (97%, 95% CI 95.9% to 97.8%) by 6 months. The percentage seen by 3 months increased from 77% in 2010 to 85.4% in 2015. 1038/1292 (80.3%, 95% CI 78.1% to 82.5%) were prescribed penicillin by 3 months of age and 1257/1292 (97.3%, 95% CI 96.3% to 98.1%) by 6 months. There were three SCD deaths <5 years caused by invasive pneumococcal disease (IPD) sensitive to penicillin.

The SCD screening programme is effective at detecting affected infants. Enrolment into specialist care is timely but below the programme standards. Mortality is reducing but adherence to antibiotic prophylaxis remains important for IPD serotypes not in the current vaccine schedule.

To assess the impact of hypothermic neural rescue at birth on health-related quality of life (HRQL) in middle childhood.

Six-year to 7-year follow-up of surviving children from the Total Body Hypothermia for Neonatal Encephalopathy (TOBY) Trial.

Community study including a single parental questionnaire to collect information on children’s HRQL.

145 children (70 in the control group, 75 in the hypothermia group) whose parents consented and returned the questionnaire.

Intensive care with cooling of the body to 33.5°C for 72 hours or intensive care alone.

HRQL attributes and utility scores using the Health Utilities Index (HUI).

At 6–7 years, speech appeared disproportionately affected when compared with other aspects of HRQL but levels of normal emotional functioning were similar in both groups. The mean (SE) HUI3 HRQL scores were 0.73 (0.05) in the hypothermia group and 0.62 (0.06) in the control group; mean difference (95% CI) 0.11 (–0.04 to 0.26).

Findings of non-significant differences were not unexpected; the study used data from long-term survivors in a neonatal trial and was underpowered. However, results favoured moderate hypothermia and so complement the clinical results of the TOBY Children study. The work provides further insight into the long-term HRQL impact of perinatal asphyxial encephalopathy and provides previously unavailable utility data with which to contemplate the longer term cost-effectiveness of hypothermic neural rescue.

This study reports on the follow-up of the TOBY clinical trial: ClinicalTrials.gov number NCT01092637.

To evaluate the diagnosis and management of reflux and gastro-oesophageal reflux disease (GORD) in infants aged <1 year presenting to general practitioners (GPs).

A nationally representative, prospective, cross-sectional survey of GP activity in Australia, 2006–2016 (Bettering the Evaluation And Care of Health Study). Annually, a random sample of around 1000 GPs recorded details for 100 consecutive visits with consenting, unidentified patients.

Diagnoses of reflux and GORD and their management including prescribing of acid-suppressant medicines (proton pump inhibitors (PPIs) and histamine receptor antagonists (H2RAs)) and counselling, advice or education.

Of all infants’ visits, 512 (2.7%) included a diagnosis of reflux (n=413, 2.2%) or GORD (n=99, 0.5%). From 2006 to 2016, diagnostic rates decreased for reflux and increased for GORD. Prescribing of acid suppressants occurred in 43.6% visits for reflux and 48.5% visits for GORD, similar to rates of counselling, advice or education (reflux: 38.5%, GORD: 43.4% of visits). Prescribing of PPIs increased (statistically significant only for visits for reflux), while prescribing of H2RAs decreased.

Overprescribing of acid suppressants to infants may be occurring. In infants, acid-suppressant medicines are no better than placebo and may have significant negative side effects; however, guidelines are inconsistent. Clear, concise and consistent guidance is needed. GPs and parents need to understand what is normal and limitations of medical therapy. We need a greater understanding of the influences on GP prescribing practices, of parents’ knowledge and attitudes and of the pressures on parents of infants with these conditions.

To determine the prevalence of invasive bacterial infections (IBI, pathogenic bacteria in blood or cerebrospinal fluid) in infants less than 90 days old with fever without a source related to the presence or absence of fever on arrival to the emergency department (ED).

Prospective registry-based cohort study.

Paediatric ED of a tertiary teaching hospital.

We included infants less than 90 days old with a history of fever evaluated in the ED from 2003 to 2016.

The prevalence of IBI in patients with a history of fever who were febrile and afebrile on arrival to the ED.

We included 2470 infants: 678 afebrile and 1792 febrile when evaluated in the ED. Fifty-nine (2.4%) were diagnosed with an IBI (bacteraemia 46, meningitis 7 and sepsis 6): 16 in the group of afebrile infants with a history of fever (2.4%, 95% CI 1.4 to 3.8 vs 43 in the febrile group, 2.4%, 95% CI 1.8 to 3.2). Of the 16 afebrile infants with a history of fever diagnosed with an IBI, 14 were well appearing. The rate of non-IBI (pathogenic bacteria in urine or stools) was similar in both groups (15.5% and 16.7%).

The prevalence of IBI in infants ≤90 days with a history of fever is similar regardless of the presence of fever on the arrival at the ED. The approach to infants with a history of fever who are afebrile in the ED should not differ from that recommended for infants who are febrile in the ED.

Congenital heart defects (CHD) are the most common birth defects worldwide and are an important cause of morbidity and early death. A significant number of deaths occur among patients with infections. CHDs predispose to the development of infective endocarditis (IE) and represent a risk factor for increased mortality due to IE. The aim of this study was to investigate the occurrence and outcomes of IE in children and adolescents with CHDs.

Data on all children with CHD and IE born in Norway between 1994 and 2016 were retrieved from the Oslo University Hospital’s Clinical Registry for Congenital Heart Defects. Survivors were followed through 2016, and supplementary information was retrieved from medical records.

In this nationwide register-based cohort study, which included all 1 357 543 live births in Norway between 1994 and 2016, the incidence of IE according to the European Society of Cardiology diagnostic criteria was 2.2 per 10 000 person-years among children and adolescents with CHDs. The incidence was stable throughout the period. Most patients with IE had severe CHDs (75%) and had undergone open chest cardiac surgery or catheter-based cardiac interventions the last year before IE. IE-related mortality among children with CHDs and IE was 8% during the follow-up period (mean 12.4 years (±5.5 years)).

The incidence of IE among children and adolescents with CHDs was higher than the reported incidence in the general population. IE was associated with severe CHDs and recent complex cardiac interventions, and had significant mortality.

Congenital lymphoedema is a lifelong condition that has detrimental physical and psychosocial outcomes for young patients and burdensome treatment responsibilities that may hamper patients’ motivation for self-management. There is limited research from the perspective of young people with primary lymphoedema. We aimed to describe the experiences and views of children and adolescents with lymphoedema to inform patient-centred practice.

Twenty patients (aged 8–21 years) with primary lymphoedema were purposively sampled from two paediatric clinics in Sydney, Australia, to participate in a semistructured interview. The transcripts were analysed thematically.

We identified six themes: reinforcing abnormality (damaging self-esteem, self-consciousness, frustrating restrictions and isolation); negotiating uncertainties (fearing condition worsening, deprioritised and sidelined, questioning cause and permanence, widespread unawareness); vulnerability and caution (avoiding pain and discomfort, preventing severe and permanent consequences, depending on permission, limiting goals and aspirations); disruptive transition (resisting change, losing progress and support, avoiding treatment costs); developing resilience (focusing on the positives, embracing individuality, recalibrating normality, prioritising coping) and taking responsibility (individualising treatment, needing support, external pressure and motivation, sticking to a routine, seeking independence).

Children and adolescents learn to adjust to the daily demands of lymphoedema management by individualising and accepting their treatment, but many continue to struggle with their self-esteem and lifestyle restrictions. Strategies are needed to empower young patients to advocate for themselves during their transition to adult care. Treatment plans that minimise social restrictions, address emotional consequences and incorporate patients’ preferences could improve adherence, satisfaction and outcomes.

Parental absence, due to death or separation from a parent, has been associated with smoking and alcohol consumption in adolescence and adulthood. The aim of this study was to investigate whether parental absence in early childhood was associated with smoking and alcohol uptake before adolescence.

Data on 10 940 children from the UK's Millennium Cohort Study were used. Logistic regression was used to test associations between parental absence (0–7 years) and reports of smoking and alcohol consumption at age 11.

Children who experienced parental absence were more likely to have smoked (OR=2.58, 95% CI 1.88 to 3.56) and consumed alcohol (OR=1.46, 95% CI 1.25 to 1.72). No differences were found by child sex or age, or parent absent. Children who experienced parental death were less likely to have drunk alcohol but those who had were more likely to have consumed enough to feel drunk.

Parental absence was associated with early uptake of risky health behaviours in a large, nationally representative UK cohort. Children who experience parental absence should be supported in early life in order to prevent smoking and alcohol initiation.

A 5-year-old boy presents to the emergency department (ED) with an acute exacerbation of asthma, is assessed as having a tight chest and has an oxygen requirement. He is followed up regularly in a paediatric respiratory clinic and is normally on a preventer inhaler. Along with back-to-back mixed nebulisers, you prescribe oral prednisolone, which he vomits 10 min later.

The paediatric specialty trainee  says she has heard of a recent randomised controlled trial (RCT) that shows that a single dose of oral dexamethasone is as good as 3 days of prednisolone in the management of acute wheeze, and you know from your own experience that oral dexamethasone is usually well tolerated by kids presenting with croup. Given how often you see children vomit prednisolone, you ask yourself whether the ED should switch to oral dexamethasone instead for childhood wheeze.

For (children presenting to...

An infant born to primigravida mother at 29 weeks’ gestational age (GA) has respiratory distress syndrome (RDS). The fellow on call elected to start nasal continuous positive airway pressure (NCPAP) support. A paediatric resident in her neonatology rotation asks whether nasal mask is better than binasal prongs or vice versa for applying NCPAP support.

In a preterm infant <37 weeks’ GA with RDS (patient) whether NCPAP applied with a nasal mask (intervention) compared with that with binasal prongs (comparison) reduces

Study selection: randomised or quasirandomised clinical trials and systematic reviews comparing NCPAP (bubble,...

Sometimes in medicine, something that seems unequivocally bad turns out to have a therapeutic use—think of botulinum toxin. Zika virus has caused a devastating epidemic of microcephaly, mostly in South America, as result of maternal exposure in pregnancy. The virus has now been characterised, and researchers in Brazil wondered if its known neuro-destructive properties could be demonstrated in neurons that are rapidly dividing in postnatal life, as with some brain tumours (Kaid C et al. Cancer Research 2018. doi: 10.1158/0008-5472.CAN-17-3201).

First they exposed tumour cell lines in vitro to the Brazilian strain of the virus (ZIKVBR), and found that it selectively infected neuromalignant cells, especially embryonic cell lines, but was ineffective against other tumour cells (breast, prostate etc.). Then in mouse model experiments, they xeno-grafted human embryonic tumour cells (effectively medulloblastoma and teratoma cells) in to mice and injected ZIKVBR into their...