Modification of the Paediatric Gastro-oesophageal Reflux Disease Symptom and Quality of Life Questionnaire (PGSQ) for children with cerebral palsy: a preliminary study.

OBJECTIVE: Gastro-oesophageal reflux disease (GORD) is a common condition affecting children, characterised by the passage of gastric contents into the oesophagus causing pain, vomiting and regurgitation. Children with neurodisability (such as cerebral palsy; CP) are predisposed to more severe GORD due to coexisting gut dysmotility and exclusive/supplementary liquid diet; however, there are no existing tools or outcome measures to assess the severity of GORD in this patient group. For children without CP, the 'Paediatric Gastro-oesophageal Symptom and Quality of Life Questionnaire' (PGSQ) assesses symptoms and response to treatment, but the questions are not suitable for children with significant cognitive impairment. We aimed to adapt the existing PGSQ assessment tool to enable use in evaluating children with CP and GORD. PATIENTS/INTERVENTIONS: Cognitive interviews were conducted by the research team with six parents/carers of children (aged 3-15) with CP (Gross Motor Function Classification System level V) who have current or past symptoms of reflux. They were asked to interpret the questionnaire using a 'think-aloud technique,' and offer suggestions on alterations to questions. Reasons for changing questions included confusing/difficult to understand questions, differing interpretations of questions and response choices not applying to the patient group. RESULTS: The PGSQ was modified iteratively following each interview. Overall, parents/carers reported that it was acceptable to recall information over the past 7 days. In the final version, it was felt the questions were relevant, useful and related to symptoms that they observed. It was easy to comprehend with no uncomfortable questions. Suggestions for future work included a section specifically focusing on the school day answered by school staff and home life answered by carers who assist them in the home. CONCLUSIONS: We have adapted the PGSQ to improve relevance and acceptability for families/carers of children with symptoms of GORD and neurodisability. Further work is needed to validate the questionnaire for this patient group.

Pharmacological treatment of gastro-oesophageal reflux in children.

BACKGROUND: Gastro-oesophageal reflux (GOR) is characterised by the regurgitation of gastric contents into the oesophagus. GOR is a common presentation in infancy, both in primary and secondary care, affecting approximately 50% of infants under three months old. The natural history of GOR in infancy is generally of a self-limiting condition that improves with age, but older children and children with co-existing medical conditions can have more protracted symptoms. The distinction between gastro-oesophageal reflux disease (GORD) and GOR is debated. Current National Institute of Health and Care Excellence (NICE) guidelines define GORD as GOR causing symptoms severe enough to merit treatment. This is an update of a review first published in 2014. OBJECTIVES: To assess the effects of pharmacological treatments for GOR in infants and children. SEARCH METHODS: For this update, we searched CENTRAL, MEDLINE, Embase, and Web of Science up to 17 September 2022. We also searched for ongoing trials in clinical trials registries, contacted experts in the field, and searched the reference lists of trials and reviews for any additional trials. SELECTION CRITERIA: We included randomised controlled trials (RCTs) that compared any currently-available pharmacological treatment for GOR in children with placebo or another medication. We excluded studies assessing dietary management of GORD and studies of thickened feeds. We included studies in infants and children up to 16 years old. DATA COLLECTION AND ANALYSIS: We used standard methodology expected by Cochrane. MAIN RESULTS: We included 36 RCTs involving 2251 children and infants. We were able to extract summary data from 14 RCTs; the remaining trials had insufficient data for extraction. We were unable to pool results in a meta-analysis due to methodological differences in the included studies (including heterogeneous outcomes, study populations, and study design). We present the results in two groups by age: infants up to 12 months old, and children aged 12 months to 16 years old. Infants Omeprazole versus placebo: there is no clear effect on symptoms from omeprazole. One study (30 infants; very low-certainty evidence) showed cry/fuss time in infants aged three to 12 months had altered from 246 ± 105 minutes/day at baseline (mean +/- standard deviation (SD)) to 191 ± 120 minutes/day in the omeprazole group and from 287 ± 132 minutes/day to 201 ± 100 minutes/day in the placebo group (mean difference (MD) 10 minutes/day lower (95% confidence interval (CI) -89.1 to 69.1)). The reflux index changed in the omeprazole group from 9.9 ± 5.8% in 24 hours to 1.0 ± 1.3% and in the placebo group from 7.2 ± 6.0% to 5.3 ± 4.9% in 24 hours (MD 7% lower, 95% CI -4.7 to -9.3). Omeprazole versus ranitidine: one study (76 infants; very low-certainty evidence) showed omeprazole may or may not provide symptomatic benefit equivalent to ranitidine. Symptom scores in the omeprazole group changed from 51.9 ± 5.4 to 2.4 ± 1.2, and in the ranitidine group from 47 ± 5.6 to 2.5 ± 0.6 after two weeks: MD -4.97 (95% CI -7.33 to -2.61). Esomeprazole versus placebo: esomeprazole appeared to show no additional reduction in the number of GORD symptoms compared to placebo (1 study, 52 neonates; very low-certainty evidence): both the esomeprazole group (184.7 ± 78.5 to 156.7 ± 75.1) and placebo group (183.1 ± 77.5 to 158.3 ± 75.9) improved: MD -3.2 (95% CI -4.6 to -1.8). Children Proton pump inhibitors (PPIs) at different doses may provide little to no symptomatic and endoscopic benefit. Rabeprazole given at different doses (0.5 mg/kg and 1 mg/kg) may provide similar symptom improvement (127 children in total; very low-certainty evidence). In the lower-dose group (0.5 mg/kg), symptom scores improved in both a low-weight group of children (< 15 kg) (mean -10.6 ± SD 11.13) and a high-weight group of children (> 15 kg) (mean -13.6 ± 13.1). In the higher-dose groups (1 mg/kg), scores improved in the low-weight (-9 ± 11.2) and higher-weight groups (-8.3 ± 9.2). For the higher-weight group, symptom score mean difference between the two different dosing regimens was 2.3 (95% CI -2 to 6.6), and for the lower-weight group, symptom score MD was 4.6 (95% CI -2.9 to 12). Pantoprazole: pantoprazole may or may not improve symptom scores at 0.3 mg/kg, 0.6 mg/kg, and 1.2 mg/kg pantoprazole in children aged one to five years by week eight, with no difference between 0.3 mg/kg and 1.2 mg/kg dosing (0.3 mg/kg mean -2.4 ± 1.7; 1.2 mg/kg -1.7 ± 1.2: MD 0.7 (95% CI -0.4 to 1.8)) (one study, 60 children; very low-certainty evidence). There were insufficient summary data to assess other medications. AUTHORS' CONCLUSIONS: There is very low-certainty evidence about symptom improvements and changes in pH indices for infants. There are no summary data for endoscopic changes. Medications may or may not provide a benefit (based on very low-certainty evidence) for infants whose symptoms remain bothersome, despite nonmedical interventions or parental reassurance. If a medication is required, there is no clear evidence based on summary data for omeprazole, esomeprazole (in neonates), H2antagonists, and alginates for symptom improvements (very low-certainty evidence). Further studies with longer follow-up are needed. In older children with GORD, in studies with summary data extracted, there is very low-certainty evidence that PPIs (rabeprazole and pantoprazole) may or may not improve GORD outcomes. No robust data exist for other medications. Further RCT evidence is required in all areas, including subgroups (preterm babies and children with neurodisabilities).

How to use the fractional exhaled nitric oxide test.

Measurement of fractional exhaled nitric oxide (FeNO) has become a mainstream, NICE-recommended, objective test of asthma severity. We explore the uses and practical issues with the FeNO test using clinically relevant questions for general paediatricians.

How to use Donath-Landsteiner test to diagnose paroxysmal cold haemoglobinuria (PCH).

Paroxysmal cold haemoglobinuria (PCH) accounts for around a third of cases of autoimmune haemolytic anaemia in children. PCH is caused by an autoantibody that fixes complement to red cells at low temperatures and dissociates at warmer temperatures (a biphasic haemolysin), triggering complement-mediated intravascular haemolysis. Named the Donath-Landsteiner (D-L) antibody after its discoverers, it is usually formed in response to infection and demonstrates specificity for the ubiquitous red cell P-antigen. A D-L test can be used to detect the presence of the D-L autoantibody in the patients' serum. Here we discuss the use of the D-L test in identifying PCH in a 2-year-old boy who presented with haemolytic anaemia. A summary of the key information can be found in the infographic.

A Clinical Investigation of the Performance and Safety of Epaderm®, an Emollient Cream.

INTRODUCTION: Emollients provide an occlusive barrier for dry and atopic skin, retain moisture, protect it from irritants, and form the basis of eczema treatment. METHODS AND ANALYSIS: A prospective interventional single arm study to evaluate the performance and safety of Epaderm® Cream, an emollient and cleanser containing 25% (w/w) paraffin and 5% (w/w) glycerine (thereafter, an emollient cream), in patients with dry skin conditions. The primary outcome measure was participant evaluation of skin moisturisation after treatment with an emollient cream for up to 4 weeks. Secondary outcome measures included: evaluation of skin softness using a questionnaire and of pruritus on a visual analogue scale (VAS); clinician assessment of xerosis using Overall Dry Skin (ODS) score and measurement of skin hydration using a non-invasive device (MoistureMeterEpiD, Delfin Technologies) at each visit. Sign test and Wilcoxon signed rank test were used to analyse changes from baseline. RESULTS: A total of 114 participants completed the study. 84.2% (80 out of 95) of participants or parents strongly agreed or agreed that the cream improved skin moisturisation at 4 weeks of treatment at the target area (p<0.0001). 86.3% of participants agreed that skin softness improved after 4 weeks (p <0.0001). ODS score improved from 2.1 (standard deviation (SD) 1.0) to 0.7 (SD 0.8) at 4 weeks. Skin hydration at the target area improved from 31.5 (SD 9.3) to 40.5 (SD 8.3) (p<0.001) at 4 weeks. Mean skin itchiness reduced from 38.0 (SD 25.4) to 17.7 (SD 19.8) at 4 weeks (p<0.0001). Ten (8.3%) adverse device events (ADEs) were reported. CONCLUSION: The emollient cream was well tolerated and demonstrated significant improvements in patient-reported skin moisturisation and softness as well as in clinical measurement of xerosis and skin hydration across all age groups including infants. The emollient cream can be recommended for dry skin conditions including atopic dermatitis and psoriasis.

Functional abdominal pain: what clinicians need to know.

Abdominal pain in childhood is extremely common and presents frequently to both primary and secondary care, with many children having recurrent pain which impacts on daily functioning. Despite this most children have no discernible underlying pathology. We discuss the underlying mechanism for functional abdominal pain (visceral hypersensitivity), the evidence base linking parental anxiety and patient symptoms, and how parents can be supported in managing their children's symptoms by addressing questions commonly asked by children and families. We look at the evidence for a one-stop rational approach to investigation including a coeliac screen, inflammatory markers and consideration of stool faecal calprotectin, in the absence of red flags. We evaluate commonly used treatments for functional abdominal pain, within a context of managing family expectations. Given the limitations in pharmacological treatment options, trials of probiotics, peppermint oil, mebeverine and (for short-term use only) hyoscine butylbromide may be appropriate. Psychological interventions including cognitive-behavioural therapy, distraction techniques and hypnotherapy have a better evidence base. There is also some evidence for other complementary therapies in children, including yoga and neurostimulation. Outcome is generally good providing there is child and family acceptance of the multiple factors implicated in the aetiology of the pain.

How to use N-terminal pro-brain natriuretic peptide (NT-proBNP) in assessing disease severity in bronchiolitis.

Bronchiolitis is a common viral illness which can lead to severe respiratory compromise and can coexist with or mask cardiac failure. Brain natriuretic peptide (BNP) and the inactive portion of its pro-hormone: N-terminal pro-BNP (NT-proBNP) are excreted in response to cardiomyocyte stretching and are established biomarkers in cardiac failure. Here, we discuss the technicalities of NT-proBNP testing and review available evidence regarding NT-proBNP testing in bronchiolitis. We identified and appraised seven studies assessing the role of BNP or NT-proBNP as biomarkers of bronchiolitis severity, in children with and without underlying congenital cardiac disease. One study of 76 children with dyspnoea showed that the median NT-proBNP level in children with cardiac failure was 7321 pg/mL vs 241 pg/mL in children with a respiratory cause of dyspnoea vs 87.21 pg/mL in healthy controls (p<0.05). A cut-off of 726 pg/mL could aid differentiation between cardiac and respiratory causes of respiratory distress. Other evidence showed a positive correlation between BNP levels and bronchiolitis severity, and that raised BNP can predict acute heart failure in children with congenital cardiac disease presenting with bronchiolitis. However, most studies consisted of small cohorts with conflicting evidence between them. Furthermore, several studies assessed BNP rather than NT-proBNP directly. BNP has a shorter half-life, which may affect analysis. In conclusion, NT-proBNP is a rapid and inexpensive test with the potential to be a useful biomarker in severe bronchiolitis and cases complicated by acute cardiac failure. However, studies with larger cohorts are required to better establish this role.

Fifteen-minute consultation on the healthy child: Bowel habit in infants and children.

A common presentation to the general paediatric clinic is a child or young person's difficult bowel habit, which is often a potent source of anxiety for parents and carers. A large proportion of these children will have a functional cause for their symptoms, with unnecessary investigation and non-evidence-based treatments adding to their difficulties. This article aims to explain what encompasses the normal bowel habit in children and young people, reassure where appropriate and identify those patterns that may be suggestive of a disorder or disease requiring treatment. We illustrate both extremes of the spectrum of normal bowel habit in children with two case studies.

How to use faecal calprotectin in management of paediatric inflammatory bowel disease.

Faecal calprotectin (FC) is a neutrophil-derived protein released in stool in response to mucosal inflammation. It is a simple, cheap and non-invasive test with high sensitivity and moderate specificity, which can be useful in the diagnosis and monitoring of inflammatory bowel disease (IBD). FC levels correlate well with bowel inflammation (both macroscopic and histological activity) and are not influenced by disease location or type of IBD. Despite the shortcoming with regards to specificity, it is the high sensitivity of FC that makes it a valuable screening tool in the diagnosis of IBD. It is especially effective in identifying children with low probability of IBD who would not benefit from further investigations. The cut-off value selected has a significant impact on the diagnostic accuracy of the test, influencing its sensitivity and specificity, and must be interpreted judiciously. Its role in disease monitoring is as an add-on test to Paediatric Ulcerative Colitis Activity Index and Paediatric Crohn's Disease Activity Index scores and can be used to differentiate disease relapse from functional symptoms. High levels of FC are also seen in a number of other conditions, such as gastrointestinal infections and coeliac disease. It is recommended that infective causes affecting the gut must be excluded first, before FC is measured.