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Session Overview
07-OP-V-d2C: Oral Presentations Session 7 - Day 2 - Zone C
Friday, 02/Dec/2022:
2:30pm - 4:30pm

Virtual location: Virtual 1

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Will be presented as Oral Presentation Virtually
ID: 198 / 07-OP-V-d2C: 1
Oral Presentation (Virtual)
Keywords: Asthma

Dupilumab improves clinical efficacy outcomes in children (6–11 years) with moderate-to-severe asthma and high eosinophils at baseline

Jackson, Daniel J.1; Hamelmann, Eckard2; Roberts, Graham3,4,5; Bacharier, Leonard B.6; Altincatal, Arman7; Gall, Rebecca8; Ledanois, Olivier9; Jacob-Nara, Juby A.10; Radwan, Amr8; Rowe, Paul J.10; Deniz, Yamo8

1University of Wisconsin School of Medicine and Public Health, Madison, WI, USA; 2Children’s Center Bethel, University of Bielefeld, Bielefeld, Germany; 3University of Southampton Faculty of Medicine, Southampton, UK; 4NIHR Southampton Biomedical Research Centre, Southampton University Hospital NHS Foundation Trust, Southampton, UK; 5David Hide Asthma and Allergy Centre, St Mary’s Hospital, Isle of Wight, UK; 6Monroe Carell Jr. Children’s Hospital at Vanderbilt University Medical Center, Nashville, TN, USA; 7Sanofi, Cambridge, MA, USA; 8Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA; 9Sanofi, Paris, France; 10Sanofi, Bridgewater, NJ, USA

BACKGROUND: Children with moderate-to-severe asthma often remain symptomatic despite standard-of-care treatments, and patients with elevated eosinophil levels frequently have increased rates of exacerbations and worse asthma control. In the phase 3 LIBERTY ASTHMA VOYAGE study (NCT02948959), treatment with dupilumab, a fully human monoclonal antibody that blocks the shared receptor component for IL-4 and IL-13, was generally well tolerated and resulted in fewer exacerbations and improved lung function vs placebo in children aged 6–11 years with uncontrolled moderate-to-severe asthma. This post hoc analysis evaluates dupilumab efficacy in patients with baseline blood eosinophils (≥500 cells/µL) enrolled in the VOYAGE study.

METHODS: Patients received add-on dupilumab (100/200 mg by body weight at randomization) or matched add-on placebo every 2 weeks (q2w) for 52 weeks. Endpoints included annualized rate of severe exacerbations and least squares mean (LSM) change from baseline in pre-bronchodilator (pre-BD) precent predicted forced expiratory volume in one second (ppFEV1).

RESULTS: In participants with baseline blood eosinophils ≥500 cells/µL, add-on dupilumab significantly lowered annualized exacerbation rates (0.249 [95% CI 0.156–0.397]; n=126) compared with add-on placebo (0.749 [0.453–1.239]; P<0.001; n=48) and significantly improved pre-BD ppFEV1 vs placebo at Week 12 (LSM difference [95% CI] 6.47 percentage points [2.27–10.66]; P<0.01) and at Week 52 (LSM difference [95% CI] 7.98 percentage points [2.17–13.78]; P<0.01) at Week 52 (Figure).

CONCLUSION: Dupilumab significantly reduced severe exacerbations and improved lung function as assessed by pre-BD ppFEV1 vs placebo in children with moderate-to-severe asthma and baseline blood eosinophils ≥500 cells/µL.

Will be presented as Oral Presentation Virtually
ID: 168 / 07-OP-V-d2C: 2
Oral Presentation (Virtual)
Keywords: Asthma

Dupilumab long-term safety, clinical efficacy, and impact on biomarker levels in children with asthma: liberty asthma excursion

Bacharier, Leonard B1; Maspero, Jorge F2; Katelaris, Constance H3,4; Fiocchi, Alessandro G5; Gagnon, Remi6; de Mir, Ines7; Guilbert, Theresa8; Jackson, Daniel J9; Li, Ning10; Akinlade, Bolanle11; Laws, Elizabeth10; Mannent, Leda P12; Maloney, Jennifer11; Tawo, Kelsey10; Khokhar, Faisal A11; Hardin, Megan13; Abdulai, Raolat M13; Lederer, David J11; Robinson, Lacey B13

1Monroe Carell Jr Children’s Hospital at Vanderbilt, Nashville, TN, USA; 2Fundación CIDEA, Buenos Aires, Argentina; 3Campbelltown Hospital, Campbelltown, NSW, Australia; 4Western Sydney University, Sydney, NSW, Australia; 5Bambino Gesù Children’s Hospital IRCCS, Rome, Italy; 6Clinique Spécialisée en Allergie de la Capitale, Quebec, QC, Canada; 7Hospital Universitari Maternoinfantil Vall d'Hebron, Barcelona, Spain; 8Cincinnati Children’s Hospital and University of Cincinnati, Cincinnati, OH, USA; 9University of Wisconsin School of Medicine and Public Health, Madison, WI, USA; 10Sanofi, Bridgewater, NJ, USA; 11Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA; 12Sanofi, Chilly-Mazarin, France; 13Sanofi, Cambridge, MA, USA

BACKGROUND: Dupilumab, a fully human monoclonal antibody, blocks the shared receptor component for interleukin (IL) 4 and Il-13. The efficacy and safety of dupilumab in 6-11-year-old children with uncontrolled, moderate to severe asthma was demonstrated up to 52 weeks in VOYAGE (NCT02948959). The EXCURSION open-label extension study (NCT03560466) assessed dupilumab long-term safety and efficacy in children who completed VOYAGE.

METHODS: 365 patients rolled over from VOYAGE into EXCURSION and received add on SC dupilumab 100/200 mg every two weeks (q2w) or 300 mg q4w (body-weight tier based) for 52 weeks. Treatment-emergent adverse events (TEAE), annualized asthma exacerbation rate (AER), and change from parent study baseline (PSBL) in percent predicted (pp) FEV1, ppFVC, FEV1/FVC, ppFEF 25-75%, blood eosinophil count and total serum IgE were assessed.

RESULTS: Dupilumab was well tolerated, and the safety profile was consistent with the parent study. 85 (68.0%; placebo/dupilumab) and 147 (61.3%; dupilumab/dupilumab) of all EXCURSION patients experienced TEAEs, with 3 (1.3%) in the dupilumab/dupilumab group leading to treatment discontinuation. Median blood eosinophil count (Q1:Q3) decreased throughout EXCURSION to below the PSBL at Week 52, by −30 (−260:70) cells/µL for placebo/dupilumab (n=105), and −140 (−410:10) cells/µL for dupilumab/dupilumab (n=191). Total serum IgE declined substantially throughout EXCURSION, with a median percent change from PSBL to Week 52 of −80.0% (278.0 IU/mL; n=114) for placebo/dupilumab and −89.9% for dupilumab/dupilumab (339.0 IU/mL; n=219). The unadjusted AER for patients with type 2 asthma (baseline blood eosinophils ≥150 cells/µL or FeNO ≥20 ppb) was 0.124 for placebo/dupilumab and 0.118 for dupilumab/dupilumab (Figure). Mean change from PSBL ppFEV1 (%) in EXCURSION at Weeks 2/52 was +8.7/+9.4 in placebo/dupilumab (mean PSBL 78.7%), and +11.3/+12.6 in dupilumab/dupilumab (mean PSBL 76.9%) (Figure). Mean change from PSBL ppFVC (%) in EXCURSION Weeks 2/52 was +3.7/+4.4 in placebo/dupilumab (mean PSBL 92.7%), and +5.4/+6.9 in dupilumab/dupilumab (mean PSBL 91.9%). Mean change FEV1/FVC (%) from PSBL at EXCURSION Weeks 2/52 was +4.6/+4.5 in placebo/dupilumab (mean PSBL 74.2%), and +5.6/+5.4 in dupilumab/dupilumab (mean PSBL 73.4%). Mean change from PSBL ppFEF25-75% (L/s) at EXCURSION Weeks 2/52 was +13.5/+15.5 in placebo/dupilumab (mean PSBL 55.6 L/s), and +17.9/+17.4 in dupilumab/dupilumab (mean PSBL 53.8 L/s).

CONCLUSIONS: Long-term use of dupilumab was well tolerated, with a decline in type 2 biomarker levels, including blood eosinophils and total serum IgE. Efficacy observed in VOYAGE among patients with type 2 asthma was sustained in EXCURSION over an additional 52 weeks, including rapid improvement in patients initiating dupilumab.

Will be presented as Oral Presentation Virtually
ID: 142 / 07-OP-V-d2C: 3
Oral Presentation (Virtual)
Keywords: oral health, general anaesthetics, waiting times

Impact of waiting times for paediatric dental general anaesthetic on patient quality of life

Grange, Esme

Salisbury District Hospital, United Kingdom

BACKGROUND: This study investigated the effect of long waiting times associated with COVID-19 on children scheduled to have dental general anaesthetics at Salisbury District Hospital. Waiting times, pain, sleep, social factors, missed school and work, antibiotic use, previous dental general anaesthetics and treatment changes were recorded.

METHOD: Parents were invited to complete a survey based on the Child Oral Health Quality of Life Questionnaire immediately after their child’s treatment.

RESULTS: 49 children were included. The mean waiting time was 312.5 days. During the waiting period, 80% of parents stated that their child experienced pain, 73% recorded eating and drinking difficulty and 65% recorded sleep disturbance. 78%, 59% & 61% stated that their child’s mood, social behaviour and concentration were affected, respectively. 43% of children had missed school, 29% of parents missed work, and 45% of children received antibiotics. The majority (69%) of treatments remained unchanged. 10% had received a previous dental general anaesthetics.

CONCLUSION: Waiting times are exceedingly long at Salisbury District Hospital but vary greatly. Many children are suffering during their wait, and parents missing work may have broader implications for the community. Actions have been implemented to improve the high repeat dental general anaesthetic rate; however, greater access to community and paediatric services would be beneficial.

Will be presented as Oral Presentation Virtually
ID: 170 / 07-OP-V-d2C: 4
Oral Presentation (Virtual)
Keywords: Interleukin 6, nephrotic syndrome, genotype, polymorphism

Interleukin-6 gene polymorphism in kuwaiti arab children with idiopathic nephrotic syndrome

Aleisa, Amal A.; Alrushood, Maysoun; Kashyap, Sumedha; Haider, Mohammad

Kuwait university-college of medicine, Kuwait

BACKGROUND: Idiopathic Nephrotic syndrome (INS) is an immune-mediated disease with unresolved theories of pathogenesis. IL-6 is a vital pro-inflammatory cytokine involved in etiology of many autoimmune diseases. Gene polymorphism of IL-6 controls the expression and function of IL-6 and, therefore, might have an effect on the pathogenesis and clinical pattern of INS. (1) This study aims to determine the frequency and association of IL-6 gene polymorphisms with INS and its effect on the disease pattern in Kuwaiti children of Arab ethnic background.

METHODS: Genotypes of the IL-6 gene polymorphisms were analyzed using PCR-RFLP in 157 INS patients and 64 age and sex-matched controls. Clinical data of all subjects were reviewed.

RESULTS: A total of 157 INS (134 steroid-sensitive and 23 steroid-resistant) patients with a mean age was 7.6±4.3 years were studied. Male: Female ratio was 2:1. The CC genotype of IL-6 gene polymorphism was detected in 75% of the INS patients compared to 68% of the controls (P=0.42). The heterozygous GC genotype was detected in 19% of INS patients compared to 28% of the controls (P =0.19). The GG genotype was detected in 6% of INS patients and 3% of the controls (P =0.52). The C-allele frequency in homozygous and heterozygous forms was found in 84.7% of INS patients compared to 82.8% of the controls (P =0.72). The G-allele frequency in homozygous and heterozygous forms was found in 15.2% of INS patients compared to 17% of the controls (P =0.72). No significant difference was found in any of the allele frequencies between SS and SR sub-groups when compared with each other or when compared to the controls.

CONCLUSION: Our data show no role of IL-6 gene polymorphisms on the clinical pattern or response to steroids in Kuwaiti children with INS.

Will be presented as Oral Presentation Virtually
ID: 258 / 07-OP-V-d2C: 5
Oral Presentation (Virtual)
Keywords: HBSC, gender identity, mental health, trans, gender non-conforming

Mental Health of gender-conforming and non-conforming Adolescents in Luxembourg

Heinz, Andreas1; Catunda, Carolina2; Willems, Helmut2

1IU Internationale Hochschule, Germany; 2University of Luxembourg

BACKGROUND: In industrialised countries, there is a growing group of adolescents whose gender identity does not match the sex identified at birth. However, in many health and well-being surveys, gender non-conforming adolescents cannot be identified as a subgroup because gender identity is not asked. In the present study, sex and gender identity were asked separately to determine whether gender-conforming adolescents differ from gender non-conforming adolescents in terms of their health and well-being.

METHODS: In the Luxembourg HBSC 2022 survey, students in secondary schools were selected at random and surveyed in the classroom (N= 7,130). To assess the congruence of sex and gender identity, biological sex at birth was asked (male or female) and compared with gender identity (boy, girl, none/other). Health and well-being were measured using standard HBSC instruments (e.g. PSS-4, WHO-5; GAD-2 HBSC-SCL).

RESULTS: Congruence of sex and gender identity was determined for 6427 students aged 12-18. In 3.5% of the students, both did not match (i.e. gender non-conforming). The percentage of gender non-conforming students depended on the language of the questionnaire (English 9.4%, German/French 3.2%), sex at birth (male 1.4%, female 5.4%), but not age. Gender non-conforming students rated their health more often as "poor" compared to their gender-conforming peers (9.9% vs 2.2%). They were much more likely to have multiple psychosomatic complaints (82.9% vs 52.2%), higher levels of stress (PSS-4 mean: 9.8 versus 7.2) and lower life satisfaction (Cantril mean: 5.6 vs 7.2). In addition, they were more likely to have felt lonely in the past 12 months, to have shown symptoms of depression, and to have planned and attempted suicide. Gender non-conforming students were also more likely to report that Covid-19 had a negative impact on various areas of their lives than gender-conforming students (e.g. on mental health and relationship with family and friends).

CONCLUSIONS: The association between the language of the questionnaire and not identifying with the sex identified at birth suggests that gender (non-) conformity is culturally shaped. The mental health of gender non-conforming students is lower in all aspects examined. To analyse the health and well-being of gender non-conforming youth, it is important to ask about both sex and gender identity in surveys.

Will be presented as Oral Presentation Virtually
ID: 215 / 07-OP-V-d2C: 6
Oral Presentation (Virtual)
Keywords: anti-SARS-CoV-2 vaccine; child; swelling; hemangioma

Not everything is as it seems…

Azevedo, Inês Alexandra1; Torrão, Helena2; Ferreira, Ana Maia3; Maia, Iris3; Costa, Miguel1

1Pediatric Department of Centro Hospitalar de Entre Douro e Vouga, Portugal; 2Radiology Department of Instituto Português Oncologia do Porto Francisco Gentil, Portugal; 3Pediatric Department of Instituto Português Oncologia do Porto Francisco Gentil, Portugal

BACKGROUND: The anti-SARS-CoV-2 vaccine was administered to millions of people in a short time, placing challenges in monitoring adverse reactions. The appearance of signs/symptoms with a temporal relationship with the vaccine can interfere with their assessment and with the diagnosis of other pathologies. The most frequently described effects are mostly benign, such as headache, fever, myalgia, arthralgia, asthenia, adenopathy and/or pain.

CASE PRESENTATION SUMMARY: An eight-year-old girl, previously healthy, was observed by a Pediatrician due to a 4 weeks-long left axillary swelling noticed 2 weeks after the administration of 1st dose of the anti-SARS-CoV-2 vaccine. She doesn´t have any other symptoms. Trauma and recent infectious were denied. On physical examination, she had an oval mass, with 5 x 5 cm, hard, painless, adherent to the deep planes, and without inflammatory signs. (1) Analytical study was normal, and axillary ultrasound showed an oval mass, with a heterogeneous texture, with multiple septations and hypoechoic elements, suggesting a probable atypical adenopathy conglomerate in the context of a vaccine reaction. Therefore, she was medicated with amoxicillin + clavulanic acid and anti-inflammatory for one week. (2) Upon re-evaluation, as the mass presented similar characteristics, the case was discussed with Pediatric Oncology and was referred for evaluation by the specialty. Axillary ultrasound was repeated and showed heterogeneous formation, with cystic foci and areas of vascularization with rare foci of calcification, and MRI confirmed a lesion suggestive of low-flow vascular malformation. The child was submitted to surgical excision, and histology showed intramuscular hemangioma.

DISCUSSION: Hemangiomas are benign tumours of the vascular endothelium characterized by a growth phase and an involution phase. Despite being benign and self-limiting in nature, some hemangiomas can cause complications. (1) This clinical case highlights the fact that, despite the temporal relationship with the anti-SARS-CoV-2 vaccine, in the face of persistent mass and warning signs, other etiologies should always be excluded.

Will be presented as Oral Presentation Virtually
ID: 235 / 07-OP-V-d2C: 7
Oral Presentation (Virtual)
Keywords: Candida spp, pediatric invasive candidiasis, mortality, risk factors

Pediatric Invasive Candidiasis: a 12-year retrospective study in a tertiary care hospital

Pedrosa, Inês1; Monteiro, Teresa2; Almeida, Teresa3; Faria, Carolina4; Reis-Melo, Ana4; Tavares, Margarida4

1Department of Pediatrics, Centro Hospitalar de Leiria, Portugal; 2Department of Pediatrics, Centro Hospitalar Universitário do Algarve, Portugal; 3Department of Clinical Pathology, Centro Hospitalar Universitário São João, Portugal; 4Pediatric Infectious Diseases and Primary Immunodeficiencies Unit, Centro Hospitalar Universitário São João, Portugal

BACKGROUND: Invasive candidiasis (IC) is a major cause of morbimortality, mainly among children with underlying medical conditions. Rates of IC are increasing worldwide, probably due to the prolonged survival of immunocompromised patients and the increased use of invasive devices. We aim to describe the epidemiology and clinical disease of IC in a tertiary pediatric hospital.

METHODS: Retrospective audit of culture-confirmed Candida isolates from a tertiary hospital’s Microbiology department over a 12-year period. Patients’ demographic, clinical characteristics and causative Candida species were analyzed. We divided patients into age groups: >28 days to <1 year (infants) and 1 to <18 years of age (children/adolescents). The study period was divided into 2 periods (2010-2015; 2016-2021) to analyze trends over time.

RESULTS: There were 40 potential episodes of IC, 7 of which were excluded (6 contaminants; 1 without available chart information). Overall, 33 IC episodes were included (31 bloodstream, 1 CSF and 1 pleural effusion isolate). The median age was 21 months (39.4% infants; 60.6% children/adolescents), with a predominance of the female sex (57.6%). At diagnosis, most patients were in the general pediatric ward (39.4%), ICU (27.3%) or haematology-oncology unit (18.2%). The median length of stay was 44 days (range 4-788). Almost half of the patients required admission to ICU during their hospital stay (48.5%). The most common species were Candida parapsilosis (45.5%) and Candida albicans (39.4%). The predisposing factors for IC were CVC use (93.9%), parenteral nutrition (51.5%), bacteremia (57.5%), mechanical ventilation (39.4%), immunosuppression (33.3%), major surgery (33.3%) and neutropenia <500/uL (12.1%). The comorbidities more frequently identified were malignancy (33.3%), prematurity (27.3%) and gastrointestinal disease (12.1%). Antifungal treatment was given in 90.9% of cases (66.7% Amphotericin B, 16.7% Fluconazole), with a mean duration of 23 days (range 8-45). The all-cause mortality rate at 30 days was 21.2% (n= 7), and no significant differences between age groups and time periods were found. Higher mortality rates were reported among patients in ICU (p= 0.046), with concomitant bacteremia (p= 0.041) and recovering from major surgery (p= 0.049).

CONCLUSIONS: These results provided us with an insight into the clinical and fungal epidemiology of IC in our hospital. Remarkably, non-albicans Candida species were the leading cause of IC (60.6%), with C. parapsilosis being responsible for 45.5% of total cases, independently of ward type and age groups. The overall mortality rate is similar to other studies in pediatric settings, and the significant risk factors related with the mortality rate were ICU hospitalization, bacteremia and major surgery.

Will be presented as Oral Presentation Virtually
ID: 130 / 07-OP-V-d2C: 8
Oral Presentation (Virtual)
Keywords: Mastoiditis, Brain abscess, Otogenic infection

Primary conservative management of a paediatric otogenic cerebellar abscess

Qureshi, Muhammad Hamza1; Elbadawey, Morouge2; Reda, Mohamed3

1Darlington Memorial Hospital, United Kingdom; 2St George’s Medical School; 3Sheikh Shakhbout Medical City

BACKGROUND: Mastoiditis, dural sinus thrombosis and intracranial abscess formation are complications of otitis media. Traditionally intradural suppurative collections are drained by neurosurgeons, with the primary otogenic source of infection addressed either simultaneously or soon afterwards by otorhinolaryngologists. A new trend in neurosurgery is to treat small brain abscesses conservatively, providing drainage of the primary site. We present the first report of an otogenic brain abscess in a child successfully treated with conservative management of the collection.

CASE PRESENTATION SUMMARY: A previously healthy 4-year-old girl presented with right-sided otomastoiditis. After initially responding to IV antibiotics, her condition deteriorated, and imaging revealed a right-sided small cerebellar abscess and lateral sinus thrombosis. The otomastoiditis and venous sinus thrombosis were surgically treated, but the cerebellar abscess was managed with further IV antibiotics and completely resolved at 6 months.

LEARNING POINTS DISCUSSION: Intracranial and cranial complications should be considered in any case presenting with mastoiditis as a complication of acute otitis media. Changes in neurosurgical opinion are altering the preferred management of otogenic brain abscesses. This case demonstrates a small cerebellar abscess can be successfully managed conservatively in a child provided adequate drainage of the infective source.

Will be presented as Oral Presentation Virtually
ID: 197 / 07-OP-V-d2C: 9
Oral Presentation (Virtual)
Keywords: young children formula, L. reuteri, gastrointestinal tolerance

Real-world effectiveness of young child formula containing Limosilactobacillus reuteri on gastrointestinal tolerance and related behaviors in toddlers: a cross-sectional observational study

Estorninos, Elvira1; Cohen, Sarah2; Reichert, Heidi2; Lebumfacil, Jowena3; Jankovic, Ivana4; Chen, Yipu4; Egli, Delphine4

1Las Pinas Doctors Hospital, Las Pinas City, Philippines; 2EpidStrategies, Katy, TX, USA; 3Wyeth Nutrition, Makati City, Philippines; 4Société Des Produits Nestlé SA, Switzerland

BACKGROUND: Gastrointestinal tolerance is an important measure of digestive comfort. This study assessed the effectiveness of young child formula (YCF) containing Limosilactobacillus (previously Lactobacillus) reuteri (DSM 17938) (LR) on digestive comfort in a real-world setting.

METHODS: A cross-sectional, single timepoint, interview-based observational study was conducted among mothers of healthy toddlers born full-term. Children aged 12-36 months, free of any major chronic illness or food allergy, had no major changes to their current feeding regimen for at least 2 weeks prior to enrollment. Gastrointestinal tolerance, associated behaviours and stool characteristics were collected via the Gut Comfort Questionnaire. Toddlers were assigned to feeding groups based on their current feeding regimen, including YCF with LR (N=150), YCF containing other pre/probiotics (N=61), YCF containing no pre/probiotics (N=173), and non-YCF users (N=38). Mean differences (MD) and 95% confidence intervals (CI) were computed using ANOVA models.

RESULTS: The Gut Comfort composite score was significantly lower, indicating lower gastrointestinal distress, in the YCF with LR group compared to non-YCF users (MD=-1.83, 95% CI: -3.20; -0.45, p=0.01), YCF users with other pre/probiotics (MD=-1.29, 95% CI: -2.44; -0.14, p=0.028), and YCF without pre/probiotics (MD=-1.41, 95% CI: -2.26; -0.57, p=0.001). For individual tolerance measures, children receiving YCF with LR had lower parental reports of fussiness and irritability, constipation, and difficulty passing stools, compared with non-YCF users; lower reports of constipation and difficulty passing stools, compared with users of YCF containing other pre/probiotics; and lower reports of fussiness and irritability, constipation, problematic sleeping, diarrhea and gassiness, compared with users of YCF with no pre/probiotics. The frequency of crying was the lowest in the YCF with LR users compared with all other groups, and the appetite scale was the highest compared to other groups except non-YCF users. Stool consistency scores were similar between YCF with LR and non-YCF users, but higher (i.e., harder) for YCF with LR users when compared with YCF containing other pre/probiotics (MD=0.21, 95% CI: 0.01; 0.41, p=0.042) and YCF with no pre/probiotics users (MD=0.15, 95% CI: 0.01; 0.30, p=0.041). The majority of stools passed by children in all feeding groups were formed. Caregivers’ opinion of YCF with LR was overwhelmingly “Very good” to “Excellent” (97.6% and 96%, respectively).

CONCLUSIONS: In this real-world observational study, children consuming YCF with LR had better digestive comfort and associated behaviours than non-YCF users and children consuming YCF with other pre/probiotics or no pre/probiotics. YCF with LR also had high satisfaction ratings from caregivers.

Will be presented as Oral Presentation Virtually
ID: 162 / 07-OP-V-d2C: 10
Oral Presentation (Virtual)
Keywords: Friedreich’s ataxia; respiratory function

Respiratory function in Friedreich’s Ataxia

Martinuzzi, Michela2; Vinante, Elena1; Colombo, Elena1; Paparella, Gabriella1; Martinuzzi, Andrea1

1IRCCS “E. Medea”, Department of Neurorehabilitation, 31015 Conegliano, Italy; 2Sandwell and West Birmingham NHS tFoundations trust, United Kingdom

BACKGROUND: Friedreich's ataxia (FRDA) is an inherited, rare, progressive disorder in children and young adults. It is characterized by ataxia, loss of gait, scoliosis, cardiomyopathy, dysarthria, and dysphagia, with reduced life expectancy. Alterations of respiratory dynamics and parameters are frequently observed. However, there are few dated studies with small cohorts in the literature. (1) We report the objective analysis of the respiratory condition of both early and late-stage FRDA patients, looking for correlations with the motor, skeletal, speech and genetic aspects of this condition.

MATERIALS AND METHODS: This retrospective observational study is based on the collection of clinical and instrumental respiratory data of 44 subjects with molecularly defined FRDA attending a tertiary rehabilitation centre in Italy. The following parameters were recorded: FVC (forced vital capacity), FEV1 (forced expiratory volume in 1s), TI (Tiffeneau index), MIP (maximal inspiratory pressure), MEP (maximal expiratory pressure), PCF (peak cough flow) and PEF (peak expiratory flow). The analysis was carried out using Pearson's correlation test, ANOVA and Post Hoc tests.

RESULTS: Data show the presence of a recurrent restrictive pattern of respiratory dysfunction, with a reduction of forced vital capacity (2.84L +- 0.86, 74.83% +- 18.21 of predicted) and of flow and pressure parameters. 80% of participants were below the minimum values for MEP and 66.7% for MIP. Cough was found to be potentially inefficient in 15/44. 6 patients were found to have mild obstructive sleep apnoea (OSA) and one severe OSA. The severity of the respiratory condition correlates with disease severity (p<0.001 for FVC, FEV1 and MEF) duration (p>0.04 for FVC), pneumophonic capacity (p>0.001 for FVC and FEV) and with severity of the thoracic scoliotic curve (p<0.020). There was no correlation between respiratory measures and GAA expansion.

CONCLUSION: The complex condition of incoordination and hyposthenia in FRDA affects daytime and night-time respiratory efficiency. We show that the respiratory deficit and the inefficiency of cough are a prevalent clinical problem deserving of early consideration.

Will be presented as Oral Presentation Virtually
ID: 200 / 07-OP-V-d2C: 11
Oral Presentation (Virtual)
Keywords: Emergency, Febrile, Oncology, CXR

The role of CXR in investigation of febrile paediatric oncology patients presenting to the emergency department

Guinness, Freya; Abdul Nabi, Sarah; El Kebbi, Ola; Khamis, Mohamad; Tamim, Hani; Sawaya, Rasha D.

HSE, Ireland

INTRODUCTRION: Fever is a common presenting complaint to the emergency department (ED) in paediatric oncology patients. While bacteraemia has been extensively studied in this population, pneumonia has not. Several studies suggest that chest x-ray (CXR) does not have a role in the investigation of neutropaenic fever in the absence of respiratory symptoms, however, this has not been studied in non-neutropaenic patients.

AIM: We aimed to look at the diagnostic yield of CXRs in all paediatric oncology patients, irrespective of their ANC level, presenting with fever to the ED, as well as to identify predictive features of radiographic pneumonia.

METHODS: This is a secondary analysis of a retrospective cohort study of acutely febrile paediatric cancer patients younger than 18 years, presenting to a tertiary center from 2014 to 2018. We included one randomly selected febrile visit per patient. We defined fever as a single oral temperature ≥38°C. Data on patient characteristics and outcomes were collected. Our primary outcome was radiographic pneumonia; our secondary outcome was whether a CXR was done or not. Definite radiographic pneumonia was defined as a report mentioning a consolidation, pleural effusion, infiltrate, or pneumonia. Possible radiographic pneumonia was defined as a report with “infiltrate VS atelectasis”, or possible pneumonia mentioned by the radiologist. Anything else was considered negative for pneumonia. Statistical analysis was done using SPSS, where both outcomes were analysed.

RESULTS: We reviewed a total of 664 charts and included data from 342 febrile pediatric patients in our analysis. Of these, 64 (N = 18.7%) had a CXR performed. 16 (N = 25%) had radiographic pneumonia while 48 (N = 75%) did not. Patients were significantly more likely to have a CXR performed if they presented with upper respiratory tract symptoms or cough (p < 0.001 for both). Patients were also more likely to have a CXR if they were asymptomatic upon admission to the PED (p < 0.001) or if they had abnormal lung auscultation at the bedside (p = 0.003). However, neither cough nor shortness of breath nor an abnormal lung exam were significant predictors of a positive CXR (0.17, 0.43 and 1, respectively). Patients with radiographic pneumonia were found to be significantly older (4.29 vs 6 years, p = 0.03), with a longer time since their last chemotherapy (15 vs 7 days, p = 0.005), and were given intravenous (IV) bolus in the PED (87.5% vs 56.3%, p = 0.02).

CONCLUSION: In our sample, CXR was not commonly performed in the initial investigation of febrile PED cancer patients unless respiratory symptoms or abnormal lung examination were noted. However, these were not significant predictors of radiographic pneumonia. Further studies are needed to identify better predictors of pneumonia in this high-risk population.

Will be presented as Oral Presentation Virtually
ID: 214 / 07-OP-V-d2C: 12
Oral Presentation (Virtual)
Keywords: abdominal pain; adolescent; epiploic appendagitis

Acute abdominal pain: not always so obvious

Azevedo, Inês Alexandra; Cardoso, Rosa; Mendes, Joana; Almeida, Claudia; de Figueiredo, Catarina Matos; Pereira, Magalys

Pediatric Department of Centro Hospitalar de Entre Douro e Vouga, Portugal

BACKGROUND: Acute abdominal pain is a frequent cause of seeking medical care in Pediatrics. The origin has a wide spectrum of possibilities, and it is important to recognize its etiology for the correct diagnostic and therapeutic approach.

CASE PRESENTATION SUMMARY: A 17-year-old male with obesity (BMI 35.2 kg/m2), insulin-resistance (HOMA-IR 13.2) and vitamin D deficiency under treatment with metformin and vitamin D was observed at the Pediatric Emergency Department due to abdominal pain in the lower quadrants since the day before. Intestinal transit and diuresis are maintained with normal characteristics. No history of fever or recent infections. No other associated symptoms. (1) On physical examination, he presented a globose abdomen with adipose panniculus, and the left iliac region was painful, with defense. An enema was performed, with an output of a moderate amount of stool. For maintaining abdominal pain, an analytical study was performed without significant changes. The abdominal ultrasound documented a hyperechogenic area in the left iliac region, with 4.5 x 2 cm, in the adipose planes adjacent to the anterior abdominal wall. The abdomen and pelvis computed tomography confirmed the diagnosis of epiploic appendagitis. Outpatient treatment with anti-inflammatory and esomeprazole was recommended. He presented good clinical evolution, with a complete resolution of symptoms in 2 weeks.

DISCUSSION: Epiploic appendagitis is normal outpouchings of peritoneal fat on the surface of the colon. The evolution is usually benign, self-limited, and complete resolution usually occurs without surgical intervention. This is usually diagnosed incidentally in children undergoing imaging for acute abdominal pain. The differential diagnosis consists of other causes of acute abdominal pain. So, inaccurate diagnosis can lead to unnecessary hospitalizations, antibiotic therapy, and surgical intervention.

Will be presented as Oral Presentation Virtually
ID: 164 / 07-OP-V-d2C: 13
Oral Presentation (Virtual)
Keywords: diabetes mellitus, neonatal diabetes, sulphonylurea

Sweet babies: fifteen year experience of neonatal diabetes in a tertiary Irish paediatric diabetes centre

Woon, Yuxin; Holcroft, Lorna; Somers, Eric; Cody, Declan; O' Connell, Susan M

Department of Diabetes & Endocrinology, Children's Hospital Ireland at Crumlin, Dublin, Ireland

BACKGROUND: Neonatal diabetes mellitus (NDM) is a rare form of diabetes presenting before six months of age.1 It is typically a monogenic form of diabetes and presents as permanent neonatal diabetes mellitus (PNDM), transient neonatal diabetes mellitus (TNDM) or syndromic neonatal diabetes. Accurate genotyping is important for prognostic counselling and as those with a mutation in genes encoding subunits of the KATP channel may be responsive to treatment with sulphonylureas.

AIM: To review the phenotypes, genotypes and management of eight children with NDM at the largest Irish paediatric diabetes centre.

METHODS: We interrogated our database for cases of NDM with known genetic mutations between 2006-2021.

RESULTS: Eight cases of NDM were treated – 4 each of PNDM and TNDM. All 4 PNDM cases were due to a KCNJ11 gene mutation. 3 TNDM cases were due to 6q24 methylation defects, and 1 due to an ABCC8 mutation. 5 cases were responsive to sulphonylurea therapy.

DISCUSSION: Treatment with sulphonylurea was more successful in cases where the medication was started at a younger age. Cases with 6q24 methylation defects presented with a similar phenotype of IUGR, prematurity and TNDM. Fifty per cent of cases reviewed presented diabetic ketoacidosis, all of which had activating KATP channel mutations. The genetic diagnosis had implications for the family members of our patients. All of our TNDM were off insulin within the first year of life and given advice on possible recurrence of diabetes mellitus later in life.

CONCLUSION: Due to increased awareness of personalised medicine, early genetic diagnosis of NDM guides treatment and informs prognosis and future risk for the patient and their families.

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