Paediatrics (RCH) - Research Publications
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ItemFrench Intensive Care Society, International congress - Réanimation 2016.(Springer Science and Business Media LLC, 2016-06)
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ItemNo Preview AvailableDisease Spectrum and Management of Children Admitted with Acute Respiratory Infection in Viet Nam(Wiley, 2017-11-23)Background: Acute respiratory infection (ARI) is the most common reason for admission to paediatric wards in Viet Nam, being responsible for 39.9% of hospital admissions and 7.9% of hospital deaths in southern Viet Nam. However, few studies have explored the ARI disease spectrum observed in central Viet Nam or differences between primary (district), secondary (provincial) and tertiary (national) level hospitals. Aims: To assess the acute respiratory infection (ARI) disease spectrum, duration of hospitalisation and outcome in children hospitalised with an ARI in Viet Nam. Methods: We conducted a retrospective descriptive study of ARI admissions to primary (Hoa Vang District Hospital), secondary (Da Nang Hospital for Women and Children) and tertiary (National Hospital of Paediatrics in Ha Noi) level hospitals in Viet Nam over a 12-month period(01/09/2015 to 31/08/2016). Results: ARIs accounted for 27.9% (37,436 / 134,061) of all paediatric admissions; nearly half (47.6%) of all children admitted to Hoa Vang District Hospital. Most (64.6%) children hospitalised with an ARI were<2 years of age. Influenza/pneumonia accounted for 69.4% of admissions; tuberculosis for only 0.3%. Overall 284 (0.8%) children died; most deaths (269/284; 94.7%) occurred at the tertiary referral hospital. The average duration of hospitalization was 7.6 days (median 7 days). The average direct hospitalization cost per ARI admission was 157.5 USD in Da Nang Provincial Hospital. In total, 62.6% of admissions were covered by health insurance. Conclusions: ARI is a major cause of paediatric hospitalization in Viet Nam, characterized by prolonged hospitalization for relatively mild disease. There is huge potential to reduce unnecessary hospital admission and cost.
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ItemTobacco product use and smoking frequency among US adults with intellectual and developmental disabilities(WILEY, 2018-08)BACKGROUND: People with intellectual and developmental disabilities (IDD) have been overlooked in tobacco use research although they are likely to experience tobacco-related health disparities. This study examined tobacco product use and smoking frequency and amount among a sample of US Special Olympics athletes with IDD. METHODS: Multiple regression analysis was used to test whether age, gender, body mass index, blood pressure, bone density, eating fruits and vegetables and family member tobacco use were correlated with the number of cigarettes smoked per day. RESULTS: The sample of people with IDD who used tobacco (n = 501) were aged 18-75 (M = 33.37) and 76.4% were male. About 73.6% reported cigarette use only, 10.6% reported dual or poly use of cigarettes and other tobacco products (cigars, pipe, and chewing tobacco) and 15.8% reported using only tobacco products other than cigarettes. Men were more likely than women to use tobacco products other than cigarettes. Of the cigarette smokers, 79.6% were daily smokers, and their mean cigarettes per day was 10.08 (SD = 9.50). Special Olympics athletes who did not have low bone density and those who consumed fruits and vegetables less than daily reported higher numbers of cigarettes per day. CONCLUSIONS: Although people with IDD are less likely to use tobacco than the general population, study results suggest that people with IDD who smoke cigarettes are just as likely as smokers in the general US population to smoke daily. Improving overall health behaviours may be important in helping smokers with IDD to reduce their tobacco use. Research is needed to understand longitudinal patterns of tobacco use and how to prevent tobacco use among people with IDD.
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ItemNo Preview AvailablePRECISION MEDICINE FOR PAEDIATRIC HIGH-GRADE DIFFUSE MIDLINE GLIOMAS - RESULTS FROM THE ZERO CHILDHOOD CANCER COMPREHENSIVE PRECISION MEDICINE PROGRAM(OXFORD UNIV PRESS INC, 2020-12)Abstract The Australian Zero Childhood Cancer (ZERO) program aims to assess the feasibility of a comprehensive precision medicine approach to improve outcomes for patients with an expected survival <30%. ZERO combines molecular profiling (whole genome sequencing, whole transcriptome sequencing, DNA methylation profiling) with in vitro high-throughput drug screening (HTS) and patient-derived xenograft drug efficacy testing. We report on the cohort of patients with midline high-grade glioma (HGG), including H3-K27M DMG, enrolled on the pilot study (TARGET) and on the ongoing ZERO clinical trial (PRISM). We identified 48 patients with midline HGG. Fresh or cryopreserved samples were submitted in 37 cases and cell culture was attempted in 30/37 cases with 45% success rate. The most commonly mutated genes/pathways identified by molecular profiling include H3-K27M mutations, DNA repair pathway, and PI3K/mTOR pathway. Two targetable fusions (NTRK and FGFR1) were reported. Five patients with germline alterations were identified. Thirty-five (72%) patients received a therapeutic recommendation from the ZERO molecular tumour board and the main recommended therapies were mTOR inhibitors, PARP inhibitors or tyrosine kinase inhibitors. HTS added evidence for the recommended therapy (n=3) or identified novel potential therapy (n=1). Out of the 35 patients, 16 received a recommended drug. Response to treatment was complete response for five months (n=1), partial response for nine months (n=1), stable disease (n=4), and progressive disease (n=10). These results highlight the feasibility of the ZERO platform and the value of fresh biopsy, necessary for pre-clinical drug testing. Targetable alterations were identified leading to clinical benefit in six patients.
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ItemA PHASE II STUDY OF CONTINUOUS LOW DOSE PANOBINOSTAT IN PAEDIATRIC PATIENTS WITH MALIGNANT RHABDOID TUMORS/ATYPICAL TERATOID RHABDOID TUMORS(OXFORD UNIV PRESS INC, 2020-12)Abstract
BACKGROUND
Panobinostat treatment has been shown to terminally differentiate malignant rhabdoid tumor (MRT)/atypical teratoid rhabdoid tumors (ATRT) in pre-clinical models. This is an open label, phase II study of panobinostat in patients with newly diagnosed or relapsed MRT/ATRT. AIMS: To assess the anti-tumor activity of low dose, continuous panobinostat, its associated toxicities, the biological activity of low dose panobinostat by measuring histone acetylation status in peripheral mononuclear cells (PMNC), and markers of differentiation in fresh tumor tissue specimens.METHODS
Following cycles of induction and consolidation chemotherapy and/or radiation treatment, patients were enrolled and commenced on panobinostat as a continuous daily oral dose starting at 10mg/m2 following a three-week wash out period between therapies. Real-time acetylation status, measuring acetylated H4 on PMNC, was performed to determine the pharmacodynamics of panobinostat. Patients were monitored for drug toxicities with the possibility of dose reductions in decrements of 2mg/m2.RESULTS
Six patients with newly diagnosed ATRT/MRT and one patient with relapsed MRT have been enrolled to date. The average age at enrollment was 2.5 years. Currently, six patients (85.7%) remain on study with a mean treatment duration of 170 days (range 44–327 days). One patient was removed from study at day 44 due to disease progression. The main dose-limiting toxicity observed to date has been myelosuppression. Panobinostat, at a dose of 10mg/m2, caused significant acetylation of H4 in PMNC.CONCLUSIONS
Treatment with panobinostat appears to be well tolerated in infants with MRT/ATRT, with successful real-time pharmacodynamic assessment of H4 acetylation. -
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ItemNo Preview AvailablePlasma Phosphorylated Neurofilament Heavy Chain Levels Over Time in Participants With Infantile-and Later-onset SMA: Data from the SHINE Study(Lippincott Williams & Wilkins, 2020-04-14)
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ItemNo Preview AvailableInvolving families in the design of a weight management program for Duchenne muscular dystrophy - Supporting nutrition and optimising wellbeing program(PERGAMON-ELSEVIER SCIENCE LTD, 2020-10)
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ItemPediatric Charcot-Marie-tooth disease clinical practice guideline(Wiley, 2020-12-01)