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关键词： Albumin-bound paclitaxel   Ewing sarcoma   Neuroblastoma   Paediatric   Rhabdomyosarcoma   Solid tumour  

摘要： Background: The phase I component of a phase I/II study defined the recommended phase II dose and established the tolerability of nab-paclitaxel monotherapy in paediatric patients with recurrent or refractory solid tumours. The activity and safety of nabpaclitaxel monotherapy was further investigated in this phase II study. Patients and methods: Paediatric patients with recurrent or refractory Ewing sarcoma, neuroblastoma or rhabdomyosarcoma received 240 mg/m(2) of nab-paclitaxel on days 1, 8 and 15 of each 28-day cycle. The primary end-point was the overall response rate (ORR;complete response [CR] + partial response [PR]). Secondary end-points included duration of response, disease control rate (DCR;CR + PR + stable disease [SD]), progression-free survival, 1-year overall survival, safety and pharmacokinetics. Results: Forty-two patients were enrolled, 14 each with Ewing sarcoma, neuroblastoma and rhabdomyosarcoma. The ORRs were 0%, 0% and 7.1% (1 confirmed PR), respectively. The DCRs were 30.8% (4 SD), 7.1% (1 SD) and 7.1% (1 confirmed PR and 0 SD) in the Ewing sarcoma, neuroblastoma and rhabdomyosarcoma groups, respectively. The median progression-free survival was 13.0, 7.4 and 5.1 weeks, respectively, and the 1-year overall survival rates were 48%, 25% and 15%, respectively. The most common grade III/4IVadverse events were haematologic (neutropenia [50%] and anaemia [48%]), and grade III/IV peripheral neuropathy occurred in 2 patients (14%) in the rhabdomyosarcoma group. Pharmacokinetics analyses revealed that paclitaxel tissue distribution was both rapid and extensive. Conclusions: In this phase II study, limited activity was observed;however, the safety of nabpaclitaxel in paediatric patients was confirmed. Published by Elsevier Ltd.

关键词： HLA typing   endomysial antibody   Coeliac disease  

摘要： Coeliac disease (CD) diagnosis is based on clinical assessment, detection of specific autoantibodies and histological examination of small intestinal biopsies. The European Society of Paediatric Gastroenterology Hepatology and Nutrition (ESPGHAN) guidelines have recently been updated and recommend CD may be diagnosed without a biopsy or HLA typing in symptomatic patients with high titre IgA tissue transglutaminase antibodies (aTTG) and positive endomysial antibodies (EMA). However, the need for EMA in patients with high level aTTG has been questioned. We aimed to determine the diagnostic benefit of HLA typing, EMA and IgG antibodies to deamidated gliadin (DGP) in children with high level aTTG. We prospectively evaluated children presenting for assessment of possible CD. All patients underwent small bowel biopsy, serological testing and HLA typing. Results were analysed and correlated with histopathological diagnosis. A total of 209 children were assessed;61.5% were found to have CD and 29% could have avoided biopsy as per 2020 ESPGHAN guidelines. Titres of aTTG >= 60 U/mL or DGP >= 28 U/mL gave 100% specificity and 100% positive predictive value (PPV) for CD. HLA typing and EMA did not improve the PPV of patients with aTTG >= 60 U/mL, but addition of DGP >= 28 U/mL improved diagnostic sensitivity whilst retaining 100% specificity. Addition of HLA and EMA testing in patients with high titre aTTG antibodies does not improve diagnostic performance and may possibly be omitted from the serological workup in these patients. Our data support combining aTTG and DGP testing and optimising cut-offs to maximise specificity as an alternative biopsy-free diagnostic approach.

关键词： COVID-19   paediatric services   survey  

摘要： Aims COVID-19 is now a global pandemic. At the time of survey, fewer than 150 children in Australia and New Zealand had documented infection. The aim of this study was to assess attitudes, readiness and confidence in the early stages of the COVID-19 pandemic through an online survey of paediatric physicians and sub-specialists across Australia and New Zealand. Methods Multiple email list groups were used to contact paediatric physicians to undertake an online Likert scale survey between 17 and 24 March. Respondents' specialty, experience and work setting were recorded. Ordinal logistic regression was used to determine respondent factors. Results There were 542 respondents from across Australia and New Zealand: an estimated 11% of the paediatric physician workforce. A minority (36.6%) agreed that their national response had been well coordinated;the majority (92.7%) agreed that senior-level hospital administrators were taking the situation seriously. Most reported a good understanding of the natural history of COVID-19 in children, and knowledge of where to find local information. A large proportion of physicians (86.1%) were worried about becoming infected through their work;few (5.8%) reported that they would not come to work to avoid infection. Closure of school and childcares would reduce the ability to continue work at current capacity for 23.6% of respondents. Conclusion Despite limited experience in pandemics, most paediatric physicians felt informed. Concern about exposure at work is common;most were willing to work regardless. The closure of schools and daycares may have an impact on staffing. Coordination and leadership will be critical.

关键词： brachial plexus block   fasting   paediatric anaesthesia   regional anaesthesia   sedation   ultrasound-guided regional anaesthesia  

摘要： Morphoea, also known as localized scleroderma, is a debilitating fibrosing disorder of uncertain aetiology, affecting the skin and subcutaneous tissues. Paediatric-onset disease is not uncommon and is associated with frequent relapses. The disease has complex pathogenetic mechanisms and multiple clinical subtypes, and affects children of all ages. Recent research has focused on elucidating the disease pathophysiology and identifying measures of disease activity. We performed a literature search on PubMed, MEDLINE and Google Scholar, using keywords such as 'pediatric morphea', 'juvenile localised scleroderma' and 'juvenile systemic sclerosis'. Relevant studies, including randomized trials, reviews of standard current guidelines and original research articles, were selected, and results were analysed before being summarized. In the first of this two-part review, we provide a bird's-eye view of the current literature concerning the epidemiology, aetiopathogenesis and clinical classification of paediatric morphoea;in Part 2, we review the diagnosis, markers of disease activity, management and natural history.

关键词： behavioural treatment   childhood obesity treatment   implementation   parental education   personalised medicine  

摘要： Aim To investigate the implementation of a plan of action for treatment of childhood obesity, and the effect after 2 years of treatment. Methods Children aged 6-12.9 years who started obesity treatment between 2008 and 2015 in a paediatric clinic in Stockholm County were included. The treatment model included staff education and support and group activities for parents and children separately followed by individual sessions to a multidisciplinary team. The main outcome was change in body mass index standard deviation score (BMI SDS), in comparison to a matched control group. Results In the intervention group, 1334 children (52% boys) with an average age of 9.3 years and BMI SDS of 2.7 and 3012 children in the control group were included. The intervention group decreased their BMI SDS more after two years compared with the control group, (-0.31 vs -0.23, P < .001). Younger age and higher BMI SDS at treatment initiation and families that completed the group sessions (all P < .001) had greater decreases in BMI SDS after 2 years. Sex did not affect the outcome. Conclusion Even though the treatment in the control group was effective, the implementation of the action plan yielded a better treatment response compared with the control group.

关键词： inflammation   outcome and process assessment   health care   cytokines  

摘要： Background Current data suggest that COVID-19 is less frequent in children, with a milder course. However, over the past weeks, an increase in the number of children presenting to hospitals in the greater Paris region with a phenotype resembling Kawasaki disease (KD) has led to an alert by the French national health authorities. Methods Multicentre compilation of patients with KD in Paris region since April 2020, associated with the detection of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) ('Kawa-COVID-19'). A historical cohort of 'classical' KD served as a comparator. Results Sixteen patients were included (sex ratio=1, median age 10 years IQR (4.7 to 12.5)). SARS-CoV-2 was detected in 12 cases (69%), while a further three cases had documented recent contact with a quantitative PCR-positive individual (19%). Cardiac involvement included myocarditis in 44% (n=7). Factors prognostic for the development of severe disease (ie, requiring intensive care, n=7) were age over 5 years and ferritinaemia >1400 mu g/L. Only five patients (31%) were successfully treated with a single intravenous immunoglobulin (IVIg) infusion, while 10 patients (62%) required a second line of treatment. The Kawa-COVID-19 cohort differed from a comparator group of 'classical' KD by older age at onset 10 vs 2 years (p<0.0001), lower platelet count (188 vs 383 G/L (p<0.0001)), a higher rate of myocarditis 7/16 vs 3/220 (p=0.0001) and resistance to first IVIg treatment 10/16 vs 45/220 (p=0.004). Conclusion Kawa-COVID-19 likely represents a new systemic inflammatory syndrome temporally associated with SARS-CoV-2 infection in children. Further prospective international studies are necessary to confirm these findings and better understand the pathophysiology of Kawa-COVID-19.

关键词： Cone beam computed tomography (CBCT)   Paediatric   Clinical indication   Dental radiology  

摘要： Introduction Cone beam computed tomography (CBCT) is used across all dental specialties and has a number of advantages compared to 2D images. The SEDENTEXCT guidelines provide a number of indications for the use, however there are currently no specific guidelines for paediatric dentistry. The aim of this study was to assess current practice of CBCT imaging within paediatric dental departments in England, audit compliance of CBCT justifications against the standards set by SEDENTEXCT and assess whether the use of CBCT affected the treatment plan for each individual patient. Methods From the retrospective analysis of CBCT examinations taken over a 4-year period across three dental hospitals in the north of England, the following data were collected: age at the time of exposure, clinical indication, region of interest (ROI) and diagnostic findings. Clinical notes were also used to identify whether the CBCT had an effect on the final treatment plan. Results A total of 335 CBCT examinations were performed, mean age: 11 years. The number of CBCTs increased each year with a twofold increase in the first 2 years. The main clinical indication in 46% of CBCT examinations was the assessment of localised developing dentition, 68% were in the upper anterior sextant and 61% of CBCT exams were in the mixed dentition age group. The investigations were justified in 100% of the cases. Conclusion The quantity of CBCT examination in paediatric dental patients is increasing to assist treatment planning but more often to enable improved surgical planning.

摘要： A polyphenotypic malignant paediatric brain tumour presenting a MN1-PATZ1 fusion, no epigenetic similarities with CNS High-Grade Neuroepithelial Tumour with MN1 Alteration (CNS HGNET-MN1) and related to PATZ1-fused sarcomas Central nervous system high-grade neuroepithelial tumour with I MN1 i alteration (CNS HGNET-MN1) is a brain tumour methylation class that has recently been identified, based on (epi)genetic profiles of a large CNS-PNETs cohort [1]. In addition, the tumour exhibited a polyphenotypic immunoprofile, a feature described in CNS HGNET-MN1 [4] but also pointing toward other tumour entities, such as atypical teratoid rhabdoid tumour or I CIC-NUTM1 i sarcoma [5]. [Extracted from the article]