Quality of life in childhood immune thrombocytopenia: Revision of the Kids' ITP Tools (KIT).

The Kids ITP Tools (KIT) is a health-related quality of life (HRQoL) questionnaire that evaluates quality of life in children with immune thrombocytopenia (ITP). There are three formats: Child Self-Report, Parent Proxy-Report and Parent Impact-Report. This study aimed to develop a domain structure by grouping-related questions from the questionnaire into domains that independently reflect various aspects of HRQoL. The study was conducted in two phases. Phase 1 involved an online survey distributed to experts to identify conceptual domains for the KIT. Phase 2 utilized a statistical approach to analyse responses from patients with ITP and their families. A revised KIT 2.0 was ultimately developed to aid in treatment decision-making and monitoring of ITP.

Assessing the implementation of a tertiary care comprehensive pediatric asthma education program using electronic medical records and decision support tools.

BACKGROUND: Self-management education is integral for proper asthma management. However, there is an accessibility gap to self-management education following asthma hospitalizations. Most pediatric patients and their families receive suboptimal or no education. OBJECTIVE: To implement a comprehensive pediatric asthma education program and evaluate subsequent self-management knowledge in patients as well as behavior change outcomes reflected in the frequency of asthma related repeat emergency department visits and hospitalization. The program implementation was informed by the Knowledge to Translation Action Framework and the i-PARIHS model for quality improvement and involved several iterative stages. METHODS: We implemented a comprehensive asthma education program for the families of all children 0-18 years old who had been admitted for an asthma exacerbation to the Children's Hospital of Eastern Ontario (CHEO), beginning on April 1, 2018. The program was adapted to the stages of the Knowledge Translation to Action Framework including undertaking an environmental scan, expert stakeholder feedback, reviews, addressing barriers, and tailoring the intervention, along with evaluating knowledge and health outcomes. Education was delivered over 1-2 h in personalized individual or small group settings, within 4 wk of hospital discharge. All education was provided by registered nurses or respiratory therapists who were also certified asthma educators. The EPIC electronic medical record was used to facilitate referral and scheduling of asthma education sessions, and to track subsequent acute asthma visits. We compared the frequency of a repeat asthma emergency department (ED) visit or hospitalization within 1-year following an initial asthma hospitalization for children who would have received comprehensive asthma education, to a historical cohort of children who were hospitalized between April 9, 2017 - Apr 8, 2018, and did not receive asthma education. RESULTS: The program had a high enrollment, capturing nearly 75% of the target population. Most families found the program to be acceptable and reported increased knowledge of how to manage asthma. We identified a crude overall 54% reduction in repeat hospitalizations among children 1 year after implementation of the asthma education program (i.e. 10.2% (23/225) repeat hospitalization rate pre- implementation versus 4.8% (11/227) post-implementation). In adjusted time-to event analysis, this reduction was prominent at 3 months among those who received comprehensive asthma education, relative to those who did not, but this improvement was not sustained by 1 year (HR =1.1, 95% CI =0.55- 2.05; p-value = 0.6). DISCUSSION: Although we did not find long-term improvements in ED visits, or hospitalizations, in children of caregivers who participated in comprehensive asthma education, the asthma education program holds potential given that most patients found it to be acceptable and that it increased asthma management knowledge. A future asthma education program should include multiple sessions to ensure that the knowledge and behavior change will be sustained, leading ultimately to long-term reductions in repeat ED visits and hospitalizations.

Natural history of Chiari I malformation with syrinx and dilatation of the central canal in the pediatric population: the CHEO experience.

PURPOSE: Given that syrinx is often considered an indication of surgery in children with Chiari I malformation (CM1), understanding of the natural history of these patients is very challenging. In this study, we investigate the natural history of children with CM1 that have syrinx and/or prominence of the central canal on presentation. METHODS: All pediatric Chiari I patients who had syrinx and/or prominence of the central canal who underwent MR imaging of the head and spine from 2007 to 2020 were reviewed. Patients were divided into 3 groups (early surgery, delayed surgery, and conservative management). We focused on those patients who did not initially undergo surgery and had at least 1 year of clinical follow-up. We assessed if there were any radiological features that would correlate with need for delayed surgical intervention. RESULTS: Thirty-seven patients met the inclusion criteria. Twenty-one patients were female and 16 were male. The mean age at presentation was 8.7 (5.8 SD). Fourteen (38%) patients had early surgical intervention, with a mean of 2.5 months after initial presentation, 8 (16%) had delayed surgery due to new or progressive neurological symptoms and 46% of patients did not require intervention during follow-up. The length of tonsillar herniation and the position of the obex were associated with the need of surgery in patients who were initially treated conservatively. CONCLUSION: In pediatric patients with CM1 with syringomyelia and prominence of the central canal, conservative treatment is initially appropriate when symptoms are absent or mild. Close follow-up of patients with CM1 and dilatation of the central canal who have an obex position below the foramen magnum and greater tonsillar herniation is suggested, as these patients show a trend towards clinical deterioration over time and may require earlier surgical intervention.

CT contrast extravasation in children: a single-center experience and systematic review.

BACKGROUND: Extravasation of iodinated contrast material during computed tomography (CT) is a rare complication. A few patients may develop severe complications such as compartment syndrome. OBJECTIVE: The purpose of this study was to retrospectively assess the prevalence, severity, management, and outcome of contrast extravasations in our institution and to perform a comparison to what has been reported in the existing literature. MATERIALS AND METHODS: This is a research ethics board (REB)-approved retrospective study comprising 11 patients who had intravenous contrast-enhanced CT between 2019 and 2022 in a tertiary pediatric center, and experienced extravasation of iodinated contrast as a complication. Age, weight, sex, co-morbidities, angiocatheter size, venous access location, total contrast volume, flow rate, patient's symptoms, severity of injury, and management were collected. For the systematic review, PRISMA guidelines were followed. RESULTS: Only 11 (0.3%) (0.17-0.54 (95%CI)) contrast extravasations occurred in a total of 3638 CTs performed with intravenous contrast during the same period in children. The median age (IQR) was 12.5 (10.0, 15.0) years. In our cohort, 1/11 patients developed compartment syndrome and required fasciotomy. The systematic review assessed 12 articles representing a population of 110 children with extravasations. Pooled prevalence from articles stratified by age was 0.32% (0.06-0.58% (95%CI)). Only three children experienced moderate to severe complications. CONCLUSIONS: We confirm that severe complications of contrast extravasation are rare and can occur at any age. No strong associations were seen with the need for surgical consultation (including age, sex, weight, flow rate, injection site, catheter size, and type of contrast).

Factors associated with future hospitalization among children with asthma: a systematic review.

OBJECTIVE: Asthma is a leading cause of emergency department (ED) visits and hospitalizations in children, though many could be prevented. Our study objective was to identify factors from the published literature that are associated with future hospitalization for asthma beyond 30 days following an initial asthma ED visit. DATA SOURCES: We searched CINAHL, CENTRAL, MEDLINE, and Embase for all studies examining factors associated with asthma-related hospitalization in children from January 1, 1992 to February 7, 2022.Selecting Studies: All citations were reviewed independently by two reviewers and studies meeting inclusion criteria were assessed for risk of bias. Data on all reported variables were extracted from full text and categorized according to identified themes. Where possible, data were pooled for meta-analysis using random effects models. RESULTS: Of 2262 studies, 68 met inclusion criteria. We identified 28 risk factors and categorized these into six themes. Factors independently associated with future hospitalization in meta-analysis include: exposure to environmental tobacco smoke (OR = 1.94 95%CI 0.67-5.61), pets exposure (OR = 1.67 95%CI 1.17-2.37), and previous asthma hospitalizations (OR = 3.47 95% CI 2.95-4.07). Additional related factors included previous acute care visits, comorbid health conditions (including atopy), allergen exposure, severe-persistent asthma phenotype, inhaled steroid use prior to ED visit, poor asthma control, higher severity symptoms at ED presentation, warmer season at admission, longer length of stay or ICU admission, and African-American race/ethnicity. CONCLUSIONS: We identified multiple factors that are consistently associated with future asthma hospitalization in children and could be used to identify those who would benefit from targeted preventative interventions.

The Association of Corticosteroids and Pediatric Sepsis Biomarker Risk Model (PERSEVERE)-II Biomarker Risk Stratification With Mortality in Pediatric Septic Shock.

OBJECTIVES: Mortality risk stratification may identify a subset of children who benefit from or are harmed by corticosteroid administration. The Pediatric Sepsis Biomarker Risk Model (PERSEVERE)-II score is a biomarker-based mortality risk stratification tool for pediatric sepsis. Our objective was to assess the association of corticosteroid administration with 28-day mortality within different levels of baseline mortality risk (PERSEVERE-II) in a cohort of children with septic shock. DESIGN: We performed a secondary analysis using prospectively collected data (January 2015 to December 2018). SETTING: PICUs in 13 tertiary care, academic centers in the United States. PATIENTS: Children with septic shock. INTERVENTIONS: None. MEASUREMENTS AND MAIN RESULTS: We assessed the association of corticosteroid administration within PERSEVERE-II risk score categories and 28-day mortality, ICU-free days, and maximum failed organs in children with septic shock. We analyzed a total of 461 patients (215 with corticosteroids exposure, 246 without corticosteroid exposure) with an average age of 7.1 years (interquartile range, 2.2-13.6 yr). In the subgroup of patients with a high PERSEVERE-II score, corticosteroid administration was associated with an increased adjusted risk of 28-day mortality (odds ratio [OR] 4.10 [95% CI 1.70-9.86]; p = 0.002), but not in the low risk group (OR 0.20 [95% CI 0.02-1.73]; p = 0.15). A significant interaction between PERSEVERE-II score and corticosteroids was seen for both secondary outcomes complicated course ( p = 0.01) and maximum failed organs ( p < 0.001). Corticosteroid exposure was associated with fewer ICU-free days ( p < 0.0001). CONCLUSIONS: In our multicenter observational study, corticosteroid administration was associated with increased mortality in a subgroup of children with a high PERSEVERE-II risk score.

Skin morbidity in Indigenous children in relation to housing conditions in remote communities in Northwestern Ontario, Canada.

BACKGROUND: Elevated rates of eczema and skin infections in Canadian First Nation (FN) communities are of concern to families, community leaders and healthcare professionals. AIM: To determine whether skin morbidity was associated with indoor environmental quality factors in Canadian FN children living in remote communities. METHODS: We quantified indoor environmental quality (IEQ) in the homes of FN children aged < 4 years of age living in four remote communities in the Sioux Lookout region of Northwestern Ontario, Canada. We conducted a quantitative housing inspection, including measuring surface area of mould (SAM), and monitored air quality for 5 days in each home, including carbon dioxide and relative humidity and quantified endotoxin in settled floor dust. We reviewed the medical charts of participating children for skin conditions and administered a health questionnaire. Relationships between IEQ and skin infections or eczema were evaluated using multivariable regression. RESULTS: In total, 98 children were included in the descriptive analyses, of whom 86 had complete data and were evaluated in multivariate analyses for dermatological outcomes (mean age 1.6 years). Of these 86 children, 55% had made ≥ 1 visits to the local health centre (HC) for skin and soft tissue infections and 25.5% for eczema. Unexpectedly, annualized eczema visits were inversely associated with SAM (RR = 0.14; 95% CI 0.01-0.93). There was a trend suggesting an inverse relationship between endotoxin and HC encounters for eczema and skin and soft tissue infections. CONCLUSION: Skin infections were common in this population of FN children. IEQ did not appear to be associated with skin infections or eczema. Mould exposure appeared to be inversely associated with HC encounters for eczema, possibly related to complex microorganism-host interactions occurring early in life.

Virtual psychoeducation for improvement of pain catastrophizing in pediatric presurgical patients and caregivers: a proof-of-concept study.

PURPOSE: Pain catastrophizing (PC) is the tendency to magnify the threat value of pain sensations and is associated with greater postsurgical pain intensity, functional disability, and pain chronicity. Higher parental PC predicts higher chronic postsurgical pain in youth. Treating PC in caregivers and youth prior to surgery may improve recovery and surgical outcomes. We developed and evaluated a psychoeducational workshop addressing PC for presurgical youth and their parents/caregivers. We hypothesized that parent/caregiver and youth PC scores would decrease over time. We also explored preintervention levels of youth anxiety and depression as moderators of outcome. METHODS: Youth (n = 43) and caregivers (n = 41) attended a virtual, group-based single-session intervention (SSI). Single-session intervention content addressed pain neuroscience, PC, and adaptive coping strategies for managing pain and PC drawn from cognitive-behavioural, acceptance and commitment, and dialectical behaviour therapy approaches. Participants completed questionnaires assessing PC at preintervention, postintervention, and two weeks postsurgery. Youth mood and anxiety were assessed at preintervention. RESULTS: Caregiver PC scores decreased from pre- to postintervention (P = 0.006), and this was maintained at postsurgery (P = 0.002). Youth PC scores decreased from preintervention to postsurgery, but only for those with higher preintervention anxiety (P = 0.01). CONCLUSION: Our results provide proof-of-concept support for a virtual SSI targeting caregivers and youth PC during the perioperative period. The present findings highlight the possible need to screen presurgical candidates for symptoms of anxiety. Replication with larger and more diverse samples, and a more robust design are warranted.

RéSUMé: OBJECTIF: Le terme de dramatisation de la douleur décrit la tendance à amplifier la valeur de menace des sensations de douleur et est associée à une plus grande intensité de la douleur postopératoire, à une incapacité fonctionnelle et à une chronicité de la douleur. Une dramatisation parentale plus élevée de la douleur prédit une douleur postopératoire chronique plus élevée chez les jeunes. Le traitement de la dramatisation de la douleur chez les soignant·es et les jeunes avant la chirurgie peut améliorer le rétablissement et les devenirs chirurgicaux. Nous avons mis au point et évalué un atelier psychoéducatif sur la dramatisation de la douleur destiné aux jeunes en période préchirurgicale et à leurs parents/soignant·es. Nous avons émis l'hypothèse que les scores de dramatisation de la douleur des parents/soignant·es et des jeunes diminueraient avec le temps. Nous avons également exploré les niveaux d'anxiété et de dépression des jeunes avant l'intervention en tant qu'éléments modérateurs des résultats. MéTHODE: Des jeunes (n = 43) et les personnes en prenant soin (n = 41) ont participé à une seule intervention virtuelle en groupe. Le contenu de l'intervention unique portait sur les neurosciences de la douleur, la dramatisation de la douleur et les stratégies d'adaptation pour la prise en charge de la douleur et la dramatisation de la douleur tirées des approches cognitivo-comportementales, d'acceptation et d'engagement, et de thérapie comportementale dialectique. Les participant·es ont rempli des questionnaires évaluant la dramatisation de la douleur avant l'intervention, après l'intervention et deux semaines après la chirurgie. L'humeur et l'anxiété des jeunes ont été évaluées avant l'intervention. RéSULTATS: Les scores de dramatisation de la douleur des soignant·es ont diminué de la période précédant à la période suivant l'intervention (P = 0,006), et cela s'est maintenu après la chirurgie (P = 0,002). Les scores de dramatisation de la douleur des jeunes ont diminué de la période précédant l'intervention à la période postchirurgie, mais seulement chez les jeunes présentant une anxiété pré-intervention plus élevée (P = 0,01). CONCLUSION: Nos résultats appuient la preuve de concept pour une intervention virtuelle unique ciblant la dramatisation de la douleur chez les soignant·es et les jeunes en période périopératoire. Ces résultats soulignent la nécessité potentielle de dépister les symptômes d'anxiété chez les candidat·es avant la chirurgie. La réplication avec des échantillons plus grands et plus diversifiés et une conception plus robuste est justifiée.

Variability in criteria for death determination in the intensive care unit.

PURPOSE: Variability in practice exists in death determination by circulatory criteria in the context of organ donation. We sought to describe the practices of intensive care health care professionals for death determination by circulatory criteria with and without organ donation. METHODS: This study is a retrospective analysis of prospectively collected data. We included patients with death determination by circulatory criteria in intensive care units at 16 hospitals in Canada, three in the Czech Republic, and one in the Netherlands. Results were recorded using a checklist for the determination of death questionnaire. RESULTS: A total of 583 patients had their death determination checklist reviewed for statistical analysis. The mean (standard deviation) age in years was 64 (15). Three hundred and fourteen (54.0%) patients were from Canada, 230 (39.5%) were from the Czech Republic, and 38 (6.5%) were from the Netherlands. Fifty-two (8.9%) patients proceeded with donation after death determination by circulatory criteria (DCD). The most common diagnostic tests reported for the whole group were absent heart sounds by auscultation (81.8%), flat continuous arterial blood pressure (ABP) tracing (77.0%), and flat electrocardiogram tracing (73.2%). In patients who successfully underwent DCD (N = 52), death was determined most frequently using a flat continuous ABP tracing (94%), absent pulse oximetry (85%), and absent palpable pulse (77%). CONCLUSION: In this study, we have described practices for death determination by circulatory criteria both within and between countries. Though some variability exists, we are reassured that appropriate criteria are almost always used in the context of organ donation. In particular, the use of continuous ABP monitoring in DCD was consistent. It highlights the need for standardization of practice and up to date guidelines, especially within the context of DCD where there is both an ethical and a legal requirement to adhere to the dead donor rule, while minimizing time between death determination and organ procurement.

RéSUMé: OBJECTIF: Il existe de la variabilité dans la pratique en matière de détermination du décès selon des critères circulatoires dans le contexte d'un don d'organes. Nous avons cherché à décrire les pratiques des professionnels de la santé en soins intensifs en ce qui touche à la détermination du décès selon des critères circulatoires avec et sans don d'organes. MéTHODE: Cette étude est une analyse rétrospective de données recueillies prospectivement. Nous avons inclus des patients dont le décès avait été déterminé par des critères circulatoires dans les unités de soins intensifs de 16 hôpitaux au Canada, trois en République tchèque et un aux Pays-Bas. Les résultats ont été consignés à l'aide de la liste de contrôle d'un questionnaire sur la détermination du décès. RéSULTATS: Au total, les listes de contrôle pour la détermination du décès de 583 patients ont été examinées à des fins d'analyse statistique. L'âge moyen (écart type) en années était de 64 ans (15). Trois cent quatorze (54,0 %) patients provenaient du Canada, 230 (39,5 %) de la République tchèque et 38 (6,5 %) des Pays-Bas. Cinquante-deux (8,9 %) patients ont procédé au don après la détermination du décès selon des critères circulatoires (DCC). Les tests diagnostiques les plus fréquemment rapportés pour l'ensemble du groupe étaient l'absence de bruits cardiaques à l'auscultation (81,8 %), le tracé plat continu de la tension artérielle (TA) (77,0 %) et le tracé plat à l'électrocardiogramme (73,2 %). Chez les patients ayant été soumis avec succès à un DCD (N = 52), le décès a été déterminé le plus souvent à l'aide d'un tracé continu plat de la TA (94 %), d'une oxymétrie de pouls absente (85 %) et d'un pouls palpable absent (77 %). CONCLUSION: Dans cette étude, nous avons décrit les pratiques de détermination du décès selon des critères circulatoires à la fois à l'intérieur et entre les pays. Bien qu'il existe une certaine variabilité, nous sommes rassurés par le fait que des critères appropriés sont presque toujours utilisés dans le contexte du don d'organes. En particulier, l'utilisation du monitorage continu de la TA était constant en cas de DCC. Cela souligne la nécessité de normaliser la pratique et de disposer de lignes directrices mises à jour, en particulier dans le contexte de DCC où il existe une exigence à la fois éthique et légale de respecter la règle du donneur décédé, tout en minimisant le temps entre la détermination du décès et la collecte d'organes.

A phase II dose evaluation pilot feasibility randomized controlled trial of cholecalciferol in critically ill children with vitamin D deficiency (VITdAL-PICU study).

BACKGROUND: Vitamin D deficiency (VDD) is highly prevalent in the pediatric intensive care unit (ICU) and associated with worse clinical course. Trials in adult ICU demonstrate rapid restoration of vitamin D status using an enteral loading dose is safe and may improve outcomes. There have been no published trials of rapid normalization of VDD in the pediatric ICU. METHODS: We conducted a multicenter placebo-controlled phase II pilot feasibility randomized clinical trial from 2016 to 2017. We randomized 67 critically ill children with VDD from ICUs in Canada, Chile and Austria using a 2:1 randomization ratio to receive a loading dose of enteral cholecalciferol (10,000 IU/kg, maximum of 400,000 IU) or placebo. Participants, care givers, and outcomes assessors were blinded. The primary objective was to determine whether the loading dose normalized vitamin D status (25(OH)D > 75 nmol/L). Secondary objectives were to evaluate for adverse events and assess the feasibility of a phase III trial. RESULTS: Of 67 randomized participants, one was withdrawn and seven received more than one dose of cholecalciferol before the protocol was amended to a single loading dose, leaving 59 participants in the primary analyses (40 treatment, 19 placebo). Thirty-one/38 (81.6%) participants in the treatment arm achieved a plasma 25(OH)D concentration > 75 nmol/L versus 1/18 (5.6%) the placebo arm. The mean 25(OH)D concentration in the treatment arm was 125.9 nmol/L (SD 63.4). There was no evidence of vitamin D toxicity and no major drug or safety protocol violations. The accrual rate was 3.4 patients/month, supporting feasibility of a larger trial. A day 7 blood sample was collected for 84% of patients. A survey administered to 40 participating families showed that health-related quality of life (HRQL) was the most important outcome for families for the main trial (30, 75%). CONCLUSIONS: A single 10,000 IU/kg dose can rapidly and safely normalize plasma 25(OH)D concentrations in critically ill children with VDD, but with significant variability in 25(OH)D concentrations. We established that a phase III multicentre trial is feasible. Using an outcome collected after hospital discharge (HRQL) will require strategies to minimize loss-to-follow-up. CLINICALTRIALS: gov NCT02452762 Registered 25/05/2015.

Including patients and caregivers in assessment in the pediatric competence by design curriculum: A national consensus study.

Although evidence supports diverse assessment strategies, including patient/caregiver involvement in Competency-Based Medical Education (CBME), few residency programs formally include patients/caregivers in assessment. We aimed to determine the milestones for which patient/caregiver inclusion would be valuable in the Canadian Pediatric Competence By Design (CBD) curriculum.Program directors from 17 Canadian pediatric residency programs were invited to participate in a Delphi study. This Delphi included 209 milestones selected by the study team from the 320 milestones of the draft pediatric CBD curriculum available at the time of the study. In round 1, 16 participants representing 13 institutions rated the value of including patients/caregivers in the assessment of each milestone using a 4-point scale. We obtained consensus for 150 milestones, leaving 59 for re-exposure. In round 2, 14/16 participants rated remaining items without consensus. Overall, 67 milestones met consensus for 'valuable,' of which 11 met consensus for 'extremely valuable.' The majority of these milestones related to communication skills.Patient/caregiver assessment is valuable for 21% of milestones in the draft pediatric CBD curriculum, predominantly those relating to communication skills. This confirms the perceived importance of patient/caregiver assessment of trainees in CBME curricula; formal inclusion may be considered. Future directions could include exploring patients/caregivers' perspectives of their roles in assessment in CBD.

Clinical and Radiological Correlation of Low Flow Vascular Malformation Treated With Percutaneous Sclerotherapy.

Objective: To retrospectively correlate imaging findings post-sclerotherapy of low-flow vascular malformations with clinical outcome. Materials and Methods: We retrospectively evaluated 81 pediatric patients who had sclerotherapy in our department over a 14-year period. Patients with a diagnosis of low-flow vascular malformation, pre and post-treatment ultrasound (US) and clinical follow-up evaluation were included in the study. Exclusion criteria were coexisting high-flow vascular malformations, history of additional surgical or medical treatment to their malformation and large infiltrative lesions difficult to measure on US. Pre and post-treatment sonographic volumes of the malformation were assessed. Changes in volume were categorized into 6- increased volume, stable and volume decrease of 1-25%/26-50%/51-75%/75-100%. Clinical outcomes were categorized into 4 - worse, no change, improved and symptom free. In cases where pre-treatment MRI was available, the estimated malformation volumes in both modalities were correlated using Spearman's rank correlation. The change in sonographic volume was correlated with clinical outcome using Spearman's rank correlation. P-values < .05 were considered significant. Results: Twenty-nine patients were included in the study; 13 with venous malformation (VM), and 16 with lymphatic malformation (LM). Nineteen patients had both pre-treatment US and MRI, showing correlation in volume between the 2 modalities (P < .001). Post-treatment change in volume correlated with clinical outcome for combined venous and LMs (rho = .44, P = .02). No correlation was found when venous (rho = .48, P = .09) and lymphatic (rho = .33, P = .21) malformations were considered separately. Conclusion: Ultrasound can potentially be used as an objective tool in evaluating sclerotherapy treatment response of low-flow vascular malformations in the pediatric population.

Optimizing transfusion therapy for survivors of Haemoglobin Bart's hydrops fetalis syndrome: Defining the targets for haemoglobin-H fraction and "functional" haemoglobin level.

Owing to the unique pathophysiology of anaemia in haemoglobin Bart's hydrops fetalis (HBHF), a transfusion strategy based on beta-thalassemia guidelines is suboptimal for chronically transfused HBHF patients. A more aggressive transfusion aimed at reducing the proportion of non-functional HbH and improving the "functional" haemoglobin (f-Hb) can lead to reduced haemolysis and improved tissue oxygenation. However, the optimal transfusion targets for these parameters are not yet defined. In this retrospective, longitudinal study on four chronically transfused patients with HBHF, we used receiver operating characteristic curves to find a pre-transfusion f-Hb of 106 g/l and a HbH of 16.1% to be the optimal thresholds to achieve a normal soluble transferrin receptor and lactate dehydrogenase, respectively.

Routine lung volume recruitment in boys with Duchenne muscular dystrophy: a randomised clinical trial.

BACKGROUND: Impaired cough results in airway secretion retention, atelectasis and pneumonia in individuals with Duchenne muscular dystrophy (DMD). Lung volume recruitment (LVR) stacks breaths to inflate the lungs to greater volumes than spontaneous effort. LVR is recommended in DMD clinical care guidelines but is not well studied. We aimed to determine whether twice-daily LVR, compared with standard of care alone, attenuates the decline in FVC at 2 years in boys with DMD. METHODS: In this multicentre, assessor-blinded, randomised controlled trial, boys with DMD, aged 6-16 years with FVC >30% predicted, were randomised to receive conventional treatment or conventional treatment plus manual LVR twice daily for 2 years. The primary outcome was FVC % predicted at 2 years, adjusted for baseline FVC % predicted, age and ambulatory status. Secondary outcomes included change in chest wall distensibility (maximal insufflation capacity minus FVC) and peak cough flow. RESULTS: Sixty-six boys (36 in LVR group, 30 in control) were evaluated (median age (IQR): 11.5 years (9.5-13.5), median baseline FVC (IQR): 85% predicted (73-96)). Adjusted mean difference in FVC between groups at 2 years was 1.9% predicted (95% CI -6.9% to 10.7%; p=0.68) in the direction of treatment benefit. We found no differences in secondary outcomes. CONCLUSION: There was no difference in decline in FVC % predicted with use of twice-daily LVR for boys with DMD and relatively normal lung function. The burden associated with routine LVR may outweigh the benefit. Benefits of LVR to maintain lung health in boys with worse baseline lung function still need to be clarified. TRIAL REGISTRATION NUMBER: NCT01999075.

Housing conditions and respiratory morbidity in Indigenous children in remote communities in Northwestern Ontario, Canada.

BACKGROUND: Rates of lower respiratory tract infection (LRTI) among First Nations (FN) children living in Canada are elevated. We aimed to quantify indoor environmental quality (IEQ) in the homes of FN children in isolated communities and evaluate any associations with respiratory morbidity. METHODS: We performed a cross-sectional evaluation of 98 FN children (81 with complete data) aged 3 years or younger, living in 4 FN communities in the Sioux Lookout region of Northern Ontario. We performed medical chart reviews and administered questionnaires. We performed a housing inspection, including quantifying the interior surface area of mould (SAM). We monitored air quality for 5 days in each home and quantified the contaminant loading of settled floor dust, including endotoxin. We analyzed associations between IEQ variables and respiratory conditions using univariable and multivariable analyses. RESULTS: Participants had a mean age of 1.6 years and 21% had been admitted to hospital for respiratory infections before age 2 years. Houses were generally crowded (mean occupancy 6.6 [standard deviation 2.6, range 3-17] people per house). Serious housing concerns were frequent, including a lack of functioning controlled ventilation. The mean SAM in the occupied space was 0.2 m2. In multivariable modelling, there was evidence of an association of LRTI with log endotoxin (p = 0.07) and age (p = 0.02), and for upper respiratory tract infections, with SAM (p = 0.07) and age (p = 0.03). Wheeze with colds was associated with log endotoxin (p = 0.03) and age (p = 0.04). INTERPRETATION: We observed poor housing conditions and an association between endotoxin and wheezing in young FN children living in Northern Ontario.

Furosemide and albumin for the treatment of nephrotic edema: a systematic review.

BACKGROUND: Edema is one of the cardinal clinical features of nephrotic syndrome (NS). It may vary from mild periorbital edema to severe generalized edema (anasarca). In patients where edema does not improve with prednisone therapy, the most common supportive medications are diuretics and albumin. However, due to the complex pathophysiology of edema formation in NS patients resulting in intravascular normovolemia or hypovolemia, optimal therapy for edema is still debated. We conducted a systematic review with the objective of evaluating the change in urine volume and urine sodium excretion after treatment with furosemide only versus furosemide with albumin in edematous patients with NS. OBJECTIVES: (1) To evaluate efficacy of furosemide alone versus furosemide with albumin in the treatment of nephrotic edema in adults and children. (2) To compare the harms and benefits of different doses of furosemide for treating nephrotic edema. SEARCH METHODS: The search included all randomized or quasi-randomized controlled trials in English and French using MEDLINE, Embase, and CENTRAL Trials Registry of the Cochrane Collaboration using the Ovid interface. CLINICALTRIALS: gov and the International Clinical Trials Registry Platform were also searched. SELECTION CRITERIA: We included all RCTs and randomized cross-over studies in which furosemide and furosemide plus albumin are used in the treatment of children or adults with nephrotic edema. We excluded patients with hypoalbuminemia of non-renal origin and severe chronic kidney disease (CKD) with a glomerular filtration rate below 30 ml/min/1.74 m2 and patients with congenital NS. DATA COLLECTION AND ANALYSIS: All abstracts were independently assessed by at least two authors to determine which studies met the inclusion criteria. Information on study design, methodology, and outcome data (urine volume, urine sodium excretion, adverse effects) from each identified study was entered into a separate data sheet. The differences in outcomes between the types of therapy were expressed as standardized mean difference (SMD) with 95% confidence intervals (CI). RESULTS: The search yielded 525 records, and after screening, five studies were included in the systematic review and four of those studies in the meta-analysis. One study had high risk of bias and the remaining three studies were deemed to have some concerns. Urine excretion was greater after treatment with furosemide and albumin versus furosemide (SMD 0.85, 95% CI = 0.33 to 1.38). Results for sodium excretion were inconclusive (SMD 0.37, 95%CI = - 0.28 to 1.02). AUTHORS' CONCLUSIONS: The current evidence is not sufficient to make definitive conclusions about the role of albumin in treating nephrotic edema. High-quality randomized studies with adequate samples sizes are needed. Including an assessment of intravascular volume status may be helpful. TRIAL REGISTRATION: Prospero: CRD4201808979. https://www.crd.york.ac.uk/PROSPERO A higher resolution version of the Graphical abstract is available as Supplementary information.

Balanced Versus Unbalanced Fluid in Critically Ill Children: Systematic Review and Meta-Analysis.

OBJECTIVES: The ideal crystalloid fluid bolus therapy for fluid resuscitation in children remains unclear, but pediatric data are limited. Administration of 0.9% saline has been associated with hyperchloremic metabolic acidosis and acute kidney injury. The primary objective of this systematic review was to compare the effect of balanced versus unbalanced fluid bolus therapy on the mean change in serum bicarbonate or pH within 24 hours in critically ill children. DATA SOURCES: We searched MEDLINE including Epub Ahead of Print, In-Process & Other Non-Indexed Citations, Embase, CENTRAL Trials Registry of the Cochrane Collaboration, ClinicalTrials.gov, and World Health Organization International Clinical Trials Registry Platform. STUDY SELECTION: Using the Preferred Reporting Items for Systematic Review and Meta-analysis Protocols guidelines, we retrieved all controlled trials and observational cohort studies comparing balanced and unbalanced resuscitative fluids in critically ill children. The primary outcome was the change in serum bicarbonate or blood pH. Secondary outcomes included the prevalence of hyperchloremia, acute kidney injury, renal replacement therapy, and mortality. DATA EXTRACTION: Study screening, inclusion, data extraction, and risk of bias assessments were performed independently by two authors. DATA SYNTHESIS: Among 481 references identified, 13 met inclusion criteria. In the meta-analysis of three randomized controlled trials with a population of 162 patients, we found a greater mean change in serum bicarbonate level (pooled estimate 1.60 mmol/L; 95% CI, 0.04-3.16; p = 0.04) and pH level (pooled mean difference 0.03; 95% CI, 0.00-0.06; p = 0.03) after 4-12 hours of rehydration with balanced versus unbalanced fluids. No differences were found in chloride serum level, acute kidney injury, renal replacement therapy, or mortality. CONCLUSIONS: Our systematic review found some evidence of improvement in blood pH and bicarbonate values in critically ill children after 4-12 hours of fluid bolus therapy with balanced fluid compared with the unbalanced fluid. However, a randomized controlled trial is needed to establish whether these findings have an impact on clinical outcomes before recommendations can be generated.

Consent models in Canadian critical care randomized controlled trials: a scoping review.

PURPOSE: Our primary objective was to describe consent models used in Canadian-led adult and pediatric intensive care unit (ICU/PICU) randomized controlled trials (RCTs). Our secondary objectives were to determine the consent rate of ICU/PICU RCTs that did and did not use an alternate consent model to describe consent procedures. SOURCE: Using scoping review methodology, we searched MEDLINE, Embase, and CENTRAL databases (from 1998 to June 2019) for trials published in English or French. We included Canadian-led RCTs that reported on the effects of an intervention on ICU/PICU patients or their families. Two independent reviewers assessed eligibility, abstracted data, and achieved consensus. PRINCIPAL FINDINGS: We identified 48 RCTs of 17,558 patients. Included RCTs had ethics approval to use prior informed consent (43/48; 90%), deferred consent (13/48; 27%), waived consent (5/48; 10%), and verbal consent (1/48; 2%) models. Fifteen RCTs (15/48; 31%) had ethics approval to use more than one consent model. Twice as many trials used alternate consent between 2010 and 2019 (13/19) than between 2000 and 2009 (6/19). The consent rate for RCTs using only prior informed consent ranged from 54 to 91% (ICU) and 43 to 94% (PICU) and from 78 to 100% (ICU) and 74 to 87% (PICU) in trials using an alternate/hybrid consent model. CONCLUSION: Alternate consent models were used in the minority of Canadian-led ICU/PICU RCTs but have been used more frequently over the last decade. This suggests that Canadian ethics boards and research communities are becoming more accepting of alternate consent models in ICU/PICU trials.

RéSUMé: OBJECTIF: Notre objectif principal était de décrire les modèles de consentement utilisés dans les études randomisées contrôlées (ERC) menées par des chercheurs canadiens dans les unités de soins intensifs adultes et pédiatriques (USI/USIP). Nos objectifs secondaires étaient de déterminer le taux de consentement aux ERC à l'USI et l'USIP qui utilisaient et n'utilisaient pas un autre modèle de consentement pour décrire les processus de consentement. SOURCES: À l'aide d'une méthodologie d'étude de portée, nous avons effectué des recherches dans les bases de données MEDLINE, Embase et CENTRAL (de 1998 à juin 2019) pour en tirer les études publiées en anglais ou en français. Nous avons inclus des ERC dirigées par des chercheurs canadiens qui rapportaient les effets d'une intervention sur les patients à l'USI/USIP ou leurs familles. Deux examinateurs indépendants ont évalué l'admissibilité, résumé les données et atteint un consensus. RéSULTATS PRINCIPAUX: Nous avons identifié 48 ERC portant sur 17 558 patients. Les ERC incluses avaient obtenu l'approbation du comité d'éthique pour l'utilisation de modèles de consentement éclairé préalable (43/48; 90 %), de consentement différé (13/48; 27 %), de renoncement au consentement (5/48; 10 %) et de consentement verbal (1/48; 2 %). Quinze ERC (15/48; 31 %) avaient reçu l'approbation du comité d'éthique pour utiliser plus d'un modèle de consentement. Deux fois plus d'études ont utilisé un autre type de consentement entre 2010 et 2019 (13/19) qu'entre 2000 et 2009 (6/19). Le taux de consentement pour les ERC utilisant uniquement un consentement éclairé préalable variait de 54 à 91 % (USI) et de 43 à 94 % (USIP), contre 78 à 100 % (USI) et 74 à 87 % (USIP) pour les études utilisant un modèle de consentement alternatif/hybride. CONCLUSION: Des modèles de consentement alternatif ont été utilisés dans une minorité des ERC en USI/USIP dirigées par des chercheurs canadiens, mais ils ont été utilisés plus fréquemment au cours de la dernière décennie. Cela donne à penser que les comités d'éthique et les communautés de recherche canadiens acceptent de plus en plus les modèles de consentement alternatifs dans les études réalisées en USI et en USIP.

Pediatric Chiari I malformation: novel and traditional measurements associated with syrinx and surgery.

PURPOSE: Multiple imaging parameters have been examined to estimate the presence of syrinx and the need for surgery in Chiari I patients (CM1); however, no consistent or definitive criteria have been proposed. The objective of this study was to review existing and identify novel radiological and clinical characteristics of CM1 patients that associate syrinx development and surgical intervention. METHODS: Patients with Chiari I malformation diagnosed on imaging between 0 and 18 years were retrospectively reviewed from January 1, 2007 to February 12, 2020. Participants were included if they had a baseline MRI of the head and spine prior to surgical intervention if required. Forty age-matched controls with cranial imaging were identified for comparison. Imaging parameters and clinical symptoms were recorded. RESULTS: A total of 122 CM1 patients were included in this study. Of the 122 patients, 28 (23%) had syrinx, and 27 (22%) had surgery. The following imaging parameters associated with syrinx and surgical intervention were identified: midbrain length (P < 0.001; P = 0.032), the obex position (P = 0.002; P < 0.001) and medullary kinking (P = 0.041; P < 0.001). Among the clinical features, the presence of overall pain (P = 0.017; P = 0.042), neck pain (P = 0.005; P = 0.027), and sensory dysfunction (P < 0.001) were found to be strongly associated with syrinx and surgery. CONCLUSION: While further investigation is needed, these specific radiological and clinical parameters should be considered when evaluating CM1 patients and may be used to guide further management.

Is early activity resumption after paediatric concussion safe and does it reduce symptom burden at 2 weeks post injury? The Pediatric Concussion Assessment of Rest and Exertion (PedCARE) multicentre randomised clinical trial.

OBJECTIVE: Investigate whether resuming physical activity (PA) at 72 hours post concussion is safe and reduces symptoms at 2 weeks, compared with resting until asymptomatic. METHODS: Real-life conditions, multicentre, single-blinded randomised clinical trial, conducted in three Canadian paediatric emergency departments (ED). Children/youth aged 10-<18 years with acute concussion were recruited between March 2017 and December 2019, and randomly assigned to a 4-week stepwise return-to-PA protocol at 72 hours post concussion even if symptomatic (experimental group (EG)) or to a return-to-PA once asymptomatic protocol (control group (CG)). The primary outcome was self-reported symptoms at 2 weeks using the Health and Behaviour Inventory. Adherence was measured using accelerometers worn 24 hours/day for 14 days post injury. Adverse events (AE) (worsening of symptoms requiring unscheduled ED or primary care visit) were monitored. Multivariable intention-to-treat (ITT) and per-protocol analyses adjusting for prognostically important covariates were examined. Missing data were imputed for the ITT analysis. RESULTS: 456 randomised participants (EG: N=227; mean (SD) age=13.3 (2.1) years; 44.5% women; CG: N=229; mean (SD) age=13.3 (2.2) years; 43.7% women) were analysed. No AE were identified. ITT analysis showed no strong evidence of a group difference at 2 weeks (adjusted mean difference=-1.3 (95% CI:-3.6 to 1.1)). In adherent participants, initiating PA 72 hours post injury significantly reduced symptoms 2 weeks post injury, compared with rest (adjusted mean difference=-4.3 (95% CI:-8.4 to -0.2)). CONCLUSION: Symptoms at 2 weeks did not differ significantly between children/youth randomised to initiate PA 72 hours post injury versus resting until asymptomatic; however, many were non-adherent to the intervention. Among adherent participants, early PA was associated with reduced symptoms at 2 weeks. Resumption of PA is safe and may be associated with milder symptoms at 2 weeks. LEVEL OF EVIDENCE: 1b. TRIAL REGISTRATION NUMBER: NCT02893969. REGISTRY NAME: Pediatric Concussion Assessment of Rest and Exertion (PedCARE).