Pediatric Registry Research Articles

Awake Supraglottic Airway Placement in Pediatric Patients for Airway Obstruction or Difficult Intubation: Insights From an International Airway Registry (PeDI).

Small case series have described awake supraglottic airway placement in infants with significant airway obstruction and difficult intubations. We conducted this study to determine outcomes when supraglottic airways were placed in awake children enrolled in the international Pediatric Difficult Intubation Registry including success of ventilation, success of tracheal intubation, and complications. We reviewed the Pediatric Difficult Intubation Registry to identify all cases of awake supraglottic airway placement before planned tracheal intubation from August 2012 to September 2023 with subsequent review of details of awake supraglottic airway placement in the medical record. We present descriptive statistics of patient demographics, ventilation and intubation outcomes, and complications. A supraglottic airway was placed in an awake child in 95 of 8061 (1.2%) cases in the Pediatric Difficult Intubation Registry. Median age was 37 days (range 0-17.6 years) and median weight was 3.7 kg (1.6-46.7 kg). Sixteen (17%) cases were in patients older than 2 years and 7 (7%) were in adolescents. Adequate ventilation via a supraglottic airway was achieved in 81/95 (85%, 95% confidence interval [CI], 77%-93%) encounters. Inadequate (n = 13) or impossible (n = 1) ventilation occurred in 14/95 (15%). No complications were reported with supraglottic airway placement. For subsequent intubation, there was a 35% (33/95) first-attempt success rate and 99% (94/95) eventual success, with 1 patient awakened after failed attempts at tracheal intubation. Hypoxia occurred during the first intubation attempt in 9/95 (9%) encounters. The incidence of hypoxia was lower in encounters in which ventilation via the supraglottic airway was adequate (4/81, 5%) than in encounters in which ventilation via the supraglottic airway was inadequate or impossible (5/14, 36%). Although infrequently attempted, awake placement of a supraglottic airway in children with difficult airways achieved adequate ventilation and provided a conduit for oxygenation and ventilation after induction of anesthesia across a spectrum of ages.

Short-term neurologic outcomes in pediatric extracorporeal membrane oxygenation are proportional to bleeding severity graded by a novel bleeding scale.

This study aimed to characterize the severity of bleeding and its association with short-term neurologic outcomes in pediatric ECMO. Multicenter retrospective cohort study of pediatric ECMO patients at 10 centers utilizing the Pediatric ECMO Outcomes Registry (PEDECOR) database from December 2013-February 2019. Subjects excluded were post-cardiac surgery patients and those with neonatal pathologies. A novel ECMO bleeding scale was utilized to categorize daily bleeding events. Poor short-term neurologic outcome was defined as an unfavorable Pediatric Cerebral Performance Category (PCPC) or Pediatric Overall Performance Category (POPC) (score of >3) at hospital discharge. This study included 283 pediatric ECMO patients with a median (interquartile range [IQR]) age of 1.3years [0.1, 9.0], ECMO duration of 5days [3.0, 9.5], and 44.1% mortality. Unfavorable PCPC and POPC were observed in 48.4% and 51.3% of patients at discharge, respectively. Multivariable logistic regression analysis included patient's age, cannulation type, duration of ECMO, need for cardiopulmonary resuscitation, acute kidney injury, new infection, and vasoactive-inotropic score. As the severity of bleeding increased, there was a corresponding increase in the likelihood of poor neurologic recovery, shown by increasing odds of unfavorable neurologic outcome (PCPC), with an adjusted odds ratio (aOR) of 0.77 (confidence interval [CI] 0.36-1.62), 1.87 (0.54-6.45), 2.97 (1.32-6.69), and 5.56 (0.59-52.25) for increasing bleeding severity (grade 1 to 4 events, respectively). Similarly, unfavorable POPC aOR (CI) was 1.02 (0.48-2.17), 2.05 (0.63-6.70), 5.29 (2.12-13.23), and 5.11 (0.66-39.64) for bleeding grade 1 to 4 events. Short-term neurologic outcomes in pediatric ECMO are proportional to the severity of bleeding events. Strategies to mitigate bleeding events could improve neurologic recovery in pediatric ECMO.

Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome in pediatric Inflammatory Bowel Disease: clinical characteristics and outcomes.

Hemophagocytic Lymphohistiocytosis (HLH)/Macrophage Activation Syndrome (MAS) in children with inflammatory bowel disease (IBD) has been reported only anecdotally. This study aimed at describing the clinical features and outcomes of children diagnosed with both IBD and HLH/MAS. Data on IBD and HLH/MAS characteristics, biochemical, microbiological and genetic assessments, treatments, and outcomes were collected from the Italian Pediatric IBD Registry and presented using descriptive statistics. Out of 4643 patients with IBD, 18 (0.4%) were diagnosed with HLH/MAS, including 12 with ulcerative colitis and 6 with Crohn disease. Among the 18 patients, 7 (39%) had early-onset IBD, but the median age at HLH/MAS diagnosis was 14.0years (IQR 11.9-16.0). Half of the patients had active IBD at HLH/MAS diagnosis, 11 (61%) patients were on thiopurines, and 6 (33%) were on anti-TNF biologics. An infectious trigger was identified in 15 (83%) patients. One (5%) patients was diagnosed with XIAP deficiency. All patients discontinued thiopurines and 5 (83.3%)discontinued anti-TNF biologics; 16 (80%) patients received steroids for HLH/MAS. Three (17%)patients had a relapse of HLH/MAS. No patient developed lymphoma or died during a median follow-up of 2.7years (IQR 0.8-4.4). Conclusions: HLH/MAS mainly affects children with early-onset IBD but primarily develops during adolescence, following an infection while on immunosuppressant treatment. Although the prognosis is generally favorable, it is crucial to investigate an underlying immune deficiency.

Sustained remission at long term follow-up in adolescents and young adults with chronic primary immune thrombocytopenia

AbstractAdolescents and young adults (AYAs) with immune thrombocytopenia (ITP) exhibit distinct clinical features and needs, defying categorization as either adults or children. Previous findings revealed a 50% risk of chronic disease at 12 months, yet the long-term course remains unclear. This study aimed to delineate the clinical and laboratory characteristics of AYAs with chronic primary ITP. Data from patients aged 12 to 25 years with chronic disease at 1 year were extracted from 3 registries (Pediatric and Adult Registry on Chronic ITP, CEREVANCE, and Cytopénies Auto-immunes Registre Midi-Pyrénéen), covering the period from 2004 to 2021. Sustained complete remission off treatment (SCROT) occurring beyond 12 months was defined as platelet count of >100 × 109/L without treatment for at least 12 months, independently of the previous treatment strategy. A total of 427 AYAs (64% female) with chronic primary ITP were included. Clinical information was available for ∼100% of patients at initial diagnosis and at 6- and 12-month follow-ups (FUs); and for 88%, 77%, and 59% at 24, 36, and 48 months, respectively. Over time, clinical features improved gradually, with fewer patients requiring treatment. Throughout the FU period, second-line drug use increased steadily among treated patients, without affecting the need for corticosteroids and IV immunoglobulins. The proportion of new patients achieving SCROT at 24-, 36-, and 48-month FU was 10% (38/375), 9.5% (31/327), and 12% (30/250), respectively, including 23 who underwent splenectomy. AYAs achieving SCROT between 12 and 36 months displayed higher platelet counts in the first year (excluding the initial period) and received fewer IV immunoglobulin treatments beyond 12 months compared with those with ongoing disease.

Endovascular treatment in pediatric stroke: an individual patient pooled analysis on presentation, management and outcomes

BackgroundThis study aimed to perform a comprehensive pooled analysis of all published series involving pediatric patients with large vessel occlusion (LVO) undergoing endovascular treatment (EVT) to study the disease landscape...

Hospital variation in post-operative cardiac extracorporeal membrane oxygenation use and relationship to post-operative mortality.

It is unclear how extracorporeal membrane oxygenation use varies across paediatric cardiac surgical programmes and how it relates to post-operative mortality. We aimed to determine hospital-level variation in post-operative extracorporeal membrane oxygenation use and its association with case-mix adjusted mortality. Retrospective analysis of 37 hospitals contributing to the Pediatric Cardiac Critical Care Consortium clinical registry from 1 August 2014 to 31 December 2019. Hospitalisations including cardiothoracic surgery and post-operative admission to paediatric cardiac ICUs were included. Two-level multivariable logistic regression with hospital random effect was used to determine case-mix adjusted post-operative extracorporeal membrane oxygenation use rates and in-hospital mortality. Hospitals were grouped into extracorporeal membrane oxygenation use tertiles, and mortality was compared across tertiles. There were 43,640 eligible surgical hospitalisations; 1397 (3.2%) included at least one post-operative extracorporeal membrane oxygenation run. Case-mix adjusted extracorporeal membrane oxygenation rates varied more than sevenfold (0.9-6.9%) across hospitals, and adjusted mortality varied 10-fold (0-5.5%). Extracorporeal membrane oxygenation rates were 2.0%, 3.5%, and 5.2%, respectively, for low, middle, and high extracorporeal membrane oxygenation use tertiles (P < 0.0001), and mortality rates were 1.9%, 3.0%, and 3.1% (p < 0.0001), respectively. High extracorporeal membrane oxygenation use hospitals were more likely to initiate extracorporeal membrane oxygenation support intraoperatively (1.6% vs. 0.6% low and 1.1% middle, p < 0.0001). Extracorporeal membrane oxygenation indications were similar across hospital tertiles. When extracorporeal cardiopulmonary resuscitation was excluded, variation in extracorporeal membrane oxygenation use rates persisted (1.5%, 2.6%, 3.8%, p < 0.001). There is hospital variation in adjusted post-operative extracorporeal membrane oxygenation use after paediatric cardiac surgery and a significant association with adjusted post-operative mortality. These findings suggest that post-operative extracorporeal membrane oxygenation use could be a complementary quality metric to mortality to assess performance of cardiac surgical programmes.

Defining pediatric neurosurgery in low-income countries: a cross-sectional study in Ethiopia.

Pediatric neurosurgical practice is prevalent in most low- and lower-middle-income countries but lacks comprehensive documentation of practice patterns, demographics, and case variety. This study aimed to present the current state of pediatric neurosurgery in Ethiopia, including workforce characterization, case variety, and relevant procedures. A survey was developed and distributed to all Ethiopian fully trained neurosurgeons (n = 50). Survey questions assessed sociodemographic variables, level of training, case variety, and neurosurgical practice. Statistical analysis was conducted to describe the current practice of pediatric neurosurgery. A total of 45 neurosurgeons responded (90%). Three respondents (7%) were women. There was only 1 fellowship-trained pediatric neurosurgeon, while most neurosurgeons were general neurosurgeons who served a pediatric patient population. Most neurosurgeons (56%) worked in the capital city, Addis Ababa, while another 13% worked in other urban settings. The top three indications for a pediatric neurosurgical procedure were neural tube defects (NTDs) (96%), hydrocephalus (93%), and trauma (60%). NTD-associated hydrocephalus was the most common hydrocephalus type seen (71%). The most common procedure for hydrocephalus was shunt insertion (96%). A prenatal diagnosis of NTD was made in < 10% of cases, as reported by 84% of respondents. The study highlights Ethiopia's need for more pediatric neurosurgeons. Suggested strategies to facilitate subspecialty training include the establishment of a fellowship program facilitated by the implementation of a nationwide pediatric neurosurgery registry. Promoting efforts for early diagnosis and treatment of pediatric conditions coupled with NTD prevention initiatives could improve pediatric neurosurgical care in Ethiopia.

Heart transplantation for pediatric patients with malignant arrhythmias: Indications and Outcomes

BackgroundArrhythmias can lead to cardiac arrest (CA) and heart failure. When intractable, heart transplant (HTX) can become the only viable treatment. This rare, high-risk cohort has not been reported as a distinct group. ObjectiveCharacterize the outcomes of pediatric patients listed for HTX with the primary indication being malignant arrhythmia (MA). MethodsUsing the Pediatric Heart Transplant Society prospective registry, we identified all patients <18 years-old listed between 2014-2022. MA as the listing indication was categorized into primary tachy-arrhythmia (PT), inherited arrhythmia (IA), congenital heart disease (CHD) and cardiomyopathy (CM) with secondary arrhythmia. Demographic, listing and transplant data were analyzed. ResultsAmong 4630 patients listed and 3317 transplanted, MA was the indication in 63 (1.4%) and 49 (1.5%), respectively. MA patients were categorized as PT in 11, IA in 4, CHD in 6 and CM in 42. When compared to the non-MA cohort, patients listed for MA were older (mean (SD) age 10.6 (6.2) vs. 6.1 (6.2) years, p<0.01), more likely to present with a CA (43% vs. 11%, p<0.01), and less likely to be in the intensive care unit (40% vs. 58%, p<0.01) or on inotropes (30% vs. 60%, p<0.01) at listing. Outcomes including waitlist mortality, transplantation, post-transplant survival and freedom from rejection were comparable to the non-MA cohort. ConclusionPatients with MA constitute a small proportion of those listed for HTX in childhood. CM was the most common category, while IA and PT were rare. Their waitlist mortality and post-transplant outcomes were comparable to the non-MA cohort.

Exploring the association between individual, family, and program characteristics and change in health outcomes 12 months after enrollment into the CANadian Pediatric Weight management Registry (CANPWR).

To examine individual, family, and program characteristics associated with changes in anthropometric and cardiometabolic health indicators in children with overweight or obesity after participating in multidisciplinary obesity management for 12 months. Participants included children 2-17 years old with overweight or obesity enrolled in the CANadian Pediatric Weight Management Registry (CANPWR). Multiple linear regression analyses were conducted to investigate the associations between individual, family, and program characteristics and changes in anthropometry (WHO BMI z-score) and cardiometabolic health indicators (systolic and diastolic blood pressure; fasting and 2-h glucose post-oral glucose tolerance test (OGTT); high density lipoprotein- (HDL) and non-HDL cholesterol and fasting triglycerides). BMI z-score data were available from 1065/1286 (82.8%) at 6-months post-baseline and 893/1286 (69.4%) at 12-months post-baseline. At 6-months, BMI z-score decreased relative to baseline (mean difference (MD) [95% confidence interval (CI)] = -0.08 [-0.10 to -0.06]; p < 0.001). BMI z-score (MD [95% CI] = -0.08 [-0.13 to -0.04); p = 0.001) and fasting triglycerides (MD [95% CI] = -0.07 [-0.13 to -0.02); p = 0.011) decreased at 12 months from baseline. Older age at baseline (estimated β = 0.025; 95% CI [0.006, 0.042], p = 0.007) and female sex (estimated β = 0.241; 95% CI [0.108, 0.329], p < 0.001) were associated with a worsened Δ BMI z-score at 12 months, while total hours with mental health provider (estimated β = -0.015; 95% CI [-0.030, -0.001], p = 0.049) was associated with an improved Δ BMI z-score at 12 months. Hours with an exercise counselor (estimated β = 0.023; 95% CI [0.008, 0.039], p = 0.003) were associated with improved HDL, while hours with a registered dietitian (estimated β = -0.026; 95% CI [-0.051, -0.001], p = 0.044) were associated with improved non-HDL cholesterol. Male sex and hours spent with a mental health provider, exercise counselor, and registered dietitian were related to significant improvements in several anthropometric and cardiometabolic health indicators at 12 months post-baseline.

Incidence of Parental Requests to Discontinue Growth-Friendly Surgical Lengthening for Early Onset Scoliosis.

The STOP questionnaire was developed to document reasons for discontinuation of growth-friendly (GF) treatment in early onset scoliosis (EOS). This study investigated the incidence of parental request (PR) on the STOP questionnaire and compared clinical information and Early Onset Scoliosis 24-Item Questionnaire (EOSQ-24) scores of PR patients with those whose parents did not request discontinuation (non-parent request [NPR]). An international pediatric spine registry was queried for EOS patients with STOP questionnaires completed by their surgeon. Age at discontinuation, sex, and EOS etiology were recorded. GF device, number of surgical procedures, complications, STOP questionnaire reasons for discontinuation, and definitive treatment were recorded. EOSQ-24 scores and clinical information in the PR cohort were compared with the NPR cohort. Data for 1326 patients were analyzed. PR was listed on the STOP questionnaires of 46 (3.5%) patients, completed at a mean age of 12 years (SD, 3.2 years). There were no statistical differences in number of procedures or complications when comparing the PR cohort with the NPR cohort. PR patients more frequently had neuromuscular EOS (P=.002), more frequently were treated with magnetically controlled growing rods (33% vs 14%, P=.036), and more frequently were observed after GF discontinuation (P=.628). EOSQ-24 scores for the PR cohort were significantly lower in most domains except pain/discomfort. For 3.5% of the EOS patients, PR was listed on the STOP questionnaire. They frequently had neuromuscular EOS and frequently were treated with magnetically controlled growing rods. Additionally, these patients had statistically lower EOSQ-24 scores across most domains. [Orthopedics. 20XX;4X(X):XXX-XXX.].

Paediatric very rare tumours registration and management in European countries with low health expenditure average rates.

Within the Paediatric Rare Tumours Network-European Registry (PARTNER) project, we aimed to evaluate the situation on the registration and management of paediatric patients affected by very rare tumours (VRT) in the European low health expenditure average rates (LHEAR) countries. A survey regarding infrastructure, organisation, and clinical decision-making information on VRT was designed. This survey was distributed to the representatives of LHEAR countries involved in the activities of the PARTNER Work Package 7. Eighteen answers from 17 countries were collected regarding the national organisation, methods of registration of VRT cases, the availability of medical experts in VRT, the access to updated diagnostic and therapeutic procedures (such as proton therapy, immunotherapy and, targeted therapies), and research on paediatric VRT. A high variability in the registration and management of patients with VRT has been observed with additional wide inequalities in pathology review, uniformity of clinical decisions, availability of selected procedures, and diagnostic and research tools. In the majority of LHEAR countries, no clinical or research structures have been implemented for children and adolescents with VRT. Therefore, VRT still have an orphan status in these countries. These significant differences on the technology access and use between European regions need to be addressed.

Long COVID or Post-Acute Sequelae of COVID-19 (PASC) in Children and Adolescents.

Introduction: Few studies of children with long COVID (post-COVID-19 condition) or post-acute sequelae of SARS CoV-2 (PASC) have been reported. Those terms describe symptoms that persist for weeks or months or as new symptoms that develop after SARS-CoV-2 infection. This condition might be found to various degrees in the severity of COVID-19. This study aimed to describe long COVID in confirmed SARS-CoV-2-infected children.Design: An observational cross-sectional study.Setting: Tertiary care hospital between January and November 2021.Participants: Children, age 5-17 years, with virologically confirmed COVID?19.Methods: This study was conducted by completing an electronic form after informed consent was obtained. The subject's characteristics and parent's or guardian's phone numbers were retrieved from the pediatric COVID-19 registry. Parents were contacted to complete a structured electronic questionnaire about the long COVID symptoms noticed in their children. Descriptive statistics were displayed in percentages and median.Results: Parent contact numbers were documented in 125/135 children who fulfilled the study criteria. There were 61 parents (48.8%) who gave consent, while the rest either did not respond or refused. There were 16 children reported as deceased primarily due to chronic renal disease and leukemia. Of the 45 children enrolled, the median (IQR) age was 11 years (5.3-17.6); 51.1% were female; and 75.6% had comorbidities. Two-thirds developed long COVID symptoms, most frequently in the age 5-9 years group, and mostly fatigue (45.2%), decreased appetite (38.7%), and muscle aches (32.3%). All patients with moderate to severe and more than half asymptomatic to mild COVID-19 developed long COVID.Conclusion: Most children had long COVID symptoms similar to adults despite being otherwise asymptomatic or having mild COVID-19.

Percent Body Fat and Weight Status of Youth Participating in Pediatric Weight Management Programs in the Pediatric Obesity Weight Evaluation Registry.

Factors associated with change in percent body fat (%BF) of children in pediatric weight management (PWM) care may differ from those associated with change in weight status. Objective: To describe %BF and weight status at initial visits to 14 PWM sites, identify differences by sex, and evaluate factors associated with change over 6 months. Methods: Initial visits of 2496 males and 2821 females aged 5-18 years were evaluated. %BF was measured using bioelectrical impedance analysis. Results: Sex-specific logistic regressions [806 males (32.3%), 837 females (29.7%)] identified associations with primary outcomes: lower %BF and metabolically impactful ≥5-point drop in percent of the 95th BMI percentile (%BMIp95) over 6 months. At the initial visit, males had lower %BF and higher %BMIp95 than females. Over 6 months, males had significantly (p < 0.001) greater median drop in %BF (-1.4% vs. -0.4%) and %BMIp95 (-3.0% vs. -1.9%) and a higher frequency of decreased %BF (68.9% vs. 57.8%), but similar percentage with ≥5-point %BMIp95 drop (36.5% vs. 32.4%; p = 0.080). For males, factors significantly associated with decreased %BF (older age, ≥6 visits, lack of developmental or depression/anxiety concerns) were not related to having a ≥5-point %BMIp95 drop. For females, lack of depression/anxiety concern was significantly associated with decreased %BF but was not associated with ≥5-point %BMIp95 drop. Conclusions: There are differences by sex in initial visit %BF and %BMIp95 and in characteristics associated with changes in these measures. PWM interventions should consider evaluating body composition and sex-stratifying outcomes.

Mortality patterns in Dutch diabetes outpatients.

Diabetes mellitus is a major cause of death. Outpatients with diabetes have more complications than patients in general practice; mortality patterns have only been studied in the total diabetes population. This study aims to assess mortality, causes, and predictors in outpatients with diabetes. A cohort study, included people with diabetes mellitus from the nationwide Dutch Paediatric and Adult Registry of Diabetes (DPARD) visiting diabetes outpatient clinics in 2016-2020. DPARD data were linked to Statistics Netherlands (CBS), comprising data on mortality, ethnicity and education. All-cause and cardiovascular mortality rates were estimated using Cox proportional hazard regression. During a median follow-up of 3.1 years among 12 992 people with diabetes, mortality rates per 10 000 person-years were 67.7 in adult type 1 diabetes and 324.2 in type 2 diabetes. The major cause of non-cardiovascular death was malignancy. During the pandemic years of influenza (2018) and COVID (2020), mortality rates peaked. Age, smoking and an estimated glomerular filtration rate of <60 ml/min were associated with all-cause mortality. In type 2 diabetes, additional factors were male sex, body mass index <20 kg/m2, diabetes duration <1 year and hypertension. Mortality among Dutch outpatients with diabetes is high. Smoking and renal failure were associated with mortality in both types. Further focus on early detection and treatment of mortality-associated factors may improve clinical outcomes.

Assessing disparities through missing race and ethnicity data: results from a juvenile arthritis registry.

Ensuring high-quality race and ethnicity data within the electronic health record (EHR) and across linked systems, such as patient registries, is necessary to achieving the goal of inclusion of racial and ethnic minorities in scientific research and detecting disparities associated with race and ethnicity. The project goal was to improve race and ethnicity data completion within the Pediatric Rheumatology Care Outcomes Improvement Network and assess impact of improved data completion on conclusions drawn from the registry. This is a mixed-methods quality improvement study that consisted of five parts, as follows: (1) Identifying baseline missing race and ethnicity data, (2) Surveying current collection and entry, (3) Completing data through audit and feedback cycles, (4) Assessing the impact on outcome measures, and (5) Conducting participant interviews and thematic analysis. Across six participating centers, 29% of the patients were missing data on race and 31% were missing data on ethnicity. Of patients missing data, most patients were missing both race and ethnicity. Rates of missingness varied by data entry method (electronic vs. manual). Recovered data had a higher percentage of patients with Other race or Hispanic/Latino ethnicity compared with patients with non-missing race and ethnicity data at baseline. Black patients had a significantly higher odds ratio of having a clinical juvenile arthritis disease activity score (cJADAS10) of ≥5 at first follow-up compared with White patients. There was no significant change in odds ratio of cJADAS10 ≥5 for race and ethnicity after data completion. Patients missing race and ethnicity were more likely to be missing cJADAS values, which may affect the ability to detect changes in odds ratio of cJADAS ≥5 after completion. About one-third of the patients in a pediatric rheumatology registry were missing race and ethnicity data. After three audit and feedback cycles, centers decreased missing data by 94%, primarily via data recovery from the EHR. In this sample, completion of missing data did not change the findings related to differential outcomes by race. Recovered data were not uniformly distributed compared with those with non-missing race and ethnicity data at baseline, suggesting that differences in outcomes after completing race and ethnicity data may be seen with larger sample sizes.

Development of the paediatric society of the African league against rheumatism (PAFLAR) JIA registry and clinical profile of JIA in Africa from the PAFLAR JIA registry

BackgroundThe spectrum of Juvenile Idiopathic Arthritis (JIA) in Africa is still largely unknown. We thus set out to illustrate how we set up the PAFLAR JIA registry and describe the clinical profile of Juvenile Idiopathic Arthritis across various regions in Africa.MethodsWe carried out a retrospective observational cohort study where collaborators were trained on use of the existing PAFLAR REDCAP database to enter data for the JIA patients currently under their care capturing their epidemiological data, clinical features, laboratory investigations, diagnosis and therapy at initial diagnosis. Descriptive statistics including means, standard deviations, medians, interquartile ranges (IQR) for continuous variables and proportions for categorical variables were calculated as appropriate. Tests for difference between groups were performed between categorical variables using Pearson’s chi-square or Fisher’s exact tests. All analyses were performed using SPSS version 22 software.ResultsWe enrolled 302 patients, 58.6% (177 of 302) of whom were female. The median age of disease onset was 7 years (range 3–11 years) and the median age at diagnosis was 8.5 years (range 5–12 years). The median duration delay in diagnosis was 6 months (range 1-20.8 months). The JIA categories included Systemic JIA 18.9% (57), Oligoarticular JIA 19.2% (83), Polyarticular RF + ve 5% (15), Polyarticular RF-ve 17.9% (54), Enthesitis Related Arthritis (ERA) 18.2% (55), Psoriatic Arthritis 7% (21) and undifferentiated JIA 5.6% (17). As regards treatment the commonest therapies were NSAID therapy at 31.1%, synthetic DMARDs at 18.1%, synthetic DMARDs combined with NSAIDs at 17.5% and steroid therapy at 9.6%. Biological DMARDs accounted for 2.3% of therapies offered to our patients at diagnosis. The average JADAS score was 10.3 (range 4.8–18.2) and the average CHAQ score was 1.3 (range 0.7-2.0).ConclusionOur study highlights strategies involved in setting up a Pan-African paediatric rheumatology registry that embraces our broad diversity and the vast spectrum of JIA in Africa while comparing the various therapies available to our patients. The PAFLAR JIA registry strives to ensure a comprehensive representation of the diverse healthcare landscapes within the continent. Further longitudinal observation studies are required to ascertain the long-term outcomes of our patients and ultimately help inform policy to create a more favorable health ecosystem to support the healthcare needs of JIA patients in Africa.

The Study of the Epidemiology of Pediatric Hypertension Registry (SUPERHERO): Rationale and Methods.

Despite increasing prevalence of hypertension in youth and high adult cardiovascular mortality rates, the long-term consequences of youth-onset hypertension remain unknown. This is due to limitations of prior research such as small sample sizes, reliance on manual record review, and limited analytic methods that did not address major biases. The Study of the Epidemiology of Pediatric Hypertension (SUPERHERO) is a multisite retrospective Registry of youth evaluated by subspecialists for hypertension disorders. Sites obtain harmonized electronic health record data using standardized biomedical informatics scripts validated with randomized manual record review. Inclusion criteria are index visit for International Classification of Diseases Diagnostic Codes, 10th Revision (ICD-10 code)-defined hypertension disorder ≥January 1, 2015 and age <19 years. We exclude patients with ICD-10 code-defined pregnancy, kidney failure on dialysis, or kidney transplantation. Data include demographics, anthropomorphics, U.S. Census Bureau tract, histories, blood pressure, ICD-10 codes, medications, laboratory and imaging results, and ambulatory blood pressure. SUPERHERO leverages expertise in epidemiology, statistics, clinical care, and biomedical informatics to create the largest and most diverse registry of youth with newly diagnosed hypertension disorders. SUPERHERO's goals are to (i) reduce CVD burden across the life course and (ii) establish gold-standard biomedical informatics methods for youth with hypertension disorders.

Social Determinants of Health and Outcomes After Pediatric Ventricular Assist Device Implantation

ABSTRACTBackgroundLimited research exists on the influence of social determinants of health (SDOH) on outcomes in pediatric patients with advanced heart failure receiving mechanical circulatory support.MethodsLinkage of the Pediatric Interagency Registry for Mechanical Circulatory Support (Pedimacs) and Society of Thoracic Surgeon's Congenital Heart Surgery Database (STS‐CHSD) identified pediatric patients who underwent ventricular assist device (VAD) implantation from 2012 to 2022 with available residential zip codes. Utilizing the available zip codes, each patient was assigned a Childhood Opportunity Index (COI) score. Level of childhood opportunity, race, and insurance type were used as proxies for SDOH. Major outcomes included death, transplant, alive with device, and recovery. Secondary outcomes were adverse events. Statistical analyses were performed using the Kaplan–Meier survival, competing risk analyses, and multivariable Cox proportional hazards model.ResultsThree hundred seventeen patients were included in the study. Childhood opportunity level and insurance status did not significantly impact morbidity or mortality after VAD implantation. White race was associated with reduced 1‐year survival (71% in White vs. 87% in non‐White patients, p = 0.05) and increased risk of pump thrombosis (p = 0.02).ConclusionChildhood opportunity level and insurance status were not linked to morbidity and mortality in pediatric patients after VAD implantation. Notably, White race was associated with higher mortality rates. The study underscores the importance of considering SDOH in evaluating advanced therapies for pediatric heart failure and emphasizes the need for accurate socioeconomic data collection in future studies and national registries.

The first national pediatric immune thrombocytopenia registry in Iran: research protocol and preliminary study results

BackgroundDisease registries are comprehensive databases that record detailed information on patients diagnosed with specific conditions, providing valuable insights into their diagnosis, treatment, and outcomes. This study aims to describe the pilot phase of the national pediatric Immune Thrombocytopenia(ITP) registry (NPITP) in Iran, serving as the inaugural interpretive report.MethodsThis patient-centered software system was implemented as a national program across multiple pediatric centers in Iran. Several focus groups were conducted to establish a minimum data set (MDS) comprising six main classes, 14 sub-classes, and 187 data elements. Following expert consensus on the final data set, a web-based software tool was developed by the dedicated IT team, accessible online and offline via https://disreg.sbmu.ac.ir/q/ITP.html. The registry included children aged between two months and 18 years with a platelet count below 100 × 109/L, based on predefined inclusion criteria.ResultsWithin a four-month period, a total of 60 ITP patients were registered, including 41 (68.3%) newly diagnosed cases, 68 (13.6%) persistent cases, and 14 (23.3%) with chronic ITP. The mean age of the registered patients was 55.93 ± 9.72 months. The most frequently observed bleeding symptoms were petechiae (68.3%), purpura (51.6%), and ecchymosis (13.3%). Among the newly diagnosed patients, 20 (33.3%) received intravenous immunoglobulin (IVIG), 17 (28.3%) were treated with prednisolone, and 17 (28.3%) received combined IVIG and steroid therapy. Of all patients, 40 (66.7%) demonstrated a complete response to treatment, while 16 (26.7%) exhibited a partial response. Four patients (6.7%) remained unresponsive to therapy. Treatment-related complications, such as Cushing’s syndrome, edema, weight gain, hirsutism, and mood disorders, were reported in 10 patients (16.6%). However, the majority of patients (81.7%) did not experience therapy-related complications.ConclusionThe pilot phase of the NPITP registry successfully implemented a web-based software tool for data collection, aiming to enhance the quality of care, facilitate clinical research, and support health service planning in the future.

COVID-19 infection in children, adolescents, and young adults with Down syndrome and hematologic malignancies.

Children, adolescents, and young adults (CAYAs) with Down syndrome (DS) and hematologic malignancies are particularly vulnerable to infections and related complications. There are limited data regarding COVID-19 infections in this group. We aimed to understand the clinical course of COVID-19 in this population. This observational study leverages the de-identified clinical and sociodemographic data captured by the Pediatric Oncology COVID-19 Case Report Registry (POCC) regarding CAYAs with cancer and COVID-19. We evaluated CAYAs (≤21years at COVID-19 infection) with hematologic malignancies and COVID-19 reported from April 1, 2020 to May 2, 2023, comparing those with and without DS. Using multivariable logistic regression, we examined rates of hospitalization, intensive care unit (ICU) admission, respiratory support, and changes in cancer-directed therapy. Among 1408 CAYAs with hematologic malignancies, 55 had DS (CAYA-DS). CAYA-DS had higher rates of hospitalization, ICU admission, and respiratory support (p<.001) than CAYAs without DS. Similarly, multivariable analyses found higher odds of hospitalization (odds ratio [OR]=2.8, 95% confidence interval [CI]: 1.5-5.1), ICU admission (OR=4.2, 95% CI: 1.9-9.1), and need for respiratory support (OR=4.2, 95% CI: 2.0-8.8) among CAYA-DS. Modifications to cancer-directed therapy were more common among CAYA-DS when related to neutropenia (p=.001), but not when unrelated to neutropenia (p=.88); CAYA-DS did not have higher odds of changes to cancer-directed therapy (OR=1.20, 95% CI: 0.7-2.1). We identify CAYA-DS with hematologic malignancies as a vulnerable subpopulation at greater risk for severe COVID-19 infection. This can inform conversations with patients and families regarding therapeutic and preventive measures, as well as the risks and benefits of modifying chemotherapy in the setting of COVID-19.