Clinical Outcomes In Pediatric Patients Research Articles

The Nancy Histopathological Index has limited value in predicting clinical outcomes in newly diagnosed pediatric patients with ulcerative colitis.

The Nancy Histological Index (NHI) is used to score histologic disease activity in patients with ulcerative colitis (UC). Our goal was to assess the utility of NHI at diagnosis in predicting clinical outcomes in pediatric patients with UC, in comparison to clinical and endoscopic scores. We retrospectively reviewed data at diagnosis of 106 children with UC (59 [55.7%] females; median age 14.4 [11.2-15.9] years, median Pediatric Ulcerative Colitis Activity Index [PUCAI] 35 [25-55]). During a follow-up of 116 (55-171) weeks, 33 patients (31.1%) required azathioprine therapy, and 32 (30.2%) were escalated to anti-tumor necrosis factor alpha (anti-TNFa). The PUCAI and Mayo endoscopic scores at diagnosis were significantly associated with escalation to anti-TNFa (p = 0.036 and p = 0.02, respectively), but not with initiation of azathioprine or subsequent acute severe colitis (ASC) events. However, the NHI was not associated with subsequent immunomodulators or anti-TNFa therapy (p = 0.42 and p = 0.78, respectively), nor with future ASC events (p = 0.70). In conclusion, the NHI failed to predict clinical outcomes in newly diagnosed pediatric patients with UC.

Clinical outcomes of pediatric patients with congenital toxoplasmosis in a fourthlevel center Introduction. Congenital

Congenital toxoplasmosis is a highly prevalent parasitic disease worldwide, with a high burden of disease and neurodevelopmental involvement in pediatric patients. To describe the clinical sequelae and neurodevelopmental state of pediatric patients with congenital toxoplasmosis at the Hospital Militar Central during 2013 to 2020. We conducted an observational, descriptive, cross-sectional study with an analytical component, including pediatric patients diagnosed with congenital toxoplasmosis. Patients consulted the Hospital Militar Central from January 2013 to December 2020. The Ages and Stages Questionnaires 3 neurodevelopmental scale was applied to children under six years old. Forty-five patients with confirmed congenital toxoplasmosis were included, with a mean age of 5.9 years; 60% were male; 11.2 % were symptomatic at birth, and 33% presented chorioretinitis. During the follow-up, 73% presented ophthalmologic sequelae, 64% cerebral calcifications, 4.4% hydrocephalus, 11.2% cerebral palsy, and 13.4% focal epilepsy. In children under six years old, 58% presented neurodevelopmental compromise, and in those over six years old, 62% had cognitive deficits. In this cohort, 68% of the patients received posnatal treatment, with a statistically significant association between not receiving treatment and ophthalmological sequelae (OR = 5.2; p < 0.001). Congenital toxoplasmosis is associated with important long-term sequelae similar to those described in several Latin American series. These findings highlight the importance of early diagnosis, evaluation, treatment, and timely interdisciplinary follow-up of patients in our country to improve their prognosis.

Incidence of Infective Endocarditis Post-TPVR with MELODY Valve in Pediatric Patients: A Systematic Review and Meta-Analysis.

Infective Endocarditis (IE) has emerged to be one of the most impactful adverse complications post-transcatheter procedures, especially Transcatheter Pulmonary Valve Replacement (TPVR). We conducted a systematic review and meta-analysis with the aim of identifying the incidence of IE post-TPVR with the MELODY valve in the pediatric population. A comprehensive literature search was performed across several prominent databases, including PubMed/MEDLINE, SCOPUS, and Science Direct. Studies compared the clinical outcomes of pediatric patients who received TPVR using the MELODY valve. Data extraction was done for variables like the total pediatric patient population that underwent TPVR with MELODY valve, mean age, the sex of the patients, the incidence rate of IE following the procedure, and the duration between the procedure and the occurrence of IE. Inverse Variance was used to estimate the incidence of IE in patients who underwent TPVR with respective 95% confidence interval (CI). In total, 4 studies with 414 pediatric patients who underwent TPVR using the MELODY valve were included in the study. The mean age of the study population was 12.7 ± 3.11 years. The pooled incidence of IE following TPVR with MELODY valve in the pediatric population was 17.70% (95% Cl 3.84-31.55; p<0.00001). Additionally, the mean length of duration to develop IE following TPVR with MELODY valve in the pediatric population was 2.18 years (95% Cl 0.35-4.01; p<0.00001). Our meta-analysis reveals that IE post-TPVR with MELODY valve in pediatric patients is a significant complication, clinically and statistically. Further research needs to be done to understand the risk factors and develop better management strategies.

Relationship Between Baseline Infarct and Clinical Outcome in Pediatric Large‐Vessel Occlusion Ischemic Stroke

Background In adults with large‐vessel occlusion stroke, the extent of the baseline infarct measured using the Alberta Stroke Program Early Computed Tomography Score (ASPECTS) predicts outcome and is used during patient selection for mechanical thrombectomy. The relationship between ASPECTS and clinical outcome is unknown in pediatric large‐vessel occlusion stroke. Methods Secondary analysis of a retrospective multicenter case–control study assessing mechanical thrombectomy versus medical management alone for pediatric large‐vessel occlusion stroke across 5 centers in Australia and Canada from 2011 to 2022. ASPECTS was measured on baseline computed tomography or magnetic resonance imaging–diffusion‐weighted imaging while blinded to clinical outcome and treatment status. The relationship between ASPECTS and pediatric modified Rankin scale scores at 3 months following stroke was assessed using ordinal logistic regression. Results In total, n = 24 thrombectomy patients (F = 10, mean age, 11.3 years [SD, 4.36]) and n = 24 control patients (F = 10, mean age, 10.2 years [SD, 4.32) were included. Mean ASPECTS was 6.3 (SD, 2.37) in the thrombectomy group and 6.1 (SD, 2.64) in the control group. In patients undergoing thrombectomy, baseline ASPECTS significantly correlated with pediatric modified Rankin scale scores at 3 months (odds ratio, 1.58 [95% CI, 1.10–2.27]; P = 0.013). In control patients, there was no significant correlation (odds ratio, 1.17 [95% CI, 0.87–1.55]; P = 0.298). Six of 7 thrombectomy patients with a large baseline infarct (ASPECTS &lt;6) had a pediatric modified Rankin scale score of 0 to 2 at final follow‐up. Conclusion Baseline ASPECTS was significantly associated with clinical outcome in pediatric patients with large‐vessel occlusion stroke who received mechanical thrombectomy. Thrombectomy patients with low ASPECTS demonstrated favorable long‐term outcomes, suggesting ASPECTS alone should not be used to exclude pediatric patients from receiving thrombectomy.

Clinical Outcomes in Pediatric Patients With Type 1 Diabetes With Early Versus Late Diagnosis: Analysis From the DPV Registry.

This study was conducted to evaluate the effects of early clinical diagnosis of type 1 diabetes by comparison of clinical parameters at diagnosis and during follow-up in patients with pediatric type 1 diabetes with early, intermediate, and late diagnosis. In a population-based analysis, data on 14,292 pediatric patients with type 1 diabetes diagnosed between 2015 and 2019 were retrieved from the Diabetes Prospective Documentation (DPV) registry in March 2023. Patients were divided into four groups: one with diabetic ketoacidosis (DKA) at diagnosis and three with early, intermediate, or late diagnosis based on age-dependent HbA1c terciles. Laboratory-measured HbA1c values and those estimated from continuous glucose monitoring were aggregated as a combined glucose indicator (CGI). Insulin-dose adjusted CGI values <9% were defined as partial remission. At diagnosis, patients had a median age of 9.8 years (IQR = 6.8; 13.0). Three years later, patients with early diagnosis had lower CGI than patients with late diagnosis or DKA (mean [95% CI] 7.46% [7.40; 7.53] vs. 7.81% [7.75; 7.87] or 7.74% [7.68; 7.79], respectively; each P < 0.001). More patients experienced partial remission (12.6% [11.0; 14.4] vs. 9.1% [7.7; 10.7] or 8.6% [7.3; 10.0]; each P < 0.001), and 11.7% [10.2; 13.5] of patients with intermediate diagnosis were in partial remission. Early clinical diagnosis of type 1 diabetes may be beneficial for metabolic control and remission after 3 years of follow-up. Patients diagnosed early may represent a distinct group with better resources or with a different disease biology and slower β-cell destruction, which needs further evaluation.

Institutional review of pediatric oncology patients participating in early phase clinical trials.

10042 Background: Phase 1 or phase 1/2 trials are a first step in pediatric cancer drug development with a primary objective of evaluating safety and dosing of new therapies or new combinations. They most commonly enroll patients with relapsed/refractory or very rare cancers and currently there is a paucity of information regarding outcomes for pediatric patients enrolled on phase 1/2 trials. Thus, we sought to describe clinical outcomes of pediatric patients enrolled in phase 1 clinical trials over a 9-year period at a single institution. Methods: We queried our clinical trials management system to generate a list of all pediatric patients who were enrolled and treated on phase 1 or phase 1/2 trials from 2011-2019. We reviewed the electronic medical record to collect baseline demographic and clinical data. We also collected key efficacy and safety endpoints post-enrollment including: time to death (if applicable); objective response to trial therapy; duration on therapy; need for dose modification; and occurrence of DLT. Overall survival was calculated using Kaplan-Meier methods. Results: A total of 224 unique patients accounted for 258 enrollments and 253 episodes of treatment on trial. The median age at trial enrollment was 11 years (range 0-27 years) and 56.2% were male. The majority of patients were white (85.7%) and non-Hispanic (88.2%). English was the primary language for 86.3% of patients and 65.8% of patients had private insurance. Solid tumors were the most common malignancy at 41.0%, followed by brain tumors (34.1%), and hematologic malignancies (24.9%). The majority of patients (60%) had metastatic disease at time of first enrollment. Among episodes of treatment, 51.4% received targeted monotherapy. Median overall survival from first enrollment for 210 patients treated with available vital status was 12.8 months. Among episodes of treatment, 25.2% of patients had an objective response to trial therapy. Stratified by disease type, objective response was documented for 52.9% of patients with hematologic malignancies, 20.5% of patients with CNS tumors, and 15.8% of patients with solid tumors. Patients on trials with cytotoxic-only treatment had the highest objective response (47.5%), while patients treated with cytotoxic plus targeted therapies combinations had the lowest rate (18.9%). The median duration of therapy was 1.5 months. Twenty-seven patients (11.0%) required dose modification and 22 patients (9.0%) had DLT. Conclusions: We present clinical outcomes for a recent cohort of pediatric patients treated on early phase clinical trials. We identified differential response rates to phase 1 therapy by disease and trial-type in a single-center cohort treated over the past decade. These data are immediately informative to discussions between providers and patients/parents regarding expected phase 1 trial outcomes.

#2374 Long-term clinical outcomes in pediatric patients with primary nephrogenic insipidus

Abstract Background and Aims Nephrogenic diabetes insipidus (NDI) is a rare inherited disorder, and there is a paucity of studies assessing long-term clinical outcomes in affected individuals. This study aims to evaluate the long-term clinical outcomes, encompassing growth and kidney function, in pediatric patients with NDI. Method This multicenter retrospective study included forty-nine children diagnosed with NDI. The medical records of these patients were retrospectively reviewed, and a linear mixed model was employed to analyze variations in growth and kidney function with increasing age. Results Mutations in the AVPR2 and AQP2 genes were identified in 40 (81.6%) and 4 (8.2%) patients, retrospectively, while pathogenic variants were either not found or not tested in 5 (10.2%) patients. The median ages at diagnosis and the last follow-up were 0.41 (interquartile ranges (IQR) 0.12−0.64) years and 9.97 (IQR 2.42−18.66) years, respectively. Thiazide treatment was administered to all patients, with 43 (87.8%) and 14 (28.6%) patients also receiving amiloride and indomethacin, respectively, in conjunction with thiazide. Our analysis using linear mixed models revealed significant changes in growth including height, weight, and body mass index (BMI) with increasing age. The estimated marginal means (EMM) for height Z score decreased from −0.114 (95% confidence interval (CI) −0.507 to 0.279) at diagnosis to −1.464 (95% CI −1.880 to −1.048) at 2 years of age, subsequently increasing to −0.706 (95% CI −1.186 to −0.225) at 14 years of age (P &amp;lt; 0.001). The height Z score at 19 year of age is −0.899 (95% CI −1.434 to −0.365), significantly lower than those at diagnosis (P = 0.038). The EMM for weight Z score increased from −1.580 (95% CI −2.009 to −1.149) at one year of age to 0.760 (95% CI 0.170 to 1.350) at 19 years of age, exhibiting a steady increase (P &amp;lt; 0.001). The EMM for BMI Z score demonstrated a consistent increase after age of 2 years (P &amp;lt; 0.001), which were −0.326 (95% CI −0.835 to 0.182) at 2 years of age and 1.210 (95% CI 0.602 to 1.817) at 19 years of age. The proportion of obesity increased from 3.2% at 3 years of age to 33.3% at 19 years of age. The EMM for estimated glomerular filtration rate (eGFR) consistently increased from 82.8 (95% CI 75.2 to 90.3) mL/min/m2 to 100.8 (95% CI 88.9 to 112.7) mL/min/m2. Although eGFR less than 60 mL/min/m2 is not present, the eGFR less than 90 mL/min/m2 is 47.5% at last follow up. Conclusion At the end of growth, the height Z score is low, and the proportion of obesity is high. To improve the growth pattern, clinicians need to monitor nutritional status and establish nutritional support during follow-up in patients with NDI. Since mild decreased kidney function developed in half, patients with NDI need long-term follow-up evaluation for kidney function.

2023 catastrophic Turkey earthquake: clinical outcomes of pediatric patients rescued under the rubble

ABSTRACT Objectives Children are the most vulnerable population affected by the earthquake. We aimed to examine the characteristics, morbidity, mortality, and the factors affecting these pediatric earthquake victims to guide the follow-up of pediatric patients who were rescued under the rubble in possible future disasters. Methods This is a retrospective observational study. The files of pediatric earthquake victims aged 0–18 years who were admitted to Adana City Training and Research Hospital between 6 February-30 April 2023 were analyzed. 318 patients were included in the study. Results Of all patients 48.7% (n = 155) were female and 51.3% (n = 163) were male. The mean age of the patients was 114.8 months. 65.4% (n = 208) of the patients were discharged from our hospital, eighteen patients (5.7%) died. Information on the hour when the patients were rescued under the rubble could be reached in 117 (36.8%) patients. The mean rescue time of these patients was 50.5 hours. 62 (53%) victims were rescued in the first 24 hours after the earthquake. There were 111 patients (34.9%) with fractures. There were 118 patients (37.1%) who underwent fasciotomy surgery. The total number of patients with amputation was 48 (15.1%). There were 36 patients (11.3%) with one limb amputated, 12 patients (%3.7) with more than one limb amputation. Internal organ injury was present in 30.5% (n = 97) of the patients. Of these patients, 85.6% (n = 83) had single organ trauma and 14.4% (n = 14) had multiple organ trauma. There were 58 patients (18.2%) with acute renal failure in follow-up. The mean rescue time for patients with amputation was 83.65 ± 62.9 hours, and for patients without amputation was 36.44 ± 50.6 hours. This difference was statistically significant (p = 0.001). No statistically significant difference was found when the ages of the patients were compared to fasciotomy, amputation, and fracture status. Conclusion While evaluating earthquake-induced trauma, pediatric patients should be approached carefully.

Association of handgrip strength with nutritional status and clinical outcomes in hospitalized pediatric patients

Background & aimsMalnutrition in children and adolescents is prevalent at hospital admission and the incidence increases with length of stay. Malnourished patients have loss of muscle mass and strength, compromising their functionality. Handgrip strength (HGS) is a nutritional marker understudied in pediatrics although it is capable of detecting nutritional deprivation before changes in body composition are observed. Therefore, this study aimed to evaluate the association between reduced HGS at hospital admission, compromised nutritional status and worse clinical outcomes of pediatric patients. MethodsCohort study conducted with patients aged 6 to 18 years admitted to a pediatric ward. Nutritional status was assessed in the first 48 hours of hospital admission using the z-score of height for age (H/A) and body mass index for age (BMI/A), percentile of mid-arm muscle circumference for age (MAMC/I) and the pediatric global subjective nutritional assessment (SGNA). HGS was measured using a digital dynamometer and considered reduced when the maximum value of three measurements was below the 5th percentile for sex and age. The clinical outcomes analyzed were length of hospital stay and frequency of readmission within 3 months after hospital discharge. ResultsA total of 135 patients were evaluated (median age 10.9 years, 55.6% male) and 17.8% had reduced HGS. Patients with reduced HGS had lower H/A z-score (-0.50 vs 0.22, p = 0.012) and a higher frequency of reduced MAMC when compared to those with normal HGS (8% vs 13%, p = 0.007). Reduced HGS was not associated with malnutrition (OR=0.63; 95%CI 0.23 - 1.77), prolonged hospital stay (OR=1.89; 95%CI 0.72 - 4.92) or readmission to hospital 3 months after hospital discharge (OR=1.82; 95%CI 0.67 - 4.93), in a model adjusted for the clinical condition. ConclusionReduced HGS was not a predictor of malnutrition and clinical outcomes. However, it was associated with lower H/A Z-score and MAMC/A percentile values and can be used as a complementary measure in the nutritional status assessment of hospitalized pediatric patients.

A comparison of Bayesian and frequentist approaches to incorporating clinical and biological information for the prediction of response to standardized pediatric colitis therapy.

The prospective cohort study PROTECT is the largest study in pediatric ulcerative colitis (UC) with standardized treatments, providing valuable data for predicting clinical outcomes. PROTECT and previous studies have identified characteristics associated with clinical outcomes. In this study, we aimed to compare predictive modeling between Bayesian analysis including machine learning and frequentist analysis. The key outcomes for this analysis were week 4, 12 and 52 corticosteroid (CS)-free remission following standardized treatment from diagnosis. We developed predictive modeling with multivariable Bayesian logistic regression (BLR), Bayesian additive regression trees (BART) and frequentist logistic regression (FLR). The effect estimate of each risk factor was estimated and compared between the BLR and FLR models. The predictive performance of the models was assessed including area under curve (AUC) of the receiver operating characteristic (ROC) curve. Ten-fold cross-validation was performed for internal validation of the models. The estimation contained 95% credible (or confidence) interval (CI). The statistically significant associations between the risk factors and early or late outcomes were consistent between all BLR and FLR models. The model performance was similar while BLR and BART models had narrower credible intervals of AUCs. To predict week 4 CS-free remission, the BLR model had AUC of 0.69 (95% CI 0.67-0.70), the BART model had AUC of 0.70 (0.67-0.72), and the FLR had AUC of 0.70 (0.65-0.76). To predict week 12 CS-free remission, the BLR model had AUC of 0.78 (0.77-0.79), the BART model had AUC of 0.78 (0.77-0.79), and the FLR model had AUC of 0.79 (0.74-0.83). To predict week 52 CS-free remission, the BLR model had AUC of 0.69 (0.68-0.70), the BART model had AUC of 0.69 (0.67-0.70), and the FLR model had AUC of 0.69 (0.64-0.74). The BART model identified nonlinear associations. BLR and BART models had intuitive interpretation on interval estimation, better precision in estimating the AUC and can be alternatives for predicting clinical outcomes in pediatric patients with UC. BART model can estimate nonlinear nonparametric association.

Combination of Qingfei Huayu Decoction and Targeted Nursing Care Improves the Clinical Outcomes in Pediatric Patients with Mycoplasma Pneumoniae Pneumonia

To explore the efficacy of the combined use of Qingfei Huayu decoction (QFHYD) and targeted nursing care in pediatric cases of Mycoplasma Pneumoniae pneumonia (MPP). One hundred and twenty pediatric cases of MPP admitted to our hospital from January 2021 to December 2022 were selected and randomly divided into control (n = 60) and experimental (n = 60) groups. The control group (CG) was treated with targeted nursing care in conjunction with conventional Western medicine, while the experimental group (EG) received QFHYD in addition to the CG’s treatment. Clinical efficacy, symptom relief time, and levels of inflammatory markers were recorded. Subsequently, a mouse model of MPP was established to investigate the effects of QFHYD on bacterial load in lung tissues, tissue damage, and the expression of members of the Notch pathway. The symptom relief times for cough, fever, and pulmonary rales were shorter in the EG than in the CG (all P &lt; 0. 001). After treatment, interleukin-6 (IL-6) and IL-8 levels decreased in both groups, with the levels being lower in the EG (both P &lt; 0. 001). Mouse model results showed that both azithromycin alone and in combination with QFHYD could reduce bacterial loads in bronchoalveolar lavage fluid and lung injury scores (all P &lt; 0. 001), with the combination showing more significant effects. Furthermore, western blotting indicated significant reductions in the relative protein levels of Notch1 and Notch2 in mouse lung tissues, with the combination showing more significant effects (all P &lt; 0. 001). In pediatric MPP, the combination of QFHYD and targeted nursing care can significantly enhance clinical efficacy, accelerate symptom relief, and improve the body’s inflammatory state. Based on animal studies, we speculate that the action of QFHYD may be related to the Notch pathway.

Preoperative nutritional status assessment and clinical outcomes in pediatric patients undergoing gastrointestinal surgery: a prospective study

Background: The objective was to assess the association between preoperative nutritional status and postoperative outcomes in children undergoing gastrointestinal surgery. Methods: This prospective study recruited pediatric patients diagnosed with gastrointestinal surgical pathology from the pediatric surgical department in a tertiary care hospital during the period from April 2023 to October 2023. Sixty surgical cases were included in our study from both genders. An organized questionnaire was created. This questionnaire involved 3 major parts: demographic and nutritional status parameters at admission and discharge. Comparison of nutritional status pre- and postoperatively were done. Results: A total of 60 pediatric patients were enrolled. According to both STRONGKIDS score and the subjective global nutritional assessment, 58.3% of upper gastrointestinal (GI) group were at risk of malnutrition. Wasting was more common in the upper GI group 75%; versus 36.8% in the lower GI group. Underweight was more common in the hepatobiliary and upper GI groups (nearly 41.7% for each group) than in the lower GI group (26.3%). Furthermore, stunted patients had a higher incidence of complications and a prolonged hospital stay (&gt;7 days) (p=0.001 and p=0.028, respectively), while underweight lower GI patients showed a prolonged hospital stay (p=0.017). Conclusions: Nutritional assessment is an essential part of surgical management of pediatric patients. Both underweight and wasting statuses were more common among hepatobiliary and upper GI groups. A long hospital stay and postoperative complications were more common among stunted patients.

Proactive therapeutic drug monitoring and vedolizumab dose optimization in children with inflammatory bowel disease.

Therapeutic drug monitoring (TDM) and dose optimization have been shown to improve clinical outcomes with antitumor necrosis factor and recent studies in adults suggest an exposure-response relationship with drug levels associated with improved clinical outcomes. However, these levels are not universally recognized as therapeutic targets for vedolizumab dosing. We aimed to assess the impact of a TDM quality improvement (QI) initiative on 52-week clinical outcomes and describe proactively obtained vedolizumab levels during the induction period in children with inflammatory bowel disease (IBD). A QI initiative to proactively obtain TDM levels at Week 6 was implemented in 2019. A retrospective review of pediatric patients with IBD treated with vedolizumab from 2018 to 2022 was performed. Baseline demographic data, medication dosing details, disease characteristics, lab results, and 12-month clinical outcomes were recorded. For this study, we defined therapeutic target levels (>20 μg/mL at Week 6 and >12 μg/mL during maintenance) based on existing data correlating these levels with improved clinical outcomes. Fifty-nine patients (31 Crohn disease [CD], 28 ulcerative colitis [UC]/indeterminate colitis [IC]) were included in the study. In total, 68% (40/59) of patients had vedolizumab levels at Week 6and 90% (53/59) had levels drawn at Week 6 or 14. Thirty-five percent of Week 6 trough levels were below our defined target of 20 μg/mL. Fifty-two of 59 patients had available data at 52 weeks. Over 80% (42/52) of patients remained on vedolizumab 52 weeks after initiation (CD 79% [23/29], UC/IC 83% [19/23]). Sixty-two percent (26/42) of patients that remained on vedolizumab at 52 weeks were treated with an intensified dosing interval of <8 weeks. Thirty-one of these 42 (74%) were in clinical remission (CR)rate at 52 weeks with 29/42 (69%) in corticosteroid-free remission. The CR rate for the entire cohort including those who discontinued therapy due to a lack of efficacy before 52 weeks was 60% (31/52). Proactive TDM and early dose optimization with vedolizumab may improve drug durability and clinical outcomes in pediatric patients with IBD.

Characteristics and clinical outcomes of pediatric patients following massive bowel resection: A retrospective cohort study

BackgroundMassive bowel resection (MBR), defined as residual small bowel < 25 % of expected length-for-age based on established norms, is a rare entity. Traditionally, children who have undergone MBR have higher mortality and less probability of achieving enteral autonomy compared to patients with less substantial bowel loss. The primary objective of this study was to determine the characteristics and clinical outcomes of pediatric patients following MBR. Secondary objectives included comparing patients who achieved enteral autonomy to those who remained parenteral nutrition (PN)-dependent to determine characteristics associated with PN independence. MethodsRetrospective cohort study of patients following MBR managed by our multidisciplinary intestinal rehabilitation program between January 1, 2006 and December 31, 2017, with an observation period ending December 31, 2021. Results61 patients with MBR fulfilled study inclusion criteria. 21 patients (34.4 %) achieved enteral autonomy while 40 patients (65.6 %) did not. Patients who achieved enteral autonomy had a greater percentage of residual small bowel (22 % vs 13 %, P < 0.01) and colon (100 % vs 50 %, P < 0.01). They were more likely to have an ICV (73.2 % vs 15 %, P < 0.01) and less likely to undergo STEP procedure (14.3 % vs 37.5 %, P = 0.02). 18 patients in the cohort without enteral autonomy achieved greater than 50 % enteral nutrition. In this cohort, the overall transplant rate was 9.8 % and mortality rate was 11.5 %. ConclusionSeveral important advancements have occurred over the last two decades that have allowed more patients to reach their adaptive potential and mitigate complications of prolonged PN following MBR. Overall outcomes are better than previously reported.

Effects of Hormone Replacement Therapy on Autoimmune Markers and Clinical Outcomes in Pediatric Patients with Hashimoto's Thyroiditis

Objective: Hashimoto’s Thyroiditis (HT), a chronic autoimmune thyroiditis, is the predominant cause of hypothyroidism in regions without iodine deficiency. HT is characterized by the loss of immunological tolerance of the thyroid gland, leading to autoimmune attacks. This study aimed to compare the autoantibody profiles, along with clinical and laboratory findings, of patients diagnosed with Hashimoto’s disease who were either receiving treatment or followed without treatment. Material and Methods: Clinical manifestations, laboratory data, and thyroid ultrasonography (USG) findings of patients diagnosed with Hashimoto’s thyroiditis receiving hormone therapy and those followed without treatment were compared in our clinic. Results: Among a total of 249 patients, 116 received hormone replacement therapy, while 133 were followed without treatment. The mean age of all patients was 13.91±3.71 years, with a mean age at diagnosis of 11.51±3.79 years. After twelve months of follow-up, the untreated group showed an increase in serum fT4 and antiTPO levels (p=0.012 and p=0.001), with no significant difference found in serum TSH, fT3, and antiTG levels. Those receiving treatment exhibited a significant decrease in serum TSH levels and a significant increase in serum fT4 levels (p=0.002 and p&amp;lt;0.001, respectively). Although there was an increase in serum antiTPO and antiTG levels over time, no change was detected in serum fT3 levels. Clinical improvement was significantly greater in the treatment group (p=0.044). Conclusion: It has been concluded that early initiation of hormone replacement therapy in Hashimoto’s thyroiditis can mitigate negative clinical effects during follow-up, contributing to patient comfort and alleviating clinical complaints.

Exoscope Efficacy and Feasibility in Pediatric Spinal Neurosurgery: A Single-Institution Cohort Case Series

The exoscope has emerged as an efficacious microscope in adult spinal neurosurgery providing improved operative field visibility and surgeon ergonomics. However, outcome data and feasibility are underrepresented in the pediatric literature. We present the largest case series aimed at assessing operative and clinical outcomes in pediatric patients undergoing various exoscope-assisted spinal surgeries. A retrospective review was conducted on all consecutive pediatric (age <18 years) spinal surgeries performed with the use of an exoscope by 3 senior surgeons at a single institution from 2020-2023. Demographics and clinical and operative outcomes were reviewed and analyzed. Ninety-six exoscope-assisted pediatric spine surgeries were performed on 89 unique patients, 41 (42.7%) of which were male. The mean age at surgery was 12 (±5.3) years. Spinal cord detethering (55.8%) was the most common procedure performed. The overall mean operative time for all procedures was 155 (±86) minutes, and the mean estimated blood loss was 18 (±41) mL. The mean length of stay was 5.4 (±6.5) days. There were 14 (14.6%) patients with complications in this cohort. At final follow-up, 64 (83.1%) of symptomatic patients reported neurologic symptom improvement. Using the exoscope in a variety of pediatric spinal surgeries resulted in an acceptable average operative time, estimated blood loss, length of stay, and rate of neurologic symptom improvement. The exoscope appears to be an efficacious option for pediatric neurosurgical spinal procedures.

Radiological and Clinical Outcomes of Pediatric Patients With a Supracondylar Humerus Fracture Surgically Treated With Closed Reduction and Percutaneous Pinning.

It was aimed to evaluate the clinical and radiological results of patients operated on with closed reduction and pinning due to pediatric supracondylar humerus fractures. The radiological and clinical results of 100 patients operated on with closed reduction and percutaneous pinning for pediatric supracondylar humerus fractures in the Department of Orthopedics and Traumatology were examined between January 2015 and February 2022. Clinical results were evaluated by performing cosmetic and functional scores defined by Flynn. Closed reduction and percutaneous pinning techniques were used in surgical treatment. In our study, 59 patients were male (59%), and 41 were female (41%). The average age of all patients is 6.21 ± 2.85 years. According to the Gartland classification, 21 patients' fractures were type IIA (21%), 12 patients' fractures were type IIB (12%), 51 patients' fractures were type III (51%), and 16 patients' fractures were type IV (16%). The average number of pins used in the treatment is 2.55 ± 0.50. The elbow bearing angle of the operated side of the patients was an average of 6.53 ± 3.29 degrees, the humerocapitellar angle was an average of 41.97 ± 3.08 degrees, and the lateral humerocapitellar angle was an average of 50.17 ± 3.58 degrees. Fifty-one patients had stage 0 (51%), 23 patients had stage 1 (23%), 23 patients had stage 2 (23%), and three patients had stage 3 (3%) residual sagittal plane deformity. According to the Flynn criteria, 92 patients had excellent functional results (92%), seven patients had good results (7%), and one patient had fair results (1%). Regarding cosmetic results, 91 patients had excellent results (91%), six patients had good results (6%), and three patients had fair results (3%). Supracondylar humerus fractures are common in children and can cause serious complications. Closed reduction and percutaneous pinning techniques are effective treatment methods in the treatment of displaced supracondylar humerus fractures.

Blinatumomab Therapy Is Associated with Favorable Outcomes after Allogeneic Hematopoietic Cell Transplantation in Pediatric Patients with B Cell Acute Lymphoblastic Leukemia

Blinatumomab, a bispecific T cell engager that binds CD19 in leukemic cells and CD3 in cytotoxic T cells and leads to leukemic blast lysis, is often used in pediatric patients with relapsed/refractory (R/R) B cell acute lymphoblastic leukemia (B-ALL) prior to allogeneic hematopoietic cell transplantation (allo-HCT). Concerns about the potential risk of blinatumomab-related immune-mediated toxicities after allo-HCT have not been adequately addressed. These include graft-versus-host disease (GVHD), delayed engraftment, and graft failure or rejection. Pediatric-specific data reporting post-HCT outcomes of patients treated with blinatumomab are scarce and limited to small cohorts. We sought to investigate the clinical outcomes of pediatric patients with R/R B-ALL who received blinatumomab therapy pre-HCT, focusing on overall survival (OS), leukemia-free survival (LFS), cumulative incidence of relapse (CIR), and nonrelapse mortality (NRM), as well as the incidence of immune-mediated post-HCT complications including GVHD, delayed neutrophil or platelet engraftment, graft failure, and graft rejection. We also investigated blinatumomab's effects on B cell reconstitution based on achievement of i.v. immunoglobulin (IVIG) independence post-HCT. This single-center, retrospective study included patients with B-ALL receiving blinatumomab therapy before undergoing allo-HCT, with transplantation performed between 2016 and 2021 at our institution. Patients receiving blinatumomab for relapse after allo-HCT were excluded. Patients receiving chemotherapy alone before allo-HCT during the same period composed the control group. Seventy-two patients were included, 31 of whom received blinatumomab before allo-HCT. Survival estimates were obtained using the Kaplan-Meier method, and the log-rank test was used to analyze differences between groups. Categorical variables were compared between groups using the chi-square test or Fisher exact test, and continuous variables were compared using the Wilcoxon rank-sum test. Cumulative incidences were estimated using the competing risks method, and Gray's test was used to analyze differences between groups. A Cox proportional hazards regression model was used for univariate and multivariable analyses for OS. Landmark analysis was performed at the set time points of 30 days and 100 days post-allo-HCT. Most patients in the study cohort had high-risk relapsed B-ALL. Blinatumomab therapy induced minimal residual disease (MRD)-negative remissions in all patients, whereas 5 patients (12.2%) receiving chemotherapy alone had persistent MRD pre-allo-HCT. Time from the start of therapy to the date of allo-HCT was shorter for patients who received blinatumomab compared with those who received chemotherapy (P < .0001). Blinatumomab therapy was associated with greater LFS compared to chemotherapy alone (P = .049), but when limited to 1 year, LFS was not significantly different from control (P = .066). There appeared to be higher OS, lower CIR, and lower NRM in patients receiving blinatumomab compared to the control group; however, the differences were not significant. None of the variables assessed in multivariable analysis was associated with differences in OS. When compared to the controls, blinatumomab therapy did not result in a higher incidence of acute or chronic GVHD, delayed neutrophil or platelet engraftment, or graft failure or rejection. The time to IVIG infusion independence post-allo-HCT was similar in the 2 groups. This study supports the use of blinatumomab salvage therapy for R/R B-ALL before allo-HCT given its efficacy in inducing MRD-negative remissions and optimizing LFS, as well as its lack of association with an increased incidence of post-allo-HCT adverse immune-mediated toxicities. Larger, prospective studies are needed to confirm these findings and to investigate blinatumomab's effects in long-term post-allo-HCT events.

One-year follow-up of children hospitalized with COVID-19: a prospective cohort study.

Currently, little is known about the long-term outcomes of COVID-19 in the pediatric population. The aim of this study was to investigate the long-term clinical outcomes of pediatric patients hospitalized with COVID-19. This was a prospective cohort study involving unvaccinated children and adolescents admitted to a tertiary hospital in southern Brazil with a COVID-19 diagnosis. Data were collected from electronic medical records for one year after the diagnosis. A total of 66 children were included: the median age was 2.9 years; 63.6% were male; and 48.5% were under 2 years of age. Over 70% had at least one comorbidity prior to the COVID-19 diagnosis. During the one-year follow-up period, 59.1% of the children revisited the emergency department, 50% required readmission, and 15.2% died. Younger children with longer hospital stays were found to be at greater risk of readmission. Having cancer and impaired functionality were found to increase the risk of death within one year. Our findings indicate that most children hospitalized with COVID-19 have comorbidities. Younger age at admission and a longer hospital stay seem to be risk factors for readmission. In addition, the presence of cancer and impaired functionality are apparently associated with the poor outcome of death within the first year after the diagnosis of COVID-19.

Personalized application of antimicrobial drugs in pediatric patients with augmented renal clearance: a review of literature.

ARC impacts antimicrobial drugs in pediatric patients. • ARC is inextricably linked to the failure of antimicrobial therapy, recurrence of infection, and subtherapeutic concentrations of drugs. • This study provides an updated overview of the influence of ARC on medication use and clinical outcomes in pediatric patients. • In this context, there are several recommendations for using antibiotics in pediatric patients with ARC: 1) increase the dose administered; 2) prolonged or continuous infusion administration; 3) use of TDM; and 4) use alternative drugs that do not undergo renal elimination.