Clinical Outcomes In Pediatric Patients Research Articles

Clinical Predictors of Morbidity and Mortality in Hospitalized Pediatric Patients With Ascites.

Ascites is a pathologic buildup of fluid in the peritoneal cavity. Knowledge is lacking in clinical outcome in pediatric patients with ascites. We aim to identify and assess clinical variables, associated with morbidity and mortality in pediatric patients who are hospitalized with ascites. A retrospective cohort study was performed on patients ages 0 to 21 hospitalized at Johns Hopkins Hospital between 1983 and 2010 with an ICD-9 discharge diagnosis of ascites (789.5, 789.51, 789.59). A total of 518 pediatric patients were studied, all with a diagnosis of ascites during hospitalization. Study outcomes included hospital length of stay (LOS) as a proxy for morbidity and death at hospital discharge for mortality. Variables analyzed included demographic data, ascites etiology and grade, comorbidities, and laboratory markers. Variables were analyzed by log-linear regression and competing risk model. Among the 3 age groups (0-5, 6-12, and 13-21), the 0 to 5 age group experienced significantly increased LOS (P < 0.001) and mortality (P = 0.027). Ascites etiology of veno-occlusive disease (VOD) and the presence of hydrothorax or thrombocytopenia was also significantly associated with increased LOS. Ascites with the etiology of congestive hepatopathy and the presence of grade 3 ascites, hepatic encephalopathy, hepatorenal syndrome, hydrothorax, hyponatremia, and thrombocytopenia were associated with increased mortality. Additionally, black pediatric patients have an increased risk of mortality (P = 0.027). Other factors including sex, leukopenia, portal vein thrombosis, and splenomegaly were not associated with LOS or mortality. Morbidity and mortality in pediatric patients hospitalized with ascites are associated with specific demographic and clinical factors. Further studies are required to apply this knowledge to predict the clinical outcomes.

Cross-sectional study of the clinical characteristics and outcomes of children hospitalized with COVID-19 in Lima, Peru

Coronavirus 2019 (SARS-CoV-2) infection in children occurred in Peru as of March 2020, leading to pediatric patients' hospitalization in areas adapted for this purpose at the Edgardo Rebagliati Martins National Hospital. In the beginning, the demand for hospitalization was low, but it increased gradually. Consistent with international reports, the majority of patients presented mild or moderate symptoms. Nonetheless, there were also severe cases, even fatal ones. To describe the characteristics and clinical outcome of pediatric patients with COVID-19 hospitalized in a referral hospital in Lima, Peru, between March and August 2020. A descriptive and inferential cross-sectional study was carried out. The population includes all hospitalized patients in the Department of Pediatrics, with clinical and surgical diagnoses associated with COVID-19. We included 100 patients, with an average age of 83.4 ± 54 months, with a predominance of male patients (55%). Hospitalized patients were grouped into five categories: respiratory failure (17%), multisystemic inflammatory syndrome (MIS-C) (31%), neurological presentation (19%), acute abdomen (20%), and patients with oncological problems (13%). Most of the patients (74%) had comorbidities. Regarding the presenting symptoms, intestinal pain predominated in the appendicitis group (90%, p < 0.001), fever was present in most patients with respiratory failure (64.7%); multisystemic inflammatory syndrome (90.3%), neurological manifestations (15.8%), acute abdomen (50%) and oncological conditions (61.5%) were also present in these patients. Kawasaki symptoms were found in 38.7% of the patients with multisystemic inflammatory syndrome. Mortality was 4%. Respiratory problems (29.4%) and multisystemic inflammatory syndrome (22.6%) required admission to intensive care, more frequently than the other presentations (p = 0.008). We conclude that the vulnerability in the pediatric population is the one that has preexisting conditions. We divided our patients according to presentation, diagnosis, and complications, which were predominantly respiratory. We also had oncological patients with COVID-19.

Effects of modified ultrafiltration and conventional ultrafiltration combination on perioperative clinical outcomes in pediatric cardiac surgery

Background:This meta-analysis was performed to review the effects of the addition of modified ultrafiltration (MUF) and conventional ultrafiltration (CUF) to CUF alone on postoperative hemoglobin, surgical and ultrafiltration data, and postoperative clinical outcomes in pediatric patients undergoing cardiac surgery.Methods:A systematic search was performed to identify randomized controlled clinical trials that compared MUF and CUF combination with CUF alone in pediatric cardiac surgery undergoing cardiopulmonary bypass (CPB) in PubMed, Embase, Cochrane Library, and Web of Science without any language or date limitation in February 2020. For each included trial, the primary outcomes including post-CPB and postoperative hematocrit, surgical and ultrafiltration data, postoperative clinical outcomes including volume of chest tube drainage within 48 hours after surgery and perioperative blood requirement, ventilation support duration, and length of stay day in the intensive care unit (ICU) and hospital were collected and analyzed. The analysis was conducted using STATA version 12.0.Results:A total of 8 trials encompassing 405 patients were included in this analysis. Analysis indicated that MUF + CUF increased the post-CPB hematocrit (Standard mean difference, SMD = 1.85, 95% confidence interval, 95% CI 0.91–2.79). Meanwhile, ultrafiltration volume was higher in CUF+MUF infants than CUF-alone infants (SMD = 1.46, 95% CI 0.51–2.41, P = .003). The clinical outcomes, including postoperative hemodynamic changes, prime volume, blood requirement, chest tube drainage volume, mechanical ventilation duration, and ICU duration, were unclear because of the unstable sensitivity analyses.Conclusions:Beneficial effects of using MUF and CUF for pediatric cardiac surgery, including increase post-CPB hematocrit and ultrafiltration volume when compared with CUF alone. Meanwhile, MUF and CUF did not significantly influence the postoperative hospital stay duration, CPB, and aortic occlusion duration.

Effects of regional differences on the outcome of cardiopulmonary resuscitation in children: How much different is Gaziantep from Izmir?

OBJECTIVES:We aimed to compare the demographic and clinical characteristics between pediatric cardiac arrest patients treated in Gaziantep at South-eastern Anatolian region and Izmir at Aegean Shore.MATERIALS And METHODS:We retrospectively reviewed sociodemographic characteristics, laboratory parameters, and clinical outcomes of pediatric patients that underwent cardiopulmonary resuscitation due to prehospital cardiac arrest at two pediatric emergency departments in Izmir Hospital of Health Sciences University and Gaziantep Cengiz Gökçek Kadın Doğum ve Çocuk Hospital of Health Ministry between August 2017 and August 2018.RESULTS:The present study included 188 patients (112 patients from Gaziantep and 76 patients from Izmir). All patients arrived at the hospital through emergency medical services. The median age was lower (14 days vs. 15 months; P < 0.001), and the proportion of Syrian refugees was higher in patients from Gaziantep (78.6% vs. 7.9%; P < 0,001). In both centers, respiratory failure was the most common etiology. In patients from Gaziantep, pH levels were lower (median: 7.10 vs. 7.24), and lactate levels were higher (median: 6 mmol/L vs. 3.6 mmol/L; P < 0.001). The mortality rate was higher among patients from Gaziantep (27.7% vs. 7.9%; P = 0.001).CONCLUSIONS:The rate of Syrian refugees among children who were brought to emergency department due to pre-hospital cardiac arrest was much higher in Gaziantep compared to Izmir. Syrian children were significantly younger and had more severe tissue hypoxia, resulting in a higher mortality rate.

Clinical Outcome in Pediatric Patients with Philadelphia Chromosome Positive ALL Treated with Tyrosine Kinase Inhibitors Plus Chemotherapy-The Experience of a Polish Pediatric Leukemia and Lymphoma Study Group.

Simple SummaryPhiladelphia chromosome positive acute lymphoblastic leukemia (ALL Ph+) is rare in children, but outcomes are still poor. The aim of our study was to analyze the toxicity events and results of children with ALL Ph+ treated according to the EsPhALL2010 protocol (the European intergroup study of post induction treatment of Philadelphia chromosome positive ALL) in Poland between the years 2012 and 2019. Our treatment outcomes are still disappointing compared to those in other reports. Improvements in supportive care and emphasis placed on the determination of MRD at successive time points, which will impact decisions on therapy, may be required.The treatment of children with Philadelphia chromosome positive acute lymphoblastic leukemia (ALL Ph+) is currently unsuccessful. The use of tyrosine kinase inhibitors (TKIs) combined with chemotherapy has modernized ALL Ph+ therapy and appears to improve clinical outcome. We report herein the toxicity events and results of children with ALL Ph+ treated according to the EsPhALL2010 protocol (the European intergroup study of post-induction treatment of Philadelphia chromosome positive ALL) in 15 hemato-oncological centers in Poland between the years 2012 and 2019. The study group included 31 patients, aged 1–18 years, with newly diagnosed ALL Ph+. All patients received TKIs. Imatinib was used in 30 patients, and ponatinib was applied in one child due to T315I and M244V mutation. During therapy, imatinib was replaced with dasatinib in three children. The overall survival of children with ALL Ph+ treated according to the EsPhALL2010 protocol was 74.1% and event-free survival was 54.2% after five years. The cumulative death risk of the study group at five years was estimated at 25.9%, and its cumulative relapse risk was 30%. Our treatment outcomes are still disappointing compared to other reports. Improvements in supportive care and emphasis placed on the determination of minimal residual disease at successive time points, which will impact decisions on therapy, may be required.

Risk Factors Related to Operative Duration and Their Relationship With Clinical Outcomes in Pediatric Patients Undergoing Roux-en-Y Hepaticojejunostomy.

Background: Operative duration might be important for perioperative morbidity, and its involvement has not been fully characterized in pediatric patients. We identified perioperative variables associated with operative duration and determined their influence on clinical outcomes in pediatric patients.Methods: We retrospectively reviewed 701 patients who underwent elective removal of choledochal cysts followed by Roux-en-Y hepaticojejunostomy. The patients were separated into the long operative time group (>165 min) and short operative time group (<165 min) based on the median operative time (165 min). Propensity score matching was performed to adjust for any potential selection bias. The independent risk factors for operative time were determined using multivariable logistic regression analyses.Results: The operative time was often increased by excision difficulty caused by a larger choledochal cyst size (OR = 1.56; 95% CI, 1.09–2.23; p < 0.001), a greater BMI (OR = 1.02; 95% CI, 1.00–1.15; p = 0.018), and older age (OR = 1.17; 95% CI, 1.02–1.39; p = 0.012) in the multivariate analysis. A long surgical duration was associated with delayed gastrointestinal functional recovery, as measured using the time to first defecation (p = 0.027) and first bowel movement (p = 0.019). Significantly lower levels of serum albumin were found in the long operative time group than in the short operative time group (p = 0.0035). The total length of postoperative hospital stay was longer in patients in the long operative time group (7.51 ± 2.03 days) than in those in the short operative time group (6.72 ± 1.54 days, p = 0.006).Conclusions: Our data demonstrated that a short operative time was associated with favorable postoperative results. The influencing factors of operative time should be ameliorated to achieve better outcomes.

Early Implantation as a Main Predictor of Response to Vagus Nerve Stimulation in Childhood-Onset Refractory Epilepsy.

We describe a multicenter experience with vagus nerve stimulator implantation in pediatric patients with drug-resistant epilepsy. Our goal was to assess vagus nerve stimulation efficacy and identify potential predictors of favorable outcome. This is a retrospective study. Inclusion criteria: ≤18 years at time of vagus nerve stimulator implantation, at least 1 year of follow-up. All patients were previously found to be unsuitable for an excisional procedure. Favorable clinical outcome and effective vagus nerve stimulation therapy were defined as seizure reduction >50%. Outcome data were reviewed at 1, 2, 3, and 5 years after vagus nerve stimulator implantation. Fisher exact test and multiple logistic regression analysis were employed. Eighty-nine patients met inclusion criteria. Responder rate (seizure frequency reduction >50%) at 1-year follow-up was 25.8% (4.5% seizure-free). At last follow-up, 31.5% had a favorable outcome and 5.2% were seizure free. The only factor significantly predicting favorable outcome was time to vagus nerve stimulator implantation, with the best outcome achieved when vagus nerve stimulator implantation was performed within 3 years of seizure onset. Implantation between 3 and 5 years after epilepsy onset correlated with better long-term seizure freedom (13.3% at T5). Overall, 65.2% of patients evidenced improved quality of life at last follow-up. However, 12.4% had adverse events, but most were mild and disappeared after 3-4 months. Early vagus nerve stimulator implantation within 5 years of seizure onset was the only predictor of favorable clinical outcome in pediatric patients. Improved quality of life and a low incidence of significant adverse events were observed.

Abstract 16461: Are Inducible Exercise Left Ventricular Outflow Tract Gradients Associated With Adverse Outcomes in Pediatric Hypertrophic Cardiomyopathy?

Introduction: Adverse outcomes in hypertrophic cardiomyopathy (HCM) includes arrhythmias, myocardial fibrosis, heart failure, and sudden cardiac arrest (SCA). These are well documented in children. Exercise stress echocardiography (ESE) can identify the severity and mechanism of left ventricular outflow tract (LVOT) obstruction that may not be present at rest. Our aim was to compare clinical outcomes in pediatric patients with varying severity of LVOT obstruction. Methods: We reviewed records of patients &lt; 22 years with HCM who underwent staged ESE Jan 2009 - Dec 2019 at our center. Patients were divided into 3 groups: no resting or exercise LVOT gradient (Group 1), no resting gradient with LVOT gradient &gt; 30 mmHg at peak exercise (Group 2), and resting LVOT gradient &gt; 30 mmHg (Group 3). We assessed echocardiographic indices, advanced imaging, and clinical data. Statistical analysis performed with p-value &lt;0.05. Results: 93 patients met inclusion criteria. 6 SCA events occurred, though not during ESE.. Patients in Group 3 were classified less often as NYHA Class 1 compared to Group 1 or Group 2, (p &lt; 0.01). Group 3 had the highest rate of myectomy (p=0.005) and ICD placement (p=0.003). There was no significant difference in exercise symptoms, peak oxygen consumption, or ischemic changes during ESE between the groups. Peak heart rate was also higher in Group 2 with no difference in blood pressure response. Groups were not associated with family history of disease, positive gene status, SCA, or the presence of fibrosis (Table). LVOT gradients differed as early as Stage 1 in Groups 1 &amp; 2. Inducible LVOT obstruction was most commonly midcavitary in Group 2. Conclusion: The presence of resting versus inducible LVOT obstruction in pediatric HCM does not appear associated with adverse outcomes. This may be hampered by the rarity of events in pediatrics. The predominant mechanism of inducible LVOT obstruction was midcavitary, which has a link to adverse long-term outcomes in adult HCM.

Enoxaparin Utilization Evaluation and Its Clinical and Laboratory Outcomes in Pediatric Patients in a Children’s Teaching Hospital

Backgrounds: In recent years, low molecular weight heparin use has increased in children. Dose of enoxaparin to achieve target anti-Xa and time to achieve anti-Xa are evolving and efficacy outcome data in terms of laboratory and clinical response rate in children still remains to be elucidated. Thus, in this drug utilization and evaluation study, we assessed patterns of enoxaparin use, its concordance with guidelines and laboratory and clinical outcomes in pediatric patients in a Children’s teaching hospital. Methods: In a prospective observational study, all pediatric patients with a thrombotic event who underwent treatment with enoxaparin were included. Demographic data, clinical outcome data based on follow-up sonography results, laboratory response based on anti-Xa and concordance with guidelines in terms of initial daily dose, duration of treatment, performing sonography to evaluate response, anti Xa check and time of anti-Xa check were evaluated. Results: During a 9-month period, 41 pediatric patients suffered a thrombotic event and received enoxaparin. Median age of participants was 18.5 months. The anti-Xa level became therapeutic on mean day 4.7 with a mean enoxaparin dose of 1.24 mg/kg. Among participants 42% achieved therapeutic anti-Xa with initial empirical dosing. Less than 25 % of participants had a follow-up sonography and among them, 77% demonstrated complete thrombosis resolution after 4-6 weeks of enoxaparin therapy. We observed one major bleeding event. Concordance with guidelines was low in the aspects of duration of treatment, performing sonography to evaluate response and anti-Xa check. Conclusion: With initial empiric dosing, it may take several days before anti-Xa become therapeutic. Among half of the children, a higher than recommended 1 mg/kg dose was required to achieve therapeutic anti-Xa level. Educational processes are mandatory regarding enoxaparin use and monitoring among clinicians to improve concordance with guidelines.

Supraglottoplasty for severe laryngomalacia. Can we predict success?

ObjectivesLaryngomalacia is the dynamic collapse of supraglottic structures during inspiration, leading to a variable degree of airway obstruction. Clinical symptoms appear in the first months of life and are usually mild and resolve by the age of 12–18 months. In severe cases, surgical intervention may be considered. The goal of the study was to review the clinical outcome of pediatric patients who underwent supraglottoplasty for laryngomalacia. Material and methodsClinical and demographic data were retrieved from medical records of children diagnosed with laryngomalacia by laryngo-bronchoscopy between 2013 and 2019. Indications, outcome and long-term follow-up were collected from children undergoing surgery. ResultsDuring the study period, 115 children were diagnosed with laryngomalacia. The median age at diagnosis was 3 months. Synchronous airway lesions were diagnosed in 20% of patients. Ten (8.7%) children underwent surgical treatment because of significant respiratory symptoms and/or failure to thrive. Three of them had comorbidities. All otherwise healthy children had significant respiratory and nutritional improvement after surgery while those with comorbidities had less successful outcomes. ConclusionWe conclude that in severe cases of laryngomalacia, supraglottoplasty has an important role to play in management. In children with comorbidities, the surgical results may be less successful. Therefore, we recommend that the decision to operate should be individualized, ensuring full disclosure to the family regarding the probable benefit along with the limitations of surgery.

Evidence demonstrating the pharmacist's direct impact on clinical outcomes in pediatric patients: An opinion of the pediatrics practice and research network of the American College of Clinical Pharmacy

Evidence demonstrating the pharmacist's direct impact on clinical outcomes in pediatric patients: An opinion of the pediatrics practice and research network of the American College of Clinical Pharmacy

The Resilient Child: Sex-Steroid Hormones and COVID-19 Incidence in Pediatric Patients.

Coronavirus disease–2019 (COVID-19), a disease caused by Severe Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) infection, has become an unprecedented global health emergency, with fatal outcomes among adults of all ages in the United States, and the highest incidence and mortality in adult men. As the pandemic evolves there is limited understanding of a potential association between symptomatic viral infection and age. To date, there is no knowledge of the role children (prepubescent, ages 9-13 years) play as “silent” vectors of the virus, with themselves being asymptomatic. Throughout different time frames and geographic locations, the current evidence on COVID-19 suggests that children are becoming infected at a significantly lower rate than other age groups—as low as 1%. Androgens upregulate the protease TMPRSS2 (type II transmembrane serine protease-2), which facilitates efficient virus-host cell fusion with the epithelium of the lungs, thus increasing susceptibility to SARS-CoV-2 infection and development of severe COVID-19. Owing to low levels of steroid hormones, prepubertal children may have low expression of TMPRSS2, thereby limiting the viral entry into host cells. As the world anticipates a vaccine against SARS-CoV-2, the role of prepubescent children as vectors transmitting the virus must be interrogated to prepare for a potential resurgence of COVID-19. This review discusses the current evidence on the low incidence of COVID-19 in children and the effect of sex-steroid hormones on SARS-CoV-2 viral infection and clinical outcomes of pediatric patients. On reopening society at large, schools will need to implement heightened health protocols with the knowledge that children as the “silent” viral transmitters can significantly affect the adult populations.

Prognostic Impact of Extramedullary Infiltration in Pediatric Low-risk Acute Myeloid Leukemia: A Retrospective Single-center Study Over 10 Years

BackgroundThe impact of extramedullary infiltration (EMI) on the clinical outcomes of pediatric patients with acute myeloid leukemia (AML) are controversial. Patients and MethodsA total of 214 pediatric patients with low-risk AML were classified as having EMI (central nervous leukemia [CNSL] and/or myeloid sarcoma [MS]) and not having EMI. Patients with isolated MS before AML diagnosis by bone marrow examination were confirmed with histopathologic examination. For patients diagnosed with AML by bone marrow examination, a thorough physical examination and radiologic imaging were used to confirm MS. ResultsMale gender, a high white blood cell count, the FAB-M5 subtype, t(8;21) and t(1;11) abnormalities, and c-KIT mutations were associated with EMI. The presence of MS was associated with a low complete remission rate (63.6% vs. 79.4%; P = .000) and poor 3-year relapse-free survival (RFS) (62.6% ± 7.5% vs. 87.0% ± 2.8%; P = .000) and 3-year overall survival (73.5% ± 7% vs. 88.8% ± 2.6%; P = .011). Multivariate analysis revealed that MS was a poor prognostic factor for RFS and overall survival. Bone infiltration was an independent risk factor for inferior RFS with MS. Patients with CNSL had a low complete remission rate (58.3% vs. 77.2%; P = .045); however, CNSL did not significantly affect the survival of low-risk patients with AML. ConclusionMS should be considered an independent risk factor to guide stratified treatment.

Vancomycin Dosage and Its Association with Clinical Outcomes in Pediatric Patients with Gram-Positive Bacterial Infections.

AimThe aim of this study was to evaluate whether vancomycin trough concentrations at initial steady state are associated with clinical and microbiological outcomes along with vancomycin-related nephrotoxicity in pediatric patients with Gram-positive bacterial (GPB) infections.MethodsA retrospective cohort study of pediatric patients who received vancomycin for ≥72 hours during 2008–2016 was conducted. Study patients were divided into three cohorts in accordance with their first trough levels at steady state: <5 mg/L (lower-trough), 5–10 mg/L (low-trough), and >10 mg/L (high-trough; reference) cohorts.ResultsOf the 201 patients eligible for study inclusion, 60 patients in the lower- and low-trough cohorts, respectively, were idect 3ntified via propensity score matching and analyzed against 30 high-trough patients in each comparison pair (neonates were excluded due to small sample size). Lower-trough patients were at a greater risk for prolonged therapy, retreatment, and dose adjustment than high-trough patients. Final steady-state troughs remained substantially lower in both the lower- and low-trough cohorts (p<0.001 and p=0.005, respectively), despite greater dose up-titration in the lower-trough cohort and percent change in daily dose in both the lower- and low-trough cohorts than in the high-trough cohort (p<0.001 for all). Clinical cure and death risk, along with the risks of isolation of resistant strains and renal events, were not significantly different between cohorts in both comparison pairs.ConclusionVancomycin troughs of <5 mg/L at initial steady state were associated with significantly compromised clinical outcomes in terms of risk of therapy prolongation, retreatment, and aggressive dose up-titration, compared to >10 mg/L troughs in pediatric patients with GPB infections.

Oral propranolol administration is effective for infantile hemangioma in late infancy: A retrospective cohort study.

The highest efficacy of oral propranolol is for infantile hemangioma (IH) in the proliferative phase. Evaluation of the effectiveness of oral propranolol is less established when it is administered in late infancy following the proliferative phase. We aimed to assess the clinical outcomes of pediatric patients managed by oral propranolol beyond the proliferative phase of IH. A retrospective cohort study in a tertiary health care referral center was conducted to track all patients with IH receiving systemic propranolol following the proliferative phase. Twenty-eight eligible patients were managed by 2 to 3 mg/kg per day of oral propranolol for IH beyond the proliferative phase, defined at 9 months of age. The mean age at the initiation of propranolol was estimated at 11.25 (SD 2.24) months. All eligible patients experienced some degree of clinical resolution, with the average improvement rate being estimated at 77.1% (SD 16.1). Comparable results were achieved among patients who were placed on propranolol at an age older than 12 months and those managed between the age of 9 and 12 months. No serious adverse events were observed during the follow-up duration. In conclusion, oral propranolol is a safe and effective treatment for patients with IH initiating this agent beyond the proliferative phase.

Evidence demonstrating the pharmacist's direct impact on clinical outcomes in pediatric patients: An opinion of the pediatrics practice and research network of the American College of Clinical Pharmacy

AbstractThe pharmacist's role in pediatric care is critical for the provision of safe medication use secondary to differences in physical development, frequent off label use of medications, need for dose calculations, and the lack of standard dosage forms. Many regulatory agencies, health care organizations, and professional societies support the pharmacist as a member of the interdisciplinary team. However, specific details showcasing how pharmacists impact the care of pediatric patients in a quantifiable and meaningful way have not been well described. The purpose of this article is to provide a review of evidence demonstrating measurable clinical outcomes directly resulting from pharmacist participation in pediatric patient care. This report is an opinion paper of The American College of Clinical Pharmacy (ACCP) Pediatrics Practice and Research Network and has been endorsed by the Pediatric Pharmacy Association (PPA). This article represents the opinion of the Pediatrics Practice and Research Network of ACCP. It does not necessarily represent an official ACCP commentary, guideline, or statement of policy or position.

Pheochromocytoma and Paraganglioma in Children and Adolescents: Experience of the French Society of Pediatric Oncology (SFCE).

PurposeThe purpose of this work is to assess the clinical outcome of pediatric patients diagnosed with pheochromocytoma and paraganglioma (PPGL) detected in France since 2000.MethodsA retrospective multicenter study was conducted that included all patients younger than 18 years with PPGL diagnosed in France between 2000 and 2016. Patients were identified from 4 different sources: the National Registry of Childhood Solid Tumors, the French Pediatric Rare Tumors Database, the French registry of succinate dehydrogenase (SDH)-related hereditary paraganglioma, and the nationwide TenGen network.ResultsAmong 113 eligible patients, 81 children with available data were enrolled (41 with adrenal and 40 with extra-adrenal PPGL). At diagnosis, 11 had synchronous metastases. After a median follow-up of 53 months, 27 patients experienced a new event (n = 7 second PPGL, n = 1 second paraganglioma [PGL], n = 8 local recurrences, n = 10 metastatic relapses, n = 1 new tumor) and 2 patients died of their disease. The 3- and 10-year event-free survival rates were 80% (71%-90%) and 39% (20%-57%),respectively, whereas the overall survival rate was 97% (93%-100%)at 3 and 10 years. A germline mutation in one PPGL-susceptibility gene was identified in 53 of the 68 (77%) patients who underwent genetic testing (SDHB [n = 25], VHL [n = 21], RET [n = 2], HIF2A [n = 2], SDHC [n = 1], SDHD [n = 1], NF1 [n = 1]). Incomplete resection and synchronous metastases were associated with higher risk of events (P = .011, P = .004), but presence of a germline mutation was not (P = .11).ConclusionsMost pediatric PPGLs are associated with germline mutations and require specific follow-up because of the high risk of tumor recurrence.

Clinical outcome of paediatric patients with traumatic brain injury (TBI) receiving 3% hypertonic saline (HTS) in the emergency room of a tertiary care hospital.

To determine the clinical outcome and mean length of hospital stay of paediatric patients with severe blunt traumatic head injury (THI) receiving 3% hypertonic saline (HTS) in the Emergency Department (ED). This case series study was conducted at the Department of Emergency Medicine, Aga Khan University Hospital, Karachi, from 2014 to 2015 via chart review of 105 patients. Detailed history and clinical examination of all paediatric patients aged 2-16 years was recorded which included moderate to severe head injury as classified by the Glasgow Coma Scale (GCS) by the Brain Trauma Foundation. As per routine care after admission of such a patient, for resuscitation 3% HTS was administered. GCS was recorded at 6 hours and at the time of discharge. Of the 105 patients, 76 (72.4%) were male and 29 (27.6%) were female, and the mean age was 61.6+45.9 months. Traumatic brain injury (TBI) was found moderate in 60 (57.1%) cases and severe in 45 (42.9%) of our patients as per the GCS. Six hours after resuscitation with 3% hypertonic saline, 45 (43%) patients normalised as per GCS, 39 (37%) patients had moderate TBI and 21 (20%) had severe TBI. Forty five patients had a hospital stay of 2-3 days. The GCS improved after resuscitation with 3% hypertonic saline in emergency department, with a mean length of stay of 4.6+3.9 and 12.6+10.7 days in moderate and severe head injury respectively with a P value of <0.001, and was normal in 94 (89.5%) patients at the time of discharge. Paediatric patients with TBI receiving 3% hypertonic saline results in improved GCS and a decrease in the length of hospital stay.

Thomsen-Friedenreich antigen activation as a predictor for clinical outcome of pediatric patients with invasive pneumococcal disease

The most severe form of pneumococcal disease is invasive pneumococcal disease (IPD), including empyema, sepsis and meningitis. Thomsen-Friedenreich antigen (TA; Galβ1-3GalNAc) activation is known to be a predictor of Streptococcus pneumoniae-associated hemolytic uremic syndrome (Sp-HUS). There have been limited data to correlate TA activation and overall disease severity of IPD in children. The study aimed to prove the positive correlation between TA activation and disease severity and to demonstrate the trend of TA level during the disease course. We retrospectively reviewed the medical records from 38 pediatric patients aged from 0 to 18 years with microbiologically-confirmed IPD between 2010 and 2015at a medical center in Taiwan. All cases underwent TA activation testing by the fluorescence-labeled peanut lectin agglutination method. Medical information including demographic data, laboratory findings, co-morbidities, and outcome was collected and reviewed. We compared the clinical manifestations and associated co-morbidities between TA-positive and TA-negative patients. Among the 38 patients, 25 (66%) showed TA activation. Compared to TA-negative patients, patients with TA activation had a statistically higher rate of prolonged anemia, thrombocytopenia, and acute kidney injury. TA-positive patients also had a longer intensive care unit stay and overall hospitalization days. The TA levels usually peaked 5-10 days after disease onset. Twenty-one pneumococcal isolates were recovered from the patients and serotyping was determined in 11 isolates: 10 serotype 19A and 1 serotype 3. TA determination not only helps to diagnose Sp-HUS but also is a predictor for IPD severity. Among hospitalized patients with severe pneumococcal disease, the peak of TA level usually appeared 5-10 days after disease onset.

Myeloid sarcoma is associated with poor clinical outcome in pediatric patients with acute myeloid leukemia.

The impact of myeloid sarcoma (MS) on clinical outcome of pediatric acute myeloid leukemia (AML) patients remains controversial. Moreover, little is known about the role of stem cell transplantation (SCT) in such patients. Clinical data of patients with AML under 18years of age were retrieved from the TARGET dataset. We analyzed the prevalence, clinical profile, molecular characteristics, and prognosis of MS in these patients. Among 884 pediatric patients with AML, the frequency of MS was 12.3%. Pediatric AML with MS was associated with age under 1-year, abnormal cytogenetics, and KMT2A rearrangement. Moreover, MS was associated with a low complete remission rate, high induction death, poor 5-year EFS, and OS. KMT2A rearrangement had a negative impact on clinical outcome in AML patients with MS. In addition, SCT had no significant effect on the survival of AML patients with MS. Multivariate analysis revealed that MS was an unfavorable prognostic factor in pediatric AML in terms of EFS (Hazard ratio 1.670, P < 0.001) and OS (Hazard ratio 1.623, P = 0.004). The presence of MS at diagnosis of pediatric AML is associated with poor clinical outcomes, particularly when associated with KMT2A rearrangements. Moreover, pediatric patients with AML and MS may not benefit from SCT.