scholarly journals Rates of Laboratory Adverse Events By Chemotherapy Course for Pediatric Acute Leukemia Patients within the Leukemia Electronic Abstraction of Records Network (LEARN)

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 333-333
Author(s):  
Tamara P. Miller ◽  
Kelly D Getz ◽  
Biniyam Demissei ◽  
Karen R. Rabin ◽  
Marla Daves ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Adverse Events ◽  
Research Funding ◽  
Risk Classification ◽  
Laboratory Result ◽  
Children's Hospital ◽  
Enrollment Status ◽  
Laboratory Results ◽  
Children’S Hospital ◽  
Chart Abstraction

Introduction: Adverse events (AEs) are often under- or misreported on clinical trials, leading to an incomplete understanding of therapy-associated toxicities. We previously demonstrated that laboratory result data can be extracted from the electronic health record (EHR), cleaned, and processed to ascertain laboratory AEs with high accuracy compared to gold standard chart abstraction at a single institution. We sought to employ this methodology at three large children's hospitals to demonstrate scalability and describe laboratory AE rates during therapy for pediatric acute myeloid and acute lymphoblastic leukemia (AML, ALL). Methods: The Leukemia Electronic Abstraction of Records Network (LEARN) comprises data from patients with AML and ALL treated at the Children's Hospital of Philadelphia (CHOP) from 2006 to 2014, Children's Healthcare of Atlanta from 2010 to 2018, and Texas Children's Hospital from 2008 to 2014. Risk classification, clinical trial enrollment status, and chemotherapy course start and end dates for all patients were collected via manual chart abstraction. After manual input of medical record numbers and course dates, a package we developed in the R programming language (ExtractEHR) was employed to extract laboratory results from the Epic EHR at each site. De-identified laboratory result data were cleaned centrally at CHOP to remove falsely abnormal results, including hemolyzed specimens and results that normalized within 1 hour for electrolytes and hematology tests and within 12 hours for liver function tests (LFTs). Laboratory results were processed and graded according to Common Terminology Criteria for Adverse Events (CTCAE) v5 definitions for 22 AEs. The highest grade of each AE in each course was calculated and results were tabulated by chemotherapy course for ALL and AML separately. Only chemotherapy courses with complete start and end dates were included; courses that were ongoing at time of abstraction were excluded. Highest grades for each AE over all chemotherapy courses combined were stratified by leukemia risk classification and trial enrollment status and were compared using chi square tests. CTCAE grade 5 definitions are not numerically based, so were excluded. Results: Laboratory result data on 1687 patients (AML: 282, ALL: 1405) who experienced 6652 courses (AML: 909, ALL: 5743) were extracted, processed and graded. For the purposes of these results, AEs indicate grades 3-4. More than 95% of AML courses had anemia, neutropenia and thrombocytopenia (Table). AML patients experienced the most laboratory AEs during Induction I and Intensification III (prevalence of grade 3-4 AEs was ≥10% for 10 AEs evaluated). Apart from hematologic AEs, hypokalemia (17.9-44.9% of courses) and increased LFTs (7.7-18.6% of courses) were the most common AEs in AML courses. While AML patients experienced higher rates of hematologic AEs, ALL patients had higher rates of electrolyte and LFT AEs over all courses. These AEs were distributed across all courses; in each course there were 7-9 AEs with a prevalence of ≥10%. Increased alanine aminotransferase was common in every ALL course (17.4-66.2%). For both for AML and ALL, AE rates were similar among patients enrolled on trials and those who were not. AE rates were similar for all AML risk classifications. In contrast, for ALL there were significantly higher rates (p<0.05) among intermediate/high risk compared to standard risk patients for 17 AEs, including AEs in all laboratory categories. Conclusions: The ExtractEHR tool is scalable for extracting laboratory result data from the EHR at multiple hospitals. These data can be processed centrally using a series of electronic code to describe comprehensive laboratory AE rates during chemotherapy, which does not otherwise exist in the literature. The lack of difference by trial enrollment status indicates that these results apply to all patients with AML and ALL. These data therefore describe expected AE rates for patients during each chemotherapy phase that can be used to provide clinical guidance and serve as a baseline comparison for experimental agents in clinical trials. These data and LEARN provide a granular resource for clinical epidemiology research focused on evaluating factors associated with incidence and resolution of laboratory AEs. Efforts are ongoing to incorporate medication and outcome data to investigate the impact of these AEs on clinical experience and outcomes. Disclosures Fisher: Pfizer: Research Funding; Astellas: Other: Data Safety Monitoring Board Chair for an antifungal study; Merck: Research Funding.

scholarly journals A Retrospective Review of Hospital-Acquired Venous Thromboembolism at a Large Pediatric Tertiary Care Center

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3471-3471
Author(s):  
Marissa A. Just ◽  
Joanna Robles ◽  
Karan R. Kumar ◽  
Andrew Yazman ◽  
Jennifer A. Rothman ◽  
...  
Keyword(s):  
Retrospective Review ◽  
Research Funding ◽  
Pediatric Patients ◽  
Central Venous ◽  
Vte Prophylaxis ◽  
Children's Hospital ◽  
Congenital Cardiac Disease ◽  
Children’S Hospital ◽  
Icd 10 ◽  
Hospital Acquired

Introduction: The incidence of venous thromboembolism (VTE) in hospitalized pediatric patients is increasing secondary to the growing medical complexity of pediatric patients and the increasing use of central venous catheters. Pediatric patients diagnosed with VTE have up to 2% mortality associated directly with their thromboses. While incidence, risk factor identification and preventive strategies are well established in hospitalized adults, this information is limited in the pediatric population. There are currently no standardized VTE risk screening tools or thromboprophylaxis guidelines for children at Duke Children's Hospital. The incidence of hospital acquired VTE (HA-VTE), as well as their associated risk factors were investigated in a retrospective review. Methods: Medical records of pediatric patients hospitalized at Duke Children's Hospital during June 2018 through November 2018 were reviewed. The EPIC SlicerDicer tool was used to identify patients with ICD-10 diagnoses codes related to thrombosis or treated with anticoagulants. Included patients were diagnosed with HA-VTE during their hospitalization or within 14 days of discharge. Data collected included demographics, thrombosis characteristics, family history, mobility, and acute or chronic co-morbid conditions. The characteristics of the study population were described by median (with 25th and 75th percentiles) for continuous variables and frequencies (with percentages) for binary or categorical variables. Results: Out of 4,176 total pediatric admissions to all units of Duke Children's Hospital (ages 0-18.99 years) during the inclusion timeframe, 33 VTE events were identified. The incidence of VTE events per 1000 patient days was 0.98. The complete patient and VTE event characteristics are listed in Tables 1 and 2. The median age of patients with VTE events was 0.4 years. Of the identified cohort, 73% had an associated central venous line (CVL). Neonates with congenital cardiac disease comprised the majority of the cohort. Other common patient characteristics observed in this cohort included impaired mobility, recent major surgery, and recent mechanical ventilation. Of the 33 VTE diagnoses, 70% received therapeutic anticoagulation with enoxaparin or unfractionated heparin. Only 2 patients (8%) received prophylactic anticoagulation prior to their diagnosis of VTE. Conclusions: The retrospective review of HA-VTE events at Duke Children's Hospital identified that the majority of the events occurred in neonates with congenital cardiac disease and the presence of CVLs. It was also noted that there was no standardization among the use of anticoagulation agents that were initiated for treatment of VTE. Furthermore, few patients received VTE prophylaxis during the hospitalization. A limitation of this review was that it was retrospective and the documentation of family history of thrombosis was inconsistent. It is also possible that several VTE events were missed due to inadequate ICD-10 coding. Based on the results of this review, there is a need to implement a risk stratification tool and develop standardized recommendations of VTE prophylaxis and treatments for pediatric patients admitted to Duke Children's Hospital. There is an additional quality improvement phase of this project and the goal is to implement a risk calculator that is based on information learned from the retrospective review. Ultimately, this risk calculator will help to decrease the incidence of VTE events at Duke Children's Hospital. Disclosures Rothman: Agios: Honoraria, Research Funding; Pfizer: Consultancy, Honoraria, Research Funding; Novartis: Honoraria, Research Funding.

A Multicenter, Phase IV Observational Study Of Ofatumumab In Chronic Lymphocytic Leukemia (CLL): A European Research Initiative On CLL (ERIC) Study

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 1645-1645 ◽  
Author(s):  
Carol Moreno ◽  
Marco Montillo ◽  
Panayiotidis Panayiotis ◽  
Adrian Bloor ◽  
Jehan Dupuis ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Adverse Events ◽  
Board Of Directors ◽  
Safety Profile ◽  
Research Funding ◽  
Response Rate ◽  
Lymphocytic Leukemia ◽  
Hematologic Toxicity ◽  
Phase Ii Trials ◽  
Advisory Committees

Abstract Background Ofatumumab was given a conditional approval in the EU on April 2010 for the treatment of CLL refractory to fludarabine (F-ref) and alemtuzumab (A-ref), encouraging the retrieval of further data in patients treated in a “daily life” setting and to investigate treatment safety. Aims The main objective of this study was to obtain information on the safety profile of ofatumumab given outside clinical trials in patients with previously treated CLL. The secondary endpoints were efficacy, progression-free-survival (PFS), and overall survival (OS). Methods This was an observational, retrospective study. Patients were eligible regardless of prior treatments or disease status and provided they had not been included in ofatumumab clinical trials. Data on patients’ characteristics at diagnosis, prior treatment, adverse events response rate, PFS and OS were recorded. Results One-hundred and twenty patients were screened of which 103 from 25 centers in 10 European countries were eventually eligible for the study. There were 71 males; median age at initiation of ofatumumab was 64 years (range, 38-84); 66% patients were in advanced clinical stage (Rai III-IV/Binet C) and 33 patients presented bulky lymphadenopathy. Number of prior lines of therapy was 4 (range, 1-13). 94% had received prior F-based therapy, 54% received A-based therapy and 51% received both. Eighty-one percent had been previously exposed to rituximab-combination regimens. Fifty-four percent were F-ref, 70% A-ref and 41% were both F- and A-refractory. Cytogenetics within 3 months prior therapy was available in 52 patients of which 34 presented abnormalities (11 patients: 17p-; 9 patients: 11q-; 2 patients: 13q-; 1 patient: trisomy 12; 11 patients: two or more abnormalities including 17p- or 11q-). Forty-two of 50 patients showed unmutated IGHV genes. The median number of cycles of ofatumumab given was 9 (range, 0-16) and the median percentage of given/planned cycles was 83.3% (range, 0-133). In most patients the treatment dose and schedule were as follows: 300 mg 1st infusion followed by 2000 mg for subsequent infusions (8 weekly followed by 4 doses monthly). One hundred and sixty-one adverse events were reported in 68 patients, with 28 (17%) of them being considered as ofatumumab-related. Infusion related-reactions occurred in 19 (28%) patients (III-IV: 6%). Neutropenia was reported in 26% (III-IV: 19%), thrombocytopenia in 15% (III-IV: 12%) and anemia in 15% (III-IV: 7%). The non-hematological adverse events, included infection 44% (III-IV: 36%), fatigue 10% (III-IV: 4%), fever 10% (III-IV: 6%), rash 10% (III-IV: 3%), cough 7% (III-IV: 1%), diarrhea 6% (grade III-IV: 0%) and nausea 1% (III-IV: 0%). Hematologic toxicity correlated with the number of prior lines of therapy. Autoimmune hemolytic anemia and Richter syndrome were reported in one patient each. Two heavily pre-treated patients (5 and 6 prior lines of therapy, respectively) developed progressive multifocal leukoencephalopathy. The overall response rate (ORR) was 23% and the median PFS and OS were 5 and 12 months, respectively. The main causes of death were disease progression (61%) and infection (28%). Conclusions The safety profile of ofatumumab given outside clinical trials to patients with poor-prognosis and heavily pre-treated CLL was consistent with that observed in clinical trials. Although not unexpectedly the ORR was lower in this study, PFS and OS were in line with those reported in phase II trials. Disclosures: Montillo: Roche: Honoraria, Membership on an entity’s Board of Directors or advisory committees, Research Funding; GSK: Honoraria, Membership on an entity’s Board of Directors or advisory committees, Research Funding; Mundipharma: Honoraria, Membership on an entity’s Board of Directors or advisory committees, Research Funding; Janssen: Honoraria, Membership on an entity’s Board of Directors or advisory committees, Research Funding. Bloor:GSK: Consultancy, Honoraria, Paid speaker Other. Schuh:GSK: Honoraria; Celgene: Honoraria; Mundipharma: Honoraria. Geisler:Roche: Consultancy; GSK: Consultancy. Hillmen:GlaxoSmithKline: Honoraria, Research Funding. Stilgenbauer:GSK: Honoraria, support Other. Smolej:GSK: Consultancy, Honoraria, travel grants Other. Jaeger:GSK: Honoraria, Research Funding. Leblond:Roche : Consultancy, Honoraria, Membership on an entity’s Board of Directors or advisory committees, Speakers Bureau; Janssen: Honoraria, Membership on an entity’s Board of Directors or advisory committees; Mundipharma: Honoraria, Membership on an entity’s Board of Directors or advisory committees, Speakers Bureau. Kimby:Roche: Consultancy, Honoraria, Research Funding; celgene: Consultancy, Honoraria, Research Funding; Teva: Consultancy, Honoraria, Research Funding; Emergent BioSolutions: Consultancy, Honoraria, Research Funding; Gilead Sciences: Consultancy, Honoraria, Research Funding; Jansen: Consultancy, Honoraria, Research Funding; Pharmacyclics: Consultancy, Honoraria, Research Funding.

scholarly journals Development and Testing of a Lymphoma Clinical Trials Specific Frailty Index: A Secondary Analysis of the LY.12 Clinical Trial

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4076-4076
Author(s):  
Abi Vijenthira ◽  
Xinzhi Li ◽  
Michael Crump ◽  
Annette E. Hay ◽  
Lois E. Shepherd ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Adverse Events ◽  
Board Of Directors ◽  
Research Funding ◽  
Frailty Index ◽  
Secondary Analysis ◽  
Advisory Committees ◽  
Frail Patients ◽  
The Impact

Abstract Background: Frailty is common in older patients with lymphoma. However, it remains unknown whether frailty is prevalent in patients included in clinical trials of lymphoma, as those with frailty may meet inclusion criteria of a trial which do not include functional information beyond performance status (PS). Understanding the prevalence and impact of frailty in clinical trials is important to direct future stratification criteria, as well as to have robust data to counsel frail patients on their potential outcomes. Methods: We conducted a secondary analysis using data from the phase III LY.12 clinical trial in which patients with relapsed aggressive non-Hodgkin lymphoma were randomized to gemcitabine-dexamethasone-cisplatin or dexamethasone-high dose cytarabine-cisplatin chemotherapy prior to autologous stem cell transplant. The primary objective of our study was to construct a lymphoma clinical trials specific frailty index (FI) using previously described methods (Searle. BMC Geriatr. 2008;8:24). Secondary objectives were to describe the association of frailty (binary variable) with overall survival (OS), event-free survival (EFS), hospitalization, adverse events (AE), serious adverse events (SAE), and proceeding to transplant, and to describe the association of frailty with these outcomes, controlling for important covariates (age, sex, immunophenotype, revised international prognostic index score (rIPI), Eastern Cooperative Oncology Group (ECOG) PS, stage, and response to previous chemotherapy). Results: 619 patients in the LY12 trial were used to construct the frailty index (Table 1). Using a binary cut-off for frailty (<0.2), 15% (N=93) of patients were classified as frail. There were no differences in age or sex between frail and non-frail patients; however they differed in terms of other lymphoma-related characteristics (Table 2). Frailty was strongly associated with OS (HR 2.012, 95% CI 1.57-2.58), EFS (HR 1.94, 95% CI 1.53-2.46), frequency of the worst overall Grade >3 AE (OR 2.65 (15% vs. 6%), p=0.003), and likelihood of proceeding to ASCT (OR 0.26, 95% CI 0.15-0.43), but not hospitalization (OR 1.52, 95% CI 0.97-2.40) or SAE (6% vs. 4%, p=0.3). In multivariable analysis, frailty was not significantly associated with OS, EFS, likelihood of proceeding to ASCT, nor hospitalization (Table 3), though there was a trend to significance for ASCT. However, rIPI remained significantly associated with OS and EFS, ECOG remained significantly associated with OS (Table 3) Conclusion: A potentially broadly applicable lymphoma clinical trials specific FI was constructed through secondary analysis of LY12 data. 15% of patients were classified as frail. Frailty was significantly associated with OS, EFS, frequency of grade >3 AE and likelihood of proceeding to transplant. However, this relationship no longer was significant when controlling for lymphoma-related prognostic variables, suggesting that the impact of poor prognostic features of lymphoma supersede the impact of frailty alone in this younger clinical trial population. Interestingly, rIPI and ECOG demonstrated their value as simple predictors that are highly associated with OS and/or EFS even when controlling for other important covariates including frailty. These findings require further testing in an external data set, and would be particularly valuable to test in an older population. Calibration of the FI against clinical frailty assessment (e.g. Clinical Frailty Scale, Comprehensive Geriatric Assessment) would also be meaningful to confirm its ability to classify frail versus non-frail patients. Figure 1 Figure 1. Disclosures Crump: Epizyme: Research Funding; Roche: Research Funding; Kyte/Gilead: Membership on an entity's Board of Directors or advisory committees; Novartis: Membership on an entity's Board of Directors or advisory committees. Hay: Merck: Research Funding; Roche: Research Funding; Abbvie: Research Funding; Amgen: Research Funding; Karyopharm: Research Funding; Seattle Genetics: Research Funding. Prica: Astra-Zeneca: Honoraria; Kite Gilead: Honoraria.

scholarly journals Can Clinical Symptoms and Laboratory Results Predict CT Abnormality? Initial Findings Using Novel Machine Learning Techniques in Children With COVID-19 Infections

2021 ◽  
Vol 8 ◽  
Author(s):  
Huijing Ma ◽  
Qinghao Ye ◽  
Weiping Ding ◽  
Yinghui Jiang ◽  
Minhao Wang ◽  
...  
Keyword(s):  
Machine Learning ◽  
Clinical Data ◽  
Pediatric Patients ◽  
Clinical Symptoms ◽  
Chest Ct ◽  
Rt Pcr ◽  
Health Crisis ◽  
Children's Hospital ◽  
Laboratory Results ◽  
Children’S Hospital

The rapid spread of coronavirus 2019 disease (COVID-19) has manifested a global public health crisis, and chest CT has been proven to be a powerful tool for screening, triage, evaluation and prognosis in COVID-19 patients. However, CT is not only costly but also associated with an increased incidence of cancer, in particular for children. This study will question whether clinical symptoms and laboratory results can predict the CT outcomes for the pediatric patients with positive RT-PCR testing results in order to determine the necessity of CT for such a vulnerable group. Clinical data were collected from 244 consecutive pediatric patients (16 years of age and under) treated at Wuhan Children's Hospital with positive RT-PCR testing, and the chest CT were performed within 3 days of clinical data collection, from January 21 to March 8, 2020. This study was approved by the local ethics committee of Wuhan Children's Hospital. Advanced decision tree based machine learning models were developed for the prediction of CT outcomes. Results have shown that age, lymphocyte, neutrophils, ferritin and C-reactive protein are the most related clinical indicators for predicting CT outcomes for pediatric patients with positive RT-PCR testing. Our decision support system has managed to achieve an AUC of 0.84 with 0.82 accuracy and 0.84 sensitivity for predicting CT outcomes. Our model can effectively predict CT outcomes, and our findings have indicated that the use of CT should be reconsidered for pediatric patients, as it may not be indispensable.

scholarly journals Cardiopulmonary Fitness and Clinical Outcomes in Adults Followed in the Cooperative Study for Sickle Cell Disease

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 1304-1304
Author(s):  
Sherif M. Badawy ◽  
Amanda B. Payne ◽  
Mark J. Rodeghier ◽  
Robert I. Liem
Keyword(s):  
Sickle Cell Disease ◽  
Clinical Outcomes ◽  
Exercise Testing ◽  
Research Funding ◽  
Cell Disease ◽  
Cardiopulmonary Fitness ◽  
Children's Hospital ◽  
Hospitalization Rates ◽  
Children’S Hospital ◽  
The Relationship

Abstract Introduction: Cardiopulmonary fitness is significantly reduced among individuals with sickle cell disease (SCD). Cardiopulmonary fitness is also an important predictor of morbidity and all-cause mortality in the general population. However, the relationship between fitness and clinical outcomes in SCD has not been well studied. The objectives of this analysis were to: 1) determine the factors associated with fitness in a cohort of adults with SCD, and 2) evaluate the relationship of fitness to hospitalization for pain and acute chest syndrome (ACS) and overall mortality. We hypothesized that clinical factors such as age, sex, hemoglobin, SCD genotype and cardiopulmonary disease significantly affect fitness, and that poor fitness is a predictor of more frequent hospitalizations for pain and ACS and higher mortality in adults with SCD. Methods: A cohort of adults with SCD was constructed from participants enrolled in phase 2 of the Cooperative Study of Sickle Cell Disease (CSSCD) who underwent exercise testing (modified Balke treadmill protocol). Primary measure for fitness was total treadmill duration. Retrospective pain or ACS hospitalization rates were calculated using events in the 3 years prior to exercise testing. Mortality and prospective hospitalization rates for pain and ACS were calculated using events after exercise testing with a minimum 6 month follow-up. Results of pulmonary function testing (PFT), echocardiography, and laboratory testing within 3 years of exercise testing were included in our analysis. Standard descriptive analyses were performed (SPSS V24). Multivariable negative binomial and Cox proportional hazards models were constructed to evaluate the relationship of fitness to ACS and pain hospitalization rates and mortality, respectively. Multivariable linear models were constructed to determine factors associated with fitness. Results: A total of 223 participants had valid exercise testing data (64% female, 70% hemoglobin SS or S/b0 thalassemia, mean age 43.3 ± 7.5 years, mean hemoglobin 9.1 ± 2.2 g/dl, mean follow-up 2.7 ± 0.7 years after exercise testing). Participants completed a mean of 11.6 ± 5.2 min on the treadmill, with 87% completing ≥ 3 stages but only 17% completing all 10 stages. We categorized fitness into tertiles of treadmill duration (5.7 vs. 11.8 vs. 18.1 min, p < 0.001). Age (45.2 vs. 43.1 vs. 41.3 years, p = 0.007), baseline hemoglobin (8.5 vs. 9 vs. 9.8 g/dl, p = 0.003), as well as the proportion of females (77 vs. 71 vs. 40%, p < 0.001) and participants with abnormal PFT (58 vs. 35 vs. 39%, p = 0.008), differed significantly across fitness tertiles. Pain or ACS hospitalization rates during the 3 years prior to exercise testing were not significantly different across fitness tertiles. Using multivariable linear regression, male sex (β = 3.1, p < 0.001), lower age at exercise testing (β = -0.14, p = 0.003), and higher hemoglobin (β = 0.44, p = 0.049) were independently associated with higher fitness, with abnormal PFT trending toward significance (β = -1.28, p = 0.07). In this model, genotype, tricuspid regurgitant jet velocity (TRJV) ≥ 2.5 m/s, and pain and ACS hospitalization rates prior to exercise testing were not significantly associated with fitness. Using a negative binomial regression model, we found that fitness did not predict future pain or ACS episodes after adjustment for age, sex, genotype, hemoglobin and TRJV. Fitness also did not predict survival in our cohort (hazard ratio, 0.97; 95% CI [0.84, 1.13], p = 0.71), in which death was reported in only 9 participants. In our Cox regression model, male sex (HR 7.1; 95% CI [1.3, 38.9]; p = 0.02) and lower hemoglobin (HR 0.56; 95% CI [0.36, 0.88]; p = 0.01) were independent predictors of death, but age at exercise testing, abnormal PFT and TRJV ≥ 2.5 m/s were not. Conclusions: In adults with SCD, lower fitness is significantly associated with female sex, older age, lower hemoglobin and abnormal PFT. Fitness did not predict survival or future pain or ACS events in the CSSCD. Given that cardiopulmonary fitness remains an important predictor of all-cause mortality in the general population, larger scale prospective studies in SCD are needed to evaluate the impact of regular exercise on improving fitness, quality of life, clinical outcomes and mortality in this population. Disclosures Badawy: Ann & Robert H. Lurie Children's Hospital of Chicago: Employment; Ann & Robert H. Lurie Children's Hospital of Chicago: Research Funding; Salveo Health Communications LLC: Consultancy. Payne:National Center on Birth Defects and Developmental Disabilities: Employment. Liem:Ann & Robert H. Lurie Children's Hospital of Chicago: Employment; Ann & Robert H. Lurie Children's Hospital of Chicago: Research Funding; National Institute of Health: Research Funding.

scholarly journals An Interview with Professor Florence Bourgeois: on Policy and Regulation of Clinical Trials

2021 ◽  
Author(s):  
Shejla Pollozi ◽  
Cindy Xie ◽  
Sumedha Sachar
Keyword(s):  
Clinical Trials ◽  
Health Informatics ◽  
Research Integrity ◽  
Regulatory Science ◽  
European Medicines Agency ◽  
Yale University ◽  
Children's Hospital ◽  
School Of Medicine ◽  
Children’S Hospital ◽  
Pediatric Populations

For more than 20 years, Dr. Florence Bourgeois, MD, MPH has held multiple positions at the intersection of pediatrics, therapeutics, regulatory science, health informatics and research integrity. A graduate of Yale University, Harvard School of Public Health and Washington University School of Medicine in St. Louis, Dr. Bourgeois’ record transcends academia and medicine. In addition to being a faculty member at Harvard Medical School and the Division of Emergency Medicine and the Computational Health Informatics Program (CHIP) at Boston Children’s Hospital, she is the Director of PedRx, which aims to advance the development and evidence-based use of novel therapeutics for children globally. As the Scientific Director of the Boston Children’s Hospital Biobank, Dr. Bourgeois’ efforts focus on expanding physicians’ capacity to conduct research in pediatric populations. As the Co-Director of the Harvard-MIT Center for Regulatory Science, Dr. Bourgeois also spearheads cross-disciplinary collaborations between academia, biotechnology and pharmaceutical companies, and regulatory agencies. She is the recipient of an Innovation in Regulatory Science Award from the Burroughs Wellcome Fund and has served as an Expert Visitor to the European Medicines Agency to analyze the EU’s pediatric drug legislation. As part of this interview, we examine the relationship between clinical trials and drug development in pediatrics as well as their ethical and social impacts.

scholarly journals Safety and tolerability of Meningococcus B vaccine in patients with chronical medical conditions (CMC)

2019 ◽  
Vol 45 (1) ◽  
Author(s):  
L. Nicolosi ◽  
C. Rizzo ◽  
G. Castelli Gattinara ◽  
N. Mirante ◽  
E. Bellelli ◽  
...  
Keyword(s):  
High Risk ◽  
Adverse Events ◽  
Clinical Importance ◽  
European Medicines Agency ◽  
Vaccine Administration ◽  
Children's Hospital ◽  
Children’S Hospital ◽  
Control And Prevention ◽  
Low Incidence ◽  
High Risk Children

Abstract Background Invasive meningococcal disease is a serious global health threat in the world; in 2016, the European Centre for Disease Control and Prevention reported 3280 confirmed cases (including 304 deaths) of Invasive Meningococcal Diseases in Europe. In Italy, in 2017 were reported 200 cases 41% of which due to menB serogroup. From January 2013 the European Medicines Agency (EMA) has authorized the marketing of the meningococcal B vaccine 4CMenB. Methods The study aimed to evaluate and complement the safety profile of 4CMenB in high risk children accessing the vaccine service of the Bambino Gesù Children’s Hospital. All individuals aged six weeks or more receiving the meningococcal 4CMenB (Bexsero®) vaccine that approached the vaccine Centre at the Bambino Gesù Children’s Hospital in Rome, were asked to participate. All parents or caregivers of vaccinated individuals in the study period, were recruited and requested to answer to a questionnaire on adverse events following immunization (AEFI) observed after 7 days, starting from the date of vaccination. Results During the study period (October 2016–October 2017), we collected 157 completed questionnaires (out of 200 distributed). Of those 132 were first doses and 25 were booster administered doses. The median age of the study population was 4.5 years (range 0.29 to 26.8 years), the majority of subjects were high-risk individuals (64%) with chronic health conditions. Overall, 311 adverse events were reported in the 7 days after vaccine administration. In particular 147 events (47%) after administration of first dose and 58 (19%) after the booster doses. A large majority of those events, were of little clinical importance and concentrated in the 24 h after vaccine administration. No hospitalizations or Emergency Department access were reported. Conclusions Results of our study demonstrated that the Bexsero® vaccine is almost well tolerated, with a low incidence of severe AEFIs. Our results also shown that the occurrence of AEFIs is similar within healthy and high risk children.

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