Objectives: To test feasibility of a randomized controlled trial (RCT) with an endpoint of time at goal anticoagulation in children on extracorporeal membrane oxygenation (ECMO) randomized to receive bivalirudin vs. unfractionated heparin.
Design: Open-label pilot RCT (NCT03318393) carried out 2018-2021.
Common variable immunodeficiency (CVID) is a disorder characterized by hypogammaglobulinemia resulting in recurrent infections. While autoimmune disorders are common in patients with CVID, no association has been reported between CVID and immune thrombotic thrombocytopenic purpura (iTTP), a disorder most often caused by autoantibodies that compromise the activity of the enzyme ADAMTS13. Reduced ADAMTS13 activity results in the accumulation of large von Willebrand factor multimers that can consume platelets and cause microvascular thrombosis and organ injury, ultimately resulting in mortality in most cases of untreated iTTP.
View Article and Find Full Text PDFObjective: Clinical research networks facilitate collaborative research, but data sharing remains a common barrier.
Materials And Methods: The TriNetX platform provides real-time access to electronic health record (EHR)-derived, anonymized data from 173 healthcare organizations (HCOs) and tools for queries and analysis. In 2022, 4 pediatric HCOs worked with TriNetX leadership to found the Pediatric Collaboratory Network (PCN), facilitated via a multi-institutional data-use agreement (DUA).
Our study aimed to evaluate the presence, clinical associations, and potential mechanistic roles of non-criteria antiphospholipid antibodies (aPL) and circulating calprotectin, a highly stable marker of neutrophil extracellular trap release (NETosis), in pediatric APS patients. We found that 79% of pediatric APS patients had at least one non-criteria aPL at moderate-to-high titer. Univariate logistic regression demonstrated that positive anti-beta-2 glycoprotein I domain 1 (anti-D1) IgG (p = 0.
View Article and Find Full Text PDFIntroduction: Women-identifying emergency physicians face gender-based discrimination throughout their careers. The purpose of this study was to explore emergency physician's perceptions and experiences of gender equity in emergency medicine.
Methods: We conducted a secondary analysis of data from a previously conducted survey of Canadian emergency physicians on barriers to gender equity in emergency medicine.
We investigated whether pediatric patients with overweight and obesity are more likely to have dyspnea compared with those who are non-overweight. We collected de-identified data from TriNetX, a global federated multicenter research database, using both the UT Southwestern Medical Center and multinational Research Networks. Our analysis focused on patients aged 8-12 years.
View Article and Find Full Text PDFRes Pract Thromb Haemost
November 2023
Background: Clinical trials have shown success in bleed prevention with emicizumab, but real-world data on the effectiveness of emicizumab in preventing serious bleeds in the pediatric population are lacking.
Objectives: To report real-world data on the effectiveness of Emicizumab in pediatric persons with hemophilia A.
Methods: We completed a retrospective chart review of 37 pediatric male patients aged ≤18 years on emicizumab prophylaxis for a median duration of 30.
Despite the growing number of pediatric antithrombotic clinical trials, standardized safety and efficacy outcome definitions for pediatric venous thromboembolism (VTE) clinical trials have not been updated since 2011. Many recent trials have adapted the recommended definitions, leading to heterogeneity in outcomes and limiting our ability to compare studies. The International Society on Thrombosis and Haemostasis Scientific and Standardization Subcommittee (SSC) on Pediatric and Neonatal Thrombosis and Hemostasis organized a Task Force to update the efficacy and safety outcome definitions for pediatric VTE clinical trials.
View Article and Find Full Text PDFAm J Health Syst Pharm
January 2023
Purpose: In the pediatric population, warfarin remains the recommended oral anticoagulant for valvular heart disease. Warfarin carries a risk of bleeding complications that can manifest as heavy menstrual bleeding (HMB) in postmenarchal adolescent females. As a result, these patients may be started on hormonal therapies, such as norethindrone, to suppress menstruation.
View Article and Find Full Text PDFObjectives: Pain and bleeding complicate 30% of pregnancies, raising concerns for viability. The objective is to evaluate the diagnostic characteristics of a single serum progesterone level in assessing pregnancy viability among symptomatic patients.
Methods: We conducted a predefined literature search in MEDLINE, Embase (OVID), CINAHL and Cochrane databases for studies that enrolled patients in first trimester with miscarriage symptoms, measured progesterone and reported pregnancy viability, from inception to July 2020.
Study Objective: To evaluate the use of intrauterine devices (IUDs) in two young women's hematology clinics and compare adverse events in adolescents with and without inherited bleeding disorders (BDs) DESIGN: Retrospective multicenter cohort study from February 2014 through February 2020 SETTING: Young women's hematology clinics at Nationwide Children's Hospital in Columbus, Ohio, and Children's Medical Center in Dallas, Texas PARTICIPANTS: Female patients evaluated for heavy menstrual bleeding (HMB) who underwent IUD placement INTERVENTIONS AND MAIN OUTCOME MEASURES: Rates of IUD expulsion, malposition, and ongoing HMB requiring additional medical treatment RESULTS: We identified 43 patients with BDs and 35 patients without BDs who underwent placement of an IUD for HMB. The mean age was 14.9 years (range 11.
View Article and Find Full Text PDFCaring for children and adolescents with disorders of hemostasis and thrombosis (HAT) has become more specialized and requires a unique skill set that many providers are not able to obtain in standard pediatric hematology/oncology/bone marrow transplant fellowship training programs. The influx of numerous therapeutic advances and increasing medical complexity has expanded the need for experienced HAT providers and subspecialty collaboration in the inpatient setting due to the nuances in the management of patients with HAT complications and concerns. While there are data highlighting the benefits of an inpatient hemostasis, thrombosis, and anticoagulation management service in adult hospitals, there are limited pediatric data supporting such programs.
View Article and Find Full Text PDFBackground: Heavy menstrual bleeding (HMB) is a presenting symptom of an inherited bleeding disorder (BD) and results in hospitalizations, limitations of daily activities, and a reduction in quality of life. Adult women with BD report a sense of stigma, difficulties understanding their bleeding, and challenges with diagnostic labels. The experiences of adolescents with HMB and BD are unknown despite advances in medical management through the rapidly growing network of young women's hematology programs.
View Article and Find Full Text PDFBackground: Evidence regarding the safety and efficacy of anticoagulant thromboprophylaxis among pediatric patients hospitalized for coronavirus disease 2019 (COVID-19) is limited. We sought to evaluate safety, dose-finding, and preliminary efficacy of twice-daily enoxaparin as primary thromboprophylaxis among children hospitalized for symptomatic COVID-19, including primary respiratory infection and multisystem inflammatory syndrome in children (MISC).
Methods: We performed a phase 2, multicenter, prospective, open-label, single-arm clinical trial of twice-daily enoxaparin (initial dose: 0.
Objectives: Gender inequities are deeply rooted in our society and have significant negative consequences. Female physicians experience numerous gender-related inequities (e.g.
View Article and Find Full Text PDFThis review focuses on significant advances in the field of pediatric hemostasis and thrombosis, with a focus on published studies within the past decade. The evaluation and management of patients with excessive bleeding remain cornerstones of consultative hematology. We will describe the development of validated bleeding assessment tools relevant to pediatric practice, laboratory advances in the evaluation of von Willebrand disease, and a shift in clinical practice regarding the interpretation of normal coagulation studies in patients with significant bleeding phenotypes.
View Article and Find Full Text PDFIron deficiency anemia is associated with heavy menstrual bleeding (HMB) and, by extension, a bleeding disorder (BD). It is unknown if iron deficiency without anemia is associated with a BD in adolescents. Moreover, the threshold of ferritin associated with fatigue in adolescents with HMB is unclear.
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