Dabigatran for Treatment and Secondary Prevention of Venous Thromboembolism in Pediatric Congenital Heart Disease.

Background: Congenital heart disease (CHD) is common in children and associated with greater risk of thrombotic complications. Management of these complications with standard-of-care treatment is suboptimal for these children.

Methods And Results: The effectiveness and safety of dabigatran were demonstrated in pivotal pediatric studies for the treatment of acute venous thromboembolism (VTE; NCT01895777) and secondary VTE prevention (NCT02197416).

Safety and effectiveness of recombinant factor XIII-A in congenital factor XIII deficiency: Real-world evidence.

Background: Regular factor XIII (FXIII) prophylaxis is standard treatment for congenital FXIII A-subunit deficiency (FXIII-A CD). Recombinant factor XIII-A (rFXIII-A) was extensively evaluated in the mentor trials.

Objective: To assess real-world safety and treatment effectiveness of rFXIII-A prophylaxis from the mentor 6 trial.

The impact of extended half-life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B.

Background: Recombinant factors VIII and IX Fc (rFVIIIFc/rFIXFc) were the only available extended half-life (EHL) products in Canada during 2016 to 2018.

Objectives: To evaluate if patient-reported outcome measures (PROMs) improved in Canadian persons with hemophilia who switched from standard half-life (SHL) to EHL products (rFVIIIFc/rFIXFc).

Patients/methods: This prospective cohort study enrolled persons with moderate or severe hemophilia aged ≥6 years who switched to rFVIIIFc/rFIXFc (2016-2018) and those who remained on SHL.

Standardized Feeding Protocol Improves Delivery and Acceptance of Enteral Nutrition in Children Immediately After Liver Transplantation.

Delivery of adequate nutrition after liver transplantation (LT) surgery is an important goal of postoperative care. Existing guidelines recommend early enteral nutrition after abdominal surgery and in the child who is critically ill but data on nutritional interventions after LT in children are sparse. We evaluated the impact of a standardized postoperative feeding protocol on enteral nutrition delivery in children after LT.

Risk Factors for Mortality and Circulatory Outcome Among Neonates Prenatally Diagnosed With Ebstein Anomaly or Tricuspid Valve Dysplasia: A Multicenter Study.

Background In a recent multicenter study of perinatal outcome in fetuses with Ebstein anomaly or tricuspid valve dysplasia, we found that one third of live-born patients died before hospital discharge. We sought to further describe postnatal management strategies and to define risk factors for neonatal mortality and circulatory outcome at discharge. Methods and Results This 23-center, retrospective study from 2005 to 2011 included 243 fetuses with Ebstein anomaly or tricuspid valve dysplasia.

Hematologic concerns in extracorporeal membrane oxygenation.

This ISTH "State of the Art" review aims to critically evaluate the hematologic considerations and complications in extracorporeal membrane oxygenation (ECMO). ECMO is experiencing a rapid increase in clinical use, but many questions remain unanswered. The existing literature does not address or explicitly state many pertinent details that may influence hematologic complications and, ultimately, patient outcomes.

Mass deworming for improving health and cognition of children in endemic helminth areas: A systematic review and individual participant data network meta-analysis.

Background: Soil transmitted (or intestinal) helminths and schistosomes affect millions of children worldwide.

Objectives: To use individual participant data network meta-analysis (NMA) to explore the effects of different types and frequency of deworming drugs on anaemia, cognition and growth across potential effect modifiers.

Search Methods: We developed a search strategy with an information scientist to search MEDLINE, CINAHL, LILACS, Embase, the Cochrane Library, Econlit, Internet Documents in Economics Access Service (IDEAS), Public Affairs Information Service (PAIS), Social Services Abstracts, Global Health CABI and CAB Abstracts up to March 27, 2018.

Fixed doses of N8-GP prophylaxis maintain moderate-to-mild factor VIII levels in the majority of patients with severe hemophilia A.

Background: N8-GP is an extended half-life recombinant factor VIII developed for prophylaxis and treatment of bleeds in patients with hemophilia A.

Objective: To assess pharmacokinetic (PK) characteristics of N8-GP in previously treated patients with severe hemophilia A, model the time spent at hemophilia thresholds of ≥1 and ≤5 IU/dL (moderate) or >5 IU/dL (mild) FVIII levels during N8-GP prophylaxis, and investigate the relationship between N8-GP half-life and von Willebrand factor (vWF).

Methods: PK assessments were obtained from patients with severe hemophilia A (FVIII < 1 IU/dL) participating in 4 clinical trials: pathfinder 1 (20-60 years); pathfinder 2 (12-17 and ≥18 years); pathfinder 5 (0-11 years), and pathfinder 7 (25-71 years).

Repetitive transcranial magnetic stimulation for the treatment of drug-resistant epilepsy: A systematic review and individual participant data meta-analysis of real-world evidence.

Objective: To perform a systematic review and meta-analysis of real-world evidence for the use of low-frequency repetitive transcranial magnetic stimulation (rTMS) in the treatment of drug-resistant epilepsy.

Methods: We systematically searched PubMed, Scopus, Medline, and clinicaltrials.gov for all relevant articles.

Progression of Friedreich ataxia: quantitative characterization over 5 years.

Objective: Friedreich ataxia (FRDA) is a progressive neurodegenerative disorder of adults and children. This study analyzed neurological outcomes and changes to identify predictors of progression and generate power calculations for clinical trials.

Methods: Eight hundred and twelve subjects in a natural history study were evaluated annually across 12 sites using the Friedreich Ataxia Rating Scale (FARS), 9-Hole Peg Test, Timed 25-Foot Walk, visual acuity tests, self-reported surveys and disability scales.