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Objective: To assess cognitive, behavioral, and adaptive functions in children and young adults with hemophilia treated according to contemporary standards of care., Study Design: Evolving Treatment of Hemophilia's Impact on Neurodevelopment, Intelligence, and Other Cognitive Functions (eTHINK) is a US-based, prospective, cross-sectional, observational study (September 2018 through October 2019). Males (aged 1-21 years) with hemophilia A or B of any severity, with or without inhibitors, were eligible. Participants underwent neurologic examinations and age-appropriate neuropsychological assessments, including standardized tests/ratings scales of early development, cognition, emotional/behavioral adjustment, and adaptive skills., Results: Five hundred and fifty-one males with hemophilia A (n = 433) or B (n = 101) were enrolled. Performance on cognitive tests was largely comparable with that of age-matched US population norms, although participants in certain age groups (4-5 and 10-21 years) performed worse on measures of attention and processing speed. Furthermore, adolescents and young adults and those with comorbid attention-deficit/hyperactivity disorder (ADHD; n = 64) reported more adaptive and executive function problems in daily life. Incidence of ADHD in adolescents (21%) was higher than expected in the general population., Conclusions: In general, males with hemophilia demonstrated age-appropriate intellectual, behavioral, and adaptive development. However, specific patient/age groups showed poorer attention performance and concerns for executive and adaptive development. This study established a normative data set for monitoring neurodevelopment in individuals with hemophilia and highlight the importance of screening and intervention for challenges with cognitive and adaptive skills in this population., Clinical Trial Registration: Evolving Treatment of Hemophilia's Impact on Neurodevelopment, Intelligence, and Other Cognitive Functions (eTHINK); NCT03660774; https://clinicaltrials.gov/ct2/show/NCT03660774., Competing Interests: Declaration of Competing Interest CM is a consultant for Novo Nordisk, coauthor of the eTHINK and paradigm study protocols, and site co-PI of the eTHINK study and has served as a speaker for Otsuka Pharmaceutical. KSW is a consultant for Novo Nordisk, coauthor of the eTHINK and paradigm study protocols, a site PI of the eTHINK study, and a consultant for AstraZeneca, Alexion, SpringWorks, and DayOne Biopharmaceuticals. CBB was a site PI of the eTHINK study and has received consultant fees from Novo Nordisk. SEC is a site PI of the eTHINK study and a consultant for Bayer, CSL Behring, Genentech, Octapharma, and Shire and has received research funding from Genentech, Pfizer, Novo Nordisk, Spark Therapeutics, and ATHN/Hemophilia of Georgia Research. AM and MG are full-time employees of Novo Nordisk. CH is a consultant for Novo Nordisk and participated in the eTHINK study. MR has received research grants from Pfizer, Bristol-Myers Squibb, and Novo Nordisk (for clinical studies) and is a consultant for Novo Nordisk. KAS has received fees as a consultant for Novo Nordisk. GNW has received fees as a consultant for Novo Nordisk. PV is a full-time employee of Cogstate. DLC was an employee of Novo Nordisk at the time of the conception of the eTHINK study and data collections. The eTHINK study was funded by Novo Nordisk A/S, Bagsværd, Denmark., (Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved.)

Objectives: To evaluate the impact of the 2002 guidelines on the current status of prophylaxis in French children with severe hemophilia A or B., Study Design: Clinical information was captured, in a prospective way, using FranceCoag Network. We retrospectively studied 291 patients with severe (<1 IU/dL) hemophilia A and B, with no history of inhibitors., Results: Our results demonstrate that the availability of national medical guidelines has improved clinical practice in France. In the past decade, the proportion of children with severe hemophilia undergoing prophylaxis has shown a significant 2- to 3-fold increase: ∼80% of these children>3 years of age are now receiving prophylaxis. In severe hemophilia A and B, the age at which prophylaxis commences has significantly decreased: 4.0 and 6.1 years for the period 1996-1999 as opposed to 1.8 and 1.4 years for the period 2004-2007 (P=.0001)., Conclusions: Long-term clinical and physical evaluations of patients will be necessary to establish the benefits of this increase in prophylactic treatment on the prevention of hemophilic arthropathy., (Copyright © 2013 Mosby, Inc. All rights reserved.)

Objective: The objective of this study was to define the efficacy and complications of implantable venous access devices (IVADs) in children with hemophilia., Study Design: Records were reviewed on all patients with congenital blood coagulation disorders monitored at two children's hospitals in whom one or more central venous catheters had been placed., Results: Since 1989 external and implantable central venous catheters have been inserted to enhance venous access for regular factor concentrate infusion in 45 patients with hemophilia ranging in age from 8 months to 19.5 years (median 7.4 years); 37 patients had factor VIII deficiency and 8 factor IX deficiency. Hemorrhagic complications of catheter placement were infrequent and minor. In the 41 patients having one or more IVADs in place for a median of 31 months, only six episodes of bacteremia occurred in 5 patients during 44,070 days of follow-up. The overall rate of bacteremia complicating IVADs in these patients was 0.14 episodes per 1000 catheter days. Other catheter-related complications were uncommon. Catheters are still in place in 33 patients for a median of 32 months., Conclusion: The low risk of infection and other complications associated with the use of IVADs makes the use of these devices attractive in the treatment of patients with hemophilia who require frequent venous access for factor concentrate infusions.

Efforts to detect subtle but objective neuropsychologic deficits could clarify the early involvement of the central nervous system and the progression of human immunodeficiency virus (HIV) infection in older children and young adolescents. Baseline examinations of 63 children and adolescents with hemophilia were conducted by examiners unaware of HIV status or staging or of our study's major hypotheses. They measured six domains of neuropsychologic functioning (motor, language, memory, attention, visual processing, and problem solving), and no differences between groups of similar age, race, and socioeconomic status defined by HIV seropositivity (n = 25) and HIV seronegativity (n = 38) were revealed. A high incidence of subtle neuropsychologic deficits relative to (1) age norms and (2) individual cognitive potential was found on measures of motor performance, attention, and speeded visual processing within both infected and uninfected groups. On the basis of these baseline data, it seems premature to attribute early, subtle neuropsychologic deficits in seropositive children with hemophilia to the central nervous system effects of HIV infection.

A 2 1/2-year-old, previously healthy child developed progressive swelling of the left leg and a hematoma of the anterior chest wall associated with a falling hemoglobin concentration, as a result of a spontaneously acquired Factor IX inhibitor. Successful management of her condition required a four-volume exchange transfusion and immunosuppressive therapy consisting of cyclophosphamide for four days and prednisone for one month. A brief review of the literature regarding the occurrence and nature of acquired coagulation factor inhibitors and the role of immunosuppressive therapy is also presented.