Background: Data regarding management of pediatric persons with hemophilia A (PwHA) with factor VIII (FVIII) inhibitors are limited. This prospective noninterventional study (NCT02476942) evaluated annualized bleeding rates (ABRs), safety, and health-related quality of life (HRQoL) in pediatric PwHA with FVIII inhibitors.
Procedure: PwHA aged <12 years with current FVIII inhibitors and high-titer inhibitor history were enrolled. Participants remained on usual treatment; no interventions were applied. Outcomes included ABR, safety, and HRQoL.
Results: Twenty-four PwHA aged 2-11 years (median 7.5) were enrolled and monitored for 8.7-44.1 weeks (median 23.4). In the episodic (n = 10) and prophylactic (n = 14) groups, respectively, 121 of 185 (65.4%) and 101 of 186 (54.3%) bleeds were treated using activated prothrombin complex concentrate (aPCC) and/or recombinant activated FVII (rFVIIa). ABRs (95% confidence interval) were 19.4 (13.2-28.4) and 18.5 (14.2-24.0) for treated bleeds, and 32.7 (20.5-52.2) and 33.1 (22.4-48.9) for all bleeds, respectively. Most prophylactic group participants (92.9%) were prescribed aPCC; 50% adhered to their prescribed treatment regimen. Adherence to prophylactic rFVIIa was not assessed. Serious adverse events included hemarthrosis (12.5%) and mouth hemorrhage (12.5%); the most common nonserious adverse event was viral upper respiratory tract infection (12.5%). HRQoL showed functional impairment at baseline; scores remained stable throughout, with little intergroup variation.
Conclusions: ABRs remained high in pediatric PwHA with inhibitors receiving standard treatment. This study demonstrates the need for more effective treatments, with reduced treatment burden, to prevent bleeds, increase prophylaxis adherence, and improve patient outcomes.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1002/pbc.28474 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
The variable relationship between the National Early Warning Score on admission to hospital, the primary discharge diagnosis, and in-hospital mortality.
Intern Emerg Med
January 2025
Department of Renal Medicine, Northern Care Alliance, Salford Royal Hospital, Salford, M6 8HD, UK.
Background: Patients with an elevated admission National Early Warning Score (NEWS) are more likely to die while in hospital. However, it is not known if this increased mortality risk is the same for all diagnoses. The aim of this study was to determine and compare the increased risk of in-hospital mortality associated with an elevated NEWS and different primary discharge diagnoses in unselected emergency admissions to a UK university teaching hospital.
View Article and Find Full Text PDFA pilot observational study of the association of 24-hour mortality with the subjective assessment of the forearm skin temperature and moisture compared to other bedside indicators of illness severity.
Resusc Plus
January 2025
School of Clinical and Biomedical Sciences, University of Bolton, United Kingdom.
Background: Although the association of peripheral skin temperature with infection, serious illness and death have been recognised for centuries, few studies have explicitly compared this finding with other bedside indicators of illness severity. This study compared subjectively assessed dorsal forearm skin temperature and moisture with other indicators of illness severity.
Methods: Non-interventional observational study of acutely ill medical patients admitted to a low-resource Ugandan hospital, which examined the association of subjectively assessed dorsal forearm skin temperature and other bedside findings with death within 24 h.
Does the healthy plate model workshop improve nutrition knowledge, behaviors, and habits in school-age children? The impact of workshop on dietary habits : Original article.
BMC Public Health
January 2025
Department of Nutrition and Dietetics, Kutahya Health Sciences University School of Health Sciences, Kütahya, Turkey.
Background: This study was conducted to identify the impacts of the healthy plate model workshop on 4th-graders nutrition knowledge, behaviors, and habits.
Methods: The study was conducted from March to June 2023 in the Uskudar district of Istanbul, involving 102 children (50% girls) with a mean age of 10.2 ± 0.
Long-Term Success and Survival of Post-Endodontic Restorations Without Posts After up to18 Years: A Practice-Based Study.
J Dent
January 2025
Department of Restorative, Preventive and Pediatric Dentistry, zmk bern, University of Bern, Switzerland; Unit for Practice-based Research, School of Dental Medicine, University of Bern, Switzerland. Electronic address:
Objectives: The aim of this non-interventional, multi-center, prospective, practice-based study was, firstly, to evaluate the longevity of composite build-ups in endodontically treated teeth, treated with root canal therapy (ETT), without post placement and secondly, to identify and analyze factors influencing the success of these reconstructions.
Methods: Each of seven general dental practitioners placed up to 50 composite build-ups without additional posts in ETT. Teeth were restricted to incisors, canines and premolars.
Use of assistive technology to assess distal motor function in subjects with neuromuscular disease.
PLOS Digit Health
January 2025
Centre Référent Maladies Rares Neuromusculaires, Service de Médecine Physique et de Réadaptation Pédiatrique des Hospices Civils de Lyon - Hôpital Femme Mère Enfant, Bron, France.
Unlabelled: Among the 32 items of the Motor Function Measure scale, 3 concern the assessment of hand function on a paper-based support. Their characteristics make it possible to envisage the use of a tablet instead of the original paper-based support for their completion. This would then make it possible to automate the score to reduce intra- and inter-individual variability.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!