Introduction: Characterisation of outcomes and costs of haemophilia care in common practice settings is essential for evaluation of new treatment options and for developing clinical practices. In Finland, haemophilia care is mostly centralised to University Hospitals, but treatment practices and costs in adult patients have not been systematically evaluated.
Aim: This study was designed to characterise healthcare resource utilisation and treatment costs of adult inhibitor-negative haemophilia patients managed in Finnish University Hospitals.
Methods: The study was based on a nationwide cohort, which consists of all adult haemophilia A (HA; n = 120) and B (HB; n = 35) patients treated in University Hospitals from 2012 to 2016. Patient characteristics and data on healthcare utilisation and factor replacement use were collected from medical records. Direct costs of care were evaluated based on wholesale drug prices and healthcare service utilisation with standard unit costs.
Results: Most of HA (79%, n = 96) and HB (84%, n = 31) patients received factor replacement therapy. The median annual bleeding rate (ABR) was low, at 0.8 for HA and 0.5 for HB, also among the patients with on-demand therapy. Over 94% (n = 149) of the patients had outpatient visits during the follow-up period. The mean total annual costs of treatment ranged from €2520 to €176,330. The highest individual cost was factor replacement therapy.
Conclusion: The outcomes of centralising the management of care to University Hospital Treatment Centres show low ABR and lower treatment costs compared with earlier reports from other high-income European populations. Management strategies, including choosing the right therapy between prophylaxis and on-demand, has been successful in Finland.
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http://dx.doi.org/10.1111/hae.14197 | DOI Listing |
Transfus Apher Sci
December 2024
Department of Hematology and Blood Banking, Faculty of Allied Medicine, Iran University of Medical Sciences, Tehran, Iran. Electronic address:
Background: Hemophilia B, or Christmas disease, is a hemorrhagic inherited disorder. Previous studies have reported measurement discrepancies in factor VIII activity between clot-based and chromogenic assays in approximately one-third of patients with non-severe hemophilia A. However, similar discrepancies in hemophilia B have been less extensively studied.
View Article and Find Full Text PDFPediatr Investig
December 2024
Hematology Department, Hemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, Key Laboratory of Major Diseases in Children, National Center for Children's Health National Key Discipline of Pediatrics (Capital Medical University), Ministry of Education, Beijing Children's Hospital, Capital Medical University Beijing China.
Importance: Emicizumab (EMI) is efficacious and safe for hemophilia A (HA) prophylaxis. However, its high cost poses a challenge in China.
Objective: To explore the possibility of using reduced-dosage EMI in Chinese HA children.
Ther Adv Hematol
December 2024
Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI, USA.
Background: Hemophilia A is caused by coagulation factor VIII (FVIII) deficiency and increases bleeding risk during invasive procedures.
Objectives: To investigate FVIII concentrate use and bleeding outcomes for invasive procedures after valoctocogene roxaparvovec gene transfer.
Design: This manuscript presents post hoc analysis of the phase III GENEr8-1 trial.
Spec Care Dentist
December 2024
Department of Child Health Nursing, Manipal College of Nursing, Manipal Academy of Higher Education, Manipal, Karnataka, India.
Objective: To analyze the various dental management strategies adopted to manage patients with hemophilia in a dental clinical setup.
Methods: An electronic database search was carried out using MEDLINE by PubMed, Scopus, Google Scholar, Web of Science, and EMBASE databases from January 2000 to August 2023 for case reports and case series published in English language. Case reports addressing the dental treatments for people with hemophilia A/hemophilia B were included.
Haemophilia
December 2024
Institute of Experimental Hematology and Transfusion Medicine, Medical Faculty, University Hospital Bonn, University of Bonn, Bonn, Germany.
Introduction: Maintaining the balance between procoagulant and anticoagulant factors is essential for effective haemostasis. Emerging evidence suggests a modulation of bleeding tendency by factors in the anticoagulant and fibrinolytic systems.
Aim: This study investigates the clinical and laboratory characteristics of a family with combined von Willebrand disease (VWD) and antithrombin (AT) deficiency.
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