Unmet needs in haemophilic arthropathy
Hemophilia A and B are rare X-linked bleeding disorders caused by coagulation factor deficiencies, leading to joint bleeding, synovial hypertrophy and chronic hemophilic arthropathy marked by progressive cartilage and bone damage. Musculoskeletal issues remain the primary source of morbidity in people with hemophilia (PwH). Despite significant advances in prophylactic therapies, joint pain, functional limitations, and […]
Factor VIII promotes angiogenesis and vessel stability regulating extracellular matrix proteins
Factor VIII (FVIII) is a crucial coagulation co-factor and mutations in the F8 gene lead to hemophilia A (HA). Reduced activity of FVIII or its absence results in bleeding episodes, which can occur either spontaneously or secondary to trauma.1 To date, there is no definitive cure for HA. The standard therapy involves prophylaxis with either replacement products, such […]
State of the art of the management of patients with haemophilia
Haemophilia A and B are congenital X-linked bleeding disorders resulting from deficiencies in clotting factors VIII (haemophilia A) and IX (haemophilia B). Patients with severe deficiency, defined as having less than 1% of normal plasma factor activivity, often have spontaneous bleeding within the first few years of life. Those with moderate and mild deficiencies typically […]
Patient-Centred Management of Well-Controlled Haemophilia: Obtaining Opinions and Definitions Through a Delphi Consensus
For people with haemophilia, health-related quality of life mainly depends on the arthropathy caused by repeated joint bleeding. Prophylaxis is the standard of care in patients with severe bleeding phenotypes, but globally, none of the measures used to assess patients’ outcomes consider their desires and life expectations. We propose the concept of the “patient-centred management […]
Patients with moderate hemophilia A and B with a severe bleeding phenotype have an increased burden of disease
Patients with moderate hemophilia express varying bleeding phenotypes. To assess the burden of disease in patients with moderate hemophilia and a mild or severe phenotype incorporating the thrombin generation profile. This sub-study of the 6th Hemophilia in the Netherlands study, analyzed data of adults with moderate hemophilia A or B. Patient characteristics and information on […]
Teenagers and Adolescents with Hemophilia–Need for a Specific Approach
Adolescents with hemophilia are a patient population with special requirements, having to manage their condition alongside the typical challenges of adolescence. Given the psychosocial impact of hemophilia and a desire to fit in with non-hemophilic peers, they may perceive treatment as more of a burden than a benefit. This can result in low adherence and […]
Hemophilia and Other Congenital Coagulopathies in Women
Deficiencies of factor VIII (FVIII)/von Willebrand factor (VWF) or factor IX (FIX) are underappreciated as potential reasons for heavy menstrual bleeding, recurrent nosebleeds, and easy bruising in girls and women. Bleeding is usually not attributed to hemophilia because clinically significant deficiencies in clotting factors VIII and IX are thought to only affect males. While severe hemophilia […]
12 June 2020
Emerging viral diseases and infectious disease risks
New pathogens and antimicrobial-resistant forms of older pathogens continue to emerge, some with the potential for rapid, global spread and high morbidity and mortality. Pathogens can emerge either through introduction into a new population or when the interaction with the vector changes; emergence is also influenced by microbiological adaptation and change, global travel patterns, domestic […]
16 October 2023
HUMAN IMMUNODEFICIENCY VIRUS, HEPATITIS C VIRUS AND HEPATITIS B VIRUS INCIDENCE IN BLOOD DONORS FROM 2000 TO 2020 IN FRANCE: TRENDS AND LESSONS FROM HAEMOVIGILANCE SURVEILLANCE
Data from 21 years (2000–2020) of haemovigilance were used to assess human immunodeficiency virus (HIV), hepatitis B virus (HBV) and hepatitis C virus (HCV) incidence rates in repeat blood donors and the occurrence of transfusion-transmitted (TT) viral infections. Blood donors who converted for HIV, HCV or HBV markers within serial three-year analysis periods were included. Epidemiological […]
17 January 2018
Epidemiological characteristics of human prion diseases
In the absence of specific therapies for human prion diseases, which are frequently associated with neurodegenerative disorders with fatal outcome, only an active surveillance can be used to monitor and prevent the transmission of such diseases. For this reason, starting from 1993, surveillance systems have been established in many countries. Moreover, there is urgent need […]
22 October 2025
Blood Group O Does Not Increase the Risk of Inhibitors inSevere Haemophilia A: Data from the PedNet Study Group
Inhibitor development against factor VIII (FVIII) is the most common complication of hemophilia A replacementtherapy. One of the variables considered to influence inhibitor development is the ABO blood group. Patients with blood groupO have, on average, a 30%–40% lower endogenous von Willebrand factor (VWF) concentration. It has been postulated that VWFlevels influence inhibitor development. The […]
22 October 2025
Contemporary approaches to treat people with hemophilia: what’s new and what’s not?
The care of people with hemophilia with access to treatment has evolved over the past 70 years, with an average life expectancy like unaffected peers. For people with hemophilia living in low- and middle-income countries, the same is not true because of the lack of access to diagnosis and treatment. It is imperative to close […]