Accurate monitoring of nonacog beta pegol (N9-GP) is essential to ensure appropriate treatment and to avoid under- or overdosing, which may result in clinical complications. The product's extended half-life, achieved through molecular modification, poses challenges for activity measurement, particularly when using a one-stage clotting assay (OSA). Therefore, a chromogenic substrate assay (CSA) is preferred, although it may be less practical for urgent or routine monitoring.

To assess the health-related quality of life (HRQOL) in children with haemophilia A and arthropathy before and after emicizumab prophylaxis using the Canadian Haemophilia Outcomes-Kids' Life Assessment Tool questionnaire Canadian Hemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT) v3.0 and haemophilia family impact tools (H-FIT).

Gene therapy (GT) represents a transformative development in the treatment of haemophilia, offering the potential for long-lasting symptom control after a single administration. Although the scientific and medical aspects of this innovation have been widely studied, its psychological impact remains under-addressed.

Knee arthropathy affects 90% of patients with severe haemophilia, with unique morphological changes making TKA technically challenging. We evaluated morphological features of haemophilic knees compared to osteoarthritic controls and provide evidence-based surgical recommendations.

Achilles tendon lengthening combined with external fixation (EF) has shown promising outcomes in managing haemophilia-related Achilles tendon contracture.

Von Willebrand disease (VWD) is one of the most common hereditary disorders of primary haemostasis. Females with VWD often report heavy menstrual bleeding (HMB) that can significantly impact health-related quality of life (HRQoL).

Radioactive synovectomy (RSO) is an alternative to arthroscopic synovectomy (AR), for treating synovitis. RSO reduces the number of hemarthroses, being less invasive and less costly.

Efanesoctocog alfa is a novel factor VIII (FVIII) concentrate with a unique molecular design that enables Von Willebrand Factor-independent clearance in patients with haemophilia A. Limited sampling strategies (LSSs) are necessary to implement accurate pharmacokinetic (PK)-guided dosing for efanesoctocog alfa in clinical practice.

Modern prophylaxis in haemophilia aims not only to prevent bleeding but also to ensure a quality of life (QoL) comparable to healthy peers. Real-world data on health-related QoL (HRQoL) in children receiving personalised pharmacokinetic (PK)-guided prophylaxis are limited.

This study aims to evaluate the natural course and resolution of flexion contracture following total knee arthroplasty (TKA) in individuals with haemophilic arthropathy.

Assessment of health-related quality of life (HRQoL) is relatively new in von Willebrand disease (VWD). So far, generic questionnaires have mainly been used for HRQoL assessment in VWD.

Haemophilic arthropathy (HA) is a common complication of haemophilia, characterized by progressive joint degeneration due to recurrent bleeding. Iron overload from erythrocyte lysis is thought to play a key role in HA pathogenesis, but its molecular basis remains unclear.

This study aimed to evaluate the clinical and radiographic outcomes of total knee arthroplasty (TKA) in patients with haemophilia A and severely stiff knees (preoperative flexion range of motion ≤40°) with a focus on functional recovery, implant stability, and complication rates.

The Natural History Study of the Safety, Effectiveness, and Practice of Treatment for People with Haemophilia (ATHN 7) monitors use of contemporary haemophilia therapies, including emicizumab, a bispecific antibody substituting for activated factor (F)VIII in people with haemophilia A (HA).

Limited real-world data exist on recombinant fusion protein-linking coagulation factor IX (FIX) with albumin (rIX-FP) in paediatric previously untreated patients (PUPs) with haemophilia B, particularly in infants.

In India, persons with haemophilia (PWH) are entitled to free diagnostic services, clotting-factor concentrates and physiotherapy. However, families often continue to experience substantial financial hardships for regular treatment. This study aimed to quantify the expenditure borne by such families of PWH treated at our centre and identify contributing factors.

As assays tailored to Altuvoct are necessary, it is essential that every request for a FVIII level for monitoring of Altuvoct clearly identifies when it has been used for treatment of the patient and details the time of sampling relative to timing infusion.

Glanzmann Thrombasthenia (GT) is a rare autosomal recessive bleeding disorder characterised by defective platelet aggregation due to abnormalities in the glycoprotein IIb/IIIa complex. With a lack of large real-world database studies, our study aimed to characterise the demographic, clinical and treatment profiles of GT patients using a large, national database.