Real-world Effectiveness of Lanadelumab in Hereditary Angioedema: Multi-country INTEGRATED Observational Study.

Background: Hereditary angioedema (HAE) is a rare genetic disease characterized by recurrent episodes of cutaneous or subcutaneous edema. There is clinical need for treatments that reduce the rate of HAE attacks in patients.

Objectives: Primary objectives were to evaluate the effectiveness of lanadelumab on attack free rate (AFR; proportion of patients who had zero HAE attacks), and on every two weeks (Q2W) and every four weeks (Q4W) adjustments on AFR.

Modulation of Haemostatic Balance in Combined von Willebrand Disease and Antithrombin Deficiency: A Comprehensive Family Study.

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.

Real-World Data on Patients With Acquired Haemophilia A in Japan Undergoing Rehabilitation or With Low Activities of Daily Living Scores: The ORIHIME II Study.

Introduction: Acquired haemophilia A (AHA) is characterized by the development of autoantibodies against factor VIII, reducing its activity and potentially resulting in bleeding.

Aim: To assess the characteristics of people with AHA undergoing rehabilitation and/or with low activities of daily living (ADL) scores, thereby characterizing unmet needs in the management of AHA and informing treatment optimization.

Methods: ORIHIME II, the largest epidemiological and treatment survey of AHA in Japan, is a descriptive, retrospective, observational study conducted using health claims data from April 2008 to October 2021.

Efficacy of Wilate Prophylaxis in Reducing Nosebleeds in Patients with Severe VWD - A Post-hoc Analysis of the WIL-31 Study.

Background: Prophylaxis with a von Willebrand factor (VWF) concentrate is recommended in von Willebrand disease (VWD) patients with a history of frequent and severe bleeds. Despite nosebleeds being a frequent manifestation of VWD, few studies have investigated the efficacy of factor prophylaxis in preventing nosebleeds in patients with severe VWD.

Methods: This post-hoc analysis of a prospective, 12-month, phase 3 study assessed the efficacy of wilate in the prevention of nosebleeds in 33 patients aged ≥6 years with severe type 1, type 2 or type 3 VWD.

The Treatment of Indirect Carotid Cavernous Fistula in a Pediatric Patient With Deep Recurrent Ophthalmic Artery and Hemophilia.

The treatment of indirect carotid-cavernous fistula (CCF) poses a unique challenge. Currently, endovascular interventions remain the principal treatment option with high cure rates and acceptable safety profiles. The anatomical characteristics of individual cases determine the optimal vascular access routes (transvenous vs.

Assessing the factors affecting the accessibility of primary dental care for people with haemophilia.

Introduction: Patients with haemophilia (PWH) often have difficulty accessing dental services.

Aim: To determine the accessibility of dental care for PWH and to examine their perceptions of how coronavirus type-2 (CoV-2) disease (COVID-19) has affected their ability to access dental treatments following the pandemic.

Methods: The questionnaire survey was conducted between July 2022 and December 2022 at haemophilia treatment centres in Hungary.

A ten-year experience with the diagnosis of von Willebrand disease in Mexico based on a cost-effective strategy.

Background: Von Willebrand disease (VWD), is the most common inherited bleeding disorder worldwide, but its diagnosis is complicated, expensive, and poorly evaluated in several countries.

Objective: To report our long-term experience with the diagnosis of VWD based on a cost-effective strategy.

Methods: We studied 802 Mexican patients, men and women, children, and adults, with clinical suspicion of VWD.

Prediction of the chance of successful immune tolerance induction in persons with severe hemophilia A and inhibitors: a clinical prediction model.

Background: Inhibitor eradication to restore factor (F)VIII efficacy is the treatment goal for persons with severe hemophilia A (HA) and inhibitors. Immune tolerance induction (ITI) is demanding and successful in about 70% of people. Until now, it has remained difficult to quantify the probability of ITI success or failure, complicating the decision to initiate or not initiate ITI.

Hemostats in the clinic.

Given the prevalence of hematological conditions, surgeries, and trauma incidents, hemostats-therapeutics designed to control and arrest bleeding-are an important tool in patient care. The prophylactic and therapeutic use of hemostats markedly enhances survival rates and improves the overall quality of life of patients suffering from these conditions. Since their inception in the 1960s, hemostats have witnessed remarkable progress in terms of the active ingredients utilized, therapeutic outcomes, demonstrated efficacy, and the storage stability.

Challenges associated with access to recently developed hemophilia treatments in routine care: perspectives of healthcare professionals.

The treatment landscape for haemophilia continues to rapidly develop, and expectations for future treatment success are high. There is limited information on the challenges to accessing new and innovative therapies. The aim of this study was to explore challenges with accessing haemophilia treatment from the perspective of healthcare professionals (HCPs).

Treatment of a compartment syndrome in the leg in an elderly patient with acquired hemophilia type A: A case report.

Introduction: Acquired hemophilia type A is a rare autoimmune disease characterized by bleeding episodes ranging from mild to severe, leading to significant morbidity and mortality. One of the various presentations is compartment syndrome secondary to a spontaneous hematoma.

Case Presentation: A 95-year-old woman arrived at the emergency department with a one-week history of progressive pain in the right leg, without a previous history of trauma.

Chemokine CXCL13 facilitates anti-FVIII inhibitory antibody development in hemophilia A patients and murine models.

The development of anti-factor VIII (FVIII) neutralizing antibodies (inhibitors) remains challenging complication in hemophilia A (HA) patients undergoing prophylactic FVIII replacement therapy. The pathogenesis of FVIII inhibitor formation remains unclear. Chemokine CXCL13, a key ligand for follicular helper T cells (TFHs), in the context of inhibitor development were assessed in the present study.

Haemophilia care in Iraq; a multi-centre study.

Objective: To evaluate the level of care available for haemophilia patients.

Methods: The descriptive, retrospective analytical study was conducted from December 15, 2020, to March 1, 2021, after approval from the Mustansiriyah University, Baghdad, Iraq, and comprised data from 3 haemophilia treating centres in Iraq participating in the World Bleeding Disorders Registry. The data collected related to patients with haemophilia A and B enrolled in the registry since March 2018, and included age at diagnosis, type of haemophilia, disease severity, age at first bleed and at first joint bleed, type of replacement therapy and outcome.

Perioperative Pain Management in Hemophilic Patient Undergoing Orthopedic Surgery: A Narrative Review.

Background: Hemophilia type A and B is associated with spontaneous bleeding in muscle tissues and joints. Acute hemarthrosis, representing 70-80% of all bleedings in severe hemophilia patients, is extremely painful. When surgical procedures are needed in hemophiliac patients, perioperative management should be planned with a multidisciplinary team.