Introduction BAY 81‐8973 (Kovaltry ® ) is a full‐length, unmodified recombinant human factor VIII (FVIII) with the same amino acid sequence as sucrose‐formulated FVIII and produced using additional advanced manufacturing technologies. Aim To demonstrate efficacy safety of for treatment bleeds prophylaxis based on two different potency assignments. Methods In LEOPOLD I (ClinicalTrials.gov identifier, NCT 01029340), males aged 12–65 years severe haemophilia A ≥150 exposure days received 20–50...

Homozygosity for the common (677C-->T) mutation in methylenetetrahydrofolate reductase (MTHFR) gene is associated with hyperhomocysteinemia, but there uncertainty as to association between this and coronary artery disease (CAD). This study examined MTHFR genotypes age at onset of CAD.Patients (n=169) documented myocardial infarction or angiographically CAD who were aged < = 55 years symptoms DNA samples from control subjects (n=313) studied. The prevalence homozygosity among patients early...

Introduction BAY 81‐8973 is a full‐length recombinant factor VIII (FVIII) with the same primary amino acid sequence as sucrose‐formulated FVIII (rFVIII‐FS) but produced advanced manufacturing technologies. Aim To analyse pharmacokinetics (PK) of after single and multiple dosing across different age ethnic groups in LEOPOLD clinical trial programme. Methods The trials enrolled patients severe haemophilia A aged 12–65 years (LEOPOLD I II) or ≤12 Kids) ≥150 ≥50 exposure days to any product no...

Summary Patients with severe haemophilia A experience frequent and spontaneous bleeding, causing debilitating damage to joints decreasing quality of life. Prophylaxis factor VIII (FVIII) reduces joint if initiated early. Circulating FVIII levels may be influenced by endogenous von Willebrand ( VWF ), a chaperone protein that binds stabilizes FVIII. The aim this study was determine whether antigen :Ag) are correlated pharmacokinetic PK ) parameters clinical outcomes in patients A. Previously...

Introduction Recombinant factor VIII (rFVIII) products with extended half‐lives, such as BAY 94‐9027, can potentially maintain higher FVIII levels for longer periods of time, thus providing improved bleeding protection vs standard‐acting products. Aim To characterize the pharmacokinetic (PK) profile 94‐9027 from phase 1, 2/3 (PROTECT VIII) and 3 Kids) clinical trials in adults, adolescents children severe haemophilia A Methods Patients (FVIII &lt;1%) &gt;50 exposure days (EDs) no history...

Ensuring hemostasis during invasive procedures is a challenge in patients with severe hemophilia A. This analysis evaluated efficacy and safety of BAY 94-9027, an extended-half-life recombinant factor VIII (FVIII), the surgical setting.Patients participating open-label 94-9027 clinical trial who underwent major surgery were included analysis. Investigator/surgeon assessment was primary outcome. In addition, information about FVIII use, levels perioperative period, bleeding complications...

Abstract Introduction BAY 94‐9027 is an extended‒half‐life, site‐specifically PEGylated, B‐domain‒deleted recombinant factor VIII (FVIII). The PROTECT main study demonstrated efficacy of bleed control using extended‐interval prophylaxis with for 36 weeks. Aim To report long‐term and safety in a descriptive analysis the ongoing extension total treatment time up to &gt;5 years. Methods Previously treated males aged 12‐65 years severe haemophilia A who completed were eligible open‐label...

Summary. Recent reports have raised concerns regarding potential risk factors for inhibitor development. In Israel, all haemophilia patients (n = 479) are followed by the National Hemophilia Center. Most children neonatally exposed to factor concentrate (due circumcision performed at age of 8 days). The impact early exposure and recombinant FVIII products (rFVIII) administration (approved in Israel since 1996) upon occurrence our cohort A (HA) was analysed. Two hundred ninety-two consecutive...

Introduction: Hemophilia A (HA) is an X-linked bleeding disorder caused by factor VIII (FVIII) deficiency or dysfunction due to F8 gene mutations. HA carriers are usually asymptomatic because their FVIII levels correspond approximately half of the concentration found in healthy individuals. However, rare cases, a carrier may exhibit symptoms moderate severe primarily skewed inactivation her non-hemophilic X chromosome. Aim: The aim study was investigate X-chromosome (XCI) patterns carriers,...

Abstract Introduction The phase 2/3 PROTECT VIII main study demonstrated efficacy and safety of BAY 94–9027 (damoctocog alfa pegol; Jivi ® ), a B‐domain‐deleted recombinant factor (FVIII), site‐specifically PEGylated to extend its half‐life. Aim To report the final data for from extension. Methods Previously treated males aged 12–65 years with severe haemophilia A (FVIII &lt;1%) who completed multicentre, open‐label were eligible Patients received either on demand or prophylaxis treatments...

Introduction BAY 81‐8973 is a recombinant factor VIII ( rFVIII ) with the same amino acid sequence as Bayer's sucrose‐formulated ‐ FS but manufactured certain more advanced technologies. Aim To describe surgery outcomes in LEOPOLD trials. Methods Male patients severe haemophilia A and no inhibitors aged 12–65 years ≥150 exposure days ED s) to FVIII I II ), or ≤12 ≥50 s Kids), received based on dosing recommendations for according surgical requirements. Haemostasis‐related complications,...

Persons with hemophilia A may require surgical procedures. Real-world data on invasive procedures in persons receiving emicizumab prophylaxis are limited.To evaluate the safety of and their outcomes a longitudinally followed cohort.Data from medical records without factor VIII (FVIII) inhibitors at our tertiary center, who received underwent all types procedures, were retrieved. Outcomes interest bleeding thrombotic complications.Overall, 35 patients 56 18 (32.1%) major. The median age was...

Summary. Haemophilia patients who received non‐virucidally treated large pool clotting factors before 1987 have a high rate of chronic hepatitis C viral infection (HCV). Some are coinfected with HIV. and other coagulation disorders were at one centre since the beginning 1970, Israeli National Hemophilia Center (INHC) was officially founded in 1987. To characterize HCV as well HCV/HIV coinfection INHC. Patients haemophilia positive for antibodies evaluated between 2001 2004. Demographic data,...

Summary Prospective data on the efficacy of secondary prophylaxis in adults with haemophilia A are limited. To analyse bleeding outcomes sucrose‐formulated recombinant factor VIII [rFVIII‐FS (control)] arm LIPLONG study, a randomized, double‐blind, 52‐week trial was conducted patients severe receiving investigational product BAY 79‐4980 or rFVIII‐FS. The per‐protocol population previously treated without history inhibitors ( n = 68 males; mean age, 34.4 years) received 25 IU kg −1 rFVIII‐FS...

Joint bleeding is a hallmark of haemophilia A,1 with recurrent bleeds having the potential to cause irreversible joint damage and crippling arthropathy, often limiting daily activities requiring surgery.2-4 Target joints are those where three or more occur in 6-month period5 prone chronic if not properly treated. Prophylaxis factor VIII (FVIII) concentrates current standard care. Its main aim prevent onset and/or progression by reducing frequency severity episodes, including that into...

Advances in treatment have enabled patients with haemophilia A to live longer and therefore may be subjected comorbidities associated ageing, addition disease-associated morbidities. There been few reports date on efficacy safety of specifically severe comorbidities.To explore the damoctocog alfa pegol prophylaxis aged ⩾40 years interest.A post hoc analysis data from phase 2/3 PROTECT VIII study its extension.Bleeding outcomes were analysed a subgroup ⩾1 comorbidity receiving (BAY 94-9027;...

In patients with haemophilia A, minor surgical interventions are vital for improving well-being and may prevent major procedures. However, the risk of bleeding in surgery be higher than surgery. Local haemostasis not ensured suturing, cautery or pressure application, procedures performed without involvement an experienced multidisciplinary team.1 Individualised haemostatic therapy should used to maintain appropriate levels factor VIII (FVIII) A during perioperative period all excessive...

Abstract Aim Very little is known regarding reproductive choices, pregnancy, and delivery of women with moderate to severe hemophilia. Our aim was describe our experience three hemophiliac their journey achieve motherhood. Methods Medical charts hemophilia A treated at center were evaluated. Data choices conception, pregnancy course, mode delivery, outcomes obtained. Results Three are presented. Whereas patient 1 chose adopt her first child later had twins through egg donations a surrogate...