Four-Year Emicizumab Treatment in an Elderly Patient With Acquired Hemophilia A: A Case Report on Perioperative Management Along With a Literature Review.

Acquired hemophilia A (AHA) is a life-threatening bleeding disorder caused by autoantibodies against coagulation factor VIII (FVIII). While immunosuppressive therapy (IST) can eradicate these autoantibodies, it may fail or cause adverse events, especially in elderly patients. Clinical trials involving AHA patients have confirmed the efficiency of emicizumab, a bispecific antibody mimicking FVIII widely used in congenital hemophilia A with or without inhibitors, but the long-term safety and effectiveness in perioperative hemostatic management remain unclear.

Comparison of genotypes and phenotypes for von Willebrand factor gene variants using Japanese genome database.

von Willebrand disease (VWD) is a common inherited bleeding disorder. The aim of this study was to determine the predicted disease states associated with various pathogenic von Willebrand factor (VWF) variants and their phenotypes using the largest Japanese whole-genome database. Of the 5857 gene variants registered in the Japanese Multi-Omics Reference Panel (jMorp), variants with the following criteria were extracted: (1) caused protein abnormalities due to genetic alterations; (2) have already been detected and included in a database, including known association with VWD; and (3) highly likely pathogenic by in silico analysis.

LC16m8 for Pre-exposure Prophylaxis against Mpox in a High-Risk Population: An Open-Label Randomized Trial.

Background: This randomized controlled trial provided LC16m8 pre-exposure prophylaxis to high-risk individuals to assess its efficacy for mpox prevention, safety, and immunogenicity.

Methods: This multicenter, randomized, open-label trial enrolled men and women aged ≥18 years at high risk of mpox. Participants were randomly assigned 1:1 to early- or late-vaccination groups.

Effects of switching to dolutegravir/lamivudine from tenofovir alafenamide fumarate/emtricitabine/dolutegravir or abacavir/lamivudine/dolutegravir on body weight and lipid profile in Japanese people living with HIV.

Background: The two-drug regimen of dolutegravir/lamivudine (DTG/3TC) is currently an optional antiretroviral therapy (ART). Despite its reported advantages on body weight and lipid profile, the same effects have not yet been reported for Asian population.

Methods: We conducted a single-center retrospective study involving Japanese people living with HIV (PLWH).

Prevalence and associated factors of low vigor in patients living with HIV and hemophilia in Japan: A cross-sectional observational study.

People living with human immunodeficiency virus (HIV) are at high risk of mental health problems. However, little is known about this risk in HIV-infected patients with hemophilia (HPH) who contracted the virus through blood products. This cross-sectional, observational study assessed patients' mood states and the factors associated with them among Japanese HPH to evaluate the need for psychosocial support.

Trisomy X conferring moderate hemophilia A by extremely skewed X-chromosome inactivation.

Background: Hemophilia carriers occasionally present with bleeding tendency due to skewed inactivation of normal carrying X chromosome.

Key Clinical Question: Can extreme skewing of X-chromosome inactivation (XCI) with trisomy X cause low factor (F) VIII activity and bleeding in a hemophilia carrier?.

Clinical Approach: A young female with low FVIII activity (2 IU/dL), who presented with history of frequent bleeding and variant, NP_000123.

Comprehensive comparison of global coagulation assays to differentiate lupus anticoagulant from acquired hemophilia A in patients with prolonged APTT.

There is no established method for differentiating acquired hemophilia A (AHA) from lupus anticoagulant (LA) positivity because both present with prolonged activated partial thromboplastin time. We compared various parameters of rotational thromboelastometry (ROTEM), thrombin generation assay (TGA), and clot waveform analysis (CWA) in patients with AHA (n = 10) and LA (n = 44). Compared with AHA, possible (n = 12) and definite (n = 32) LA showed significantly shorter clotting time (CT) in NATEM mode of ROTEM (> 3600 vs.

A multicenter, observational study to evaluate hemostasis following recombinant activated FVII treatment in patients in Japan with congenital factor VII deficiency.

Unlabelled: Reports describing symptoms and treatment of patients with congenital factor VII (FVII) deficiency frequently relate to patients in Europe, while only a small number describe data from Asian countries.This multicenter, prospective observational study (NCT01312636) collected data from 30 sites for 55% of patients registered in 2011 in Japan with congenital FVII deficiency treated with activated recombinant FVII (rFVIIa) for bleeding episodes and/or during surgery.The mean follow-up in 20 eligible patients was 11 months (range 1-49 months).

The length of the sanitary napkins can be used as a handier index than pictorial blood loss assessment chart to predict the heavy menstrual bleeding.

Aim: Many women with inherited bleeding disorders are not diagnosed because of a lack of appropriate indicators. This study aimed to assess the predictability of the pictorial blood loss assessment chart (PBAC) as an indicator of menorrhagia and identify an easy indicator of menorrhagia resulting from bleeding disorders.

Methods: A multicenter study enrolled 9 patients with von Willebrand disease (VWD), 23 hemophilia carriers, and 71 controls aged 20-45 years who completed PBACs for two menstrual cycles as well as questionnaires.

In vitro validation of chromogenic substrate assay for evaluation of surrogate FVIII-activity of emicizumab.

[Introduction] Emicizumab, a bispecific antibody mimicking activated factor VIII (FVIII), is increasingly used in prophylaxis against bleeding in hemophilia A. Human factor-based chromogenic substrate assay (hCSA) shows concentration-dependency between emicizumab and reported FVIII activity. However, the assay measurement settings have not been optimized for emicizumab, and the reported FVIII activity cannot be directly referred as surrogate FVIII activity.

Prolonged α-thrombin-related activation and delayed active protein C-associated degradation confer mild phenotype in a patient with severe hemophilia A with F8 p.H118R.

In hemophilia A, bleeding mostly correlates with factor VIII activity (FVIII:C), although some patients show discrepancy in bleeding severity and FVIII:C. We report a novel procoagulant mechanism associated with F8 p.H118R (c.

Analysis of nationwide hemophilia care: A cohort study using two Japanese healthcare claims databases.

Background And Aims: In many developed countries, hemophilia care is provided by specialized centers which can offer standardized high-quality care for patients and collect data for patient registries. However, in countries with less centralized provision of hemophilia care, registry data lacks accuracy and medical care is inconsistent among providers. Claims databases can be an alternative for obtaining nationwide data on hemophilia care, and we applied this approach to evaluate inequalities in hemophilia care in Japan.

Coagulation assay discrepancies in Japanese patients with non-severe hemophilia A.

Patients with non-severe hemophilia A often show discrepancies in factor VIII (FVIII) activity. However, information on variant-specific coagulation assay characteristics in Japanese patients is limited. Pathogenic variants were classified into three groups, thrombin-cleavage site (TC), A1-A2-A3 interface (IF), and non-discrepant, with reference to previous studies.