Final Analysis Results from the AGEHA Study: Emicizumab Prophylaxis for Acquired Hemophilia A with or without Immunosuppressive Therapy.

Background:  Primary analysis of the phase III AGEHA study suggested a favorable benefit-risk profile for emicizumab prophylaxis in patients with acquired hemophilia A (PwAHA); however, only patients undergoing immunosuppressive therapy (IST; Cohort 1) were included.

Objectives:  To present final analysis results of AGEHA, including data on IST-ineligible patients (Cohort 2) and on long-term prophylaxis with emicizumab.

Methods:  For patients in both Cohorts 1 and 2, emicizumab was administered subcutaneously at 6 mg/kg on Day 1, 3 mg/kg on Day 2, and 1.

[Novel treatment strategies for acquired hemophilia A].

Acquired hemophilia A (AHA) is a bleeding disorder caused by autoantibody (inhibitor) production targeting blood coagulation factor VIII (FVIII). It is characterized by sudden onset, and often causes extensive and severe bleeding in soft tissue. Acquired hemophilia A is diagnosed when coagulation tests show normal PT, prolonged APTT, decreased FVIII activity, normal VWF activity, and positive FVIII inhibitor.

Timing of Assessment of Humoral and Cell-Mediated Immunity after Influenza Vaccination.

Assessment of the immune response to influenza vaccines should include an assessment of both humoral and cell-mediated immunity. However, there is a lack of consensus regarding the timing of immunological assessment of humoral and cell-mediated immunity after vaccination. Therefore, we investigated the timing of immunological assessments after vaccination using markers of humoral and cell-mediated immunity.

Haemophilia and cardiovascular disease in Japan: Low incidence rates from ADVANCE Japan baseline data.

Introduction: With the increasing life expectancy of people with haemophilia, the risk of cardiovascular disease (CVD) and thrombotic events has become a growing concern. Longitudinal studies on the incidence and risk factors of CVD in this population are limited, and optimal prevention and treatment strategies are yet to be established.

Aim: This study aimed to present the baseline data of a prospective longitudinal study focusing on a subset of Japanese patients with haemophilia, specifically investigated the incidence, risk factors and treatment modalities for CVD and thrombotic diseases in people aged 40 years in Japan over 10 years through the ADVANCE Japan study.

Changes in Leptin to Adiponectin Ratio After Antiretroviral Therapy: A Pilot Observational Study.

Aim: Weight gain with the use of dolutegravir, bictegravir, and tenofovir alafenamide for antiretroviral therapy has been reported. However, studies on changes in body composition and the leptin/adiponectin ratio after antiretroviral therapy initiation are limited. These factors are important because they can be used as indicators of metabolic syndrome and cardiovascular disease risk.

A prospective, multicenter, open-label phase III study of emicizumab prophylaxis in patients with acquired hemophilia A.

Background: Emicizumab is a bispecific antibody that mimics the cofactor function of activated factor (F) VIII. It prevents bleeds in patients with congenital hemophilia A regardless of the inhibitor status; however, no prospective clinical studies have been conducted for emicizumab in patients with acquired hemophilia A (PwAHA).

Objectives: To describe the primary analysis results from a prospective, multicenter, open-label phase III study evaluating the efficacy, safety, and pharmacokinetics of emicizumab in PwAHA (AGEHA; JapicCTI-205151).

The seroprevalence of neutralizing antibodies against the adeno-associated virus capsids in Japanese hemophiliacs.

Adeno-associated virus (AAV) vectors are promising modalities of gene therapy to address unmet medical needs. However, anti-AAV neutralizing antibodies (NAbs) hamper the vector-mediated therapeutic effect. Therefore, NAb prevalence in the target population is vital in designing clinical trials with AAV vectors.

Comparison between the impact of tenofovir alafenamide and that of abacavir on rapid kidney function decline: A retrospective observational study.

Objectives: To compare the impact of tenofovir alafenamide (TAF) on the slope of the estimated glomerular filtration rate (eGFR) with that of abacavir in Japanese patients living with HIV infection.

Methods: The participants in this single-centre, retrospective, observational study were Japanese patients with HIV infection who started antiretroviral therapy with TAF/emtricitabine or abacavir/lamivudine or were switched from tenofovir disoproxil fumarate/emtricitabine to TAF/emtricitabine or abacavir/lamivudine (anchor drugs remained constant) between January 2012 and December 2020. The eGFR slope was defined as the regression coefficient between eGFR and time.

Severe acute heart failure during or following cytokine release syndrome after CAR T-cell therapy.

Although cardiac dysfunction after chimeric antigen receptor (CAR) T-cell therapy has been increasingly reported, the underlying dynamics and pathogenesis are not well documented. Herein, we describe the clinical presentation and treatment for two patients who developed severe acute heart failure after CAR T-cell therapy. Both cases shared several common characteristics, including the bone marrow involvement at the time of CAR T-cell therapy and early onset of cytokine release syndrome (CRS) with fever developing on the day of CAR T-cell infusion.

Association between the pharmacokinetics of rabbit anti-thymocyte globulin and acute graft-versus-host disease in patients who received haploidentical hematopoietic stem cell transplantation.

Anti-thymocyte globulin (ATG) is an important prophylactic drug against acute graft-versus-host disease (aGVHD) after haploidentical hematopoietic stem cell transplantation (haplo-HSCT). This study analyzed the pharmacokinetics of rabbit ATG 2.5 mg/kg and its effect against aGVHD in 24 patients undergoing unmanipulated haplo-HSCT.

Effect of switching from tenofovir disoproxil fumarate to tenofovir alafenamide on estimated glomerular filtration rate slope in patients with HIV: A retrospective observational study.

Introduction: There is limited data on the effects of switching from tenofovir disoproxil fumarate (TDF) to tenofovir alafenamide (TAF) on estimated glomerular filtration rates (eGFR) slope in patients with human immunodeficiency virus (HIV) infection. This study aimed to compare the eGFR slope when administering TDF and TAF and to investigate the predictors of improvement in eGFR slope after switching from TDF to TAF.

Methods: We conducted a single-center, retrospective, observational study in Japanese patients with HIV infection who switched the antiretroviral drug from TDF to TAF.

Impact of the use of hydroxyethyl starch in granulocyte apheresis using Spectra Optia.

Objectives: To determine the impact of the use of hydroxyethyl starch (HES) in granulocyte apheresis using Spectra Optia.

Background: Granulocyte transfusion (GT) is a therapeutic option for neutropenic patients with severe bacterial or fungal infections. Recent studies in emergency medicine have shown the potential risk of using HES, which is routinely used in granulocyte apheresis to increase yield by sedimenting red blood cells.

Success and limitations of plasma treatment in pregnant women with congenital thrombotic thrombocytopenic purpura.

Background: Congenital thrombotic thrombocytopenic purpura (cTTP), otherwise known as Upshaw-Schulman syndrome, is an extremely rare hereditary disease. Pregnancy is identified as a trigger for TTP episodes in patients with cTTP.

Objectives: To investigate the ideal management of pregnant patients with cTTP.

Utility of urinary liver-type fatty acid-binding protein as a predictor of renal dysfunction in Japanese patients with HIV receiving tenofovir disoproxil fumarate with low urinary β2 microglobulin levels: a retrospective observational study.

Background: Tenofovir disoproxil fumarate (TDF) is known to reduce estimated glomerular filtration rate (eGFR). It is clinically important to identify patients at high risk for renal dysfunction as early as possible. Among the tubular markers, urinary β2 microglobulin (Uβ2MG) is a well-known biomarker of TDF-related tubulopathy.

Ischaemic events are rare, and the prevalence of hypertension is not high in Japanese adults with haemophilia: First multicentre study in Asia.

Introduction: With the increasing life expectancy of patients with haemophilia (PWH), the number of PWH with age-related comorbidities, such as ischaemic events, is increasing.

Aim: We conducted this multicentre observational study to identify the risk factors for major ischaemic events in PWH.

Methods: This study was the first multicentre observational study, conducted with the participation of five haemophilia treatment centres in Japan, conducted in ≥30-year-old adult PWH.

11β-hydroxysteroid dehydrogenase type 1 is associated with antiretroviral therapy-induced increase in low-density lipoprotein cholesterol .

Objective: To investigate the association between 11β-hydroxysteroid dehydrogenase type 1 (11β-HSD1) activity and antiretroviral therapy (ART)-induced increase in low-density lipoprotein cholesterol (LDL).

Materials And Methods: We enrolled 62 patients and used liquid chromatography-tandem mass spectrometry to measure 11β-HSD1 activity, which was expressed as a ratio of the sum of urinary tetrahydrocortisol and allo-tetrahydrocortisol concentrations to urinary tetrahydrocortisone concentration. Patient data, including baseline laboratory values, were extracted from medical records for logistic regression analyses of factors associated with LDL increase during ART.

[Diagnosis and management of von Willebrand disease].

von Willebrand disease (VWD) is an inherited bleeding disorder resulting from either a quantitative or a qualitative deficiency in the plasma glycoprotein von Willebrand factor (VWF). A diagnosis of VWD can be made when a patient presents with appropriate bleeding and VWF <30 IU/dl. However, persons with VWF levels of 30-50 IU/dl cannot be precluded from the diagnosis of VWD.

Urinary liver-type fatty acid-binding protein levels as a potential risk factor for renal dysfunction in male HIV-infected Japanese patients receiving antiretroviral therapy: a pilot study.

Renal dysfunction is recognized with increasing frequency among the non-infectious co-morbidities associated with human immunodeficiency virus (HIV) infection. Recently, urinary liver-type fatty acid-binding protein (L-FABP) was suggested to be a predictor of the progression of renal dysfunction in patients without HIV. However, little is known regarding the utility of urinary L-FABP as a predictor of renal dysfunction in patients with HIV.

A potential drug interaction between phenobarbital and dolutegravir: A case report.

In this report, we describe a human immunodeficiency virus (HIV)-infected patient in whom changes in phenobarbital (PB) dosage resulted in associated changes in plasma concentrations of dolutegravir (DTG). His plasma concentrations of DTG were 0.934, 0.