Hemophilia

Treatment options have improved care, but also increased the need for patient education.

About a quarter of joints examined had at least 1 joint bleed.

However, the patients also said they worried about insufficient care coordination between specialists and other care providers.

The projected cost reductions held both for patients switching from a different extended-half-life product and for patients switching from a standard-half-life product.

Little is known about the risk of bleeding in patients with bleeding disorders who undergo major surgeries or outcomes for those managed perioperatively at hemophilia treatment centers.

Recommendations are lacking on strength exercise intensity, tolerability, and adverse effects for patients with severe forms of hemophilia.

Study authors determined that regular musculoskeletal assessments can help facilitate personalized care for patients who have hemophilia, in that they can detect changes early.

Hemarthropathy in multiple joints is a common feature of more severe forms of hemophilia, with the ankle joint often being reported as the main site of pain.

This analysis found that the 3000 patients with hemophilia had higher rates of pain, depression, and anxiety medication use.

Specific dosing regimens of treatments prescribed by blood specialists have not been widely studied, despite the advent of newer hemophilia therapies, the researchers said.

The measure will allow for a more complete picture of treatment burden and how it affects things like health-related quality of life and adherence, explained researchers.

Prior to this study, explained researchers, data on the long-term course of osteoporosis, as evaluated through bone mineral density (BMD) in these patients, have been lacking.

A group of researchers detailed a case of acquired hemophilia A (AHA) following infection and again following vaccination, and provided additional insights through their systematic review of available data.

These new findings suggest that variability in factor VIII (FVIII) half-life in patients, though impacted by all 3 factors, is largely attributed to variability in the clearance of endogenous von Willebrand factor (VWF).

The approach for cardiovascular disease (CVD) was based on expert opinion, personal experience, and adaptation of evidence from people who did not have hemophilia.

Researchers found that the anticoagulant effects of apixaban, betrixaban, edoxaban, and rivaroxaban were partially neutralized at an andexanet alfa dose of 50 mcg/mL and were completely neutralized at 100 mcg/mL.

The second half of 2022 brought the first gene therapies for hemophilia to the market in both the United States and abroad.

Early initiation of prophylaxis regimens and joint status monitoring over time are the most relevant aspects of evaluating treatment efficacy in this patient population, study authors concluded.

Data show that patients who have factor VIII (FVIII) inhibitor titers of 20 or higher BU/mL took longer to achieve complete responses (CR) and had lower CR rates vs patients with lower titers.

The researchers underscored the importance of their findings for a wide group of patients with inherited bleeding disorders, as most colorectal cancer (CRC) screening programs across the globe use fecal immunochemical testing (FIT).

Strength training for people with hemophilia (PwH) should consider exercise variables and pain threshold, a review finds.

Johnson & Johnson (J&J) ends late-stage HIV vaccine trial; mothers in states with abortion bans are almost 3 times more likely to die before, during, and after birth; Sanofi hopes to release a drug for hemophilia A in 2023.

The most-read hemophilia articles of the year highlighted new drug developments for hemophilia, the impact of carrier status, and opportunities for better patient education.

Safety and efficacy were the most important factors for patients with hemophilia learning about gene therapies, although more research is needed to see how collaboration between providers and patients contributes to the best outcomes.

Optimized hemophilia care requires rapid, accurate, and high-quality genetic testing, but access to these tests varies across the European Union, according to a recent study.

A recent review poses gene therapy as a novel treatment option with potential to reduce health care disparities globally.

The HHS announced an increase in individuals seeking coverage from Healthcare.gov; monkeypox will receive a name change to destigmatize the virus; a gene therapy for hemophilia was approved and is now the most expensive in the world.

The hesitation to prescribe nonsteroidal anti-inflammatory drugs (NSAIDs) to patients with hemophilia is not supported by current research, based on a recently published review, but the available studies had significant limitations.

Gene therapy is a novel approach to hemophilia A treatment that carries a substantial cost up front but may lead to cost savings compared with current prophylaxis agents in the long run.

This rare immune-related adverse event has only been previously reported 4 times in the context of immune checkpoint inhibitor (ICI) treatment.