Fig. 1: Phenotypic and dysmorphic features of patient 1 (A and B),...

Self Reported Physical Activity Not Significantly Different Across Different Severities Of Hemophilia

1. In this cohort of individuals with hemophilia, the physical activity levels and step count did not significantly differ between severity phenotypes. 

Evidence Rating Level: 1 (Excellent) 

Hemophilia is a bleeding disorder characterized by blood that does not form clots. It can be divided into hemophilia A, due to a deficiency in factor VIII or hemophilia B, a deficiency in factor IX. Physical activity (PA) is recommended for individuals with either type of hemophilia, although the amount of PA is still under investigation. In patient populations where joint pain can limit movement, one way to assess overall strength is by measuring grip strength. Research has shown that grip strength can be a predictor of fall risk. Based on this information, the goal of the study was to determine how the severity phenotype influences the level of PA in patients with hemophilia (PwH), and understand the relationship between PA and bone quality, specifically bone mineral density (BMD) and trabecular bone mass (TBS) and lean mass. Individuals included in the study were adult males with mild, moderate, and severe hemophilia A or B. Each eligible participant tracked their PA with an electronic activity tracker worn for seven consecutive days while also utilizing an activity diary. A total of 223 PwH (median age 43 [30, 56] years) were included in the study. Among these individuals, 45 had mild, 46 had moderate, and 132 had severe hemophilia A or B. The amount of step activity/day was 7392 [4981, 10579] within the entire cohort and did not significantly differ among the hemophilia severity types (p=0.162). Similarly, the subjective PA did not differ significantly between the phenotypes (p=0.459). There was a positive correlation between lean body mass and BMD (rho=0.309, p<0.001), but not between lean body mass and TBS (rho=-0.11, p=0.134). While there was a positive correlation between PA and TBS (rho=0.202, p=0.005). There was also a positive correlation between handgrip strength and BMD (rho=0.361, p<0.001), TBS (rho=0.221, p=0.021), and lean mass (rho=0.287, p=0.003). Overall, self-reported PA and step activity did not differ significantly between the severity groups. 

Click to read the study in PLOSONE

Image: PD

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Costs Of Hemophilia With Inhibitors Are High, Warranting Better ManagementAMCP Annual 2025

The FDA has recently approved two new prophylactic treatments for people with hemophilia who develop antibodies to clotting factors.

An analysis of costs and healthcare resource utilization found that patients with hemophilia A or B with inhibitors (HAwI and HBwI) incurred substantial costs, with pharmacy and medical costs representing the highest contributing factor, according to a poster presented at the annual meeting of the Academy of Managed Care Pharmacy (AMCP) in Houston.

The study was conducted using the Komodo Health Claims Database on data from January 1, 2016, to December 31, 2023. During the analysis, most patients experienced at least one bleeding event. Moreover, all-cause costs were found to be twice as high for those with HAwI compared with HBwI. In both groups, the healthcare utilization was high, with nearly two-thirds requiring an emergency visit and a quarter to a third requiring an inpatient stay.

"People with hemophilia and inhibitors incur a substantial economic burden," the study authors, who are from Novo Nordisk, wrote in their poster. "There is an urgent need for improved prophylactic options and targeted interventions to optimize patient care and mitigate costs."

Hemophilia is an X chromosome-linked congenital bleeding disorder with joint damage as a consequence of the disease. As an X-linked disorder, it affects males much more often than females. Hemophilia A is more common than hemophilia B. The treatment for hemophilia has been factor replacement therapy for the missing clotting factors VIII and IX; however, in some patients, antibodies develop that inhibit clotting factors VIII and IX, limiting the efficacy of the replacement therapy. They are referred to as inhibitors. At the time of the study, the authors noted, there were no additional prophylactic treatments approved for HBwI and only one approved for those with HAwI.

Since the study was conducted, however, two new agents have gained approval. In December 2024, the FDA approved Novo Nordisk's Alhemo (concizumab) to prevent or reduce frequent bleeding episodes for patients with HAwI and HBwI. In March 2025, the FDA approved Sanofi's Qfitlia (fitusiran) for both types of hemophilia. The agent is approved to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 12 years of age or older with or without the patient being on a factor VIII or IX inhibitor.

Study Details

For the study presented at AMCP, the investigators sought to examine the full economic impact of hemophilia with inhibitors in the United States using claims data for 84 patients with HAwI and 16 with HBwI. The median age of those with HAwI was 23.0, and it was 21.8 years for those with HBwI. Most of the enrolled patients were male, and 33% of those with HAwI and 50% of those with HBwI reported joint-related problems. Patients were well distributed across the United States.

At least one bleeding event was experienced by 69% of those with HAwI and 75% of those with HBwI. For the HAwI group, 49% were on prophylaxis. Of those on a prophylaxis (n = 41), at least one bleed was experienced by 49% of patients. The annual bleed rate (ABR) was 1.3 for HAwI and 1.8 for HBwI. For those with HAwI on prophylaxis who experienced one or more bleeds, the ABR was 1.6.

For the HAwI group, the annual mean length of stay (LOS) was six days for the 26% of patients who required inpatient care. All patients with HAwI required an outpatient visit during the study period, and 61% required an emergency visit.

For the HBwI group, the mean LOS was 13 days for the 38% of patients requiring inpatient care. In this group, 94% of patients were also treated in the outpatient setting, and 63% required an emergency visit.

The mean all-cause costs for those with HAwI was $359,304, and the mean pharmacy costs were $206,479. For these patients, pharmacy costs represented 57% of the total costs. For those with HBwI, the mean all-cause costs were $156,789, and the medical costs were $152,784, representing 97% of the total costs.

Hemophilia Patients' Bone Health Screening Rates Low At US HTCs

Despite high rates of bone health issues in hemophilia and von Willebrand disease (VWD), the routine use of DEXA scans remains low across hemophilia treatment centers (HTCs) in the U.S., a study reports.

DEXA, or dual-energy X-ray absorptiometry, is a type of imaging test that uses low-dose X-rays to measure bone density and check for osteoporosis, which is where bones become weaker and more likely to break.

"We show that the rates of screening for osteoporosis were suboptimal, at less than 15% … at specialized HTCs," the researchers wrote, noting that the rates were calculated based on responses from a fraction of treatment centers, which may "not necessarily reflect practices at all HTCs."

The study, "Bone Health Screening in Persons With Bleeding Disorders: A Survey of United States Haemophilia Treatment Centres," was published in Haemophilia.

Hemophilia is caused mainly by mutations in genes that provide instructions to produce specific clotting factors, or proteins needed for the blood to clot, resulting in poor clotting. People with hemophilia commonly have heavy and prolonged bleeding episodes. In severe cases, joint bleeding may occur which may also contribute to poor bone health.

Evaluating rates of bone health screening

Several studies have reported that people with hemophilia have significantly lower bone mineral density than people without the disease. High rates of osteoporosis and bone fractures have also been reported with VWD, another bleeding disorder marked by the lack or dysfunction of von Willebrand factor, a blood clotting protein.

"Despite reports of elevated rates of osteoporosis and fractures in persons with hemophilia … and von Willebrand disease … routine bone health screening using [DEXA] scans is not consistently implemented for this population across all [HTCs] in the United States," wrote the researchers, who surveyed all federally funded HTCs in the U.S. To determine the rates of bone health screening using vitamin D and DEXA scans in hemophilia and VWD. The survey included nine multiple-choice questions to access current practices used to evaluate bone health. One member from each HTC could complete the survey.

Of the 147 HTCs where the survey was distributed, 66 completed it, yielding a response rate of about 45%. Most respondents were physicians and worked in HTCs that treated both adults and children with bleeding disorders.

Among the responding centers, 21 (31.8%) performed routine screening for vitamin D deficiency during annual visits, while nine (13.6%) performed regular bone health screenings using DEXA scans. DEXA scans were ordered by physicians at a center in 5 of the 9 (55.6%) HTCs where bone health screenings were used. In the remaining four (44.4%), people with bleeding disorders had been referred by primary care providers or specialists for bone health evaluation through DEXA scans.

DEXA scans were more commonly ordered for people with hemophilia, most commonly those with severe forms of hemophilia A and B. Scans were requested less frequently for VWD and milder forms of hemophilia.

The most common indications to perform DEXA scans included HIV infections (77.7%), low physical activity or immobility (66.7%), fractures after a minor fall or injury in patients with bleeding disorders under age 50 (55.5%), and vitamin D deficiency (55.5%).

"In light of the growing burden of an aging population with hemophilia and VWD (and the broad scope of bleeding disorders), future efforts should focus on gaining a deeper understanding of barriers to bone health in [people with hemophilia and VWD] and developing guidelines for bone health assessment, with a specific focus on measuring vitamin D levels and performing DEXA scans," the researchers wrote.

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