Many Patients With Hemophilia Experience Functional Declines Over Time

Functional ability declined in more than one-third of individuals with hemophilia over a median 3.5 years, new research shows.

Previously, data on the course of physical functioning and any decline that might take place was lacking, despite the World Federation of Hemophilia advising regular musculoskeletal assessments for patients, authors explained.

The latest study, published in Haemophilia, found that among the 104 patients assessed, nearly 56% remained stable over the course of 3.5 years, while the functional ability of 8.7% improved. Thirty-six percent of patients exhibited a decrease in functional ability.

Regular musculoskeletal assessments, “enables clinicians to detect changes early and enable adjustments in personalized health care when needed,” the researchers explained.

To better describe changes in perceived limitations among patients, they collected data from medical health records of regular check-ups of those with moderate to severe hemophilia at 2 time periods.

Data from individuals who experienced a major joint or muscle bleed less than 6 weeks before the checkup were excluded. Participants were also excluded if they underwent total knee or total hip replacement less than a year before the check-up or underwent an ankle arthrodesis less than 6 months before the check-up.

Any perceived limitations were measured via the Haemophilia Activities List (HAL). At the first time point, the median HAL score was 79.5. At the second time point, it was 74.2.

Results showed a body mass index (BMI) greater than 30 kg/m² may be an important factor that could negatively influence a change in perceived functioning among patients. Worse joint status at baseline and less limitations in activities at baseline were also associated with more deterioration. However, the factors included in the study meant researchers could only explain a small part of this decline.

“It remains important to monitor functional ability in all adults with moderate and severe hemophilia every 3 to 5 years,” they wrote.

Hemophilia is characterized by a limited ability to form blood clots, which can lead to increased bleeding after surgeries and easy bruising. In addition, bleeds in joints and muscles can lead to limitations in activities and may lead to hemophilic arthropathy. This joint condition results in chronic pain, reduced range of motion, and muscle atrophy, the authors wrote.

“Consequently, this arthropathy limits a person's quality of life and ability to participate in society,” they said.

To the authors’ knowledge, this study was the first to investigate changes over time of perceived limitations in activities for adults with hemophilia.

At baseline, the mean patient age was 42, and 85% had severe hemophilia. The more than 100 participants in the study marks a strength of the research, as the disease is considered rare.

Because the study was carried out at a single center in the Netherlands among patients who attended regular clinical visits, the findings might not be generalizable to other countries with a different health care setting, the authors cautioned.

In addition, baseline assessments were carried out in 2014, prior to the introduction of new, less burdensome treatment options for patients, they added.

Overall, “The current study highlights the importance to raise awareness in [people with hemophilia] about maintaining a healthy weight as it showed that people with a BMI over 30 have a higher probability to experience a meaningful decline in perceived functioning,” the researchers concluded. “Future research in this field should focus on longitudinal data collection with 3 or more measurements in time.”

Reference:

Blokzijl J, Pisters MF, Veenhof C, Schutgens REG, and Timmer MA. Functional decline in persons with haemophilia and factors associated with deterioration. Haemophilia. Published online March 28, 2023. doi:10.1111/hae.14783