Third of hemophilia patients in study perceived declines in function

Those most likely affected were obese or had worse joint health

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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About one-third of moderate-to-severely affected people with hemophilia perceived declines in their daily functional abilities over a period of three to five years, a Dutch study found.

While no strong risk factors for such declines were identified, patients more likely to be affected were those who were obese, had worse starting joint health, or higher function at the study’s start.

As noted by the authors, it is important to regularly monitor patients’ functional abilities “in order to timely identify patients that may need an intervention.”

The study, “Functional decline in persons with haemophilia and factors associated with deterioration,” was published in Haemophilia.

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Pain, mobility issues affect quality of life in people with hemophilia

People with hemophilia lack factors needed for proper blood clotting, leading to an increased susceptibility for bleeding episodes. Bleeding also can occur internally, leading to chronic joint disease which causes pain and mobility issues that limit quality of life.

In recent years, “there is an increasing recognition for the importance to look beyond bleeds” when assessing hemophilia patients, according to the authors.

Current guidelines from the World Federation of Hemophilia suggest that in addition to looking at bleed rates and and joint function, doctors should assess patients’ functional limitations in daily life.

“Knowledge on these topics can enable healthcare providers to identify people at risk of a decline in an early stage and develop interventions with the aim to detain this decline,” the researchers wrote.

In the study, researchers examined changes to perceived functional limitations over time among 104 patients with moderate or severe hemophilia A or B seen at a medical center in the Netherlands.

Most participants, with a mean age of 42.1, had severe hemophilia (85%), hemophilia A (92%), and were receiving preventive treatments at the study’s start (73%).

The main outcome measure was a change in perceived limitations in activities as assessed by the Hemophilia Activities List (HAL). The assessment contains 42 questions relating to daily activities; scores range from 0-100 with a higher score reflecting better function.

HAL scores were collected once at the study’s start (baseline), between February 2014 and July 2016, and again at a single time point a median of 3.5 years later.

The median HAL total score was 79.5 at baseline and 74.2 at the follow-up assessment, and there was an overall mean decline of 5.9 points.

More than one-third of participants (35.6%) saw a HAL decline of at least 10.2 points, reflecting a clinically meaningful deterioration. The remaining patients either saw no meaningful change (55.8%) or an improvement (8.7%).

When looking at factors that could potentially influence functional declines, none seemed to be major contributors.

Still, patients who were obese — with a body mass index of more than 30 — saw a nearly 11-point drop in HAL scores compared with those of healthy weight.

Higher functioning patients had greater declines

Moreover, patients with higher HAL scores at baseline saw greater declines over time. For every 1 point higher HAL at baseline, patients saw an extra .34 point decline later on.

Likewise, higher starting Hemophilia Joint Health Scores, reflecting worse joint function, were associated with greater HAL declines.

The findings were generally similar when looking specifically at the upper extremity subscale of HAL, which includes the arms, wrists, and hands. In the lower extremities — legs, ankles, and feet — HAL declines also were linked to worse joint health and higher baseline HAL scores, but not with obesity.

However, the models “derived from current research only explained a small part of the variance in changes in limitations in activities of [people with hemophilia],” the researchers wrote, adding that “precise identification of the people most at risk is not possible.”

Thus, routine monitoring of functional ability is important for all patients. Maintaining a healthy weight may also be one important factor for preventing functional limitations later on.

Still, “a study with more measurement points over a longer time period is needed,” the researchers wrote.

Moreover, the authors emphasized data were collected among a patient group with routine access to clinical visits and preventive care, and “might therefore not be generalizable to countries with a different healthcare setting.”