Key Findings: Study Shows That Regular Treatment to Prevent Bleeding Episodes Is Used Less Frequently Among Adults Than Children with Hemophilia B

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Therapies and healthcare delivery for men and boys with hemophilia have improved over the last five decades. Yet people affected by hemophilia still experience significant health challenges, such as bleeding into joints, which can cause issues with the ability to move freely and easily.

It is important to monitor and learn more about the health of people with hemophilia in order to better address these challenges. This knowledge helps to develop programs to reduce illness and death from hemophilia.

About the Study

Researchers from the Centers for Disease Control and Prevention (CDC) and the U.S. Hemophilia Treatment Center Network analyzed data from a health monitoring system that tracked care and health outcomes of boys and men with hemophilia in the United States. The study aimed to examine

The use of prophylaxis (regular treatment to prevent bleeding episodes) among those with hemophilia B;
Differences in prophylaxis use among subgroups of the population based on age, race and ethnicity, type of health insurance, body weight and other characteristics; and
Which characteristics were most likely to affect whether a person was using prophylaxis to prevent bleeding episodes.

Researchers studied information collected from 2,428 male patients between 2 and 79 years of age with moderate or severe hemophilia B. These patients received care at U.S. hemophilia treatment centers from 1998-2011. Information was collected from each patient’s most recent clinic visit in order to look at how prophylaxis use varied according to each patient’s characteristics.

We invite you to read the scientific summary of the study here .

What is Hemophilia

Hemophilia is an inherited bleeding disorder in which the blood does not clot properly. This can lead to spontaneous bleeding as well as prolonged bleeding following injuries or surgery.

Blood contains many proteins called clotting factors that can help to stop bleeding. People with hemophilia have low levels of either factor VIII (“factor eight”) or factor IX (“factor nine”). Because of the way hemophilia is inherited, the condition predominantly affects males, while females with the genetic change in the clotting factor are typically referred to as carriers. Hemophilia carriers can have an increased bleeding tendency, such as heavy menstrual bleeding.

Main Findings from the Study

One-third of these patients were on prophylaxis treatment to prevent bleeding, with the greatest prophylaxis treatment use among patients younger than 20 years old. Among patients with hemophilia B using prophylaxis, nearly three-fourths were younger than 20 years.
Patients in both age groups (younger than 20 years and 20 years or older) with severe hemophilia B were more likely to be on prophylaxis than those with moderate hemophilia B (nearly 10 times more likely for those patients with severe hemophilia B less than 20 years old, and nearly 6 times more likely for those 20 years or older).
Nearly all patients using prophylaxis had either private or public insurance.
Racial or ethnic differences in prophylaxis use were mostly small, with the exception that Hispanic patients with hemophilia B were almost twice as likely to use prophylaxis, compared to non-Hispanic white patients.
Among patients age 20 years or older, overweight and obese men were about half as likely as their normal weight counterparts to be using prophylaxis.

Public Health Message

Among males with hemophilia B, lower rates of prophylaxis use among adults compared to children may result in worse joint health as the population ages.

Critical Gaps in Information & Future Work

Gaps in Information

A number of observations from this study need further investigation to determine why they occur:

Prophylaxis, through the regular infusion or injection of clotting factor treatments into a vein to prevent bleeding, results in better joint health. However, this study found that not everyone with hemophilia B used prophylaxis, especially adults (compared with children) and those with moderate disease (compared with severe disease).
Compared to prophylaxis rates reported in males with hemophilia A, those for males with hemophilia B are lower, and the reasons for this are not completely understood.
It is not known why some patients with hemophilia do not use prophylaxis, and what might be the barriers that keep them from using this treatment approach.

Future Work

More work in this area could help explain why some boys and men with hemophilia do not use prophylaxis, which could be useful for developing strategies to successfully change this behavior. Additional work could help define the factors that increase the risk for joint bleeds, which could then aid the development of treatment strategies for men with hemophilia who have a higher risk for joint disease.

More Information

Please visit the following links for more information:

Hemophilia

Treatment

Public health monitoring programs for hemophilia and other bleeding disorders

Reference for the Study

Ullman M, Zhang QC, Grosse SD, Recht M, Soucie JM; Hemophilia Treatment Center Network Investigators. Prophylaxis use among males with haemophilia B in the United States. Haemophilia. 2017 Aug 6. doi: 10.1111/hae.13317. [Epub ahead of print]