Similar quality-of-life problems reported by hem A and B patients
Patients report joint pain significantly hurting their quality of life
Despite preventive treatment, people with hemophilia A or B report lower quality of life, mainly due to poor joint health, a study in Sweden shows.
No significant difference was found between these two patient groups, who report high frequency of pain, mobility problems, and mental health issues like anxiety and depression.
“This indicates that areas of insufficient care exist, and we present data suggesting that depression and anxiety may be unrecognized and undertreated” in people with hemophilia B, researchers wrote.
The study, “No difference in quality of life between persons with severe haemophilia A and B,” was published in the journal Haemophilia.
126 people with severe hemophilia A and B were included in study
Hemophilia is a bleeding disorder caused by a deficiency in coagulation factor VIII (hemophilia A) or coagulation factor IX (hemophilia B), which are required for proper blood clotting. Hemophilia A is more common and more widely studied than hemophilia B.
The main disease symptom is traumatic and spontaneous bleeding. This might include internal bleeding, particularly in the joints, which can lead to pain and impaired mobility, affecting the patient’s daily life.
One of the main treatments for hemophilia is replacement therapy, which supplies the clotting factors to hemophilia patients, FIX in the case of hemophilia B patients.
The B-NORD study was designed to better understand hemophilia B and how it compares with hemophilia A, and to help improve the care of hemophilia B patients. The study enrolled patients registered at hemophilia treatment centers in Denmark, Finland, Norway, and Sweden, and who had a diagnosis of severe hemophilia B.
Control participants had severe hemophilia A and were matched by age, gender, and treatment (on demand or preventive).
In total, 126 people with hemophilia (63 with hemophilia B and 63 with hemophilia A), including five children 15 to 17 years old, completed a questionnaire to evaluate their quality of life. The mean age was 40 years in the hemophilia B group, and 41 years in the hemophilia A group.
We describe a well-treated haemophilia population with the majority of patients on prophylaxis but, despite this, impaired [quality of life] is reported with a high frequency of pain, mobility problems and anxiety/depression.
Nearly all patients were on preventive treatment
Nearly all (98%) hemophilia B patients were on preventive treatment, or prophylaxis, with factor replacement therapy, and hemophilia A patients were matched accordingly.
Quality of life was assessed by the EQ-5D questionnaire, which includes a descriptive system to evaluate five aspects of the patients’ lives: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. The results in each dimension — which comprise three levels: no problems (level 1), some problems (level 2), and extreme problems (level 3) — were then combined into an index value that reflects overall health.
The questionnaire also includes the EQ visual analogue scale (VAS), a scale that indicates the “overall health state today” and provides additional data on patients’ assessment of their overall health.
A high proportion of patients reported problems with mobility (46% of those with hemophilia B, and 44% with hemophilia A), and more than half identified pain/discomfort (62% of those with hemophilia B and 56% with hemophilia A). Anxiety/depression was reported by 33% of the patients with hemophilia B, compared to 17% of those with hemophilia A.
The majority of patients who reported problems classified them as “some problems” (level 2), with a small proportion classifying them as “extreme problems” (level 3; 1.6% to 3.2%).
No significant differences were detected between the two groups in the different questionnaire dimensions. Also, no differences were observed with the EQ-5D index (mean of 0.80 for patients with hemophilia B and 0.83 for those with hemophilia A) and EQ-VAS value (mean of 70 for the hemophilia B group vs. 77 in the A group).
Older age linked to lower quality-of-life scores
The impact of joint health, age, and body mass index (BMI, a measure of body fat) on quality of life were then analyzed by merging patients into one group.
Older age correlated with a higher score on the Hemophilia Joint Health Score (HJHS) tool that evaluates joint health, with higher scores indicating more severe joint damage. Older age also correlated with lower scores on quality-of-life questionnaires.
The indexes of quality of life were significantly associated with joint health, “confirming that impaired joint health significantly decreases [quality of life],” the researchers wrote.
Specifically, each increase by one HJHS score was associated with a 0.003 decrease in the EQ-5D index score, and with a 0.37 decrease in the EQ-VAS score.
No association was found between BMI and quality-of-life scores. There were also no significant differences in the quality of life reported by patients with hemophilia B treated with standard or extended half-life products (clotting factors with extended half-life, meaning they are active for a longer period of time).
“We describe a well-treated haemophilia population with the majority of patients on prophylaxis but, despite this, impaired [quality of life] is reported with a high frequency of pain, mobility problems and anxiety/depression,” the team concluded.
This indicates insufficient care in some areas, particularly for depression and anxiety, which might be “unrecognized and undertreated” in patients with hemophilia B.
Thus, “increased awareness among the staff at [hemophilia treatment centers] as well as attention and responsiveness to signals of ill health are important first steps to improve the [quality of life] of our patients,” the researchers wrote.
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