Hemophilia Need Not Be Severe to Affect Life Quality for Men and Women

Mild-to-moderate hemophilia has a significant impact on the physical and psychosocial health, and life quality, of both men and women, according to a recent study.

Findings also highlighted the unmet needs of these patient groups, supporting better guidelines and quality of care for people with mild-to-moderate disease, its researchers noted.

The study, “Impact of hemophilia on patients with mild‐to‐moderate disease: Results from the P‐FiQ and B‐HERO‐S studies,” was published in the journal Hemophilia.

Spontaneous internal bleeding episodes as well as bleeds following an injury or surgery that are slow to naturally resolve are known to people with hemophilia. These bleeding events can limit patients’ activities and cause painful damage to their joints.

As such, hemophilia can have a severe impact on quality of life, affecting patients’ physical, psychological, social, and economic well-being. While several studies have assessed its affects on patients and their caregivers, most focused only on men or those with severe forms of the disease.

Since previous studies highlighted that joint bleeding occurs in people with mild-to-moderate hemophilia, including girls and women, it is critical to understand the unmet needs of all those with these milder disease forms to improve their care.

A research team from various U.S. centers and universities, the National Hemophilia Foundation, and Novo Nordisk, assessed the overall impact of hemophilia and the unmet needs of people with mild-to-moderate disease, including women.

They analyzed data on these patients, who were subsets of one of two studies sponsored by Novo Nordisk: the P‐FiQ study (NCT01988532) and the B‐HERO‐S study (NCT02568202).

The P‐FiQ study assessed the impact of pain on functional impairment and health‐related quality of life of 381 men with hemophilia of any degree of severity in the U.S. who experienced joint pain or bleeding.

B‐HERO‐S evaluated the psychosocial needs of adults and children, including women and girls, with hemophilia B of any severity by recruiting 299 adult patients and 150 caregivers of pediatric patients.

In both studies, participants completed a battery of validated patient-reported outcome measures to assess overall health status, functional abilities, physical activity, and pain. Joint health was only assessed in the P‐FiQ study, while depression and anxiety were evaluated only in the B‐HERO‐S study.

In the P‐FiQ study, participants’ median age was 34; 16% had mild hemophilia and 13% had moderate disease.

In the B‐HERO‐S study, the median age of adult patients was 39 and of caregivers 35. Nearly 30% of these patients were women, 25% had mild hemophilia, and 63% had moderate disease. Most (77%) caregivers were mothers of children with hemophilia (median age of 10). Of those children, 18% had mild hemophilia and 56% had moderate hemophilia.

Results showed that on-demand treatment was the most common form of treatment in mild-to-moderate hemophilia patients in the P‐FiQ study (53–86%). In contrast, most adults with hemophilia B in B-HERO-S received some form of routine treatment to prevent bleeds (63% with mild disease, and 86% with moderate disease).

Notably, prophylaxis (preventive treatment) was also the most common treatment approach for women with hemophilia B (85%) and children with moderate disease (70%), but not for children with mild hemophilia B (26%).

The high proportion of women reporting to use prophylactic therapies could be due to the fact that women who participated in the study were more likely to be those with more bleeding episodes, the researchers noted.

According to B-HERO-S results, difficulties in accessing treatment were reported by about half of all patients, and by 72% of women. Problems in accessing hemophilia treatment centers were reported by 17% of trial participants, most being adults with moderate disease.

Hemophilia was also found to have a considerably negative impact on patient’s health-related quality of life and functional abilities across a spectrum of disease severity, with more than 78% of patients in both studies reporting pain.

Anxiety and depression were common among adult patients and caregivers of children with moderate hemophilia B, with more than 80% of those in B-HERO-S stating the disease negatively affected their relationships.

No major differences in hemophilia’s impact were observed between men and women.

These findings highlighted that hemophilia negatively affects the physical and psychosocial domains and overall life quality of people of either sex with mild-to-moderate disease.

“These patients experience difficulty accessing treatment resources and face multiple barriers when it comes to participation in recreational activities, work, education and mental and emotional health,” the researchers wrote.

“These findings add to the growing body of literature supporting the need for improved guidelines and increased quality of care in mild‐to‐moderate hemophilia patients,” they added.

Researchers’ suggestions to address the unmet needs of this patient population include increasing awareness of bleeding issues and the psychosocial impact of the disease, and updating guidelines with key goals and timelines, appropriate physical activity recommendations, and information specific to women.

They also noted that community engagement programs could help educate and encourage patients to seek treatment for bleeding episodes, and that proactive planning could help patients avoid not having treatment at hand when needed.

Novo Nordisk developed a number of approved hemophilia therapies, including  NovoSeven, a recombinant (engineered) activated factor VII;  Rebinyn (nonacog beta pegol; Refixia in the EU), a recombinant coagulation factor IX to treat adults and children with hemophilia B; and Esperoct (turoctocog alfa pegol), approved in the U.S. for both prophylactic and on-demand bleeding control in adults and children with hemophilia A.