People with mild to moderate hemophilia, in particular women with the disease, face unique challenges, many that are specifically linked to a lack of information and insufficient engagement with other patients and healthcare providers, according to a panel of U.S. experts.
In a recent study, these experts proposed new strategies, such as improving access to data, increasing interaction in the hemophilia community, and providing more and better resources, they said could prove helpful in overcoming these barriers.
Titled “Identified unmet needs and proposed solutions in mild‐to‐moderate haemophilia: A summary of opinions from a roundtable of haemophilia experts,” the study was published in the journal Haemophilia.
People who have mild to moderate hemophilia experience different challenges than those with severe disease. For example, people who rarely have bleeding-related issues may be less able to recognize when one occurs, and their healthcare providers may be less likely to identify certain problems, like joint pain, as being related to hemophilia.
Despite these differences, there is very little available information on the experiences of people with mild-to-moderate hemophilia. In a recent study, researchers attempted to perform a large analysis to synthesize the available data on the burden of mild hemophilia. However, the scarce available information made such analysis impossible.
A U.S.-based panel of experts met in June 2019 to discuss the challenges faced by people with mild-to-moderate hemophilia, and to propose solutions. The expert panel included a wide range of healthcare workers, as well as patients, caregivers, and members of advocacy organizations. The panel focused on the U.S., though data from around the globe were discussed.
The panel’s conclusions were summarized in the study.
Insufficient education about the disease and a lack of involvement with the hemophilia community were highlighted as major challenges for individuals with mild-to-moderate hemophilia. According to the experts, those with less severe disease are more likely to have an incomplete understanding of it, compared with people with severe hemophilia, who must more frequently deal with symptoms and bleeds. The experts say the difference comes from being less engaged with the hemophilia community and spending less time in specialty treatment centers.
“In adolescents and young adults, patients may struggle with a lack of knowledge about the genetics of their disorder as well as how to best prioritize their care. As adults, they may experience a lack of knowledge about age‐related comorbidities [co-existing conditions],” the researchers wrote.
Many resources in the hemophilia community — for instance, published guidelines on disease management — are oriented toward people with severe disease, the experts noted.
“It is not uncommon for initiatives to focus on goals that may be specific to severe bleeding phenotypes [manifestations],” the team wrote.
“Challenges for all patients occur throughout their life, and without prior planning, issues with access to factor replacement therapy arise when trauma, emergent surgeries or scheduled procedures occur,” they wrote.
The experts also noted that mild-to-moderate hemophilia can have a substantial impact on school, work, daily activities, and interpersonal relationships. For example, they pointed out that the decision of whether or not to disclose that one has a bleeding disorder can complicate relationships with peers at work or at school, especially when health problems arise. Also, mild hemophilia can cause physical problems that make certain kinds of work or sports difficult or dangerous.
Additionally, the panel highlighted that many of these difficulties are particularly pronounced for women with hemophilia. Girls and women who have hemophilia are sometimes labeled as “just a carrier,” for example, leading to a lack of education and exclusion from the hemophilia community — even though females with hemophilia can still experience substantive symptoms. Notably, menstruation and pregnancy also can pose unique complications to women with hemophilia.
The team of experts recommended several strategies to address these challenges. Broadly, they urged that more data be collected on how mild-to-moderate hemophilia affects patients, and that resources be dedicated to increasing education within the community as well as among healthcare providers.
“Patient and caregiver education and resources could be improved with specific programming for mild‐to‐moderate haemophilia through collaborations between [hemophilia treatment centers] and the local haemophilia community,” the team wrote.
They also called for auditing of existing educational materials from advocacy organizations, and for developing better guidelines for the treatment of mild-to-moderate hemophilia. Specifically, they pointed to the need to “highlight different key goals and timelines for patients with mild‐to‐moderate haemophilia,” adding that “a focus should be placed on developing healthcare self‐management skills between clinic visits.”
The panel also called for increased engagement of women with the hemophilia community.
“Girls and women should be encouraged to join the Blood Sisterhood programme of the Hemophilia Federation of America (HFA),” the team wrote, adding that heavy menstrual and other bleeding can be tracked with the HFA Sisterhood app.
Overall, the suggested initiatives could empower these patients and lead to better health, the researchers concluded.
“We have highlighted this patient sub‐population to demonstrate and highlight the significant unmet needs and opportunities for [hemophilia treatment centers], national organizations and local communities to become partners in their disease management. If implemented, the suggested initiatives can empower these patients and will improve outcomes across all life stages,” they wrote.
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