Hemophilia patients still face unmet health needs despite treatment
Issues include breakthrough bleeding episodes, ongoing joint problems
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- Despite preventive treatment, many with hemophilia still face breakthrough bleeds, joint issues, and activity limitations.
- Patients often require caregiver support for medication and transportation, impacting independence.
- New patient-centered treatments are needed to better prevent bleeds and improve quality of life.
People with hemophilia still experience significant unmet health needs despite receiving preventive treatment, according to an international study.
“Despite current treatment, many [people with hemophilia] continue to experience limitations to their daily activities, productivity, and physical independence,” researchers wrote. Specific problems that emerged in the survey included breakthrough episodes of bleeding, ongoing joint problems, and difficulty participating in sports and leisure activities.
The team suggested that drug developers should be aware of these ongoing needs when designing new treatments.
“A [patient-centered] approach is needed to ensure clinical effectiveness can be balanced with lifestyle priorities,” they wrote.
The study, “Real-World Unmet Treatment Needs for Patients With Haemophilia: Results From the Global Adelphi Disease Specific Programme Database,” was published in Haemophilia. Novo Nordisk, which markets several preventive medications for hemophilia, funded the study.
Preventive therapies changed course of hemophilia for many people
Hemophilia is a blood disorder that causes easy and excessive bleeding. It occurs due to missing or defective clotting factors, a family of proteins that help blood form clots. Modern preventive, or prophylactic, treatment options can help ease symptoms and reduce the risk of bleeds if taken regularly.
One category of approved preventive medications is factor replacement therapies, which provide the body with the deficient clotting factor. Some factor replacement therapies have an extended half-life (EHL), meaning they remain in the bloodstream longer than standard half-life (SHL) therapies. This can enable less frequent dosing.
Nonfactor therapies, another type of prophylaxis, help restore the body’s ability to form a healthy blood clot without replacing missing clotting factors.
Preventive therapies have changed the course of hemophilia for many people worldwide. However, “following global treatment advancements, real-world data on residual burdens on patients remain limited,” the researchers wrote.
In this study, the team aimed to identify concerns that persist among people with hemophilia, caregivers, and doctors despite the use of prophylaxis. To do this, they used data from a large, international survey of doctors who treat people with hemophilia. A subset of these patients and their caregivers also completed a survey.
In total, 283 doctors reported on 2,227 of their patients — about 70% with hemophilia A and the remainder with hemophilia B. Additionally, 487 patients and 137 caregivers participated in the survey study.
Most participants were receiving either SHL or EHL factor replacement therapy. Some hemophilia A participants were on a nonfactor therapy instead. Participants’ average age was 27.1.
‘Available therapies reduce, but do not eliminate, bleeding risk’
Doctors reported that they were partially or completely satisfied with treatments for about 90% of their patients. However, they still cited several concerns.
“Common reasons for lack of physician satisfaction across all therapy types included administration/dosing issues and lack of efficacy,” the researchers wrote.
Among both doctors and patients, treatment satisfaction tended to be lowest for SHL factor replacement therapies. Satisfaction rates were highest for nonfactor therapies for hemophilia A.
Despite ongoing treatment, 57% of participants had experienced at least one bleeding episode in the past year. These rates were highest among people aged 12 to 17.
“This indicates that available therapies reduce, but do not eliminate, bleeding risk, illustrating the need for treatments that can prevent bleeds in more patients,” the researchers wrote.
Additionally, nearly half of the patients continued to experience joint problems related to hemophilia. People older than 30 experienced the highest rates of joint pain, potentially reflecting cumulative damage throughout life.
These findings highlight persistent unmet needs and may help inform strategies to improve education, awareness, and access to evolving therapeutic options for people with haemophilia.
In a quality-of-life survey, people with hemophilia reported that the disease limited their participation in sports and leisure activities. Relatedly, many reported avoiding contact sports or excessive physical activity. This suggests that concerns around bleeding remained.
“Given the benefits of physical activity on functional ability and mental health, these self-imposed limitations could lead to long-term consequences for both,” the team noted.
Most participants, regardless of hemophilia type or medication category, required caregiver support. The most common reasons for needing this support were assistance with medication management and transportation. Treatment strategies that are easier to manage or require less travel might support more independence for people with hemophilia, according to researchers.
“These findings highlight persistent unmet needs and may help inform strategies to improve education, awareness, and access to evolving therapeutic options for people with haemophilia,” the researchers wrote.
The researchers noted several limitations, including that the data were self-reported rather than based on medical records. Differences in treatment access in the surveyed countries may also have influenced results. However, the international design was also a potential strength, helping the study reflect the global hemophilia population.
Bringing patient voices into conversations about treatment could help improve satisfaction, the team suggested.
“Balancing clinical effectiveness with lifestyle priorities requires [patient-centered] care incorporating open communication about treatment preferences and shared decision-making,” they wrote.
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