Hemophilia A care in China has major challenges, treatment gaps
Study finds only 30% of patients received prophylactic treatment for disease
People with hemophilia A in China see major health challenges, such as frequent joint bleeding, joint malformation, and high rates of hospital visits, primarily owing to the limited use of preventive therapies, a real-world study shows.
After a bleeding episode, most patients rely on on-demand treatment, which is less effective at preventing complications. This results in higher healthcare costs and more frequent medical visits.
“This study highlights the burden of illness and varied disease management, indicating the importance of improving disease awareness and treatment accessibility for patients with hemophilia A in China,” the researchers wrote. The study, “Disease Burden and Treatment Patterns Amongst Patients with Haemophilia A in China: A Retrospective Database Analysis Study,” was published in Advances in Therapy.
Hemophilia A is a rare inherited bleeding disorder that predominantly affects males and is marked by a deficiency or dysfunction in clotting factor VIII (FVIII), a protein in blood clotting. This can cause prolonged bleeding episodes, particularly in joints and muscles, which can lead to chronic pain, progressive joint damage, disability, and a reduced quality of life.
Treatment for hemophilia A typically involves factor replacement therapy, wherein FVIII is infused into the bloodstream to restore blood clotting. It can be administered as prophylaxis, with infusions given regularly to prevent bleeds and preserve joint health, or on demand to treat acute bleeds.
In China, however, access to optimal treatment is limited and many patients rely exclusively on on-demand treatment to manage bleeds. Despite national efforts to strengthen hemophilia healthcare infrastructure, “only limited real-world data exists to understand the changes in disease management over time and the economic burden of hemophilia A in China,” the researchers said.
Analyzing hemophilia A care in China
To address this, they analyzed the clinical characteristics, treatment patterns, and healthcare-related costs for hemophilia A patients, drawing on data from 3,164 males registered in the National Hemophilia Registry from January 2015 to December 2019. The males were a mean age of 21.5, nearly half (48.3%) were children or adolescents, and 58.9% had been diagnosed with severe hemophilia A. Over half (57.1%) were either students or employed.
Their first bleed occurred most often in the skin or mucous membranes (40%), followed by the joints (32.1%), and muscles (24.5%). Over the course of the disease, joint bleeds were the most prevalent, affecting 77.9%. As a result, more than a third (36.1%) developed joint malformations, most often in the knees and elbows. Among those with detailed bleeding histories, the mean annual number of bleeds was 24, with about 15 of those being joint bleeds.
Only 30.1% of the patients had received prophylactic treatment over the course of their disease. At the time of the index visit, which is a randomly selected visit during the study period with complete treatment data, 71.1% had been exclusively managed with on-demand therapy, 13.9% had only been using prophylactic treatment, and 14.4% had both.
Plasma-derived FVIII products were the common treatment, given to 48.1% of patients, followed by recombinant (lab-made) FVIII products (28.8%). A smaller percentage (18%) used both. The patients on prophylaxis were younger, more likely to be students or employed, to reside in developed areas, and to have access to urban resident insurance.
To evaluate healthcare utilization and costs associated with hemophilia A, the researchers also analyzed 118 patients from the Institute of Hematology and Blood Disease Hospital, covering data from January 2017 to December 2019. These patients had a mean age of 30.4, with 71.2% being adults and 51.7% diagnosed with severe hemophilia A.
Hospitalization rates rose from 8% in 2017 to 21% in 2019, with stays ranging from 2 to 13 days. However, inpatient costs were relatively low, averaging 702.6 CNY (about $98) per patient annually, mostly from laboratory tests.
Most healthcare costs came from outpatient care. Between 63% and 75% of patients had outpatient visits annually, with a mean of 11.6 visits per patient. The mean annual outpatient cost per patient was 56,736.8 CNY (about $7,900), of which more than 97% was attributed to FVIII replacement therapy.
“Primary prophylaxis with replacement clotting factors is considered to be the gold standard of care in hemophilia with evidence suggesting that it is associated with improved joint outcomes, decreased physical pain and hospitalization rates, and better [quality of life],” the researchers wrote. “Although prophylactic therapy is cost-effective compared with on-demand therapy, only 30% of patients from our study received prophylactic treatment. Hence, increasing the awareness of benefits of prophylactic treatments in patients with hemophilia, including cost-effectiveness and safety, is warranted.”
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