Hemophilia treatment has entered an exciting era, with new products making diagnosis and treatment available for a far larger population than ever before, said WHF Vice-President Medical Marijke van den Berg during her Tuesday morning VP Medical Plenary address.
The session covered a variety of studies in people with severe hemophilia A that demonstrate how early prophylaxis can prevent bleeding and is key for joint outcome. This replaces episodic therapy, which has been a frequently used hemophilia treatment regimen since the 1970s.
“Modern hemophilia treatment has completely changed the phenotype—but not in countries where early treatment is not available,” she said.
Ms. van den Berg cited a very large U.S. study of patients with severe hemophilia A, divided into four birth-date cohorts. Even in the age group born in the 1980s, disability was too high, she said, with more than five joint bleeds over six months, despite very high clotting factor consumption.
The large, international Musculoskeletal Function in Hemophilia (MUSFIH) study of children with severe hemophilia A showed that even very high factor dosing resulted in substantial bleeding, van den Berg said. The study also showed that the number of bleeds—but not the dose of episodic treatment—is responsible for joint outcome. This is a key understanding because joint function deteriorates after age 12.
Ms. van den Berg cited a small, randomized study showing that low-dose prophylaxis, rather than episodic treatment, reduces bleeding by 80 percent. Research also shows that early diagnosis is crucial. “Remember, more than 50 percent of those with severe hemophilia A have a negative family history,” van den Berg said.
But when and how do you start prophylaxis? van den Berg said research suggests that the key is to start earlier than age 3 because physical examination scores increase with treatment delay. Other research shows that low-dose prophylaxis should be done a minimum of once a week.
There is a correlation between joint scores and dosage of factor replacement. “With a 1,000-1,500 dose, there’s a lot to gain,” van den Berg said. The good news for people in developing countries, where factor supply is limited, is that data show that lifetime prophylaxis with 1,000 IU per kilo is much more effective than episodic treatment. “You can significantly improve outcome with limited factor consumption,” she said.
However, to implement low-dose prophylaxis, comprehensive care centres are crucial, van den Berg said. She recently toured two international hemophilia training centers that are excellent examples of this: the centre in Campinas, Brazil, led by Margareth Castro Ozelo, and the centre in Johannesburg, South Africa, led by Johnny Mahlangu. The Johannesburg centre serves 1,200 patients, with an impressive 35 percent on prophylaxis and home therapy.
Unfortunately, these centres are the exception. Recent data from Africa show that not even 5 percent of hemophilia patients are diagnosed. “The main reason is because limited or no treatment is available,” van den Berg said.
The WFH Humanitarian Aid Program will substantially address that deficit. From 2016 to 2020, the program plans to provide a predictable supply of 500 million IUs of factor, van den Berg said. Availability of products will lead to more diagnosis, and that will lead to more training and, in some cases, corrective surgery.
In conclusion, van den Berg said that the evidence shows that only primary prophylaxis can prevent joint disease, and episodic treatment is not an appropriate regimen for severe hemophilia A. After joint bleeds, signs of arthropathy appear even with very high-dose prophylaxis. And signs of loss of joint function are often visible at puberty due to growth spurts.
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