Over the past 50 years or so, persons with Hemophilia and providers have witnessed great advances in the treatment of this disease. More recently, novel therapeutic approaches and treatment modalities have continued to push the progress of Hemophilia care to new frontiers. Even with these remarkable progresses, however, new and old challenges continue to plague the Hemophilia community: access to diagnosis, access to therapy, and the burden of infusions – especially in patients on prophylaxis due to severe Hemophilia – remains a significant challenge.
A Kedrion-supported scientific symposium at the 2019 International Congress of Grupo CLAHT in San José, Costa Rica (October 10-12) was key in sending the message on the current status and future perspectives of replacement therapy, which could be seen as a possible solution to some of these challenges.
“Despite new therapies for Hemophilia coming in, replacement factors still have a role” – explained Carlos Ramírez, MD, Hematologist at Clínica Colsanitas in Bogotá, Colombia and speaker at the symposium. “On one side, we have been using them for more than 50 years, and even today they are irreplaceable for certain categories of patients. On the other, it is ethically very important to be able to provide every person with the same healthcare options, and every patient with adequate treatment, and I’m not sure we are ready for all this innovation, which has costs not every country is able to bear, at least not for everyone.”
“It will definitely take time, but I think that, if these new therapies are shown to be effective, they will also be used in developing countries”, the other speaker Miguel Escobar, MD, a Hematologist that treats Hemophilia patients in the USA, responded. “However, replacement therapy is still the mainstay of treatment for patients with Hemophilia, even in the US, and will continue to be so at least for the treatment of acute bleeds or for ITI (Immune Tolerance Induction), for which there are no alternatives to replacement factors.”
“Access to diagnosis, and therefore access to care, is still lacking in many countries of this Region, – remarked Kedrion Biopharma’s Sr. Medical Affairs, Hematology Lead, Prasad Mathew, MD, in his conclusions – but we see a path forward for patients with Hemophilia. A number of studies, as well as decades of clinical practice, have shown the efficacy of replacement therapy in reducing joint damage by preventing bleeding episodes. Why not go for the most cost-effective therapeutic solution and ensure that every patient gets access to early diagnosis and initiation of prophylaxis at as young age as possible?”