[Public Voices] Hemophilia Treatments Must Be Chosen Carefully

Various Half-Lives and Administration Methods in Development
Pros and Cons Depending on Patient Activity Level

When treating hemophilia, I always have high expectations whenever advanced treatments are introduced. How effective and safe are they? Are patients able to use them conveniently? Physicians focus on the efficacy and safety of the treatments, while patients, entrusting efficacy and safety to their doctors, pay great attention to the convenience of drug administration. Therefore, enabling patients to control bleeding more safely and effectively is the most important aspect of hemophilia care.

Understanding the characteristics of therapeutic agents is crucial for controlling bleeding in hemophilia patients. For example, among the widely used factor VIII products for hemophilia treatment, standard half-life (SHL) agents have a half-life of about 10 to 14 hours. Extended half-life (EHL) agents, which were introduced later, have a half-life 1.5 to 1.7 times longer. The recently launched non-factor agent, Hemlibra, shows an even longer half-life, allowing prescriptions at intervals of up to four weeks. The development of treatments like Hemlibra, which have long half-lives and are convenient to administer (subcutaneous injection), is very welcome in prophylactic hemophilia management. Furthermore, it is excellent news that even more advanced treatments are on the horizon.

However, from the perspective of providing care to control patients’ bleeding and help them live healthy lives, immediate convenience is important, but long-term treatment plans must consider various factors such as the patient’s physical activity level and underlying conditions. This is because the goal is to enable a healthy life throughout their lifetime. While there is abundant domestic and international data on the efficacy and safety of long-prescribed treatments, and I have extensive clinical experience with them, I exercise caution when using newly launched treatments until solid trust and experience are established. This is my duty to protect patients from potential risks.

For bleeding prevention in hemophilia patients, the level of coagulation factor activity in the blood is a critical consideration. Patients with high activity levels or those engaging in relatively intense physical activities need to maintain high coagulation factor activity in the blood even if multiple doses of medication are required. This approach reduces long-term damage to joints and other tissues. A study published in 2017 (Res Pract Thromb Haemost. 2017;1:231-241) suggested that about 25% coagulation factor activity is necessary to achieve normal clot stability. Based on this, it is very important for active children and young adults or those performing high-intensity physical activities to check and manage their coagulation factor activity before activity through personalized treatment.

In connection with this, recently launched or soon-to-be-launched non-factor therapies tend to have relatively low calculated coagulation factor activity values after administration but offer longer maintenance times. Therefore, they could be considered treatment options for patients with low activity levels, such as infants or the elderly. For new treatments to completely replace existing ones, they must cover all the advantages and disadvantages of the current therapies. Otherwise, each treatment should be appropriately selected and used according to clinical objectives, and patients can receive more suitable care through expanded treatment options that include both new and traditional therapies. Various ongoing studies raise expectations for even more advanced treatments, which will bring patients a better quality of life than ever before.

Gu Honghoe, Director of the Korea Hemophilia Foundation

This content was produced with the assistance of AI translation services.