What Is Hemophilia?

What is Hemophilia? Diseases with low prevalence in society, chronic and life-threatening conditions are defined as "rare diseases." Approximately 400 million people worldwide and approximately 5 million people in Turkey are affected by rare diseases, yet 90% of these diseases have no treatment yet. Hemophilia, a rare genetic bleeding disorder, negatively affects the lives of approximately 180 thousand people worldwide and more than 6 thousand people in Turkey. Emphasizing the importance of disseminating accurate information to patients and their relatives in the management of hemophilia A, a disease requiring lifelong treatment, Hemophilia Associations Federation Chairman Prof. Dr. Kaan Kavaklı stated: "Through our work on hemophilia treatment, we continue to take steps to raise treatment standards in order to contribute to patients, patient relatives and the healthcare system. The success of these efforts for all stakeholders is directly proportional to increased public awareness regarding hemophilia." Approximately 7,000 different rare diseases affect more than 400 million people worldwide. For 90% of known rare diseases, no treatment has been found yet, but work continues through clinical research on diagnostic and therapeutic solutions. Hemophilia A, a genetic bleeding disorder that develops as a result of blood clotting factor being absent or insufficient, is among rare diseases. Today, while 180 thousand people worldwide and more than 6 thousand people in Turkey struggle with this disease, scientific research continues with a focus on improving patients' lives globally.

"The Burden of Hemophilia A Disease Constitutes 1.62% of Turkey's Total Health Expenditure"

Noting that the data obtained from the "Societal Burden of Hemophilia A Disease" research is noteworthy, Hemophilia Associations Federation Chairman Prof. Dr. Kaan Kavaklı continued: "According to the results of research conducted in 2018, which included costs arising during treatment, follow-up and disease progression, the per-patient annual cost of more than 6 thousand hemophilia A patients in Turkey is approximately TRY 560,000. Despite being a rare disease, hemophilia's annual disease burden reaches nearly TRY 3 billion. This figure constitutes 1.62% of Turkey's total health expenditure."

Hemophilia A Disease and Its Risks Must Be Well Understood

  Sharing information within the scope of 28 February Rare Diseases Day regarding hemophilia A, a rare disease requiring lifelong treatment, Prof. Dr. Kaan Kavaklı stated, "Hemophilia A is a disease in which we have determined the average age to be approximately 25 years. If treatments are not applied regularly, hemophilia A patients face very serious risks. Patients can face the risk of death due to permanent joint disabilities and brain hemorrhages that may occur following head trauma. For this reason, access to hemophilia A treatments is of great importance. In Turkey, patients have access to standard hemophilia A treatments. By developing treatment options, we aim to reduce the burden on patients and their relatives. We have very important objectives such as increasing patient adherence to treatment and enabling patients to live bleed-free lives while improving their quality of life. We continue our work with the hope of finding a cure for patients. Our efforts continue for innovative solutions in fighting hemophilia A." Noting that hemophilia patients are generally diagnosed in their first year of life, Prof. Dr. Kaan Kavaklı continued: "We begin treatment after the first bleeding episode, preferably before age 3. At this point, the family's proper introduction to hemophilia and receiving professional support is of critical importance. In treatment management for children, we focus most on matters that will protect them from bleeds. By concentrating on preventing joint bleeds and life-threatening bleeds and preventing permanent disabilities, we continue our efforts to enable hemophiliacs to lead more active lives."

"Through the 'Hem de Nasıl' Campaign, We Invite Everyone to Join the Hemophilia Journey"

  The 'Hem de Nasıl' digital media platform, launched through the collaboration of Roche Pharmaceuticals Turkey, the Turkish Hemophilia Association and the Hemophilia Associations Federation, aims to provide patients and their relatives with accurate and current information in the field of hemophilia. The platform, established to increase awareness about hemophilia, a disease requiring lifelong treatment, shares content through its website and social media channels. Referring to the platform's contribution to hemophilia awareness, Prof. Dr. Kaan Kavaklı stated, "Because rare diseases are difficult to diagnose, awareness in this disease is of great importance. For this reason, it is very critical for the general public to have comprehensive information about living with hemophilia for the patient journey. The 'Hem de Nasıl' platform was established for exactly this purpose, with important collaborations to answer many questions about hemophilia from childhood to adulthood, from school life to working life and family life."