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Breakthrough in bleeding disorders
Published in The Saudi Gazette on 25 - 11 - 2013

RIYADH – The Center of Excellence in Thrombosis and Hemostasis (CETH) of College of Medicine, King Saud University in Riyadh, in collaboration with Orthopedic Surgery Research Chair, has recently held a major event for the management of bleeding disorders. Entitled “Current Practices in Thrombosis and Hemostasis-2013”. The 3-day event embraced scientific gathering where keynotes speakers and experts from Saudi Arabia, GCC states, Egypt, India, Europe, USA and Canada discuss scientific papers on bleeding disorders and Hemophilia. During the event, major orthopedic surgeries were carried out by local and international experts, including joint replacements and constructive procedures for patients with Hemophilia A, Hemophilia B and Von Willebrand Disease.
“This event provides specialized insights into the proper management of bleeding disorders at both micro and macro levels alike. It comprises practical and hands-on workshops to educate hemophilia and other bleeding disorder patients and help them understand their disease and properly deal with it in day to day life at home,” said Professor Abdul Kareem Al Momen, CETH Director. “It plays a crucial role in training patients as well as medical and paramedical staff on the management of the disease at different situations from various perspectives,” he added.
Thrombosis and hemostasis are global problems with high prevalence among both men and women of all ages. Studies on the Saudi population have demonstrated high prevalence of venous and arterial thromboembolic as well as bleeding disorders with high rate of morbidity and mortality. Aiming at raising awareness and enhancing knowledge and understanding of thrombosis and hemostasis to help maximize prevention for those at risk, CETH is well positioned to transfer state of the art technologies and therapies being a leading international research centers for the diagnosis, prevention and management of thrombosis & hemostasis.
Hemophilia is an inherited disease that prevents the blood from clotting properly. People with hemophilia have deficiency blood protein also called clotting factor which is necessary to clot the blood and stop bleeding. Most patients with hemophilia A are dependent on factor VIII replacement therapy, while patients with hemophilia B are dependent on factor IX replacement therapy.
According to the World Federation of Hemophilia, 80 per cent of hemophilic patients are afflicted with hemophilia A while 20 per cent of those patients have hemophilia B. Recent studies showed that approximately 320,000 – 340,000 persons – predominantly males – are affected with hemophilia A worldwide. In Saudi Arabia, with a clear absence of accurate statistics of patients with hemophilia however, sporadic studies reveal that the number of those patients may go up to 2000.
“Hemophilia treatments have witnessed exceptional developments following the introduction of plasma-derived options. However, there has been always a need for effective treatments with high safety and efficacy profile that could minimize risk of serious viral infections including HBV, HCV and HIV etc.”
Al Momen said “scientists then developed recombinants derived from human or animal sources. When selecting a plasma-derived or recombinant concentrates, the two most important safety issues are transmission of infectious agents and inhibitor formation.”
“The newly approved completely albumin-free treatments represent the latest generation of recombinants that employ advancements of manufacturing and purification processes. Using a synthetic ligand that is totally free of animal materials and multiple purification steps including chromatography and nanofiltration, these treatments help to further reduce the risk of potential viral contamination,” added Dr. Tarek Owaidah, consultant hematologist, Pathology Lab. Medicine, King Faisal Specialist Hospital and Research Centre. “They are the only recombinant treatments that go through a nanofiltration purification step which is an additional virus removal stage that includes non-enveloped viruses and possible new infectious agents.”
Saudi Food and Drug Authority (SFDA) has recently approved two drugs used for the treatment and prophylaxis of people with tendency to bleed i.e. hemophilia A and B. The new treatments are recombinants DNA-derived factor VIII and factor IX to replace missing clotting factors in hemophilic patients. They are developed through a state-of-the-art manufacturing and purification process that helps reduce the risk of serious viral infections that might be transmitted via infected blood.
Dr. Owaidah said that few decades ago, children with hemophilia had significantly reduced life expectancy and crippled with arthritis and joint deformity, however with the introduction of new biotechnologically advanced treatment options hemophilic patients faced a few limitations and became more active members in the society closed to normal with increased life expectancy.
“When it comes to treatment, safety is a primary concern for patients. We believe that the state of the art manufacturing and purification process used for the new recombinants make them an effective and safe therapeutic option for many patients with hemophilia,” Dr. Owaidah added. — SG


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