Experts in the field of Hematologyat recently gathered in Dubai during the RAISE 2: Raising the Bar in Hemophilia Treatment Summit, to discuss novel therapies and personalized care for patients diagnosed with hemophilia.
Hemophilia A, the most common type of hemophilia, is a rare bleeding disorder that causes longer-than-normal bleeding due to lack of proper clotting factor in the blood. The disease is most commonly diagnosed in males, and more than half of patients with hemophilia A have the severe form of the condition.
Experts at the RAISE 2 Summit discussed current perspectives and evidence-based best practice in treatment, including bioengineered protein replacement therapies, non-replacement treatments and gene therapy. Prophylactic treatment with regular infusion of either plasma-derived or recombinant factor VIII or IX products is currently best practice based on proven effectiveness in the prevention of bleeding and joint destruction in patients with moderate to severe bleeding.
Physicians also discussed ways to implement and optimize personalization through multidisciplinary approaches, as dose and frequency of therapies must be adjusted to the patients pharmacokinetic (PK) profile and bleed frequency to work effectively. “With early detection and treatment, most people with hemophilia can have good quality of life. With recent advancement in genetic sciences, physicians can now offer tailored prophylactic treatment which works best according to the to the patient’s lifestyle. Such tailored treatment also ensures better chances for the patient to live a normal life, participate in the routine daily activities and have regular attendance at school or work.” said Dr. Maryam Matar, Founder and Chairperson of UAE Genetic Disease Association.
Experts used case-based evidence to discuss the implementation of the myPKFiT™ mobile app which is the first and only FDA-cleared PK dosing software for hemophilia A patients 16 and older who are receiving treatment.With myPKFiT™, healthcare professionals can estimate a full PK curve with as few as two measurable blood samples, compared to 9 to 11 as recommended by guidelines from the International Society on Thrombosis and Haemostasis (ISTH). Using the patient’s PK curve and additional patient information, healthcare professionals can develop a personalized, PKguided prophylactic treatment regimen tailored to the individual patient’s needs.
“At Takeda, our research efforts focus on areas where there is most clinical need. We are proud of our hematology heritage and we plan on expanding it through continued research and innovation, including continued focus on direct factor replacement”, said Dina El Salmy, Head of Portfolio Management at Takeda India, CIS, Middle East and Africa (ICMEA). “We are relentlessly pursuing solutions to minimize bleeding disorders. Beyond delivering new medications, we are dedicated in our efforts to encourage early diagnosis, propel patients towards optimal personalized care and help them remain on the correct therapy to achieve the best long-term outcomes”.