Coagulation factor deficiency syndrome

-- A look back at the last five decades of treatment options to what lies ahead with additional potentially curative gene therapies -- PHILADELPHIA, June 7, 2023 /PRNewswire/ -- The Hemostasis and Thrombosis Center (HTC) at Children's Hospital of Philadelphia (CHOP) is proudly celebrating 50 years of providing comprehensive care to children and adolescents with hemophilia and other inherited bleeding or clotting disorders. The Center was founded in 1973 and was one of the first HTCs established in the country and is considered a Center of Excellence for the diagnosis, treatment and prevention of bleeding and clotting disorders, serving as a national resource for other institutions. "What started at CHOP as caring for 48 patients a year with hemophilia has grown into providing care for over 1,000 children with all types of bleeding and clotting disorders at our Center," says Leslie Raffini, MD, Director of the Hemostasis and Thrombosis Center at Children's Hospital of Philadelphia. "It's been an incredible journey to witness the advancement of treatment options for our patients, which today includes an FDA-approved potentially curative gene therapy option for patients with hemophilia B." The Last 50 Years of Hemophilia Treatment Hemophilia is an X-linked inherited bleeding disorder that is caused by a clotting factor deficiency that can cause frequent, painful joint bleeds and disability. There are two types of hemophilia. Factor VIII deficiency or hemophilia A, is the most common type of hemophilia, while factor IX deficiency, or hemophilia B, is about four times less common. In the 1970s, patients were taught to treat bleeding episodes at home with donated plasma-derived clotting factor concentrates. In the 1980s, during the AIDS epidemic in the U.S., the blood supply many of these patients received was contaminated with HIV from infected blood donors. During this time, donated blood was the only treatment available for hemophilia patients, which led to many of them becoming infected with HIV. In the 1990s, genetically engineered recombinant clotting factors became available, making it safer and easier for patients to administer the treatments at home. By the mid 1990s most of CHOP's patients with hemophilia were receiving 2-3 infusions of clotting factor per week at home to prevent joint bleeds. Although this worked well to prevent joint damage, caregivers had to infuse the clotting factor into their child's veins, which was painful and stressful. In 2017, a new treatment called emicizumab was approved to prevent joint bleeds in hemophilia A. This treatment is given as a small subcutaneous injection every 1-4 weeks and provides excellent protection from bleeding. "Most patients with hemophilia A at CHOP are treated with this product and do well without any joint bleeds or damage," said Raffini. "Most children lead a very active and normal lifestyles, with the exception of participating in high-contact sports like football or hockey." On the Cusp of Additional New Gene Therapies In 2022, the FDA approved the first gene therapy product for hemophilia B patients 18 years of age or older. Gene therapy has the potential to cure genetic disorders such as hemophilia. There are currently numerous other gene therapy products in development for hemophilia that are expected to be available to patients in the coming decade. CHOP has led the development of hemophilia gene therapy through basic, translational and clinical research efforts and recently launched the Novel Therapeutics for Bleeding Disorders (NoT Bleeding) Program, which is housed in CHOP's HTC. The team is led by world-class experts who together have more than two decades of clinical experience in gene therapy for hemophilia and in innovating care for patients with bleeding disorders. A Unique Perspective Regina Butler, RN-BC, clinical manager for the Division of Hematology at CHOP has been with the institution since the HTC's inception and has spent the last 50 years as a hemophilia nurse and an international leader in the field. "It has been an amazing experience to observe the tremendous advances in this field since I started in the 1970s," said Butler. "Hemophilia has changed from a disease that caused disability and pain that severely impacted the lives of patients and their families to a disorder with minimal complications and that is much more manageable. Seeing our patients lead healthy and active lives today is tremendously rewarding." The HTC at CHOP is comprised of a multi-disciplinary team made up of clinicians, nurses, social workers, physical therapists, and researchers. CHOP is part of the national network of federally funded HTCs in the U.S. and previously served as the Core Center for the 16 HTCs in the Mid-Atlantic Region for 15 years under the leadership of Regina Butler. As a Core Center, CHOP oversaw the services provided by the Mid-Atlantic Region III HTCs and collaborated with the Maternal and Child Health Bureau and CDC to improve treatment and support for people with hemophilia and other inherited bleeding and clotting disorders. For more information about CHOP's HTC, visit . About Children's Hospital of Philadelphia A non-profit, charitable organization, Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, the 595-bed hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. The institution has a well-established history of providing advanced pediatric care close to home through its CHOP Care Network, which includes more than 50 primary care practices, specialty care and surgical centers, urgent care centers, and community hospital alliances throughout Pennsylvania and New Jersey, as well as an inpatient hospital with a dedicated pediatric emergency department in King of Prussia. In addition, its unique family-centered care and public service programs have brought Children's Hospital of Philadelphia recognition as a leading advocate for children and adolescents. For more information, visit . Contact: Jennifer Lee Children's Hospital of Philadelphia 610-800-6592 [email protected] SOURCE Children's Hospital of Philadelphia

LACHEN, Switzerland--( BUSINESS WIRE )--Octapharma AG today announced that European medical authorities have approved the lyophilised presentation of the well-established octaplasLG ® - pharmaceutically-licensed S/D treated plasma for transfusion. The line extension, which will be marketed in Europe as a powder and solvent for solution for infusion, offers new and potentially life-saving treatment options in pre-hospital and emergency settings. “The European authorization of octaplasLG ® powder continues Octapharma's ongoing commitment to help prevent uncontrolled haemorrhage associated with trauma, which accounts for 30%-50% of all deaths in the first 24 hours" 1-5 , says Dr. Oliver Hegener, Vice President IBU Critical Care. Trauma results in 4.4 million deaths worldwide annually 6 , with uncontrolled bleeding being the major cause of potentially preventable death. Trauma-induced coagulopathy (TIC) is an early abnormal response characterised by hypocoagulation, in addition to acute blood loss, shock, hypothermia and metabolic acidosis, that begins soon after injury. Early - pre-hospital - administration of plasma has been shown to increase survival, compared with crystalloids, when pre-hospital transport was longer than 20 minutes. 7 Until now, access to early plasma transfusion was limited by logistic hurdles as well as a lack of available transfusion medicine in the pre-hospital setting for both for civilian and military patients. "octaplasLG ® powder, an AB universal plasma, can be stored at room temperature and reconstituted within minutes, representing a potentially lifesaving treatment option in emergency situations and in locations with limited frozen storage facilities and cold chain infrastructure required for regular FFP," explains Dr. Hegener. About octaplasLG ® Pharmaceutical grade human plasma for infusion with standardised quality and unique pathogen safety profiles due to Octapharma’s integrated pathogen elimination/inactivation concept. The standardised content is achieved by pooling between 630 and 1520 single plasma units from multiple carefully screened donors. Safety measures present during manufacturing include: Donor screening, immunoneutralisation, Solvent/detergent treatment and Ligand affinity chromatography for prion reduction. The product is approved for: Complex deficiencies of coagulation factors such as coagulopathy due to severe hepatic failure or massive transfusion. Substitution therapy in coagulation factor deficiencies, when a specific coagulation factor concentrate (e.g., factor V or factor XI) is not available for use or in emergency situations when a precise laboratory diagnosis is not possible. Rapid reversal of the effects of oral anticoagulants (coumarin or indanedione type) when a prothrombin complex concentrate is not available for use or administration of vitamin K is insufficient due to impaired liver function or in emergency situations. Potentially dangerous haemorrhages during fibrinolytic therapy, using e.g., tissue plasminogen activators, in patients who fail to respond to conventional measures. Therapeutic plasma exchange procedures, including those in thrombotic thrombocytopenic purpura (TTP). About Octapharma Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. Octapharma employs more than 11,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Haematology, and Critical Care. Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 190 plasma donation centres across Europe and the US. References: 1. Bosch F, Angele MK, Chaudry IH. Gender differences in trauma, shock and sepsis. Mil Med Res. 2018;5(1):35. 2. Iapichino GE, Ponschab M, Cadamuro J, et al. Concentrated lyophilized plasma used for reconstitution of whole blood leads to higher coagulation factor activity, but unchanged thrombin potential compared with fresh-frozen plasma. Transfusion. 2017;57(7):1763-71. 3. Nguyen C, Bordes J, Cungi PJ, et al. Use of French lyophilized plasma transfusion in severe trauma patients is associated with an early plasma transfusion and early transfusion ratio improvement. J Trauma Acute Care Surg. 2018;84(5):780-5. 4. Moore EE, Moore HB, Kornblith LZ, et al. Trauma-induced coagulopathy. Nat Rev Dis Primers. 2021;7(1):30. 5. Johansson PI, Sorensen AM, Perner A, et al. Disseminated intravascular coagulation or acute coagulopathy of trauma shock early after trauma? An observational study. Crit Care. 2011;15(6): R272. 6. World Health Organization. Injuries and violence. 19 March 2021. Available at: https://www.who.int/news-room/fact-sheets/detail/injuries-and-violence. Last accessed January 2023 . 7. Pusateri AE, Moore EE, Moore HB, et al. Association of Prehospital Plasma Transfusion With Survival in Trauma Patients With Hemorrhagic Shock When Transport Times Are Longer Than 20 Minutes: A Post Hoc Analysis of the PAMPer and COMBAT Clinical Trials. JAMA Surg. 2020;155(2): e195085.

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Inspiration Biopharmaceuticals, Inc. (Inspiration) announced interim clinical results for the company’s two lead product candidates, an intravenous recombinant porcine FVIII (OBI-1) in the treatment of serious bleeds in acquired hemophilia A (AHA) and an intravenous recombinant FIX (IB1001) for the treatment and prevention of bleeding in children less than 12 years of age with hemophilia B. The data were presented in posters at the 54th American Society of Hematology Annual Meeting in Atlanta, Georgia. Inspiration’s OBI-1 investigational drug met the primary endpoint of providing effective or partially effective treatment of serious bleeding in 16 subjects with acquired hemophilia A based on the investigator’s assessment of response to treatment.