- The HAVEN 7 study was designed to further confirm the benefit of preventative treatment (prophylaxis) with Hemlibra from birth in previously untreated or minimally treated infants with severe haemophilia A without inhibitors
- In the study, 77.8% of participants had no bleeding episodes that required treatment1
- In addition, real-world efficacy and safety data from the EUHASS database and ATHN 7 study were also presented 2,3
The burden of severe haemophilia A in infants and on their parents and caregivers is significant.
“These initial results support the benefit of starting Hemlibra from birth given that early preventative treatment is essential in infants,” said
HAVEN 7 is a phase III, multi-centre, open-label study evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of Hemlibra in infants with severe haemophilia A without factor VIII inhibitors. The results of this interim analysis, which included data from 54 participants, showed that 77.8% of participants (n=42) did not have any bleeds which required treatment, while 42.6% (n=23) did not have any treated or untreated bleeds at all. There were no treated spontaneous bleeds in any participants, and all treated bleeds were traumatic. A total of 77 bleeds occurred in 31 participants (57.4%); 88.3% were traumatic. Mean model-based annualised bleeding rate (ABR) (95% CI) at the time of interim analysis was 0.4 (0.23–0.65) for treated bleeds. 1
Hemlibra’s safety profile was consistent with previous studies, with no new safety signals observed. Nine people (16.7%) reported a Hemlibra-related adverse event (AE), all of which were local injection site reactions. Eight participants (14.8%) reported 12 serious AEs, unrelated to Hemlibra. There were no deaths, thromboembolic events or cases of thrombotic microangiopathy, reinforcing Hemlibra’s favourable safety profile. No intracranial haemorrhages occurred.1
Primary analysis will be conducted at 52 weeks. The study also has an additional seven-year follow-up period to collect long-term data such as safety and joint health outcomes, further building upon our understanding of the benefit of Hemlibra in this population.
EUHASS database and ATHN 7 study
Hemlibra is approved as a treatment for people with haemophilia A with factor VIII inhibitors in more than 110 countries worldwide, and for people without factor VIII inhibitors in more than 100 countries worldwide. It has been studied in one of the largest clinical trial programmes in people with haemophilia A with and without factor VIII inhibitors, including eight phase III studies.
About Hemlibra® (emicizumab)
Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It is designed to bring together factor IXa- and factor X, proteins involved in the natural coagulation cascade, and restore the blood clotting process for people with haemophilia A. Hemlibra is a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once-weekly, every two weeks, or every four weeks (after an initial once-weekly dose for the first four weeks). Hemlibra was created by
About haemophilia A
Haemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Haemophilia A affects around 900,000 people worldwide, 4,12 approximately 14% and 48% of whom have a moderate or mild form of the disorder,13 respectively. However, the severity of haemophilia A is not always reflective of bleeding behaviour, as some people with non-severe haemophilia may experience symptoms similar to those with severe haemophilia and warrant prophylaxis.14 All severities of haemophilia A can significantly reduce the quality of life for people affected, as well as their family and caregivers. People with haemophilia A either lack or do not have enough of a clotting protein called factor VIII. In a healthy person, when a bleed occurs, factor VIII brings together the clotting factors IXa- and X, which is a critical step in the formation of a blood clot to help stop bleeding. Depending on the severity of their symptoms, people with haemophilia A can bleed frequently, especially into their joints or muscles. 13 These bleeds can present a significant health concern as they often cause pain and can lead to chronic swelling, deformity, reduced mobility and long-term joint damage.15 A serious complication of treatment is the development of inhibitors to factor VIII replacement therapies. Inhibitors are antibodies developed by the body’s immune system that bind to and block the efficacy of replacement factor VIII, making it difficult, if not impossible, to obtain a level of factor VIII sufficient to control bleeding.4
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References
[1] Pipe S, et al. Emicizumab Prophylaxis for the Treatment of Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Interim Analysis of the HAVEN 7 Study. Presented at
[2] Nissen F, et al. Real-World Safety of Emicizumab: Interim Analysis of the European Haemophilia Safety Surveillance (EUHASS) Database. Presented at
[3] Recht M, Emicizumab and Females with Hemophilia A: Case Series from ATHN 7. Presented at
[4] Srivastava A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020; 26 (Suppl 6): 1-158.
[5] Manco-Johnson MJ, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia.
[6] Andersson NG, et al. Intracranial haemorrhage in children and adolescents with severe haemophilia A or B - the impact of prophylactic treatment.
[7] Spagrud LJ, et al. Pain, distress, and adult-child interaction during venipuncture in pediatric oncology: an examination of three types of venous access.
[8] Van den Berg HM, et al. Timing of inhibitor development in more than 1000 previously untreated patients with severe hemophilia A. Blood 2019;134:317–320.
[9] Valentino LA, et al. Central venous access devices in haemophilia. Haemophilia. 2004;10: 134–146.
[10] Valentino LA & Kapoor M. Central venous access devices in patients with hemophilia. Expert Rev Med Devices 2005;2: 699–711.
[11] Mancuso M, et al. Prophylaxis in children with haemophilia in an evolving treatment landscape. Haemophilia. 2021;27: 889–896.
[12] Iorio A et al. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males. Ann Intern Med 2019 Oct 15;171(8):540-546.
[13]
[14] Collins PW, et al. Clinical phenotype of severe and moderate haemophilia: Who should receive prophylaxis and what is the target trough level? Haemophilia, 2021;27: 192-198.
[15] Franchini M, et al. Haemophilia A in the third millennium. Blood Rev. 2013; 179-84.
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