Emicizumab is well tolerated and can control bleeding even in the youngest children, starting from birth. These are the new results on the monoclonal antibody used against hemophilia A that arrive from San Diego, a few days after the close of the American Society of Hematology (Ash) congress. An additional wealth of knowledge that comes specifically from the primary analysis of the phase III HAVEN 7 study, and which acts as a corollary to the recognition Of Takehisa Kitazawa e Johnny Mahlangu with the Ernest Beutler Lecture and Prize for drug development.
Hemophilia A, a drug to reduce bleeding even in mild and moderate forms by Dario Rubino 25 July 2022
Hemophilia A in children
Hemophilia A is a rare disease characterized by bleeding, which is in turn due to the lack of a clotting factor, in this case factor VIII. There are several shape, more or less serious depending on how much the missing clotting factor is present (which can be present in variable percentages). Emicizumab is a bispecific monoclonal antibody, which compensates for the absence of factor VIII by acting as a bridge between factor IX and factor to be started in the first year of life, as explained by the experts. “Prophylaxis with factor VIII replacement therapy often begins after the first year of life due to problems related to venous access,” he underlined Antonio CoppolaMedical director at the Haemophilia and Congenital Haemorrhagic Diseases Hub center of the Parma University Hospital, on the sidelines of the preliminary results presented at the same congress last year.
Hemophilia A: new encouraging data on treatment in newborns by Valentina Guglielmo 16 December 2022
And give that HAVEN 7
Those arriving today from San Diego are helping to redefine the possibilities of using the drug in the little ones, which can also be administered subcutaneously, from birth, at different dosage frequencies. The new data coming from HAVEN 7 – conducted on cases of severe hemophilia A without factor VIII inhibitors, previously untreated or minimally treated – show that more than 50% of patients who received it (55 in total) had no bleeding requiring treatment. 16.4% had no bleeding, treated or untreated, and in total there were 207 bleeds in 46 patients, almost 90% due to trauma. There were no spontaneous bleeds requiring treatment in any patient.
Redefining the standards of care for hemophilia A
“The results of the primary analysis of the HAVEN 7 study presented at the ASH 2023 conference are extremely promising for the management of haemophilia A without inhibitors in newborns. The efficacy and safety of emicizumab in this age group, with significant control of bleeding, represent further confirmation of what has been seen so far in the emicizumab clinical development program and in clinical practice”, he commented Flora Peyvandidirector of the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and head of the Complex Structure (SC) of General Medicine – Hemostasis and Thrombosis at the IRCCS Ca’ Granda Foundation Ospedale Maggiore Policlinico in Milan, and director of the Department of Medical-Surgical and Transplant Pathophysiology of the University of Milan.
Hemophilia A, a drug to reduce bleeding even in mild and moderate forms by Dario Rubino 25 July 2022
Peyvandi reiterated the importance of flexible administration thanks to the drug, given the difficult venous access. Furthermore, according to some studies, early prophylaxis improves long-term outcomes and reduces the risk of intracranial hemorrhage. “These results – concludes the expert – reinforce the importance of starting prophylaxis as soon as possible to improve long-term results, also reducing the emotional and physical burden for patients and their families. We cannot but welcome these advances which will significantly contribute to redefining the standards of care for our patients with haemophilia A.”