A monoclonal antibody has shown promise as an effective new treatment for haemophilia, German researchers have reported.
The antibody normalises thrombin formation, leading the developers to hope it will reduce long-term complications from the disease.
Results of a phase 3 clinical trial, called ‘explorer8’, have been reported in The Lancet Haematology. It involved 148 patients with haemophilia A or B without inhibitors, and the researchers report “significantly” reduced bleeding rates in those who received the antibody, concizumab.
The treatment blocks the anticoagulant factor TFPI (tissue factor pathway inhibitor), enabling the normal production of thrombin.
Researcher Professor Hermann Eichler, of Saarland University Hospital, who helped develop the antibody, said: “This development is of major significance for haemophilia patients. By blocking TFPI, thrombin production at the injury site is significantly increased, which allows blood clotting to begin and the wound to close.
“In contrast to previous therapeutic approaches, blocking the action of TFPI normalises thrombin formation in haemophilia patients – something we typically only see in individuals with healthy coagulation. The bleeding rate was significantly lower in trial participants who received concizumab, and the drug, which is injected under the skin with a disposable pen similar to those used by diabetics, is very well tolerated.”
He added: “One of the patients who took part in the trial recently suffered a bad fall from his bicycle and sustained a large wound. Without haemophilia treatment, the man would almost certainly have suffered a serious bleeding complication. But now, just a few days later, he is sitting there just like you and me, and his wounds are healing quite normally.”
Source:
Chowdary P, Angchaisuksiri P, Apte S, Astermark J, Benson G, Chan AKC, Jiménez Yuste V, Matsushita T, Høgh Nielsen AR, Sathar J, Sutton C, Šaulytė Trakymienė S, Tran H, Villarreal Martinez L, Wheeler AP, Windyga J, Young G, Thaung Zaw JJ, Eichler H. (2024) “Concizumab prophylaxis in people with haemophilia A or haemophilia B without inhibitors (explorer8): a prospective, multicentre, open-label, randomised, phase 3a trial.” Lancet Haematology, 1 December 2024, doi: 10.1016/S2352-3026(24)00307-7
Link: https://www.thelancet.com/journals/lanhae/article/PIIS2352-3026(24)00307-7/fulltext
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