Concizumab prophylaxis for haemophilia A or B without inhibitors: a plain language summary of the phase 3 explorer8 study

Plain Language SummaryWhat is this summary about?This summary describes results of ‘explorer8’, a study which looked at how the drug concizumab worked in people with haemophilia without inhibitors. The original article was published in Lancet Haematology.Haemophilia is a condition where blood does not clot properly because of missing clotting factors, either factor VIII (haemophilia A) or factor IX (haemophilia B). Haemophilia can be treated by replacing the missing clotting factors through intravenous injections. This can be burdensome, painful and sometimes not effective because of inhibitors (antibodies). New treatments that work differently and can be given as subcutaneous injections are important for people with haemophilia.What were the results?Concizumab is a medicine for haemophilia A or B, with or without inhibitors, and is injected subcutaneously. People given concizumab had fewer bleeds that needed treatment in one year compared with those who did not receive concizumab (haemophilia A: 86% fewer; haemophilia B: 79% fewer). Most of the reported side effects (COVID-19 and upper airway infection) were not attributable to concizumab, although an increase in D-dimers may be related to the treatment. In this study, participants and their doctors knew who was receiving concizumab and who received their usual medicine.What do the results mean?Concizumab can help prevent bleeding and was well tolerated in people with haemophilia without inhibitors.How to say (download PDF and double click sound icon to play sound)…Concizumab: con-siz-oo-mabHaemophilia: he-moh-fil-ee-ahIntravenous: in-trah-vee-nusSubcutaneous: sub-cu-tay-nee-usConcizumab: A treatment that aims to control bleeds in people with either haemophilia A or B, and with or without inhibitors to their factor replacement therapy.Inhibitors: Proteins made by the immune system that attack factor replacement therapy and stop it from working properly.Immune system: The body’s defence system, which consists of organs and processes that fight infections and other diseases.Clotting factor: One of several components in the blood that work together to create a clot and stop bleeding.Intravenous: Given into a vein.Antibody: A protein made by the immune system that sticks to a specific target on something it recognises as foreign or dangerous.Subcutaneous injection: An injection made under the skin.D-dimers: Protein fragments produced when blood clots form and dissolve naturally.This is an abstract of the Plain Language Summary of Publication article.View the full Plain Language Summary PDF of this article to read the full-textLink to original article hereTrial registration: ClinicalTrials.gov identifier: NCT04082429.