Inhibition of neonatal Fc receptor as a treatment for immune thrombocytopenia

Perhaps the most convincing study on the mechanism of immune thrombocytopenia was the classic Harrington experiment in 1950. 1 Harrington WJ Minnich V Hollingsworth JW Moore CV Demonstration of a thrombocytopenic factor in the blood of patients with thrombocytopenic purpura. J Lab Clin Med. 1951; 38: 1-10 PubMed Google Scholar William Harrington collected a cohort of healthy volunteers and transfused into each of them plasma or whole blood from people with immune thrombocytopenia. Most participants (including Harrington himself) developed severe, rapid onset, and transient thrombocytopenia. The experiment spurred decades of studies in search of the plasma factor that causes immune thrombocytopenia. Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trialEfgartigimod significantly increased sustained platelet count responses compared with placebo in patients with chronic immune thrombocytopenia, including those who had received multiple previous immune thrombocytopenia therapies. Upon completion of the ADVANCE IV study, patients could enroll in the ongoing open-label extension. Subcutaneous efgartigimod is currently being evaluated in patients with immune thrombocytopenia in the ADVANCE SC+ trial. Full-Text PDF