On December 11, 2022, a study presented in a plenary session of the annual meeting of the American Society of Hematology showed that patients taking efgartigimod for chronic primary immune thrombocytopenia (ITP) significantly improved platelet counts. Efgartigimod has a novel mechanism of action. It lowers IgG levels without affecting important immune-system components such as lymphocytes, IgG production, or the body’s innate immune system.

The study, called ADVANCE IV, was conducted by researchers from the Georgetown University Medical Center. The study was a phase III, double-blinded clinical trial that included 131 patients with ITP. All patients in the trial had low platelet counts and had at least one ITP treatment before being randomly assigned to the trial.

The findings of the trial showed that patients with chronic ITP who received efgartigimod achieved a significant improvement in sustained platelet response (21.8%) in comparison to the placebo (5%). The study also showed that in 4 of the 6 scheduled visits, more than half of the patients who responded to the drug showed doubled platelet counts.

Dr. Catherine Broome, M.D., an associate professor of medicine at Georgetown University and principal investigator of the study, noted that significant response to the drug was seen in all types of patients regardless of age, disease severity, time since diagnosis, prior ITP treatment, or use of other medications. She added that the most commonly reported side effects of the drug included bruising, headaches, blood in the urine, and rash-like symptoms on the skin related to bleeding. 

(Source: https://theprint.in/health/researchers-find-drug-effective-to-treat-autoimmune-blood-disorder/1260987/ )