Congenital thrombotic thrombocytopenic purpura

This study tested a freeze-dried plasma product that can be mixed with water in 6 minutes, avoiding the long thaw time of standard plasma. It included 42 trauma patients with severe bleeding who needed blood transfusions. The goal was to see if faster plasma delivery could improv…

This study tested a medicine called BAX 930 in 52 people born with severe TTP, a rare condition that causes dangerous blood clots. The medicine replaces a missing enzyme to prevent or treat sudden flare-ups. Participants received either BAX 930 or standard treatment for 6 months,…

This study tested a pathogen-reduced version of cryoprecipitate, a blood product that helps blood clot, in 208 patients undergoing liver transplant or heart surgery. The goal was to see if having this product readily available could stop bleeding early and reduce the need for oth…

This study tested a new diagnostic tool that uses imaging flow cytometry to examine platelets in blood samples. Researchers compared it to standard lab tests in 31 patients suspected of having platelet disorders or heparin-induced thrombocytopenia. The goal was to see if the new …

This study tested a new lab method to diagnose platelet function disorders in 322 people with bleeding symptoms. The method uses flow cytometry to detect signaling defects in platelets, which are often missed by standard tests. The goal was to standardize the technique across mul…

This study investigates inherited thrombocytopenias, rare conditions where low platelet counts cause bleeding problems. About half of patients have an unknown genetic cause. Researchers aim to identify new disease genes and build a lab-grown bone marrow model to test how well dru…