Acquired thrombotic thrombocytopenic purpura

This pilot study tests efgartigimod, a drug that lowers harmful antibodies, in 15 adults with immune-mediated thrombotic thrombocytopenic purpura (iTTP), a rare and life-threatening blood disorder. Participants receive weekly infusions for 8 weeks, then are monitored for another …

This study looks at whether adding a drug called Wilate (a von Willebrand factor concentrate) to standard platelet transfusions can reduce bleeding and the need for more blood products in intensive care patients. About 120 adults who need platelets during or after surgery will be…

This study is collecting information from 40 patients with a rare blood disorder called immune-mediated thrombotic thrombocytopenic purpura (iTTP) who have been treated with daratumumab. The goal is to see how well daratumumab works and how safe it is for patients whose disease d…

This study tests whether a simpler treatment for immune thrombotic thrombocytopenic purpura (iTTP) works as well as the standard approach. Instead of daily plasma exchange (an invasive procedure), patients receive plasma infusions, immune-suppressing drugs, and caplacizumab. The …

This study follows 300 teens and young adults (ages 10-24) with inherited bleeding disorders who choose either a levonorgestrel IUD or norethindrone acetate pills for heavy menstrual bleeding. Researchers will track bleeding patterns, satisfaction, and quality of life over six mo…

This study is testing a new blood test (the HMEC test) to see if it can better identify which patients with thrombotic microangiopathy (TMA) have a specific immune system problem driving their disease. TMA is a severe condition that damages kidneys and the brain. The researchers …

This study follows 163 teens and young adults with heavy menstrual bleeding, some with a bleeding disorder and some without, to see how well hormonal treatments like the pill, patch, or IUD work. Researchers will measure blood loss, quality of life, and iron levels over time. The…

This study is a large registry that collects health information from up to 200,000 people with blood disorders such as hemophilia, thrombosis, and sickle cell disease. Participants are seen at Hemophilia Treatment Centers across the U.S. The goal is to gather real-world data to h…

This study looks at whether a specific gene, TNFAIP3, is linked to immune-mediated thrombotic thrombocytopenic purpura (iTTP), a rare blood clotting disorder. Researchers will compare genetic data from 400 people with iTTP and healthy volunteers to see if gene variations affect d…

This study creates a registry to collect data on dengue infections in people with congenital bleeding disorders like hemophilia. Dengue can cause serious bleeding, and those with bleeding disorders may face higher risks. The goal is to understand bleeding complications, treatment…

This study looks for substances in the blood that might be linked to fatigue in people with low platelet counts (thrombocytopenia). Researchers think a protein called BDNF, which is stored in platelets, could play a role. They will measure BDNF levels in 280 patients and healthy …

This study looks at 50 adults with unexplained bleeding to see if low serotonin levels in their platelets are linked to their symptoms. Researchers will use a standard bleeding questionnaire to measure bleeding severity. The goal is to better understand how serotonin deficiencies…

This study reviews medical records from over 1,200 people with immune thrombotic thrombocytopenic purpura (iTTP), a rare and life-threatening blood disorder. The goal is to understand long-term health problems, such as heart disease and thinking issues, that survivors face. By an…