Thrombotic microangiopathy

This study tested a new oral medication called iptacopan in 34 adults with a rare disease called atypical hemolytic uremic syndrome (aHUS), which causes blood clots, low platelets, and kidney damage. The goal was to see if the drug could improve blood counts and kidney function w…

This study tested a drug called ravulizumab in people aged 12 and older who developed a serious blood clotting condition (thrombotic microangiopathy) after a stem cell transplant. The goal was to see if the drug could prevent death or worsening of the condition over 26 weeks. Par…

This study tested a drug called TAK-755 in 29 adults with a rare blood clotting disorder called immune-mediated thrombotic thrombocytopenic purpura (iTTP). The goal was to see if TAK-755, along with standard immune-suppressing drugs, could control the disease with little or no pl…

This study tested a drug called avatrombopag in 19 Japanese adults with chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, raising bleeding risk. The goal was to see if the drug could safely increase platelet counts over 26 weeks. Parti…

This Phase 3 trial tested an oral drug called HMPL-523 (sovleplenib) in 272 adults with chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding risks. Participants who had not responded well to prior treatments received eith…

This study tested a new medicine called HBM9161, given as a weekly shot under the skin, for adults with primary immune thrombocytopenia (ITP) — a condition where the immune system destroys platelets, raising the risk of bleeding. The trial included 36 people who had not responded…

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 phase 3 trial tested the long-term safety and effectiveness of efgartigimod, an IV drug, in 101 adults with primary immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding. Participants received efgartigimod infusions over 52 w…

This study tested the drug eltrombopag in 48 Chinese adults with chronic hepatitis B and a bleeding disorder called immune thrombocytopenia (low platelets). The goal was to see if taking eltrombopag for 6 to 22 weeks could safely raise platelet counts. The trial is complete, but …

This study tested two low-dose rituximab schedules in 100 adults with immune thrombocytopenia (ITP), a condition causing low platelet counts and bleeding. The goal was to see which regimen better raises platelets and controls bleeding. Participants had failed or relapsed on stand…

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 drug called ravulizumab in 41 children who developed a serious blood clotting problem (TMA) after a stem cell transplant. The goal was to see if the drug could help control the condition and improve blood counts and kidney function. The treatment lasted 26 wee…

This study tested whether a drug called caplacizumab, combined with immune-suppressing medicines, could treat a rare blood clotting disorder called immune-mediated thrombotic thrombocytopenic purpura (iTTP) without the usual plasma exchange procedure. Fifty-one adults aged 18 to …

This study tested a drug called lusutrombopag in 17 Chinese adults with persistent or chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, raising bleeding risk. Participants had failed standard treatments and took the drug for up to 12 w…

This study tested the drug eculizumab in 25 children and adults in China with a rare kidney condition called atypical hemolytic uremic syndrome (aHUS). The goal was to see if the drug could stop blood clots and improve kidney function. All participants received eculizumab intrave…

This pilot study tested a drug called pegcetacoplan in 12 adults who developed a serious blood clotting condition (TA-TMA) after a stem cell transplant. The goal was to see how the drug moves through the body, if it helps, and if it is safe. Researchers measured drug levels and m…

This study tested a drug called caplacizumab in 21 Japanese adults with acquired thrombotic thrombocytopenic purpura (aTTP), a rare condition causing dangerous blood clots. Participants received caplacizumab alongside standard treatments like plasma exchange and steroids. The goa…

This completed phase 3 trial tested whether adding all-trans retinoic acid (ATRA) to eltrombopag works better than eltrombopag alone for adults with immune thrombocytopenia (ITP) that did not respond to steroids. The study enrolled 96 participants and measured sustained platelet …

This study looked at whether treating Helicobacter pylori (a common stomach infection) with antibiotics can raise platelet counts in people with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding and bruising. Researchers enrol…

This study tested a new blood test designed to quickly determine whether the complement system is involved in a serious kidney condition called thrombotic microangiopathy (TMA). The test measures complement deposits on cells in a lab sample. Researchers compared test results to h…

This study looks back at medical records of children and adults who developed a serious blood-clotting condition called thrombotic microangiopathy (TMA) within a year after a stem cell transplant. Researchers aim to understand survival rates and causes of death in these patients,…

This study follows children in France diagnosed with severe autoimmune cytopenia—conditions where the immune system attacks blood cells. Researchers will track their health over time to see who achieves remission and what factors influence their recovery. The goal is to better un…

This completed study looked at blood samples from 70 children and adults newly diagnosed with immune thrombocytopenic purpura (ITP), a condition that causes easy bruising and bleeding. Researchers wanted to see if certain immune and blood markers could predict whether the disease…

This study followed 37 adults over age 60 with unexplained low platelet counts to see if certain tests could predict immune thrombocytopenic purpura (ITP). Participants had blood tests, bone marrow biopsies, and a short course of the steroid prednisone. The goal was to find which…

This study looked at 33 adults who had both immune thrombocytopenia (ITP) — a condition where the immune system destroys platelets — and cancer. Researchers wanted to see if treating the cancer could also improve ITP. They also investigated whether cancer cells might mimic platel…

This study looked at how chronic immune thrombocytopenia (ITP) affects thinking skills and fatigue in adults. Researchers measured brain function and inflammation using blood tests and questionnaires in 97 patients. The goal was to better understand these connections, not to test…

This study looked at how well a daily low-dose aspirin works in people who have both immune thrombocytopenia (low platelets) and heart disease. Only 4 adults took part. Researchers measured platelet function after aspirin to see if the standard dose is enough to prevent blood clo…

This study looked back at 223 people with acquired thrombotic thrombocytopenic purpura (aTTP) who received caplacizumab (Cablivi). The drug quickly normalizes platelet counts, but some patients experience a sharp rise then a dip, which might mislead doctors into giving unnecessar…