Researchers sought to determine whether gene therapy with valoctocogene roxaparvovec would improve health-related QoL for men with severe hemophilia A.
Researchers sought to determine whether prophylactic EHL factor VIII or IX concentrates would improve adherence rates and outcomes in patients with hemophilia.
Researchers sought to determine the cost-effectiveness of prophylactic emicizumab in patients with moderate or mild hemophilia A without inhibitors in the United States.
Researchers sought to determine whether valoctocogene roxaparvovec would yield hemostatic efficacy in patients with severe hemophilia A without inhibitors.
Researchers sought to determine whether responses would be sustained after TPO-RA discontinuation among patients with persistent or chronic immune thrombocytopenia.
Researchers sought to determine whether withholding prophylactic platelet transfusions before central venous catheter placement would improve bleeding outcomes in patients with severe thrombocytopenia.
Researchers sought to determine whether there is a link between recombinant factor VIII Fc fusion protein and time to tolerization in patients with severe hemophilia A .
Researchers sought to develop recommendations for future research opportunities to address the priorities of patients with ultra-rare bleeding disorders.
Researchers sought to determine whether there were changes in participant-reported physical activity among people with moderate to severe hemophilia during the COVID-19 lockdown.
Researchers sought to determine whether TQG202 would lead to effective bleeding control when used on-demand for patients with moderate-to-severe hemophilia A.
Researchers sought to determine whether rondaptivon pegol would be an effective treatment to increase endogenous FVIII levels in patients with hemophilia A.
Researchers sought to determine whether inhibitor incidence with N8-GP prophylaxis would be within range for patients with previously untreated hemophilia A.
Researchers sought to determine whether there were any changes in prophylactic treatment practices among previously untreated patients with hemophilia.