Researchers sought to determine whether deferiprone would have the safe efficacy as deferoxamine for treating iron-overloaded patients with sickle cell disease or other transfusion-dependent anemias.
Researchers sought to determine whether LentiGlobin was safe and effective for treating a subset of patients with sickle cell disease.
Vadadustat is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor.
Researchers sought to determine whether paroxysmal nocturnal hemoglobinuria clones might predict treatment responses for patients with MDS and aplastic anemia.
Researchers sought to determine whether cfDNA is not only a marker of, but makes a contribution to, pathophysiology of sickle cell disease.
Researchers sought to determine whether haplo-identical donors could be an alternative option for patients with Fanconi anemia undergoing HSCT.
Researchers sought to determine whether ticagrelor would be a safe and effective option for patients younger than 24 months with sickle cell disease.
Researchers sought to determine what should be the primary treatment goal for patients with sickle cell disease.
Researchers sought to determine whether haplo-identical donors could be an alternative option for patients with Fanconi anemia undergoing HSCT.
Researchers sought to determine whether deferasirox would be a good option for long-term management of iron overload in patients who receive iron chelation therapy.
