Improved survival in thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS): clinical experience in 108 patients. William R. Bell, M.D., Hayden G. Braine, M.D., Paul M. Ness, M.D., and Thomas S. Kickler, M.D. Abstract: Thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS) is characterized by microangiopathic hemolytic anemia, thrombocytopenia, fever, central nervous system abnormalities, and renal dysfunction. In early reports, the mortality rate was nearly 100%. A treatment protocol was introduced in 1979 for patients admitted to Johns Hopkins Hospital with the diagnosis of TTP-HUS. Treatment regimens included prednisone alone for patients with minimal symptoms and no central nervous system symptoms, and prednisone plus plasma exchange for patients with rapid clinical deterioration who did not improve after 48 hours of prednisone alone and for patients presenting with central nervous system symptoms and rapidly declining hematocrit values and platelet counts. TTP-HUS is a spectrum of disorders that includes thrombotic thrombocytopenic purpura and hemolytic uremic syndrome. In early reports, the mortality rate was nearly 100%. One of the first documented survivors of the disease, described in 1959, was treated with exchange transfusions. A variety of other therapeutic approaches had been tried in an uncontrolled fashion with limited success. Because of the often fulminant and unrelenting rapid progression of the disorder, multiple treatments have often been employed simultaneously, making it difficult to determine which therapy was effective. In 1963, it was suggested that plasma therapy might be beneficial. We report our experience with the treatment of TTP-HUS with corticosteroids and plasmapheresis with plasma replacement in patients admitted to our facility from September 1979 to December 1990. A placebo-controlled study was not done because plasmapheresis is the only form of therapy for TTP-HUS that is acknowledged to be effective. From September 1979 to December 1990, all patients admitted to Johns Hopkins University Hospital who satisfied the following criteria for the diagnosis of TTP-HUS were treated according to a single protocol. The diagnosis was made if the patient had at least four of the following findings: microangiopathic hemolytic anemia with a negative Coombs' test, thrombocytopenia, central nervous system abnormalities, fever, and renal dysfunction. In some patients, the diagnosis was confirmed by the presence of intravascular hyaline thrombi in biopsy samples of gingival tissue or bone marrow. Therapy was instituted as soon as the diagnosis of TTP-HImproved survival in thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS): clinical experience in 108 patients. William R. Bell, M.D., Hayden G. Braine, M.D., Paul M. Ness, M.D., and Thomas S. Kickler, M.D. Abstract: Thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS) is characterized by microangiopathic hemolytic anemia, thrombocytopenia, fever, central nervous system abnormalities, and renal dysfunction. In early reports, the mortality rate was nearly 100%. A treatment protocol was introduced in 1979 for patients admitted to Johns Hopkins Hospital with the diagnosis of TTP-HUS. Treatment regimens included prednisone alone for patients with minimal symptoms and no central nervous system symptoms, and prednisone plus plasma exchange for patients with rapid clinical deterioration who did not improve after 48 hours of prednisone alone and for patients presenting with central nervous system symptoms and rapidly declining hematocrit values and platelet counts. TTP-HUS is a spectrum of disorders that includes thrombotic thrombocytopenic purpura and hemolytic uremic syndrome. In early reports, the mortality rate was nearly 100%. One of the first documented survivors of the disease, described in 1959, was treated with exchange transfusions. A variety of other therapeutic approaches had been tried in an uncontrolled fashion with limited success. Because of the often fulminant and unrelenting rapid progression of the disorder, multiple treatments have often been employed simultaneously, making it difficult to determine which therapy was effective. In 1963, it was suggested that plasma therapy might be beneficial. We report our experience with the treatment of TTP-HUS with corticosteroids and plasmapheresis with plasma replacement in patients admitted to our facility from September 1979 to December 1990. A placebo-controlled study was not done because plasmapheresis is the only form of therapy for TTP-HUS that is acknowledged to be effective. From September 1979 to December 1990, all patients admitted to Johns Hopkins University Hospital who satisfied the following criteria for the diagnosis of TTP-HUS were treated according to a single protocol. The diagnosis was made if the patient had at least four of the following findings: microangiopathic hemolytic anemia with a negative Coombs' test, thrombocytopenia, central nervous system abnormalities, fever, and renal dysfunction. In some patients, the diagnosis was confirmed by the presence of intravascular hyaline thrombi in biopsy samples of gingival tissue or bone marrow. Therapy was instituted as soon as the diagnosis of TTP-H