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Recombinant ADAMTS13 for Immune Thrombotic Thrombocytopenic Purpura.
Bendapudi, Pavan K; Foy, Brody H; Mueller, Sarah B; Liu, Jun; Feingold, Louis M; Burke, Kristen E; Cruz, Wendy; Chen, Maria Y; Lau, Emily S; Goldberg, Rachel L; Tatake, Ishan; Wilkinson, Shelby C; Carney, Brian J; Stone, James R; Park, Doyun; Avelino, Alzira R; Hassan, Sajjad; Andrzejewski, Chester; Ruby, Kristen N; Friedman, Kenneth D; Brunker, Patricia A R; Leaf, Rebecca K; Higgins, John; Dzik, Walter H; Stefely, Jonathan A; Makar, Robert S.
Affiliation
  • Bendapudi PK; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Foy BH; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Mueller SB; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Liu J; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Feingold LM; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Burke KE; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Cruz W; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Chen MY; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Lau ES; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Goldberg RL; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Tatake I; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Wilkinson SC; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Carney BJ; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Stone JR; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Park D; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Avelino AR; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Hassan S; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Andrzejewski C; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Ruby KN; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Friedman KD; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Brunker PAR; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Leaf RK; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Higgins J; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Dzik WH; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Stefely JA; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
  • Makar RS; From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division
N Engl J Med ; 390(18): 1690-1698, 2024 May 09.
Article in En | MEDLINE | ID: mdl-38718359
ABSTRACT
In patients with immune thrombotic thrombocytopenic purpura (iTTP), autoantibodies against the metalloprotease ADAMTS13 lead to catastrophic microvascular thrombosis. However, the potential benefits of recombinant human ADAMTS13 (rADAMTS13) in patients with iTTP remain unknown. Here, we report the clinical use of rADAMTS13, which resulted in the rapid suppression of disease activity and complete recovery in a critically ill patient whose condition had proved to be refractory to all available treatments. We also show that rADAMTS13 causes immune complex formation, which saturates the autoantibody and may promote its clearance. Our data support the role of rADAMTS13 as a novel adjunctive therapy in patients with iTTP.
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Full text: 1 Database: MEDLINE Main subject: Purpura, Thrombotic Thrombocytopenic / ADAMTS13 Protein Limits: Adult / Female / Humans Language: En Journal: N Engl J Med Year: 2024 Type: Article
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Full text: 1 Database: MEDLINE Main subject: Purpura, Thrombotic Thrombocytopenic / ADAMTS13 Protein Limits: Adult / Female / Humans Language: En Journal: N Engl J Med Year: 2024 Type: Article