Recombinant ADAMTS13 for Immune Thrombotic Thrombocytopenic Purpura

This brief report details the compassionate use of recombinant human ADAMTS13 (rADAMTS13) in a critically ill patient with immune thrombotic thrombocytopenic purpura (iTTP) refractory to standard treatments. The patient, a 28-year-old woman, showed rapid clinical improvement and complete recovery following intravenous rADAMTS13 therapy. Laboratory data revealed that rADAMTS13 formed immune complexes with autoantibodies, saturating and possibly promoting their clearance. The study provides pharmacokinetic and mechanistic insights, suggesting rADAMTS13 may serve as a valuable adjunctive therapy in severe iTTP cases.

This brief report details the compassionate use of recombinant human ADAMTS13 (rADAMTS13) in a critically ill patient with immune thrombotic thrombocytopenic purpura (iTTP) refractory to standard treatments. The patient, a 28-year-old woman, showed rapid clinical improvement and complete recovery following intravenous rADAMTS13 therapy. Laboratory data revealed that rADAMTS13 formed immune complexes with autoantibodies, saturating and possibly promoting their clearance. The study provides pharmacokinetic and mechanistic insights, suggesting rADAMTS13 may serve as a valuable adjunctive therapy in severe iTTP cases.