ABSTRACT
Congenital thrombotic thrombocytopenia purpura (cTTP) is a very rare disorder worldwide. Standard treatment of recognized cases has been to administer fresh frozen plasma as the source of ADAMTS13, to replenish the absent ADAMTS13 enzyme. An alternative source, a plasma-derived factor VIII concentrate used for hemophilia A, and found to contain this enzyme, was reported to be effective in 1 patient in the United States. We now report details on a US cohort of 8 cTTP patients who have been successfully treated for varying periods with a marketed antihemophilic factor concentrate Koate-DVI. This biological product has been used successfully on demand in varying doses to treat acute exacerbations, as well as prophylactically (3 to 6 U ADAMTS13 every 3 to 21 d). Self-infused at home, in lieu of fresh frozen plasma therapy in the hospital setting, this product has effectively prevented episodes of thrombocytopenia, microangiopathic hemolytic anemia, and the concomitant organ damage in these patients. This specific virus inactivated product can be used to prevent further manifestations of this congenital enzyme deficiency.
Subject(s)
ADAMTS13 Protein/administration & dosage , Purpura, Thrombotic Thrombocytopenic/drug therapy , ADAMTS13 Protein/deficiency , Adolescent , Adult , Anemia, Hemolytic/prevention & control , Child , Cohort Studies , Factor VIII/administration & dosage , Factor VIII/chemistry , Humans , Thrombocytopenia/prevention & control , United States , Young AdultABSTRACT
With 550,000 new cases each year, congestive heart failure is a major medical problem. Several medical therapies, including digoxin, angiotensin-converting enzyme inhibitors, and beta-blockers, have reduced the number of re-hospitalizations and slowed the progression of congestive heart failure. Angiotensin-converting enzyme inhibitors, some beta-blockers, and the combination of hydralazine with nitrates have improved survival. Despite these benefits, medical therapy frequently fails to improve quality of life. Biventricular pacing has been introduced to resynchronize mechanical and electrical asynchrony frequently observed in patients with heart failure. The most recent pacing trials show an improvement in quality of life and functional class. Long-term data are needed to determine the effect of biventricular pacing on survival. The acute hemodynamic studies suggest that resynchronization pacing therapy may predict a positive long-term benefit for many patients with congestive heart failure.
