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Semin Thromb Hemost 2014; 40(01): 034-040
DOI: 10.1055/s-0033-1363165
Thieme Medical Publishers 333 Seventh Avenue, New York, NY 10001, USA.

Therapy for Thrombotic Thrombocytopenia Purpura: Past, Present, and Future

Kent Chapman
1   Department of Haematology, Pathology NSW, John Hunter Hospital, New South Wales, Australia
,
Sam Yuen
2   Department of Haematology, Calvary Mater Newcastle, New South Wales, Australia
› Author Affiliations
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Publication History

Publication Date:
31 December 2013 (online)

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Abstract

While clinical recognition of thrombotic thrombocytopenia purpura (TTP) has been evident for almost 90 years, the pathological basis of this disorder has only, relatively, recently been elucidated. Consequently, options for treating TTP had evolved rather slowly for many years. Despite this, current treatment practices of intensive plasma exchange often with immune modulation have seen survival rates increase dramatically. Nevertheless, the current understanding of TTP may witness the cusp of a new era for this disorder, with new and emerging treatments nearing clinical practice that specifically target the root cause of TTP. Some of these targeted approaches may even see the beginning of plasma-free treatments for TTP, with potentially faster recoveries and fewer long-term adverse effects.

Keywords

thrombotic thrombocytopenia purpura - TTP - therapy - von Willebrand cleaving protease - ADAMTS-13

Note

Since completing this review, a single report on the use of NAC in a case of TTP has recently emerged.[50] A 44-year-old woman presenting with clinical symptoms and laboratory results consistent with TTP was started on standard therapy including plasma exchange and corticosteroid treatment but remained largely unresponsive, and so rituximab was initiated. The patient's condition worsened and she became comatose; antibiotics were initiated, but cultures remained sterile. After 3 days of coma and further clinical deterioration, treatment with NAC was begun. The patient received a loading dose of 150 mg/kg NAC intravenously (IV) over 1 hour. Within 18 hours, the patient awakened abruptly and began communicating with medical personnel. Plasma exchange, corticosteroids, rituximab, and NAC infusion (150 mg/kg IV over 17 hr daily × 10 d) were continued and by day 17 the platelet count was more than 50 × 109/L. The patient fully recovered and was discharged on day 31.