Renata Guzicka-Kazimierczak, Arkadiusz Kazimierczak, Jeremy Clark


Purpose: Thrombotic thrombocytopenic purpura is a rare disease. Therefore, the small numbers of patients in trials results in statistically weak evidence supporting treatment guidelines. The aetiology of this disease is based on a deficiency of metalloproteinase 13 (ADAMTS-13), which leads to the creation of von Willebrand factor polymers and platelet microthrombosis in small vessels. Treatment relies on plasma exchange. Immunosuppression based on cyclophosphamide is not recommended in current guidelines. The aim of this study was to assess the early and long-term effects of treatment, and to evaluate therapy prognostic factors.

Material and methods: This was a retrospective study of 10 patients hospitalized 14 times due to new or recurrent onset of TTP. Currently recommended treatment was used in all cases, including total plasma exchange (range 0.5–2 plasma volume). The volume of exchange plasma was 24.6 L (mean) over 20 days (median). All patients received glucocorticosteroids. Immunosuppression (based on cyclophosphamide in 7 cases) was utilized in 70% of patients (9 patients).

Results: Of all 11 patients 9 (70%) survived and achieved remission. Fifty percent of patients stayed in long-lasting remission. Average follow-up time was 36 months (median 14.5 months, range 10 days to 108 months). Factors improving the chance of remission were: age below 55 years (p < 0.05), no tachycardia on admission to hospital (p < 0.001), and immunosuppression based on cyclophosphamide (p = 0.032).

Conclusions: Presence of tachycardia on TTP onset and age above 55 years reduce the survival and remission rate. Remission from TTP is suggested to be caused by immunosuppression based on cyclophosphamide.


thrombotic thrombocytopenic purpura; cyclophosphamide; total plasma exchange; plasmapheresis; immunosuppression

Full Text:



Choroby wewnętrzne. Ed. A. Szczeklik. Medycyna Praktyczna, Kraków 2006, 1555–1568.

Dmoszyńska A., Robak T.: Podstawy Hematologii. Wyd. Czelej, Lublin 2008, 446–448.

Scully M., Hunt J.B., Benjamin S., Liesner R., Rose P., Peyvandi F. et al.: Guidelines on the diagnosis and management of thrombotic thrombocytopenic purpura and other thrombotic microangiopathies. Br J Haematol. 2012, 158 (3), 323–325.

O’Brien K.L., Price T.H., Howell C., Delaney M.: The use of 50% albumin/plasma replacement fluid in therapeutic plasma exchange for thrombotic thrombocytopenic purpura. J Clin Apher. 2013, 28 (6), 416–421.

George J.N.: Forecasting the future for patients with hereditary TTP. Blood. 2012, 120 (2), 243–244.

Machin S.J.: Clinical annotation. Thrombotic thrombocytopenic purpura. Br J Haematol. 1984, 56, (2), 191–197.

Pratt K.P.: Presenting ADAMTS13 on a TTP- associated MHC. Blood. 2013, 121 (17), 3302–3303.

Hoffman R., Benz E.J., Sanford J. Shattil, Furie B., Silberstein L.E. et al.: Thrombotic thrombocytopenic purpura and the hemolytic uremic syndrome. In: Hematology. Elsevier Inc., Churchill Livingstone 2009, 2099 2111.

Levy G.G., Nichols W.C., Lian E.C., Foroud T., McClintick J.N., McGee B.M. et al.: Mutations in a member of the ADAMTS gene family cause thrombotic thrombocytopenic purpura. Nature. 2001, 413, 488–494.

McPherson R.A., Pincus M.R.: Henry’s clinical diagnosis and management by laboratory methods. Elseviere Saunders, Philadelphia 2011, 557–600.

Knovich M.A., Farland A., Owen J.: Long-term management of acquired thrombotic thrombocytopenic purpura using serial plasma ADAMTS13 measurements. Eur J Haematol. 2012, 88 (6), 518–525.

Bhagirath V.C., Kelton J.G., Moore J., Arnold D.M.: Rituximab maintenance for relapsed refractory thrombotic thrombocytopenic purpura. Transfusion. 2012, 52 (12), 2517–2523.

Kaushansky K.: The vWF- cleaving protease: new opportunities in TTP. Blood. 2001, 98 (6), 1643–1644.

George J.N.: Controlling chronic TTP. Blood. 2005, 106 (6), 1896.

Deford C.C., Reese J.A., Schwartz L.H., Perdue J.J., Kremer Hovinga J.A., Lämmle B. et al.: Multiple major morbidities and increased mortality during long-term follow-up after recovery from thrombotic thrombocytopenic purpura. Blood. 2013, 122 (12), 2023–2029.

Rock G.A., Sumak K.H., Buskard N.A., Blanchette V.S., Kelton J.G., Nair R.C. et al.: Comparison of plasma exchange with plasma infusion in the treatment of thrombocytic thrombocytopenic purpura. N Engl J Med. 1991, 325 (6), 393–397.

Goyal J., Adamski J., Lima J.L., Marques M.B.: Relapses of thrombotic thrombocytopenic purpura after treatment with rituximab. J Clin Apher. 2013, 28 (6), 390–394.

George J.N., Al.- Nouri Z.A.: Diagnostic ant therapeutic challenges in the thrombotic thrombocytopenic purpura and hemolytic uremic syndromes. Hematology Am Soc Hemat Educ Program. 2012, 1, 604–609.


Copyright (c) 2016 Renata Guzicka-Kazimierczak, Arkadiusz Kazimierczak, Jeremy Clark

License URL: