Correspondence

Long-Term Outcomes after Treatment for Refractory Immune Thrombocytopenic Purpura

N Engl J Med 2001; 344:1402-1403May 3, 2001

Article

To the Editor:

In April 1993, my colleagues and I reported in the Journal our experience with combination chemotherapy as a treatment for patients with severe refractory immune thrombocytopenic purpura.1 We now provide long-term follow-up data on the initial eight patients with immune thrombocytopenic purpura and on four additional patients (Table 1Table 1Responses to Combination Chemotherapy of Patients with Refractory Immune Thrombocytopenic Purpura.). All patients had severe disease with periodic mucosal bleeding, and in all splenectomy had failed, as had an average of five other therapies. Of the 12 patients treated, 5 had a complete remission (a normal platelet count) and 1 had a partial remission (a platelet count of more than 50,000 per microliter); these remissions persisted without further therapy until the last follow-up visit or until their death from another cause (a cerebrovascular accident in Patient 5 and fulminant hepatitis C acquired from a transfusion in Patient 6). Remissions in the four surviving patients have lasted from more than 60 to more than 150 months.

Of the remaining six patients, two had a complete remission but relapsed 4 months and 35 months after treatment; Patient 7 is alive and in complete remission after high-dose therapy with cyclophosphamide (50 mg per kilogram of body weight per day for 4 days), and in Patient 8 normal platelet counts were maintained with prednisone (10 to 30 mg per day) and danazol (200 mg per day) until fungal abscesses developed and she died from a generalized fungal infection. Patient 9 had no response to combination chemotherapy but subsequently had a response to danazol (200 to 400 mg per day), colchicine (0.6 mg per day), and prednisone (10 to 15 mg per day), and she has had normal platelet counts for more than five years; her condition had previously been resistant to these drugs. The remaining three patients had no persistent response to combination chemotherapy or any other therapy and died within a few months as a result of central nervous system bleeding due to unremitting thrombocytopenia.

In summary, one half of this small group of patients with severe, life-threatening immune thrombocytopenic purpura remained in remission without further therapy after combination chemotherapy of the type used for lymphomas. Some of the other patients appeared to have responses to treatments that had previously failed. Combination chemotherapy should be considered for the select group of patients with severe refractory immune thrombocytopenic purpura in whom other forms of therapy have failed and who are at risk for death from their disease.

Robert McMillan, M.D.
Scripps Research Institute, La Jolla, CA 92037

1 References

1. 1

   Figueroa M, Gehlsen J, Hammond D, et al. Combination chemotherapy in refractory immune thrombocytopenic purpura. N Engl J Med 1993;328:1226-1229
   Full Text | Web of Science | Medline

Citing Articles (17)

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   Lisa J. Toltl, Donald M. Arnold. (2011) Pathophysiology and management of chronic immune thrombocytopenia: focusing on what matters. British Journal of Haematology 152:1, 52-60
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   Jyoti D Chouhan, Jon D Herrington. (2010) Treatment Options for Chronic Refractory Idiopathic Thrombocytopenic Purpura in Adults: Focus on Romiplostim and Eltrombopag. Pharmacotherapy 30:7, 666-683
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   L. Weiss, R. Greil. (2010) Short review – Management of Immune Thrombocytopenia in adults. memo - Magazine of European Medical Oncology 3:2, 69-72
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   D. Provan, R. Stasi, A. C. Newland, V. S. Blanchette, P. Bolton-Maggs, J. B. Bussel, B. H. Chong, D. B. Cines, T. B. Gernsheimer, B. Godeau, J. Grainger, I. Greer, B. J. Hunt, P. A. Imbach, G. Lyons, R. McMillan, F. Rodeghiero, M. A. Sanz, M. Tarantino, S. Watson, J. Young, D. J. Kuter. (2010) International consensus report on the investigation and management of primary immune thrombocytopenia. Blood 115:2, 168-186
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   D. M. Arnold, I. Nazi, A. Santos, H. Chan, N. M. Heddle, T. E. Warkentin, J. G. Kelton. (2010) Combination immunosuppressant therapy for patients with chronic refractory immune thrombocytopenic purpura. Blood 115:1, 29-31
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   James B. Bussel. (2009) Traditional and New Approaches to the Management of Immune Thrombocytopenia: Issues of When and Who to Treat. Hematology/Oncology Clinics of North America 23:6, 1329-1341
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   Aisha Hasan, Marc Michel, Vivek Patel, Roberto Stasi, Susanna Cunningham-Rundles, John P. Leonard, James Bussel. (2009) Repeated courses of rituximab in chronic ITP: Three different regimens. American Journal of Hematology 84:10, 661-665
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   Bertrand Godeau, Drew Provan, James Bussel. (2007) Immune thrombocytopenic purpura in adults. Current Opinion in Hematology 14:5, 535-556
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   J. N. GEORGE. (2006) Management of patients with refractory immune thrombocytopenic purpura. Journal of Thrombosis and Haemostasis 4:8, 1664-1672
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    Douglas B. Cines, Robert McMillan. (2005) Management of Adult Idiopathic Thrombocytopenic Purpura. Annual Review of Medicine 56:1, 425-442
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    Roscoe L. Warner, Harry C. Winter, Cecilia L. Speyer, James Varani, Irwin J. oldstein, Hedwig S. Murphy, Kent J. Johnson. (2004) Marasmius oreades lectin induces renal thrombotic microangiopathic lesions. Experimental and Molecular Pathology 77:2, 77-84
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    J. R. Passweg, M. Rabusin, M. Musso, Y. Beguin, S. Cesaro, G. Ehninger, I. Espigado, A. Iriondo, L. Jost, V. Koza, S. Lenhoff, I. Lisukov, F. Locatelli, A. Marmont, P. Philippe, C. Pilatrino, P. Quartier, J. Stary, P. Veys, J. Vormoor, A. Wahlin, F. Zintl, C. Bocelli-Tyndall, A. Tyndall, A. Gratwohl, . (2004) Haematopoetic stem cell transplantation for refractory autoimmune cytopenia. British Journal of Haematology 125:6, 749-755
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    R. Stasi, D. Provan. (2004) Management of Immune Thrombocytopenic Purpura in Adults. Mayo Clinic Proceedings 79:4, 504-522
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    James A. Williams, Laurence A. Boxer. (2003) Combination Therapy for Refractory Idiopathic Thrombocytopenic Purpura in Adolescents. Journal of Pediatric Hematology/Oncology 25:3, 232-235
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    Michael Gadenstätter, Bernd Lamprecht, Anton Klingler, Gerold J Wetscher, Richard Greil, Thomas Schmid. (2002) Splenectomy versus medical treatment for idiopathic thrombocytopenic purpura. The American Journal of Surgery 184:6, 606-609
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    Klaus Lechner. (2001) MANAGEMENT OF ADULT IMMUNE THROMBOCYTOPENIA. Reviews in Clinical and Experimental Hematology 5:3, 222-235
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    &NA;. (2001) Combination chemotherapy beneficial for refractory ITP. Inpharma Weekly &NA;:1287, 17
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