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Prepublished online as a Blood First Edition Paper on August 8, 2002; DOI 10.1182/blood-2001-12-0171. This Article Full Text (PDF) All Versions of this Article: 2001-12-0171v1 101/1/71    most recent Alert me when this article is cited Alert me if a correction is posted Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Huhn, R. D Articles by Dunbar, C. E Search for Related Content PubMed PubMed Citation Articles by Huhn, R. D Articles by Dunbar, C. E Social Bookmarking                   What's this? Submitted December 7, 2001 Accepted July 26, 2002 High-dose cyclophosphamide with autologous lymphocyte-depleted peripheral blood stem cell (PBSC) support for treatment of refractory chronic autoimmune thrombocytopenia Richard D Huhn*, Patrick Fogarty, Ryotaro Nakamura, Elizabeth J Read, Susan F Leitman, Margaret E Rick, Janice Kimball, Adeira Greene, Kristin Hansmann, Alois Gratwohl, Neal S Young, A John Barrett, and Cynthia E Dunbar Hematology Branch, National Institutes of Health, National Heart Lung & Blood Institute, Bethesda, MD, USA; Umbilical Cord Blood Stem Cell Laboratory, Coriell Institute for Medical Research, Camden, NJ, USA Hematology Branch, National Institutes of Health, National Heart Lung & Blood Institute, Bethesda, MD, USA Department of Transfusion Medicine, National Institutes of Health, Warren Grant Magnuson Clinical Center, Bethesda, MD, USA Department of Laboratory Medicine, National Institutes of Health, Warren Grant Magnuson Clinical Center, Bethesda, MD, USA Department of Internal Medicine, University of Basel Kantonsspital, Basel, Switzerland * Corresponding author; email: huhnr{at}att.net. Patients with refractory chronic autoimmune thrombocytopenia (AITP) have a significant risk of morbidity and mortality related to hemorrhage. High-dose (HD) cytotoxic therapy may produce remissions but entails risks related to myelosuppression. Hematopoietic stem cell support with lymphocyte-depleted grafts may accelerate hematologic recovery and concomitantly reduce repopulation by autoreactive immunocytes. Fourteen patients with chronic AITP, who had failed multiple prior therapies including corticosteroids, splenectomy, intravenous immunoglobulin and various cytotoxic or immunomodulatory regimens were treated with HD cyclophosphamide (50 mg/kg/day) and autologous G-CSF-mobilized leukocytes depleted of lymphocytes by immunomagnetic CD34-positive selection. There were no significant adverse events related to G-CSF, intravenous device insertion or leukapheresis. Treatment-related complications predictably included transient hemorrhagic cystitis (1 patients), vaginal bleeding (2 patients), gastrointestinal bleeding (one patient), epistaxis (one patient) and antibiotic-responsive febrile neutropenia (all patients). The mean time to ANC 500/mm3 was 9 ± 0.6 days. Eight patients experienced antibiotic-responsive gram-positive bacteremia. A median of 2 platelet transfusions was required for stem cell mobilization, intravenous catheter insertion and apheresis and a median of 9 platelet transfusions was required during hematopoietic recovery. Six patients obtained durable complete responses (platelet counts >100,000/mm3 without other therapy) with maximum follow-up of 42 months. Two additional patients obtained durable partial responses (platelet counts significantly increased over baseline with reduced medication requirements and cessation of bleeding complications). This therapeutic approach is feasible and relatively non-toxic for patients with severe chronic AITP, a substantial proportion of whom may obtain durable remissions. Larger controlled trials are recommended. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: J. Burzynski New options after first-line therapy for chronic immune thrombocytopenic purpura Am. J. Health Syst. Pharm., January 15, 2009; 66(2_Supplement_2): S11 - S21. [Abstract] [Full Text] [PDF] M. Ruggeri, S. Fortuna, and F. Rodeghiero Heterogeneity of terminology and clinical definitions in adult idiopathic thrombocytopenic purpura: a critical appraisal from a systematic review of the literature Haematologica, January 1, 2008; 93(1): 98 - 103. [Abstract] [Full Text] [PDF] E. Elli, M. Parma, P. Perseghin, M. Dassi, E. Terruzzi, D. Belotti, P. Pioltelli, and E.M. 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