Intense immunosuppressive therapy followed by autologous peripheral blood selected progenitor cell reinfusion for severe autoimmune disease
| Autor: | Renato Scalone, Ferdinando Porretto, Francesca Bondì, Alessandra Crescimanno, Vita Polizzi, Maurizio Musso |
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| Rok vydání: | 2001 |
| Předmět: |
Adult
Male medicine.medical_specialty Transplantation Conditioning Adolescent Cyclophosphamide medicine.medical_treatment Thrombotic thrombocytopenic purpura Aftercare Pure red cell aplasia Antigens CD34 Methylprednisolone Transplantation Autologous Gastroenterology Autoimmune Diseases Autologous stem-cell transplantation Internal medicine medicine Humans Antilymphocyte Serum Autoimmune disease Chemotherapy Immunomagnetic Separation business.industry Hematopoietic Stem Cell Transplantation Hematology Middle Aged medicine.disease Cryoglobulinemia Surgery Hospitalization Treatment Outcome Absolute neutrophil count Female business Immunosuppressive Agents Follow-Up Studies medicine.drug |
| Zdroj: | American Journal of Hematology. 66:75-79 |
| ISSN: | 1096-8652 0361-8609 |
| Popis: | Autologous stem cell transplantation (HSCT) has been shown to be effective in curing a large spectrum of autoimmune disorders. Case reports are being collected in the EBMT/EULAR Autoimmune Disease Stem Cell Project registry, which reports transplant-related mortality (TRM) of 6%. In order to reduce TRM and preserve the anti-autoimmune effect we evaluated a more immunoablative as opposed to myeloablative conditioning regimen for the autotransplant of severe immunomediated diseases. We enrolled patients affected by systemic lupus erythematosus (SLE: 3 patients), by autoimmune thrombocytopenic purpura (AITP: one patient), by thrombotic thrombocytopenic purpura (TTP: one patient), by pure red cell aplasia (PRCA: one patient), and by a severe cryoglobulinemia (one patient). All patients were mobilized with cyclophosphamide (Cy) 4 g/m2 + G-csf. Conditioning regimen consisted of Cy 50 mg/kg/day (days −6 and −5); anti-T-globulin (ATG) 10 mg/kg/day and 6-methylprednisolone (PDN) 1 g/day (days −4, −3, and −2). Immunomagnetically selected CD34+ cells were re-infused on day 0. In three patients neutrophil count fell below 0.5 × 109/l, while a PLT count below 20 × 109/l was registered in two patients. Extrahematological toxicity was very low. Four patients (2 SLE, 1 TTP, 1 cryoglobulinemia) are in complete corticosteroid-free remission with a median follow up of 335 days. The third SLE patient improved considerably; however, he still needs low-dose corticosteroid maintenance. The AITP and PRCA patients achieved a CR but soon relapsed; nevertheless, the procedure restored a steroid-sensitive status. The use of this immunoablative conditioning regimen in auto-HSCT transplant was shown to be effective in controlling disease progression and could be a valuable strategy in reducing TRM. Am. J. Hematol. 66:75–79, 2001 © 2001 Wiley-Liss, Inc. |
| Databáze: | OpenAIRE |
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