Abstract |
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 x 10(9)/l, while a PLT count below 20 x 10(9)/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.
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Authors | M Musso, F Porretto, A Crescimanno, F Bondi, V Polizzi, R Scalone |
Journal | American journal of hematology
(Am J Hematol)
Vol. 66
Issue 2
Pg. 75-9
(Feb 2001)
ISSN: 0361-8609 [Print] United States |
PMID | 11421302
(Publication Type: Journal Article)
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Chemical References |
- Antigens, CD34
- Antilymphocyte Serum
- Immunosuppressive Agents
- Cyclophosphamide
- Methylprednisolone
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Topics |
- Adolescent
- Adult
- Aftercare
- Antigens, CD34
(analysis)
- Antilymphocyte Serum
(administration & dosage, toxicity)
- Autoimmune Diseases
(complications, therapy)
- Cyclophosphamide
(administration & dosage, toxicity)
- Female
- Follow-Up Studies
- Hematopoietic Stem Cell Transplantation
(methods)
- Hospitalization
- Humans
- Immunomagnetic Separation
- Immunosuppressive Agents
(administration & dosage, therapeutic use)
- Male
- Methylprednisolone
(administration & dosage, toxicity)
- Middle Aged
- Transplantation Conditioning
(adverse effects, methods, standards)
- Transplantation, Autologous
(adverse effects, methods, standards)
- Treatment Outcome
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