Autologous hematopoietic stem cell transplantation in multiple sclerosis: A phase II trial
| Autor: | Mancardi, Giovanni L., Sormani, Maria P., Gualandi, Francesca, Saiz, Albert, Carreras, Eric, Merelli, Elisa, Donelli, Amedea, Di Bartolomeo, Paolo, Rottoli, Maria R., Rambaldi, Alessandro, Amato, Maria P., Massacesi, Luca, Di Gioia, Massimo, Vuolo, Luisa, Currò, Daniela, Roccatagliata, Luca, Filippi, Massimo, Aguglia, Umberto, Iacopino, Pasquale, Farge, Dominique, Saccardi, Riccardo, ASTIMS Haemato Neurological Collaborative Group, on behalf of the Autoimmune Disease Working Party of the European Group for Blood, Marrow Transplantation, ASTIMS Haemato Neurological Collaborative Group on behalf of the Autoimmune Disease Working Party ADWP of the European Group for Blood, Marrow Transplantation EBMT, LUGARESI, ALESSANDRA |
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| Přispěvatelé: | Mancardi, Gl, Sormani, Mp, Gualandi, F, Saiz, A, Carreras, E, Merelli, E, Donelli, A, Lugaresi, A, Di Bartolomeo, P, Rottoli, Mr, Rambaldi, A, Amato, Mp, Massacesi, L, Di Gioia, M, Vuolo, L, Currà, D, Roccatagliata, L, Filippi, Massimo, Aguglia, U, Iacopino, P, Farge, D, Saccardi, R., Mancardi, Giovanni L., Sormani, Maria P., Gualandi, Francesca, Saiz, Albert, Carreras, Eric, Merelli, Elisa, Donelli, Amedea, Lugaresi, Alessandra, Di Bartolomeo, Paolo, Rottoli, Maria R., Rambaldi, Alessandro, Amato, Maria P., Massacesi, Luca, Di Gioia, Massimo, Vuolo, Luisa, Currò, Daniela, Roccatagliata, Luca, Aguglia, Umberto, Iacopino, Pasquale, Farge, Dominique, Saccardi, Riccardo, ASTIMS Haemato-Neurological Collaborative Group, on behalf of the Autoimmune Disease Working Party (ADWP) of the European Group for Blood and Marrow Transplantation (EBMT)., ASTIMS Haemato-Neurological Collaborative Group on behalf of the Autoimmune Disease Working Party ADWP of the European Group for Blood and Marrow Transplantation EBMT |
| Rok vydání: | 2015 |
| Předmět: |
Oncology
Adult Male medicine.medical_specialty Transplantation Conditioning medicine.medical_treatment Antineoplastic Agents Gadolinium Hematopoietic stem cell transplantation Filgrastim Transplantation Autologous Antineoplastic Agent Young Adult Multiple Sclerosis Relapsing-Remitting Internal medicine medicine Clinical endpoint Humans Carmustine Mitoxantrone Expanded Disability Status Scale business.industry Hematopoietic Stem Cell Transplantation Immunosuppression Middle Aged Multiple Sclerosis Chronic Progressive Image Enhancement Magnetic Resonance Imaging Transplantation Autologou Transplantation Treatment Outcome Immunology Female Neurology (clinical) business Human medicine.drug |
| Popis: | Objective: To assess in multiple sclerosis (MS) the effect of intense immunosuppression followed by autologous hematopoietic stem cells transplantation (AHSCT) vs mitoxantrone (MTX) on disease activity measured by MRI. Methods: We conducted a multicenter, phase II, randomized trial including patients with secondary progressive or relapsing-remitting MS, with a documented increase in the last year on the Expanded Disability Status Scale, in spite of conventional therapy, and presence of one or more gadolinium-enhancing (Gd+) areas. Patients were randomized to receive intense immunosuppression (mobilization with cyclophosphamide and filgrastim, conditioning with carmustine, cytosine-arabinoside, etoposide, melphalan, and anti-thymocyte globulin) followed by AHSCT or MTX 20 mg every month for 6 months. The primary endpoint was the cumulative number of new T2 lesions in the 4 years following randomization. Secondary endpoints were the cumulative number of Gd+ lesions, relapse rate, and disability progression. Safety and tolerability were also assessed. Twenty-one patients were randomized and 17 had postbaseline evaluable MRI scans. Results: AHSCT reduced by 79% the number of new T2 lesions as compared to MTX (rate ratio 0.21, p = 0.00016). It also reduced Gd+ lesions as well as the annualized relapse rate. No difference was found in the progression of disability. Conclusion: Intense immunosuppression followed by AHSCT is significantly superior to MTX in reducing MRI activity in severe cases of MS. These results strongly support further phase III studies with primary clinical endpoints. The study was registered as EUDRACT No. 2007-000064-24. |
| Databáze: | OpenAIRE |
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