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From the Department of Neurological Sciences and Vision (Drs. Mancardi and Murialdo), University of Genova; II Division of Hematology and Stem Cell Transplantation Centre (Drs. Gualandi and Marmont), San Martinos Hospital; Unit of Clinical Epidemiology and Trials (Dr. Sormani), National Cancer Institute; Department of Radiology (Dr. Sardanelli), Biomedical Institute, Genova; Bone Marrow Transplantation Unit (Drs. Saccardi and Pagliai), Careggi Hospital; Department of Neurological and Psychiatric Sciences (Dr. Massacesi), University of Firenze; Neuroimaging Research Unit (Drs. Filippi and Inglese), Department of Neuroscience, Scientific Institute, Ospedale San Raffaele, Milan; Department of Neuroscience (Dr. Marrosu), University of Cagliari; Department of Neuroscience (Dr. Meucci), University of Pisa; and Department of Oncology and Neuroscience (Dr. Lugaresi), University "Gabriele dAnnunzio," Chieti, Italy.
Address correspondence and reprint requests to Dr. G.L. Mancardi, Department of Neurological Sciences and Vision, University of Genova, via de Toni 5, 16132 Genova, Italy; e-mail: neurolab{at}cisi.unige.it
BACKGROUND: Autologous hematopoietic stem cell transplantation (ASCT) has been recently utilized with encouraging results in patients with poorly controlled MS.
OBJECTIVE: To determine in severe cases of MS the effect of ASCT on gadolinium (Gd)-enhanced MRI and to obtain information on clinical course and safety.
METHODS: In a cooperative study, 10 patients with rapidly evolving secondary progressive MS were transplanted, after BEAM conditioning regimen (carmustine, etoposide, cytosine-arabinoside, and melphalan), with unmanipulated autologous peripheral blood SC mobilized with high-dose cyclophosphamide (CY; 4 g/m2) and granulocyte-colony-stimulating factor. Triple-dose Gd-enhanced scans were performed monthly for a pretreatment period of 3 months and compared with serial monthly Gd-enhanced MRI for the following 6 months and then once every 3 months.
RESULTS: The median follow-up is now 15 months (range 4 to 30 months). The number of Gd-enhancing lesions decreased immediately after mobilization with CY and finally dropped to zero in all cases after the conditioning regimen. The number of new T2-weighted positive lesions paralleled data obtained for Gd-enhanced MRI. Clinically, patients improved slightly or remained stable.
CONCLUSION: These results demonstrate that the therapeutic sequence CYBEAMASCT has the capacity to completely suppress MR-enhancing activity, an effect that is sustained with time. The final impact of this procedure on disease course remains to be established.
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