Safety and efficacy of autologous hematopoietic stem-cell transplantation following natalizumab discontinuation in aggressive multiple sclerosis
Autor: | Alessandro Barilaro, R. Nistri, Riccardo Saccardi, Chiara Innocenti, Claudia Mechi, E. Magnani, Luca Massacesi, A. Repice, Benedetta Forci, Alice Mariottini, A. Fani |
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Rok vydání: | 2018 |
Předmět: |
Adult
Male medicine.medical_specialty Cyclophosphamide medicine.medical_treatment Hematopoietic stem cell transplantation Disease 03 medical and health sciences 0302 clinical medicine Natalizumab Multiple Sclerosis Relapsing-Remitting Internal medicine parasitic diseases medicine Humans 030212 general & internal medicine Retrospective Studies business.industry Multiple sclerosis Progressive multifocal leukoencephalopathy Hematopoietic Stem Cell Transplantation Middle Aged medicine.disease Discontinuation Transplantation Treatment Outcome Neurology Withholding Treatment Female Neurology (clinical) business 030217 neurology & neurosurgery Immunosuppressive Agents medicine.drug |
Zdroj: | European journal of neurology. 26(4) |
ISSN: | 1468-1331 |
Popis: | Background and purpose Natalizumab (NTZ) is a highly effective treatment for relapsing-remitting multiple sclerosis (MS), but its withdrawal is often followed by disease reactivation or rebound, even if other disease-modifying treatments (DMTs) are administered. In this study, for the first time, the safety and efficacy of autologous hematopoietic stem-cell transplantation (aHSCT) performed following NTZ discontinuation were retrospectively compared with conventional DMTs. Methods Patients with relapsing-remitting MS treated with NTZ who discontinued the drug after at least six administrations and with at least 6 months of follow-up were included. Patients underwent aHSCT after a minimum of 6 months following NTZ withdrawal, receiving meanwhile cyclophosphamide or corticosteroids, or other DMTs approved for MS (control group) after an adequate wash-out period. Both hematological and neurological follow-up were assessed according to standard policies. Results A total of 52 patients were included, 11 who received aHSCT and 41 who received DMTs. Baseline clinical and demographic characteristics were similar between the two groups. No fatality or life-threatening complications, including progressive multifocal leukoencephalopathy, were observed. At 3 years following NTZ discontinuation, no evidence of disease activity was reported in 54.5% of the patients in the aHSCT group compared with 11.5% of those in the DMT group (P = 0.0212). Disease reactivation in the patients with aHSCT was observed only during wash-out/bridging therapy and 100% of the cases were free from disease activity after aHSCT. Conclusions These data suggest that an aggressive therapy should be established after NTZ with the shortest possible wash-out period. aHSCT after 6 months from NTZ withdrawal appears to be safe. |
Databáze: | OpenAIRE |
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