scholarly journals Autologous haematopoietic stem cell transplantation for secondary progressive multiple sclerosis: an exploratory cost-effectiveness analysis

2009 ◽  
Vol 45 (6) ◽  
pp. 1014-1021 ◽  
Author(s):  
P Tappenden ◽  
R Saccardi ◽  
C Confavreux ◽  
B Sharrack ◽  
P A Muraro ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Cost Effectiveness ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Haematopoietic Stem Cell Transplantation ◽  
Progressive Multiple Sclerosis ◽  
Haematopoietic Stem Cell ◽  
Secondary Progressive ◽  
Effectiveness Analysis

Impact of autologous haematopoietic stem cell transplantation on disability and brain atrophy in secondary progressive multiple sclerosis

2020 ◽  
Vol 27 (1) ◽  
pp. 61-70 ◽  
Author(s):  
Alice Mariottini ◽  
Stefano Filippini ◽  
Chiara Innocenti ◽  
Benedetta Forci ◽  
Claudia Mechi ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Brain Atrophy ◽  
Haematopoietic Stem Cell Transplantation ◽  
Haematopoietic Stem Cell ◽  
Open Label ◽  
Free Survival ◽  
Secondary Progressive

Background: Autologous haematopoietic stem cell transplantation (aHSCT) is a valuable option in aggressive relapsing–remitting multiple sclerosis (MS), but its efficacy in secondary progressive (SP)-MS is still controversial. Objective: Assessing efficacy of aHSCT in SP-MS by clinical-radiological outcomes. Methods: Open-label monocentric retrospective study enrolling consecutive SP-MS patients treated with BEAM-aHSCT in the period 1999–2016. Results: In total, 26 SP-MS patients with moderate–severe disability were included. Progression-free survival (PFS) at years 5 and 10 after aHSCT were, respectively, 42% and 30%. Out of 16 patients who worsened, only 6 patients (23% overall) maintained continuous disability accrual (CDA), whereas 10 patients stabilized following one single-step Expanded Disability Status Scale (EDSS) worsening. CDA-free survival was 74% at 5–10 years. No relapses or magnetic resonance imaging (MRI) activity were reported, thus no evidence of disease activity (NEDA)-3 corresponded to PFS. Annualized rate of brain atrophy (AR-BVL) normalized after 1 year in 55% of the cases analysed (12/22). Conclusion: BEAM-aHSCT halted CDA and normalized AR-BVL in most of the treated patients, inducing long-term remission of inflammatory activity at a median follow-up of 99 months (range 27–222). These data suggest that CDA might still be mainly driven by inflammation in a subgroup of SP-MS and could therefore be reversed by treatments. CDA should be analysed independently from any isolated disability worsening.

scholarly journals Autologous haematopoietic stem cell transplantation fails to stop demyelination and neurodegeneration in multiple sclerosis

Brain ◽  
2007 ◽  
Vol 130 (5) ◽  
pp. 1254-1262 ◽  
Author(s):  
I. Metz ◽  
C. F. Lucchinetti ◽  
H. Openshaw ◽  
A. Garcia-Merino ◽  
H. Lassmann ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Haematopoietic Stem Cell Transplantation ◽  
Haematopoietic Stem Cell

scholarly journals Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis

2017 ◽  
Vol 13 (7) ◽  
pp. 391-405 ◽  
Author(s):  
Paolo A. Muraro ◽  
Roland Martin ◽  
Giovanni Luigi Mancardi ◽  
Richard Nicholas ◽  
Maria Pia Sormani ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Haematopoietic Stem Cell Transplantation ◽  
Haematopoietic Stem Cell

scholarly journals A prospective, randomized, controlled trial of autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: a position paper

2012 ◽  
Vol 18 (6) ◽  
pp. 825-834 ◽  
Author(s):  
R Saccardi ◽  
MS Freedman ◽  
MP Sormani ◽  
H Atkins ◽  
D Farge ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Haematopoietic Stem Cell Transplantation ◽  
Controlled Trial ◽  
Case Series ◽  
Haematopoietic Stem Cell ◽  
Controlled Clinical Trial ◽  
Randomized Controlled

Background: Haematopoietic stem cell transplantation (HSCT) has been tried in the last 15 years as a therapeutic option in patients with poor-prognosis autoimmune disease who do not respond to conventional treatments. Worldwide, more than 600 patients with multiple sclerosis (MS) have been treated with HSCT, most of them having been recruited in small, single-centre, phase 1–2 uncontrolled trials. Clinical and magnetic resonance imaging outcomes from case series reports or Registry-based analyses suggest that a major response is achieved in most patients; quality and duration of response are better in patients transplanted during the relapsing–remitting phase than in those in the secondary progressive stage. Objectives: An interdisciplinary group of neurologists and haematologists has been formed, following two international meetings supported by the European and American Blood and Marrow Transplantation Societies, for the purpose of discussing a controlled clinical trial, to be designed within the new scenarios of evolving MS treatments. Conclusions: Objectives of the trial, patient selection, transplant technology and outcome assessment were extensively discussed. The outcome of this process is summarized in the present paper, with the goal of establishing the background and advancing the development of a prospective, randomized, controlled multicentre trial to assess the clinical efficacy of HSCT for the treatment of highly active MS.

Autologous haematopoietic stem cell transplantation reduces abnormalities in the expression of immune genes in multiple sclerosis

2014 ◽  
Vol 128 (2) ◽  
pp. 111-120 ◽  
Author(s):  
Alessandra de Paula A. Sousa ◽  
Kelen C. R. Malmegrim ◽  
Rodrigo A. Panepucci ◽  
Doralina S. Brum ◽  
Amilton A. Barreira ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
T Cells ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Cellular Immunity ◽  
Haematopoietic Stem Cell Transplantation ◽  
Haematopoietic Stem Cell ◽  
Immune Genes ◽  
Relative Normalization

This study shows that autologous haematopoietic stem cell transplantation applied for treatment of multiple sclerosis induces a relative normalization of the expression of immune genes in T-cells from the patients, suggesting a ‘reset’ of adaptive cellular immunity.

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