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STUDY: Sustained disease remission in multiple sclerosis after HSCT, Italy

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  • STUDY: Sustained disease remission in multiple sclerosis after HSCT, Italy

    The SPMS results are more impressive than in other studies. Encouraging! -D

    Sustained disease remission in multiple sclerosis after autologous haematopoietic stem cell transplantation. The Italian experience

    ECTRIMS Online Library. Boffa G. Oct 26, 2017; 200307

    Abstract: P652
    Type: Poster
    Abstract Category: Therapy - disease modifying - 26 Immunomodulation/Immunosuppression

    Background: Despite the advent of new highly-active therapies for multiple sclerosis (MS), long-term disease remission remains elusive and only a small percentage of patients achieves the so-called no evidence of disease activity (NEDA) status. This is particularly relevant for patients with aggressive MS with suboptimal response to conventional treatment. Against this scenario, autologous haematopoietic stem cell transplantation (AHSCT) has recently demonstrated the potential to maintain long-term disease remission in aggressive MS patients.

    Objective: To evaluate the long-term outcomes of a large multicenter cohort of aggressive MS patients treated with AHSCT.

    Methods: Data were obtained in a multicenter, observational, retrospective cohort study including patients treated with AHSCT with the same conditioning regimens in Italy from 1996 to 2016. EDSS progression was defined as 1 EDSS point increase (0.5 if baseline EDSS>=5.5) confirmed at 6 months. Demographic, disease-related and treatment-related data and reports of adverse events were collected.

    Results: 122 consecutive MS patients were included, with a median follow-up of 4,7 years (range, 0,5-17 years). 59% of patients had relapsing-remitting (RR) MS. The median EDSS score was 5 (range 1-8.5) 1 year before AHSCT and 6 (range 1-9) at AHSCT. One death (0,8%) was reported within 100 days of transplant. Stem cell were mobilized with cyclophosphamide and G-CSF; 102 patients (84%) were conditioned with carmustine-cytarabine-etoposide-melphalan (BEAM) plus anti-thymocyte globulin (ATG) whilst 20 patients (16%) with cyclophosphamide plus ATG . Only patients who underwent the BEAM protocol were included in the long-term analysis. The 5-year probability of progression-free survival were 91% for RRMS and 62% for SPMS respectively (p< 0.001). NEDA status (defined as no relapses, no EDSS progression and no MRI activity) at 5 year was maintained by 72% of RRMS patients and by 55% of SPMS patients (p=0.07).

    Conclusion: Our data demonstrate that AHSCT is reasonably safe and extremely effective for inducing long-term disease remission in aggressive MS patients.

    Dave Bexfield

  • #2
    So... What's next on this front?

    Do people like me want to go for this type of treatment? Could I even get something like this? Do I want to? Would I still want to stay on an oral med "just in case"?

    This raises a lot of questions for me, in all good and intriguing ways.

    For those that don't know I have about 12 lesions on my brain, haven't had a relapse since diagnosis a little over 2 years ago, and have no disease progression in 1.5+ years on Aubagio. My lesions did double in 7 months on Copaxone.
    girl1dir =)