{
  "abstract": "Background Autologous haematopoietic stem cell transplantation (AHSCT) is increasingly used as a one-off disease-modifying therapy for aggressive forms of multiple sclerosis (MS). We report real-world effectiveness of AHSCT for MS in the UK.Methods This retrospective open-label study included patients with (pw)MS treated with AHSCT between 2002 and 2023 in 14 UK centres. Outcomes included relapse-free survival (RFS), MRI activity-free survival (MFS), progression-free survival (PFS) and no evidence of disease activity (NEDA-3). We assessed 6-month confirmed Expanded Disability Status Scale (EDSS) score progression or improvement compared with pre-treatment. Treatment-related mortality (TRM) was defined as death from any cause within 100 days post-autologous graft reinfusion.Results 364 pwMS were included (median age 40 years; 58% female). Of these, 271 pwMS had adequate neurological follow-up data: 168 (62%) had relapsing-remitting MS (pwRRMS) and 103 (38%) had progressive MS (pwPMS). Median disease duration from symptom onset was 10 years (IQR 6–14), EDSS 6 (IQR 4.0–6.5) and follow-up from AHSCT 46 months. At 2 and 5 years from AHSCT, RFS was 94.6% and 88.6%; MFS 93.1% and 80.1%; PFS 83.5% and 62.4%; NEDA-3 72.3% and 46.2%. pwRRMS had significantly higher rates of PFS (p=0.007) and NEDA-3 (p=0.001) than pwPMS. RRMS was a predictor of EDSS improvement, whose prevalence was 24.2% at 2 years and 20.4% at 5 years. TRM was 1.4% (n=5/364).Conclusions In this cohort with high EDSS at baseline and including pwPMS, AHSCT led to durable remission of inflammatory activity and stabilisation or improvement of neurological disability, particularly in pwRRMS.",
  "authors": [
    {
      "affiliations": [
        "Department of Brain Sciences, Faculty of Medicine, Imperial College, London, UK",
        "Neurology, Imperial College Healthcare NHS Trust, London, UK"
      ],
      "name": "Paolo Antonio Muraro"
    },
    {
      "affiliations": [
        "Guy’s and St Thomas’ NHS Foundation Trust, London, UK",
        "King’s College Hospital NHS Foundation Trust, London, UK"
      ],
      "name": "Majid Kazmi"
    },
    {
      "affiliations": [
        "Department of Brain Sciences, Faculty of Medicine, Imperial College, London, UK",
        "Neurology, Imperial College Healthcare NHS Trust, London, UK"
      ],
      "name": "Eleonora De Matteis"
    },
    {
      "affiliations": [
        "Institute for Translational Neuroscience, The University of Sheffield, Sheffield, UK",
        "Sheffield Teaching Hospitals NHS Foundation Trust and NIHR Sheffield Biomedical Research Centre, Sheffield, UK"
      ],
      "name": "Gavin Brittain"
    },
    {
      "affiliations": [
        "Department of Brain Sciences, Faculty of Medicine, Imperial College, London, UK",
        "Department of Neurosciences, Psychology, Drug Research and Child Health, University of Florence, Florence, Italy"
      ],
      "name": "Alice Mariottini"
    },
    {
      "affiliations": [
        "Department of Brain Sciences, Faculty of Medicine, Imperial College, London, UK",
        "Neurology, Imperial College Healthcare NHS Trust, London, UK"
      ],
      "name": "Richard Nicholas"
    },
    {
      "affiliations": [
        "King’s College Hospital NHS Foundation Trust, London, UK"
      ],
      "name": "Eli Silber"
    },
    {
      "affiliations": [
        "King’s College Hospital NHS Foundation Trust, London, UK"
      ],
      "name": "Varun Mehra"
    },
    {
      "affiliations": [
        "Haematology, Imperial College Healthcare NHS Trust, London, UK"
      ],
      "name": "Ian Gabriel"
    },
    {
      "affiliations": [
        "NIHR (National Institute for Health and Research) University College London Hospitals Biomedical Research Centre, London, UK",
        "Queen Square MS Centre, UCL Queen Square Institute of Neurology, University College London, London, UK"
      ],
      "name": "Olga Ciccarelli"
    },
    {
      "affiliations": [
        "British Society of Blood and Marrow Transplantation and Cellular Therapy, London, UK"
      ],
      "name": "Julia Lee"
    },
    {
      "affiliations": [
        "British Society of Blood and Marrow Transplantation and Cellular Therapy, London, UK"
      ],
      "name": "Rachel Pearce"
    },
    {
      "affiliations": [
        "University of Genoa, Genoa, Italy",
        "IRCCS Ospedale Policlinico San Martino, Genoa, Italy"
      ],
      "name": "Maria Pia Sormani"
    },
    {
      "affiliations": [
        "University of Genoa, Genoa, Italy",
        "IRCCS Ospedale Policlinico San Martino, Genoa, Italy"
      ],
      "name": "Alessio Signori"
    },
    {
      "affiliations": [
        "British Society of Blood and Marrow Transplantation and Cellular Therapy, London, UK"
      ],
      "name": "Ruth Paul"
    },
    {
      "affiliations": [
        "Cambridge University Hospitals NHS Trust, Cambridge, UK"
      ],
      "name": "Ram Malladi"
    },
    {
      "affiliations": [
        "King’s College Hospital NHS Foundation Trust, London, UK"
      ],
      "name": "Victoria Potter"
    },
    {
      "affiliations": [
        "Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK"
      ],
      "name": "John Snowden"
    },
    {
      "affiliations": [
        "Institute for Translational Neuroscience, The University of Sheffield, Sheffield, UK",
        "Sheffield Teaching Hospitals NHS Foundation Trust and NIHR Sheffield Biomedical Research Centre, Sheffield, UK"
      ],
      "name": "Basil Sharrack"
    }
  ],
  "title": "Real-world effectiveness of autologous haematopoietic stem cell transplantation for multiple sclerosis in the UK",
  "uid": "79994b17-e505-5118-943f-c03493cf7f5c"
}