{
  "abstract": "Background Autologous haematopoietic stem cell transplantation (aHSCT) represents a treatment option for highly aggressive multiple sclerosis (MS). Here, we report outcome analyses from the two largest German centres performing aHSCT in MS.Methods In this retrospective analysis, people with (pw) MS who underwent aHSCT between 2007 and 2025 were included. Outcomes comprise no evidence of disease activity (NEDA-3), 3-month confirmed disability changes on the Expanded Disability Status Scale and transplantation-related mortality (TRM). Predictors of treatment response were evaluated according to the European Committee for Treatment and Research in Multiple Sclerosis and the European Society of Blood and Marrow Transplantation consensus criteria.Results A total of 109 pwMS were included: 55 (50.5%) with relapsing-remitting MS (RRMS), 23 (21.1%) with secondary progressive MS (SPMS) and 31 (28.4%) with primary progressive MS (PPMS). Median follow-up after aHSCT was 20.1 months. Overall, 82.8% (SE 4.9%) of pwMS maintained NEDA-3. PwRRMS had significantly higher NEDA-3 rates (Kaplan-Meier (KM) estimate 91%, SE 5%) than those with SPMS (KM 80.1%, SE 10.5%, p=0.018) or PPMS (KM 70.6%, SE 11.4%, p=0.035). Patients meeting core consensus criteria achieved NEDA-3 more often (KM 94.4%, SE 5.4%) than those meeting the extended criteria (KM 84.4%, SE 8.5%, p=0.199) or those outside the criteria (KM 73.7%, SE 8.8%, p=0.004). In progressive MS, a disease duration of <5 years indicated a potential prediction of NEDA-3 stability. TRM was 0.9% (n=1/109).Conclusions In this heterogeneous cohort, comprising a substantial proportion of people with progressive MS, aHSCT provided sustained NEDA-3 stability and a marked reduction in inflammatory disease activity, particularly in RRMS, with a potential benefit in progressive MS.",
  "authors": [
    {
      "affiliations": [
        "Institute of Neuroimmunology and Multiple Sclerosis, University Medical Center Hamburg-Eppendorf Center for Molecular Neurobiology Hamburg, Hamburg, Germany",
        "Department of Neurology, University Medical Center Hamburg-Eppendorf, Hamburg, Hamburg, Germany"
      ],
      "name": "Felix Fischbach"
    },
    {
      "affiliations": [
        "Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany"
      ],
      "name": "Johanna Richter"
    },
    {
      "affiliations": [
        "Department of Neurology, University of Heidelberg, Heidelberg, Germany"
      ],
      "name": "Miriam Korporal-Kuhnke"
    },
    {
      "affiliations": [
        "Institute of Neuroimmunology and Multiple Sclerosis, University Medical Center Hamburg-Eppendorf Center for Molecular Neurobiology Hamburg, Hamburg, Germany",
        "Department of Neurology, University Medical Center Hamburg-Eppendorf, Hamburg, Hamburg, Germany"
      ],
      "name": "Lena K Pfeffer"
    },
    {
      "affiliations": [
        "Department of Medical Statistics, University Medical Center Göttingen, Göttingen, Germany"
      ],
      "name": "Paula Starke"
    },
    {
      "affiliations": [
        "Institute of Neuroimmunology and Multiple Sclerosis, University Medical Center Hamburg-Eppendorf Center for Molecular Neurobiology Hamburg, Hamburg, Germany",
        "Department of Neurology, University Medical Center Hamburg-Eppendorf, Hamburg, Hamburg, Germany"
      ],
      "name": "Vivien Häußler"
    },
    {
      "affiliations": [
        "Institute of Neuroimmunology and Multiple Sclerosis, University Medical Center Hamburg-Eppendorf Center for Molecular Neurobiology Hamburg, Hamburg, Germany",
        "Department of Neurology, University Medical Center Hamburg-Eppendorf, Hamburg, Hamburg, Germany"
      ],
      "name": "Bente Braun"
    },
    {
      "affiliations": [
        "Institute of Neuroimmunology and Multiple Sclerosis, University Medical Center Hamburg-Eppendorf Center for Molecular Neurobiology Hamburg, Hamburg, Germany",
        "Department of Neurology, University Medical Center Hamburg-Eppendorf, Hamburg, Hamburg, Germany"
      ],
      "name": "Amanda Keller"
    },
    {
      "affiliations": [
        "Institute of Neuroimmunology and Multiple Sclerosis, University Medical Center Hamburg-Eppendorf Center for Molecular Neurobiology Hamburg, Hamburg, Germany",
        "Department of Neurology, University Medical Center Hamburg-Eppendorf, Hamburg, Hamburg, Germany"
      ],
      "name": "Yekta Abbasi"
    },
    {
      "affiliations": [
        "Institute of Neuroimmunology and Multiple Sclerosis, University Medical Center Hamburg-Eppendorf Center for Molecular Neurobiology Hamburg, Hamburg, Germany",
        "Department of Neurology, University Medical Center Hamburg-Eppendorf, Hamburg, Hamburg, Germany"
      ],
      "name": "Margarethe Daumen"
    },
    {
      "affiliations": [
        "Institute of Neuroimmunology and Multiple Sclerosis, University Medical Center Hamburg-Eppendorf Center for Molecular Neurobiology Hamburg, Hamburg, Germany",
        "Department of Neurology, University Medical Center Hamburg-Eppendorf, Hamburg, Hamburg, Germany",
        "Department of Medicine, University Medical Center Hamburg-Eppendorf, Hamburg, HH, Germany"
      ],
      "name": "Katja Lange"
    },
    {
      "affiliations": [
        "Institute of Neuroimmunology and Multiple Sclerosis, University Medical Center Hamburg-Eppendorf Center for Molecular Neurobiology Hamburg, Hamburg, Germany",
        "Department of Neurology, University Medical Center Hamburg-Eppendorf, Hamburg, Hamburg, Germany"
      ],
      "name": "Stefanie Reinhardt"
    },
    {
      "affiliations": [
        "Department of Neurology, University of Heidelberg, Heidelberg, Germany"
      ],
      "name": "Jessica Stockmann"
    },
    {
      "affiliations": [
        "Department of Hematology, Oncology and Rheumatology, Heidelberg University Hospital, Heidelberg, Germany"
      ],
      "name": "Juliane Brandt"
    },
    {
      "affiliations": [
        "Department of Hematology, Oncology and Rheumatology, Heidelberg University Hospital, Heidelberg, Germany"
      ],
      "name": "Sandra Sauer"
    },
    {
      "affiliations": [
        "Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany"
      ],
      "name": "Ina Rudolph"
    },
    {
      "affiliations": [
        "Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany"
      ],
      "name": "Boris Fehse"
    },
    {
      "affiliations": [
        "Institute of Neuroimmunology and Multiple Sclerosis, University Medical Center Hamburg-Eppendorf Center for Molecular Neurobiology Hamburg, Hamburg, Germany",
        "Department of Neurology, University Medical Center Hamburg-Eppendorf, Hamburg, Hamburg, Germany"
      ],
      "name": "Sina Cathérine C Rosenkranz"
    },
    {
      "affiliations": [
        "Department of Medical Statistics, University Medical Center Göttingen, Göttingen, Germany"
      ],
      "name": "Tim Friede"
    },
    {
      "affiliations": [
        "Institute of Neuroimmunology and Multiple Sclerosis, University Medical Center Hamburg-Eppendorf Center for Molecular Neurobiology Hamburg, Hamburg, Germany",
        "Department of Neurology, University Medical Center Hamburg-Eppendorf, Hamburg, Hamburg, Germany"
      ],
      "name": "Manuel Alexander Friese"
    },
    {
      "affiliations": [
        "Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany"
      ],
      "name": "Nicolaus Kröger"
    },
    {
      "affiliations": [
        "Department of Hematology, Oncology and Rheumatology, Heidelberg University Hospital, Heidelberg, Germany"
      ],
      "name": "Norbert Blank"
    },
    {
      "affiliations": [
        "Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany"
      ],
      "name": "Francis Ayuk"
    },
    {
      "affiliations": [
        "Department of Neurology, University of Heidelberg, Heidelberg, Germany"
      ],
      "name": "Brigitte Wildemann"
    },
    {
      "affiliations": [
        "Institute of Neuroimmunology and Multiple Sclerosis, University Medical Center Hamburg-Eppendorf Center for Molecular Neurobiology Hamburg, Hamburg, Germany",
        "Department of Neurology, University Medical Center Hamburg-Eppendorf, Hamburg, Hamburg, Germany"
      ],
      "name": "Christoph Heesen"
    }
  ],
  "title": "Autologous haematopoietic stem cell transplantation in multiple sclerosis: outcomes and predictors from German real-world data",
  "uid": "bf1ace76-7f4e-5ca2-bbbb-d19be384d274"
}