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<p>Autologous haematopoietic stem cell transplantation (AHSCT) is a treatment option for relapsing forms of multiple sclerosis (MS) that are refractory to disease-modifying therapy (DMT). AHSCT after failure of high-efficacy DMT in aggressive forms of relapsing–remitting MS is a generally accepted indication, yet the optimal placement of this approach in the treatment sequence is not universally agreed upon. Uncertainties also remain with respect to other indications, such as in rapidly evolving, severe, treatment-naive MS, progressive MS, and neuromyelitis optica spectrum disorder (NMOSD). Furthermore, treatment and monitoring protocols, rehabilitation and other supportive care before and after AHSCT need to be optimized. To address these issues, we convened a European Committee for Treatment and Research in Multiple Sclerosis Focused Workshop in partnership with the European Society for Blood and Marrow Transplantation Autoimmune Diseases Working Party, in which evidence and key questions were presented and discussed by experts in these diseases and in AHSCT. Based on the workshop output and subsequent written interactions, this Consensus Statement provides practical guidance and recommendations on the use of AHSCT in MS and NMOSD. Recommendations are based on the available evidence, or on consensus when evidence was insufficient. We summarize the key evidence, report the final recommendations, and identify areas for further research.</p>

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Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis and neuromyelitis optica spectrum disorder — recommendations from ECTRIMS and the EBMT

  • 期刊论文

  • 发表日期：

    2025年1月15日

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  • Paolo A. Muraro,
  • Alice Mariottini,
  • Raffaella Greco,
  • Joachim Burman,
  • Ellen Iacobaeus,
  • Matilde Inglese,
  • John A. Snowden,
  • Tobias Alexander,
  • Maria Pia Amato,
  • Lars Bø,
  • Giacomo Boffa,
  • Olga Ciccarelli,
  • Jeffrey A. Cohen,
  • Tobias Derfuss,
  • Dominique Farge,
  • Mark S. Freedman,
  • Maria Gaughan,
  • Christoph Heesen,
  • Majid Kazmi,
  • Kirill Kirzigov,
  • Per Ljungman,
  • Gianluigi Mancardi,
  • Roland Martin,
  • Varun Mehra,
  • Lucia Moiola,
  • Riccardo Saccardi,
  • Mar Tintoré,
  • Bruno Stankoff,
  • Basil Sharrack,
  • Yolanda Blanco,
  • Franziska Di Pauli,
  • Gavin Giovannoni,
  • Cheryl Hemingway,
  • Azza Izmail,
  • Iljias Jelcic,
  • Ludwig Kappos,
  • Luisa Klotz,
  • Anne Kristine Lehmann,
  • Marcin Mycko,
  • Albert Saiz,
  • Joost Smolders,
  • Sandra Vukusic,
  • Barbara Willekens,
  • Helene Zephir

摘要

Autologous haematopoietic stem cell transplantation (AHSCT) is a treatment option for relapsing forms of multiple sclerosis (MS) that are refractory to disease-modifying therapy (DMT). AHSCT after failure of high-efficacy DMT in aggressive forms of relapsing–remitting MS is a generally accepted indication, yet the optimal placement of this approach in the treatment sequence is not universally agreed upon. Uncertainties also remain with respect to other indications, such as in rapidly evolving, severe, treatment-naive MS, progressive MS, and neuromyelitis optica spectrum disorder (NMOSD). Furthermore, treatment and monitoring protocols, rehabilitation and other supportive care before and after AHSCT need to be optimized. To address these issues, we convened a European Committee for Treatment and Research in Multiple Sclerosis Focused Workshop in partnership with the European Society for Blood and Marrow Transplantation Autoimmune Diseases Working Party, in which evidence and key questions were presented and discussed by experts in these diseases and in AHSCT. Based on the workshop output and subsequent written interactions, this Consensus Statement provides practical guidance and recommendations on the use of AHSCT in MS and NMOSD. Recommendations are based on the available evidence, or on consensus when evidence was insufficient. We summarize the key evidence, report the final recommendations, and identify areas for further research.