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Autologous Hematopoietic Stem Cell Transplantation for MS, the Ottawa Protocol

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  • Autologous Hematopoietic Stem Cell Transplantation for MS, the Ottawa Protocol

    Curr Protoc
    . 2022 May;2(5):e437.
    doi: 10.1002/cpz1.437. Autologous Hematopoietic Stem Cell Transplantation for Multiple Sclerosis, the Ottawa Protocol

    Mathieu Puyade 1 2, Francis Brunet 3, Rush Carolina 3 4, Nathan Fergusson 5, Ilia Makedonov 4, Mark S Freedman 3 4 5, Harold Atkins 1 4 5
    Affiliations expandAbstract

    Autologous hematopoietic stem cell transplantation (aHSCT) is increasingly used to treat patients with highly active multiple sclerosis (MS) refractory to disease-modifying therapy. Briefly, cyclophosphamide and filgrastim are used to mobilize autologous hematopoietic stem cells (HSC) into the circulation. HSC are harvested by leukapheresis, purified using a CD34 immunomagnetic selection process, and cryopreserved. Busulphan, cyclophosphamide, and rabbit anti-thymocyte globulin are used to destroy the patient's autoreactive immune system, followed by infusion of the previously collected HSC, which reconstitute a nave and self-tolerant immune system. Many MS patients experience durable remissions with no evidence of new disease activity following aHSCT. Treatment-related toxicity is rare, but potentially life-threatening complications necessitate appropriate patient selection by MS neurologists and HSCT physicians. AHSCT must be performed with a highly trained multidisciplinary team expert to minimize morbidity and mortality. We present the current aHSCT procedure for an MS indication at The Ottawa Hospital, developed from our program's 20-year experience. 2022 Wiley Periodicals LLC. Basic Protocol 1: Candidate selection Basic Protocol 2: Autologous hematopoietic stem cell mobilization, collection, purification, and cryopreservation Basic Protocol 3: Autologous hematopoietic stem cell transplantation Basic Protocol 4: Supportive care following recovery from aHSCT (Beyond 100 days) Basic Protocol 5: Ongoing evaluation of multiple sclerosis.

    Keywords: autologous hematopoietic stem cell transplantation; immune reconstitution; immunotolerance; multiple sclerosis; patient selection.
    Dave Bexfield