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HSCT for severe resistant autoimmune and inflammatory diseases – a guide

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  • HSCT for severe resistant autoimmune and inflammatory diseases – a guide

    The full article is available. "There is now increasing evidence that its early use in patients with highly active RRMS results in significant reduction in clinical and MRI disease activity as well as improving patients’ disability to a significantly greater degree compared with currently available DMTs." -D

    Autologous haematopoietic stem cell transplantation (aHSCT) for severe resistant autoimmune and inflammatory diseases – a guide for the generalist

    John A Snowden, consultant haematologist, director of BMT and chair of the Autoimmune Diseases Working Party (ADWP) of the European Society for Blood and Marrow Transplantation (EBMT)A⇑, Basil Sharrack, consultant neurologistB, Mohammed Akil, consultant rheumatologistC, David G Kiely, consultant respiratory physicianD, Alan Lobo, consultant gastroenterologistE, Majid Kazmi, consultant haematologistF, Paolo A Muraro, professor of neurology, neuroimmunology and immunotherapyG and James O Lindsay, professor of inflammatory bowel diseaseH

    ADepartment of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Royal Hallamshire Hospital, Sheffield, UK
    BDepartment of Neurology, Sheffield Teaching Hospitals NHS Foundation Trust, Royal Hallamshire Hospital, Sheffield, UK
    CDepartment of Rheumatology, Sheffield Teaching Hospitals NHS Foundation Trust, Royal Hallamshire Hospital, Sheffield, UK
    DSheffield Pulmonary Vascular Disease Unit, Sheffield Teaching Hospitals NHS Foundation Trust, Royal Hallamshire Hospital, Sheffield, UK
    EDepartment of Gastroenterology, Sheffield Teaching Hospitals NHS Foundation Trust, Royal Hallamshire Hospital, Sheffield, UK
    FKings Healthcare Partners, London, UK
    GBrain Sciences, Imperial College London, London, UK
    HCentre for Immunobiology, Barts and the London School of Medicine and Dentistry, Blizard Institute, Queen Mary University of London, London, UK
    Address for correspondence: Professor John Snowden, Sheffield Teaching Hospitals NHS Foundation Trust, Haematology, Royal Hallamshire Hospital, Sheffield S10 2JF, UK.

    ABSTRACT
    Autologous haematopoietic stem cell transplantation (aHSCT) is commonly used for the treatment of haematological cancers, but is increasingly used in the treatment of patients severely affected by autoimmune diseases (ADs). In fact, ADs have become the fastest growing indication for aHSCT.

    A wide range of diseases have been treated, but the field has focused on three areas: multiple sclerosis, diffuse cutaneous systemic sclerosis and Crohn's disease, where there are populations of patients for whom disease control remains unsatisfactory despite the advent of biological and targeted small molecule therapies. Scientific studies of immune reconstitution have provided support for a ‘rebooting’ of the immune system through a re-diversification of naive and regulatory immune effector cells. In addition, there may be health economic benefits from a single one-off procedure. Even so, the treatment with aHSCT is intensive with a range of toxicities and risks which, despite being routine to transplant haematologists, are less familiar to disease specialists.

    Close collaboration between transplant haematologists and relevant disease specialists in patient selection, clinical management and follow-up is mandatory. Ideally, patients should be treated on a clinical trial if available.

    FULL TEXT (FREE PDF)
    http://www.clinmed.rcpjournal.org/co....full.pdf+html
    Dave Bexfield
    ActiveMSers
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