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Old 10-08-2019, 05:36 PM
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Dave @ ActiveMSers
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Default HSCT: updated guidelines and recommendations

If this is a treatment you are considering, please read the full paper. One of the key takeaways for more advanced MS: "Registry studies and other cohort analyses have repeatedly shown that aHSCT is more efficacious in patients with RRMS than SPMS or PPMS. Even so, several reports support the association of Gd-enhancement with favourable outcomes." And while PPMS has historically been a red flag for this treatment, "Given the poor prognosis, the support from registry data and the limited treatment options, very occasional patients with high levels of persistent inflammatory activity with rapidly accumulating disability may be considered." If there is an active inflammatory component, it could be effective even in progressive cases of MS. -D

Bone Marrow Transplant.
2019 Sep 26. doi: 10.1038/s41409-019-0684-0. [Epub ahead of print]

Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and recommendations from the EBMT Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of EBMT and ISCT (JACIE).

Sharrack B, Saccardi R, et al

These updated EBMT guidelines review the clinical evidence, registry activity and mechanisms of action of haematopoietic stem cell transplantation (HSCT) in multiple sclerosis (MS) and other immune-mediated neurological diseases and provide recommendations for patient selection, transplant technique, follow-up and future development.

The major focus is on autologous HSCT (aHSCT), used in MS for over two decades and currently the fastest growing indication for this treatment in Europe, with increasing evidence to support its use in highly active relapsing remitting MS failing to respond to disease modifying therapies.

aHSCT may have a potential role in the treatment of the progressive forms of MS with a significant inflammatory component and other immune-mediated neurological diseases, including chronic inflammatory demyelinating polyneuropathy, neuromyelitis optica, myasthenia gravis and stiff person syndrome. Allogeneic HSCT should only be considered where potential risks are justified.

Compared with other immunomodulatory treatments, HSCT is associated with greater short-term risks and requires close interspeciality collaboration between transplant physicians and neurologists with a special interest in these neurological conditions before, during and after treatment in accredited HSCT centres. Other experimental cell therapies are developmental for these diseases and patients should only be treated on clinical trials.

Dave Bexfield
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