To develop guidelines for the use of haematopoietic stem cell transplantation (HSCT) immune-mediated neurological diseases and provide recommendations for patient selection, transplant technique, follow-up and future development.

Autologous HSCT has been increasingly used in the treatment of multiple sclerosis (MS). Increasing evidence is accumulating to support its use in highly active relapsing remitting MS. AHSCT has also been used in the progressive forms of MS and other immune-mediated neurological diseases, including chronic inflammatory demyelinating polyneuropathy, neuromyelitis optica, myasthenia gravis and stiff person syndrome.  Authoritative guidelines are needed to advise clinicians and health authorities on clinical practice and health services resourcing. 

A systematic review of published clinical evidence and registry data was undertaken by clinicians and relevant professional groups, including nursing, statistical and data management representation, with experience in HSCT in immune-mediated neurological disease.

The evidence for indications was systematically classified in four categories (standard of care (S), clinical option (CO), developmental (D) and generally not recommended (GNR)). Strength of the evidence was graded as levels I, II and III based on consideration of health benefits, side effects and risks and balanced against the non-HSCT options.

HSCT requires close inter-specialty collaboration before, during and after treatment in accredited HSCT centers (II). 

Intermediate-intensity conditioning regimens are recommended and high-intensity conditioning regimens should be restricted to highly selected cases (II).

AHSCT should be offered in highly active relapsing remitting MS failing to respond to disease modifying therapies (S/I) or in aggressive MS before failing such therapies (CO/II). In progressive MS and other immune mediated neurological disorders, data are heterogeneous and AHSCT should be delivered on clinical trials if available (CO/II).

The evidence for allogeneic HSCT and Mesenchymal Stromal Cells is developmental (D/III).  

There is a need for clinical trials across all settings (III) and neurology specific guidelines supported by neurological societies (Â鶹´«Ã½Ó³»­, ENA).