Autologous haematopoietic stem cell transplantation for refractory stiff-person syndrome: the UK experience.

ABSTRACT: Stiff Person Syndrome (SPS) is a rare immune-mediated disabling neurological disorder characterised by muscle spasms and high GAD antibodies. There are only a few case reports of autologous haematopoietic stem cell transplantation (auto-HSCT) as a treatment for SPS.

Objective

To describe the UK experience of treating refractory SPS with auto-HSCT.

Methods

Between 2015 and 2019, 10 patients with SPS were referred to our institution for consideration of auto-HSCT. Eight patients were deemed suitable for autograft and four were treated. Of the treated patients, three had classical SPS and one had the progressive encephalomyelitis with rigidity and myoclonus variant. All patients were significantly disabled and had failed conventional immunosuppressive therapy. Patients were mobi

SUBMITTER: Kass-Iliyya L 

PROVIDER: S-EPMC7815605 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature