Autologous haematopoietic stem cell transplantation for refractory stiff-person syndrome: the UK experience.
Lewis Kass-IliyyaJohn A SnowdenAlice ThorpeHelen JessopAndrew D ChantryPtolemaios G SarrigiannisMarios HadjivassiliouBasil SharrackPublished in: Journal of neurology (2020)
Auto-HSCT is an intensive but well tolerated and effective treatment option for patients with SPS refractory to conventional immunotherapy. Further work is warranted to optimise patient selection and establish the efficacy, long-term safety, and cost-effectiveness of this treatment.