A case report published in Med describes two patients with severe neuromyelitis optica spectrum disorder (NMOSD) who remained free of disease relapse for more than 15 years after receiving donor (allogeneic) haematopoietic stem cell transplants.
The patients had treatment-resistant NMOSD and had previously not responded to standard therapies. Following transplantation, both remained free of relapses without ongoing immunosuppressive treatment, while the disease-associated antibodies became undetectable throughout long-term follow-up.
The authors suggest the findings provide proof of concept that allogeneic stem cell transplantation may produce sustained remission in selected patients with severe NMOSD by replacing the immune system. They emphasise that the report describes only two patients and that further studies are needed to assess the safety, risks and potential benefits of this approach in larger groups of patients.
The study is discussed in a Nature news article.