A study published in Med and reported around 15 June 2026 found two patients with neuromyelitis optica spectrum disorder (NMOSD) remained relapse-free for 15 years after allogeneic haematopoietic stem cell transplant, off ongoing medication.
Key clinical outcomes
- Long-term remission: Two NMOSD patients relapse-free for 15 years following allogeneic HSCT; no maintenance immunotherapy required.
- Functional recovery: One patient regained normal neurological function and fathered children; the other regained use of a previously paralysed arm.
- Timing and source: Transplants performed in 2009 and 2010; donors were a sibling in one case and an unrelated donor in the other.
Allogeneic HSCT — Procedure & mechanism
- Definition: Donor-derived haematopoietic stem cell transplantation replacing the recipient immune system.
- Conditioning regimen: Chemotherapy-based immune ablation precedes donor cell infusion to eradicate autoreactive lymphocytes.
- Immune reconstitution: Donor immune system eliminates autoreactive B-cell clones and establishes regulatory T-cell–mediated tolerance.
Risks & limitations
- Serious complications: Graft‑versus‑host disease, severe infections, antibody deficiency, lymphadenopathy and secondary malignancy (one patient developed bladder cancer).
- Evidence gap: Report comprises two patients; larger controlled trials needed to assess safety, efficacy and candidate selection.
IASPOINT Booster Facts
- NMOSD marker & prevalence: Often associated with aquaporin‑4 (AQP4) IgG; prevalence ≈3 per 100,000.
- HSCT types: Autologous HSCT uses patient cells; allogeneic HSCT uses donor cells and can confer graft‑versus‑autoimmunity effects.
- Clinical setting: Allogeneic HSCT requires specialised transplant centres and strict trial/regulatory oversight due to high risk.