Objective: To investigate the safety and efficacy of AHSCT in NMO.
Patients and methods: Patients <= 70 years with aggressive NMO unresponsive to two or more immunosuppressive agents were included. Stem cells were mobilized with G-CSF 16 µ g/kg/day for 4 days. Rituximab (375 mg/m2) was administered the day before starting G-CSF to deplete B cells. Leukapheresis continued until > 3.0 x 106 CD34+ cells/kg weight were collected. To prevent exacerbations, prednisone 1 mg/kg/day was given for 10 days, beginning 1 day before the start of G-CSF. The conditioning regimen of BEAM/Thymoglobulin 5 mg/kg was followed by AHSCT. Subjects received G-CSF (5 µ g/kg/day) from Day +5 until neutrophil engraftment.
Results: Five patients underwent AHSCT.Four remain free of disease progression 24-42 months afterwards. One case remained stable for 27 months before having a severe fatal relapse. Complications of urosepsis, nitrofurantoin-related diffuse alveolar hemorrhage, aspergillus infection and hypoimmunoglobulinemia were easily managed. Engraftment occurred in 10-13 days and platelet recovery by 13-17 days.
Conclusions: AHSCT may halt disease progression in some, but not all, patients with severe therapy-resistant NMO. Further studies may lead to an improved understanding of the efficacy of this treatment and NMO disease mechanisms.
Full conference title:
- EBMT 39th (2013)