Autologous haematopoietic stem cell transplantation following high-dose immunosuppressive therapy in neuromyelitis optica

Hans Hägglund*, James Bowen, George Kraft, Annette Wundes, Lou Brundin, Fredrik Piehl, Rainer Storb, George Georges, Kenneth Maravilla, Richard Nash

Author address: 

Stockholm, SE; Seattle, US; Denver, US


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: 

Annual Meeting of European Society for Blood and Marrow Transplantation
    • EBMT 39th (2013)