Contributions
Abstract: P609
Type: Poster
Abstract Category: Therapy - disease modifying - Immunomodulation/Immunosuppression
Background: Neuromyelitis optica (NMO) presents autoimmune disorder affecting predominantly optic nerves and spinal cord with high risk of severe disability. Current long-term NMO treatment include immunosuppressives, cytostatics and rituximab. In the case of inefficacy more intensive treatment could be considered based on the data from aggressive multiple sclerosis (MS) treatment. There is an option of high-dose immunoablation with autologous stem cells support (ASCT) or even allogeneic hematopoietic stem cells transplantation (alloSCT).
Patient and method: 40-year-old woman with NMO (aquaporine-4-IgG seropositive), first symptoms in 1998 (optic neuritis), with non-responsivity to conventional treatment (azathioprine, cyclophosphamide, intravenous immunoglobulins, rituximab) and ASCT failure (September 2009) underwent alloSCT in April 2011. Patient experienced 9 acute relapses since ASCT and Expanded Disability Status Scale (EDSS) progressed from 6.0 to 6.5 at the time of alloSCT. Non-myeloablative regime (fludarabine, cyclophoshamide and antithymocyte globulin) was used.
Results: 5 years after alloSCT there has been no NMO relapse, EDSS improved and remained stable (6.0), there is partial recovery and further stable finding on brain and spinal cord magnetic resonance imaging. Disease-specific aquaporine-4-IgG antibodies have been negative since alloSCT. So far no graft versus host disease has been clinically apparent (patient has no specific treatment, only hydrocortisone substitution) and autologous hematopoiesis is up to 0,1%.
Conclusion: AlloSCT can be considered as an off-label therapeutic option for aggressive and unfavorable course of NMO in young patients. In our patient status of no evidence of disease activity“ (NEDA) has been achieved during 5-year follow-up thanks to alloSCT treatment.
Disclosure: Supported from PRVOUK P26/LF1/4.
Abstract: P609
Type: Poster
Abstract Category: Therapy - disease modifying - Immunomodulation/Immunosuppression
Background: Neuromyelitis optica (NMO) presents autoimmune disorder affecting predominantly optic nerves and spinal cord with high risk of severe disability. Current long-term NMO treatment include immunosuppressives, cytostatics and rituximab. In the case of inefficacy more intensive treatment could be considered based on the data from aggressive multiple sclerosis (MS) treatment. There is an option of high-dose immunoablation with autologous stem cells support (ASCT) or even allogeneic hematopoietic stem cells transplantation (alloSCT).
Patient and method: 40-year-old woman with NMO (aquaporine-4-IgG seropositive), first symptoms in 1998 (optic neuritis), with non-responsivity to conventional treatment (azathioprine, cyclophosphamide, intravenous immunoglobulins, rituximab) and ASCT failure (September 2009) underwent alloSCT in April 2011. Patient experienced 9 acute relapses since ASCT and Expanded Disability Status Scale (EDSS) progressed from 6.0 to 6.5 at the time of alloSCT. Non-myeloablative regime (fludarabine, cyclophoshamide and antithymocyte globulin) was used.
Results: 5 years after alloSCT there has been no NMO relapse, EDSS improved and remained stable (6.0), there is partial recovery and further stable finding on brain and spinal cord magnetic resonance imaging. Disease-specific aquaporine-4-IgG antibodies have been negative since alloSCT. So far no graft versus host disease has been clinically apparent (patient has no specific treatment, only hydrocortisone substitution) and autologous hematopoiesis is up to 0,1%.
Conclusion: AlloSCT can be considered as an off-label therapeutic option for aggressive and unfavorable course of NMO in young patients. In our patient status of no evidence of disease activity“ (NEDA) has been achieved during 5-year follow-up thanks to alloSCT treatment.
Disclosure: Supported from PRVOUK P26/LF1/4.