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Hematopoietic stem cell transplantation for children with multiple sclerosis and neuromyelitis optica: Long-term outcomes and late effects
K. Kirgizov, R. Bembeeva, E. Volkova, E. Pristanskova, N. Sidorova, V. Konstantinova, O. Blagonravova, S. Piliya, E. Skorobogatova, N. Zavadenko, A. Rumyantsev
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https://doi.org/10.1016/j.ejpn.2017.04.674
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Objective: To estimate long-term outcomes and late effects at children underwent Autologous HSCT for multiple sclerosis (MS) and Allogenic HSCT for neuromyelitis optica (NMO).
Methods: Fifteen pts. included (MS – 12, NMO – 3). MS: female – 9, male – n*=*3; NMO: all females. MS median age – 16,8*±*1,6 y.o., median length of MS prior HSCT – 17,5*±*3,4 months, debut age: 13,4*±*1,5 y.o. (4–15 y.o.). Median EDSS 6,16*±*0,2. NMO pts. median age – 12,7*±*1,4 y.o., median length of NMO prior HSCT – 19,2*±*4,1 months, debut age: 10,2*±*0,4 y.o. (9–11 y.o.). All patients had severe*refractory disease treated with different immunosuppression, but still have inflammation signs. MS patients received Cyclophosphamide and ATGAM based chemotherapy and NMO patients received Treosulfan, Fludarabine, Rituximab and ATGAM. Late effects estimation – due to developed protocol for late effects estimation (somatic and neurocognitive status).
Results: EDSS improved at MS patients fast – on 3,1*±*0,3 during first 60 days. Median follow-up 40,7*±*2,4 months (10–76 months). Two MS patients relapsed (clinical and MRI). No severe complications registered. NMO patients stopped the progression and improved neurologically (clinical and MRI). One pt. with NMO died due to refractory ADV-infection, two patients did not have any severe toxic episodes. Median follow-up 23*±*4,7 months (1–60 months). MS patients late effects: cardio-vascular – 5 pts., endocrine*– 3 pts (all females). NMO patients late effects: cardio-vascular – 2 pts., late immune reconstitution – 1 pt. One NMO patient experienced skin cGVHD. All patients had deficit in neurocognitive*sphere and received special rehabilitation.
Conclusion: HSCT is successful approach for refractory pediatric MS and NMO treatment. In-time HSCT can significantly improve the outcome. Most of the patients remain in remission during the long time of follow-up. Late effects can be found in these patients, so it's important to find it and give adequate rehabilitation.
K. Kirgizov, R. Bembeeva, E. Volkova, E. Pristanskova, N. Sidorova, V. Konstantinova, O. Blagonravova, S. Piliya, E. Skorobogatova, N. Zavadenko, A. Rumyantsev
*Show more
https://doi.org/10.1016/j.ejpn.2017.04.674
Get rights and content
Objective: To estimate long-term outcomes and late effects at children underwent Autologous HSCT for multiple sclerosis (MS) and Allogenic HSCT for neuromyelitis optica (NMO).
Methods: Fifteen pts. included (MS – 12, NMO – 3). MS: female – 9, male – n*=*3; NMO: all females. MS median age – 16,8*±*1,6 y.o., median length of MS prior HSCT – 17,5*±*3,4 months, debut age: 13,4*±*1,5 y.o. (4–15 y.o.). Median EDSS 6,16*±*0,2. NMO pts. median age – 12,7*±*1,4 y.o., median length of NMO prior HSCT – 19,2*±*4,1 months, debut age: 10,2*±*0,4 y.o. (9–11 y.o.). All patients had severe*refractory disease treated with different immunosuppression, but still have inflammation signs. MS patients received Cyclophosphamide and ATGAM based chemotherapy and NMO patients received Treosulfan, Fludarabine, Rituximab and ATGAM. Late effects estimation – due to developed protocol for late effects estimation (somatic and neurocognitive status).
Results: EDSS improved at MS patients fast – on 3,1*±*0,3 during first 60 days. Median follow-up 40,7*±*2,4 months (10–76 months). Two MS patients relapsed (clinical and MRI). No severe complications registered. NMO patients stopped the progression and improved neurologically (clinical and MRI). One pt. with NMO died due to refractory ADV-infection, two patients did not have any severe toxic episodes. Median follow-up 23*±*4,7 months (1–60 months). MS patients late effects: cardio-vascular – 5 pts., endocrine*– 3 pts (all females). NMO patients late effects: cardio-vascular – 2 pts., late immune reconstitution – 1 pt. One NMO patient experienced skin cGVHD. All patients had deficit in neurocognitive*sphere and received special rehabilitation.
Conclusion: HSCT is successful approach for refractory pediatric MS and NMO treatment. In-time HSCT can significantly improve the outcome. Most of the patients remain in remission during the long time of follow-up. Late effects can be found in these patients, so it's important to find it and give adequate rehabilitation.