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Efficacy of autologous Hematopoietic stem cell transplantation for MS. A single-centre report of 81 patients.
C. Zhukovsky1, A. Tolf1, H. Cherif2, K. Carlson2, J. Burman1 1Neurology, 2Haematology, Uppsala University, Uppsala, Sweden
Introduction: Autologous hematopoietic stem cell transplantation (AHSCT) has been used as a treatment of MS since 1995. The first patient in Sweden was treated in 2004 and in 2016 it was recommended as a treatment option for patients with active MS by the Swedish Board of Health and Welfare.
Objectives: To describe clinical outcomes after AHSCT for MS.
Methods: All patients who had been treated with AHSCT for MS at Uppsala University Hospital from April 2004 to February 2019 (n=81) were included in the study; 56 women and 25 men. Seventy-seven (95%) were diagnosed with relapsing-remitting MS. The median age was 29 years (range 8 - 58 years); the mean disease duration before AHSCT was 6.0 years (±5.6 years); the median number of treatments prior to AHSCT was 2 (range 0 - 5); the mean annualized relapse rate in the year preceding AHSCT was 2.2 (±2.1), and the median baseline EDSS was 3.5 (range 0 - 8.5). Nineteen patients were treated with the intermediate intensity BEAM/ATG (BCNU, etoposide, cytarabine and melphalan + anti-thymocyte globulin) conditioning regimen and 62 patients were treated with a low intensity cyclophosphamide/ATG conditioning regimen.
Results: The mean follow-up time was 3.9 years (±3.2) and the total number of follow-up years was 314. Progression free survival was 93 % (95% CI, 85 - 100%): relapse free survival was 87% (95% CI, 78 - 97%); MRI event free survival was 74% (95% CI, 69 - 89%); and event free survival (no EDSS progression, no relapses and no new MRI lesions) was 68 % (95% CI, 54 - 84%) at five years. The annualized relapse rate post-AHSCT was 0.022. At end of follow-up, 62% of patients had improved in their expanded disability status scale (EDSS) score; 35% were unchanged and 3.3% worsened. The median ΔEDSS at 1, 2 and 3 years after AHSCT were 1.5 (range -1 - 8), 2 (range -1.5 - 8) and 2 (range -1.5 - 8.25). The overall mortality was 0 %.
Conclusion: AHSCT leads to a drug-free prolonged disease remission and functional improvement in a majority of patients.
C. Zhukovsky1, A. Tolf1, H. Cherif2, K. Carlson2, J. Burman1 1Neurology, 2Haematology, Uppsala University, Uppsala, Sweden
Introduction: Autologous hematopoietic stem cell transplantation (AHSCT) has been used as a treatment of MS since 1995. The first patient in Sweden was treated in 2004 and in 2016 it was recommended as a treatment option for patients with active MS by the Swedish Board of Health and Welfare.
Objectives: To describe clinical outcomes after AHSCT for MS.
Methods: All patients who had been treated with AHSCT for MS at Uppsala University Hospital from April 2004 to February 2019 (n=81) were included in the study; 56 women and 25 men. Seventy-seven (95%) were diagnosed with relapsing-remitting MS. The median age was 29 years (range 8 - 58 years); the mean disease duration before AHSCT was 6.0 years (±5.6 years); the median number of treatments prior to AHSCT was 2 (range 0 - 5); the mean annualized relapse rate in the year preceding AHSCT was 2.2 (±2.1), and the median baseline EDSS was 3.5 (range 0 - 8.5). Nineteen patients were treated with the intermediate intensity BEAM/ATG (BCNU, etoposide, cytarabine and melphalan + anti-thymocyte globulin) conditioning regimen and 62 patients were treated with a low intensity cyclophosphamide/ATG conditioning regimen.
Results: The mean follow-up time was 3.9 years (±3.2) and the total number of follow-up years was 314. Progression free survival was 93 % (95% CI, 85 - 100%): relapse free survival was 87% (95% CI, 78 - 97%); MRI event free survival was 74% (95% CI, 69 - 89%); and event free survival (no EDSS progression, no relapses and no new MRI lesions) was 68 % (95% CI, 54 - 84%) at five years. The annualized relapse rate post-AHSCT was 0.022. At end of follow-up, 62% of patients had improved in their expanded disability status scale (EDSS) score; 35% were unchanged and 3.3% worsened. The median ΔEDSS at 1, 2 and 3 years after AHSCT were 1.5 (range -1 - 8), 2 (range -1.5 - 8) and 2 (range -1.5 - 8.25). The overall mortality was 0 %.
Conclusion: AHSCT leads to a drug-free prolonged disease remission and functional improvement in a majority of patients.