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Haematopoietic stem cell transplantation for multiple sclerosis: the Swedish experience
J. Burman, E. Iacobaeus, A. Svenningsson, J. Lycke, M. Gunnarsson, P. Nilsson, M. Vrethem, S. Fredrikson, C. Martin, J. Fagius (Uppsala, SE)
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Introduction: Hematopoietic stem cell transplantation (HSCT) has been performed for multiple sclerosis (MS) since 1995. The goal of this therapy is to achieve long-term remission through short-lasting ablation of the immune system. Initially, patients with treatment-resistant progressive forms of MS were treated, largely with disappointing results. Eventually, HSCT was tried for relapsing-remitting MS (RRMS) and today an emerging corpus of evidence suggests that HSCT can be a very effective therapy for RRMS. In Sweden, HSCT has been used as a rescue therapy for aggressive MS since 2004. In this study we describe the outcome of the Swedish patients.
Materials and methods: We identified a total of 48 patients who had been treated with HSCT for MS up until February 2013. A vast majority of patients were relapsing remitting (83 %); mean age was 31 years (+/-8.3 years); mean disease duration before HSCT was 75 months (+/-62 months); mean annualized relapse rate in the year preceding HSCT was 4.1 (+/-3.8); 41 patients were treated with a medium intensity protocol (BEAM + ATG) and 7 patients were treated with a low intensity protocol (cyclophosphamide + ATG).
Results: Patients with at least one year of follow up (n=41) were analysed further. The mean follow-up time was 47 months (+/-29 months, range 12-108). Relapse free survival was 89 %; MRI event free survival was 88 %; expanded disability status scale (EDSS) score progression free survival was 77 %; event free survival (no relapses, no new MRI lesions and no EDSS progression) was 68 %.
Presence of gadolinium enhancing lesions prior to HSCT was associated with a favorable outcome (event free survival 79 % vs 46 %, p=0.028). Other factors such as relapsing-remitting disease course, age, disease duration and EDSS were investigated but no statistically significant differences could be demonstrated.
Overall mortality was 0 %. Apart from the expected side effects of sepsis and culture negative fever in connection to the procedure, 7 patients (15 %) experienced Herpes Zoster reactivation; 4 patients developed thyroid disease (8.3 %); one patient developed Crohn’s disease; one patient developed alopecia areata; and one patient contracted epilepsy.
Conclusion: HSCT is an effective treatment of MS, with a high proportion of patients being free from disease activity. Patients with inflammatory disease activity prior to treatment are more likely to respond to the procedure. HSCT can be performed relatively safely at experienced centres.
J. Burman, E. Iacobaeus, A. Svenningsson, J. Lycke, M. Gunnarsson, P. Nilsson, M. Vrethem, S. Fredrikson, C. Martin, J. Fagius (Uppsala, SE)
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Introduction: Hematopoietic stem cell transplantation (HSCT) has been performed for multiple sclerosis (MS) since 1995. The goal of this therapy is to achieve long-term remission through short-lasting ablation of the immune system. Initially, patients with treatment-resistant progressive forms of MS were treated, largely with disappointing results. Eventually, HSCT was tried for relapsing-remitting MS (RRMS) and today an emerging corpus of evidence suggests that HSCT can be a very effective therapy for RRMS. In Sweden, HSCT has been used as a rescue therapy for aggressive MS since 2004. In this study we describe the outcome of the Swedish patients.
Materials and methods: We identified a total of 48 patients who had been treated with HSCT for MS up until February 2013. A vast majority of patients were relapsing remitting (83 %); mean age was 31 years (+/-8.3 years); mean disease duration before HSCT was 75 months (+/-62 months); mean annualized relapse rate in the year preceding HSCT was 4.1 (+/-3.8); 41 patients were treated with a medium intensity protocol (BEAM + ATG) and 7 patients were treated with a low intensity protocol (cyclophosphamide + ATG).
Results: Patients with at least one year of follow up (n=41) were analysed further. The mean follow-up time was 47 months (+/-29 months, range 12-108). Relapse free survival was 89 %; MRI event free survival was 88 %; expanded disability status scale (EDSS) score progression free survival was 77 %; event free survival (no relapses, no new MRI lesions and no EDSS progression) was 68 %.
Presence of gadolinium enhancing lesions prior to HSCT was associated with a favorable outcome (event free survival 79 % vs 46 %, p=0.028). Other factors such as relapsing-remitting disease course, age, disease duration and EDSS were investigated but no statistically significant differences could be demonstrated.
Overall mortality was 0 %. Apart from the expected side effects of sepsis and culture negative fever in connection to the procedure, 7 patients (15 %) experienced Herpes Zoster reactivation; 4 patients developed thyroid disease (8.3 %); one patient developed Crohn’s disease; one patient developed alopecia areata; and one patient contracted epilepsy.
Conclusion: HSCT is an effective treatment of MS, with a high proportion of patients being free from disease activity. Patients with inflammatory disease activity prior to treatment are more likely to respond to the procedure. HSCT can be performed relatively safely at experienced centres.