Background: High-dose immunosuppressive therapy (HDIT) with autologous haematopoietic stem cell transplantation (AHSCT) is a new and promising approach to MS treatment.
Methods: In this paper the authors’ present the results of a prospective phase II open-label single centre study with the analysis of the safety and efficacy of HDIT+AHSCT with reduced-intensity conditioning regimen in 95 MSers. The MSers underwent early, conventional and salvage/late transplantation. Efficacy was evaluated based on clinical and quality of life outcomes.
Results: No transplant related deaths were observed. The mobilization and transplantation procedures were well tolerated. All the MSers, except one, responded to the treatment. At long-term follow-up (mean 46 months) the overall clinical response in terms of disease improvement or stabilization was 80%. The estimated progression-free survival at 5 years was 92% in the group after early AHSCT versus 73% in the group after conventional/salvage AHSCT. Statistically significant difference between the survival probabilities of two groups was determined (P=0.01). No active, new or enlarging lesions in magnetic resonance imaging (MRI) were registered in patients without disease progression. All MSers who did not have disease progression were off therapy throughout the post-transplant period. AHSCT was accompanied by a significant improvement in patient’s quality of life (QoL) with statistically significant changes in the majority of QoL parameters (P<0.05).
Conclusion: The results of this study support the feasibility of AHSCT with reduced intensity conditioning in MSers. Multicentre cooperative studies are needed for better assessment of treatment results and optimization of the treatment protocol of AHSCT with reduced intensity conditioning regimens in MS.
“The death rate associated with this study is much lower than what is published from othe studies and registers, which runs on at something in the region of 5%. The results are very impressive. The one caveat is that the study is open-label and therefore difficult to interpret. The big question is what happens to these MSers long-term; are they cured, will they relapse or will they return many years later with SPMS?”