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Disease modifying therapy after autologous haematopoietic stem cell transplantation in multiple sclerosis patients:
Nataša Giedraitienė1, Gintaras Kaubrys1, Rasa Kizlaitienė1
1Clinic of Neurology and Neurosurgery, Institute of Clinical Medicine, Faculty of Medicine, Vilnius University, Vilnius, Lithuania.
Background:
There is limited information reported on the management of disease modifying treatment (DMT) in patients with multiple sclerosis (MS) who have been treated with autologous hematopoietic stem cell transplantation (AHSCT).
Methods:
We conducted a single-centre prospective study of patients with highly active relapsing-remitting MS who underwent AHSCT in Lithuania from May 2014 till January 2025.
Results:
Forty-two patients were selected and treated with AHSCT. 65.0 % of patients who underwent AHSCT achieved No Evidence of Disease Activity-3 (NEDA-3) over a 65-month median follow-up period. 35.6 % of patients experienced improvement in their Expanded Disability Status Scale (EDSS) scores by the third month post-transplant, and this improvement was maintained throughout the follow-up period. Twelve patients (28.5 %) who experienced disease activity after AHSCT were treated with DMT: eight patients (19.0 %) were treated with ocrelizumab, and in four patients (9.5 %), SPMS was diagnosed and patients were treated with siponimod. Five patients who were treated with ocrelizumab after AHSCT achieved NEDA-3, three of whom had been on DMT for >12 months.
Conclusion:
The majority of patients maintained NEDA-3 up to five years after AHSCT. Patients who did not achieve NEDA-3 were successfully treated with high-efficacy DMT. No mortality was observed in our cohort. These findings support the efficacy and safety of AHSCT, as well as the use of DMT following AHSCT, in patients with highly active relapsing-remitting MS.
