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Autologous hematopoietic stem cell transplantation was found to be superior to alemtuzumab in controlling disease activity and improving cognition and quality of life in multiple sclerosis in a recent  observational cohort study.

Multiple sclerosis (MS) is an autoimmune neurological disease affecting approximately 2.8 million people worldwide. Autologous hematopoietic stem cell transplantation (aHSCT) is a highly effective treatment for MS. However, most research on aHSCT focuses on disability and disease activity outcomes, with only a few short-term studies evaluating cognitive function and quality of life (QoL). 

A monocentric observational cohort study published in the journal Multiple Sclerosis and Related Disorders assessed the long-term outcome differences in disease progression, cognitive impairment, and QoL after aHSCT or alemtuzumab treatment.

Study Population

A total of 41 MS patients were enrolled. Of these, 20 received aHSCT and 21 received alemtuzumab. The mean follow-up of the aHSCT group was significantly longer than that of the alemtuzumab group (9 vs. 5.9 years), whereas the disease duration was significantly longer in the alemtuzumab group. The rest of the baseline characteristics were comparable between the groups.

Treatment With aHSCT Outperformed Alemtuzumab in MS Disease Activity

There was a significant difference (p=0.012) between the groups regarding the probability of achieving no evidence of disease activity (NEDA) with three components (NEDA-3) status by the end of follow-up. Five years after treatment initiation, 16 out of 20 (75%) patients in the aHSCT group maintained NEDA-3 status, versus only six out of 21 (40%) patients in the alemtuzumab group. At 10 years post-transplantation, five out of 10 (55%) patients maintained NEDA-3 status.

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Six (30%) patients in the aHSCT group demonstrated a 12-month confirmed Expanded Disability Status Scale (EDSS) improvement compared to only two (9.5%) patients in the alemtuzumab group (p=0.140). EDSS progression was similar between the groups. Patients in the alemtuzumab group suffered more relapses than those in aHSCT group (p<0.001). The mean annualized relapse rate was 0.216 in the alemtuzumab-treated patients versus 0.005 in the aHSCT-treated patients (p=0.027). Seven of the alemtuzumab-treated patients showed new T2 lesions versus none of the aHSCT-treated patients at least six months after treatment initiation (p=0.002).

Improved Upper and Lower Extremity Functioning Noted in aHSCT-Treated Patients

Improved hand functioning was noted in the aHSCT group compared to the alemtuzumab group (p=0.049) as measured by the Multiple Sclerosis Functional Composite. Patients treated with aHSCT completed the timed 25-foot walk test in less time compared to baseline, showing better lower extremity function than alemtuzumab-treated patients (p=0.073).

Neurocognitive Benefits of aHSCT Over Alemtuzumab Revealed

Both groups demonstrated typical MS-related cognitive impairment at baseline, with no significant between-group differences. The aHSCT-treated patients showed neurocognitive improvement in all domains except short-term memory. Significant improvements in 5 of 12 domains were seen after a mean follow-up of 8.9 and 5.9 years in the aHSCT and alemtuzumab groups, respectively. The aHSCT group demonstrated a significantly better outcome in tonic and phasic alertness, information processing speed, verbal learning, and the paced visual serial addition test (PASAT)-divided attention than the alemtuzumab group.

Better QoL Outcomes With aHSCT Demonstrated Over 10-Year Period Versus Alemtuzumab

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The Hamburg Quality of Life Questionnaire in Multiple Sclerosis 10.0 (HAQUAMS) total score remained constant in the aHSCT group for up to 10 years, with scores increasing afterward. In contrast, the alemtuzumab group demonstrated an increase in the total HAQAMS score, suggesting a decline in QoL. A significant improvement in fatigue was observed in aHSCT-treated patients (p=0.013).

Source:

Braun, B., Fischbach, F., Richter, J., Pfeffer, L. K., Fay, H., Reinhardt, S., Friese, M. A., Stellmann, J., Kröger, N., Heesen, C., & Häußler, V. (2023). Benefits of aHSCT over alemtuzumab in patients with multiple sclerosis besides disability and relapses: sustained improvement in cognition and quality of life. Multiple Sclerosis and Related Disorders, 105414. https://doi.org/10.1016/j.msard.2023.105414