Autologous haematopoietic stem cell transplantation compared with alemtuzumab for relapsing-remitting multiple sclerosis: an observational study.

ABSTRACT:

Objective

To compare outcomes after treatment with autologous haematopoietic stem cell transplantation (AHSCT) and alemtuzumab (ALZ) in patients with relapsing-remitting multiple sclerosis.

Methods

Patients treated with AHSCT (n=69) received a conditioning regimen of cyclophosphamide (200?mg/kg) and rabbit anti-thymocyte globulinerG (6.0?mg/kg). Patients treated with ALZ (n=75) received a dose of 60?mg over 5?days, a repeated dose of 36?mg over 3?days after 1?year and then as needed. Follow-up visits with assessment of the expanded disability status scale score, adverse events and MR investigations were made at least yearly.

Results

The Kaplan-Meier estimates of the primary outcome measure 'no evidence of disease activity' was 88% for AHSCT and 37% for ALZ at 3?years, p<0.0001. The secondary endpoint of annualised relapse rate was 0.04 for AHSCT and 0.1 for ALZ, p=0.03. At last follow-up, the proportions of patients who improved, were stable or worsened were 57%/41%/1% (AHSCT) and 45%/43%/12% (ALZ), p=0.06 Adverse events grade three or higher were present in 48/69 patients treated with AHSCT and 0/75 treated with ALZ in the first 100 days after treatment initiation. The most common long-term adverse event was thyroid disease with Kaplan-Meier estimates at 3?years of 21% for AHSCT and 46% for ALZ, p=0.005.

Conclusions

In this observational cohort study, treatment with AHSCT was associated with a higher likelihood of maintaining 'no evidence of disease activity'. Adverse events were more frequent with AHSCT in the first 100 days, but thereafter more common in patients treated with ALZ.

SUBMITTER: Zhukovsky C 

PROVIDER: S-EPMC7841472 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature