Chiara Innocenti (Italy)
University Hospital of Careggi Cell Therapy and Transfusional Medicine UnitAuthor Of 1 Presentation
EFFECTIVENESS OF AUTOLOGOUS HAEMATOPOIETIC STEM CELL TRANSPLANTATION AND CONVENTIONAL IMMUNOSUPPRESSION IN SECONDARY PROGRESSIVE MULTIPLE SCLEROSIS: A RETROSPECTIVE PROPENSITY-MATCHED CASE-CONTROL STUDY
- Alice Mariottini (Italy)
Abstract
Background and Aims:
Autologous haematopoietic stem cell transplantation (AHSCT) suppresses relapses but its effect on disability progression in secondary-progressive multiple sclerosis (SP-MS) seems moderate, and the benefit/risk ratio is still controversial. The effectiveness of AHSCT was therefore evaluated in SP-MS comparing it with conventional immunosuppression with cyclophosphamide (Cy).
Methods:
SP-MS patients treated with AHSCT or Cy were retrospectively included and propensity-score matched according to baseline clinical-demographic characteristics. AHSCT procedure: BEAM+ATG; Cy treatment: monthly i.v. infusions (0.75 gr/m2), over a 3 years-period. Time to event was estimated by Kaplan-Meier analysis and adjusted using Cox-regression model.
Results:
93 SP-MS were included: 31 AHSCT; 62 Cy. Disability progression free-survival at year 5 was 70% in the AHSCT group and 75% in the Cy group (not significant); survival free from EDSS worsening was similar in the two groups, being 43% and 47% in the AHSCT and Cy groups respectively (not significant). Annualized-relapse rate significantly dropped from pre-treatment in both groups (p<0.0001): from 0.56 (95%CI 0.3-0.7) to 0.00 (95%CI 0.0–0.0) in AHSCT and from 0.45 (95%CI 0.3-0.5) to 0.20 (95%CI 0.10–0.30) in Cy, but survival-free from relapses was significantly higher for AHSCT than for Cy cases (100% vs 52% respectively at 3 years, p<0.0001).
Conclusions:
In SP-MS effectiveness of AHSCT on disability was similar to that of Cy, whereas it was remarkably superior as for relapses suppression. These data suggest that the risk/benefit ratio of AHSCT needs further evaluation in SP-MS and confirm that disability progression becomes independent from relapses in advanced MS, as probably based more on neurodegeneration than on inflammation.