The effect of HDIT
with low-intensity conditioning regimens in patients with various types of multiple sclerosis (MS
) in terms of clinical and patient-reported outcomes was studied.
In total, 418 patients with relapsing-remitting (RRMS
) and secondary progressive MS
) were enrolled in a single-center study from October 2006 to October 2018. Median follow-up was 29.8 months.
Outcomes of AHSCT
were evaluated both from physician's and patient's perspective at 3, 6, 12 months after AHSCT
and at long-term follow-up. EDSS
changes, proportion of patients who achieved NEDA
-3, event-free survival (EFS
), safety, and quality of life (QoL) changes were evaluated separately in patients with RRMS
Paired t-test, Wilcoxon test and Generalized Estimating Equations and were used for comparisons.
Kaplan-Meyer method was used to evaluate EFS
in terms of relapse-free survival (RFS
) and progression-free survival (PFS
) after AHSCT
. Good tolerability of transplantation procedure was demonstrated in both patient groups.
There were no cases of transplantation-related mortality.
Response to treatment was achieved in the vast majority of patients.
Significant improvement in disability for the entire group at all time-points after transplantation as compared with baseline was observed. The EDSS
score improved in 32% and 17% of RRMS
patients and in 32% and 36% SPMS
patients, at 2 years and 4 years, respectively.
At follow-up of 12 months post-ransplant, 94.6% RRMS
patients and 85.6% SPMS
patients achieved NEDA
At 7-year follow-up after AHSCT
the estimated RFS in RRMS
were 83%; PFS
No differences in EFS
were found according to conditioning regimens in both RRMS
was similar in the subgroups of patients depending on age and disease duration. RFS
was dramatically better in patients with EDSS
< 4 as compared to patients with EDSS
> 4 in RRMS
no differences were shown for PFS
patients depending on EDSS
In terms of patient's perspective AHSCT
resulted in significant and sustained improvement of patient's QoL
both in RRMS
. The results obtained point to feasibility of AHSCT
with low-intensity conditioning regimens in RRMS
patients. Multi-center cooperative studies are worthy to optimize the protocol of AHSCT
with low-intensity conditioning regimens in patients with MS