Clinical and Quality of Life Outcomes in Patients with Multiple Sclerosis (MS) after High Dose Chemotherapy(HDCT)+Autologous Stem Cell Transplantation (ASCT).

Objectives: HDCT+ASCT is a new and promising therapy for MS patients. Among a number of questions are the terms of conducting HDCT+ASCT. According to our concept there are 3 strategies of HDCT+ASCT depending on terms of disease stage: early, conventional and salvage. The aim of the research was to study clinical and quality of life (QoL) outcomes in MS patients with different terms of HDCT +ASCT.

Methods: Twelve patients with MS were included in the study. All the patients previously underwent conventional treatment. Clinical and QoL evaluation was provided at baseline, at discharge, at 3, 6, 9, 12 months and later every half a year after HDCT+ ASCT. MRI was conducted at baseline, in 6, 12 months and at the end of follow-up. QoL was assessed by FACT-BMT and FAMS. Integral QoL index was assessed by the method of integral profiles. Median EDSS at base-line was 6.0 (range 2 –7.5). The median follow-up duration was 18 months (range 3–60 months).

Results: Clinical examination and MRI revealed disease stabilization (DS) in ten patients and progression (DP) in two patients. EDSS decreased in seven patients; increased from 6.5 to 7.0 and from 6.0 to 6.5 in patients with DP and remained the same in three patients. Distinct QoL improvement was observed in all the patients with DS at the end of follow-up.

Three case reports with different terms of ASCT were analyzed. Patient K. underwent early ASCT (male, 21 years old; secondary progredient type; base-line EDSS - 2.0; follow-up 18 months); patient F.- conventional ASCT (female, 35 years old; secondary progredient type; base-line -EDSS 5.0; follow-up 60 months); patient P.- salvage ASCT (female, 49 years old; secondary progredient type; base-line EDSS 7.5; follow-up 48 months). Clinical examination and MRI revealed DS. EDSS decreased from 2.0 to 1.5 in patient K.; from 5.0 to 4.5 in patient F. and from 7.5 to 6.0 in patient P. at the end of follow-up. Integral QoL index increased dramatically as compared to base-line: in patient K. - from 0.6 to 0.7; in patient F. - from 0.2 to 0.7; in patient P. - 0.4 to 0.6.

Conclusion: HDCT+ASCT in MS patients resulted in DS in ten out of twelve patients under observation. Along with clinical stabilization dramatic improvement of QoL took place. Analysis of three case reports demonstrated feasibility of early, conventional and salvage ASCT in MS patients.