By Judith Moser, MD
NICE, France -- June 11, 2008 -- In patients with multiple sclerosis (MS), immunosuppressive therapy followed by autologous haematopoietic stem cell transplantation elicited high response rates and improved quality of life for up to 6 years.
The results of the study were presented here at the 18th Meeting of the European Neurological Society (ENS) by Tatiana Ionova, MD, PhD, Department of Haematology, Pirogov National Medical Surgical Center, Moscow, Russia.
During the last decade, high-dose immunosuppressive therapy followed by autologous haematopoietic stem cell transplantation has been used with increasing frequency as a therapeutic option for patients with MS.
"The aim of the study was to assess the clinical and patient-reported outcomes in patients who underwent early, conventional, and late transplantation," explained Dr. Ionova in a poster session on June 10.
Fifty-six patients with all types of MS (primary progressive, secondary progressive, progressive relapsing, relapsing remitting) were included. Their mean age was 32.0 years (range 17-51). Median Expanded Disability Status Scale (EDSS) score at baseline was 6.0, the mean follow-up duration 18 months (range 6-84 months).
Clinical improvement was defined as a decrease in EDSS score of at least 0.5 points on 2 consecutive visits 6 months apart. Disease progression was defined as an increase of at least 0.5 points after 6 months and/or the appearance of new lesions on magnetic resonance imaging (MRI). Stabilisation corresponded to no change in EDSS score.
Neurological assessments and quality of life assessments were done at baseline, at discharge, every 3 months until 1 year after the transplantation, and every 6 months thereafter. "As multiple sclerosis is considered an incurable disease, quality of life is the overall treatment goal," emphasised Dr. Ionova.
MRI scans were done at baseline, at 6 months, at 12 months, and at the end of the follow-up.
"All patients appeared to respond to treatment", reported Dr. Ionova. Improvement was seen in 62.3%, and stabilisation occurred in 37.7% of patients. Progression after improvement occurred in 7.1% and progression after stabilisation in 11.8% of patients.
There were no deaths during the course of the study.
Out of 26 patients included in the quality-of-life analysis, 24 exhibited a response and preserved a good quality of life during the follow-up. No unexpected treatment-related adverse events were observed.
According to Dr. Ionova, immunosuppressive therapy plus autologous haematopoietic stem cell transplantation appears to be a safe and effective therapy for multiple sclerosis, Dr. Ionova concluded.
The data obtained point to feasibility of early, conventional, and salvage/late transplantation in MS patients, she said.
[Presentation title: Treatment Outcomes in Multiple Sclerosis Patients After High Dose Immunosuppressive Therapy With Autologous Haematopoietic Stem Cell Transplantation. Abstract P 731]
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