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Autologous Haematopoietic Stem Cell Transplantation After Immunosuppressive Therapy Effective and Safe in Multiple Sclerosis
12 June 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.

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]

Source: Doctors Guides (c) 1995-2008 Doctor's Guide Publishing Limited (12/06/08)

Frank this is a great find thanks for posting it. If you compare that study to the Canadian study done by Mark Freeman with ASCT the results are similar. See it here.

I like that the outcome measures are simple and functionally related and easily discerned so often in the pharmaceutical trials we see sort of, for lack of a better term, medical "spin" where we get people talking about fewer enhancing lesions and a "trend" that MAY mean less disability as they grasp at any straw to make it look good, when what they really mean is "the MRI's look good but that's about it....".

In thinking along the lines of more aggressive therapy for MS like these ASCT's, in one article on the Canadian study Dr Freedman said that they had a number of patients have jumps in functional ability at the 2year post treatment mark, which they thought was due to stem cell changes in the brain that took that long to show up.

Comparing the therapies like Revimmune which is high dose cyclophosphamide to ASCT which has the addition of stem cells to the basic premise of killing the "old" immune system, it will be interesting to see what approach works best.
1. Kill the peripheral immune system but not the marrow and allow it to rebiuld fro naive cells with no stem cells involved at all (HiCy).
2. DO ASCT and kill the system and give stems.
3. DO stem cells all alone.

Three possibilities, decades before we know huh?