Background: High-dose immunosuppressive therapy (HDIT) with autologous haematopoietic stem cell transplantation (AHSCT) is a new and promising approach to MS treatment.
Methods: In this paper the authors’ present the results of a prospective phase II open-label single centre study with the analysis of the safety and efficacy of HDIT+AHSCT with reduced-intensity conditioning regimen in 95 MSers. The MSers underwent early, conventional and salvage/late transplantation. Efficacy was evaluated based on clinical and quality of life outcomes.
Results: No transplant related deaths were observed. The mobilization and transplantation procedures were well tolerated. All the MSers, except one, responded to the treatment. At long-term follow-up (mean 46 months) the overall clinical response in terms of disease improvement or stabilization was 80%. The estimated progression-free survival at 5 years was 92% in the group after early AHSCT versus 73% in the group after conventional/salvage AHSCT. Statistically significant difference between the survival probabilities of two groups was determined (P=0.01). No active, new or enlarging lesions in magnetic resonance imaging (MRI) were registered in patients without disease progression. All MSers who did not have disease progression were off therapy throughout the post-transplant period. AHSCT was accompanied by a significant improvement in patient’s quality of life (QoL) with statistically significant changes in the majority of QoL parameters (P<0.05).
Conclusion: The results of this study support the feasibility of AHSCT with reduced intensity conditioning in MSers. Multicentre cooperative studies are needed for better assessment of treatment results and optimization of the treatment protocol of AHSCT with reduced intensity conditioning regimens in MS.
“The death rate associated with this study is much lower than what is published from othe studies and registers, which runs on at something in the region of 5%. The results are very impressive. The one caveat is that the study is open-label and therefore difficult to interpret. The big question is what happens to these MSers long-term; are they cured, will they relapse or will they return many years later with SPMS?”
5 thoughts on “Research: de-risking stem cells transplants”
This sounds drastic but would be great if disease activity is reduced or eliminated. Will participants be monitored so that the development, or hopefully not, of SPMS be known about?
how about this decision tree:I. Current drugs do not stop disease progression. SPMS is quasi certain.II. AHSCT has a negligible death rate. You will live to see another rate.III. AHSCT may or may not stop progression to SPMS. It is a *free* option not offered by the DMDs.Ceteris paribus non-lethal risks associated with AHSCT, why would patients not take this approach more seriously? Free upside that may or may not materialise over time, but worse case scenario clearly not worse than staying on DMDs by the looks of it!Impressive results
In the abstract it says that this was used on all types of MS, presumably PPMS and SPMS. It sounds too good to be true?
Mortality is one thing, but complications are another.Unfortunately the abstract doesn't detail the protocol used for preconditioning, and doesn’t tell us about side effects to stem cell treatment experienced by the groups.This personal blog from someone who's had full-blown autologous haematopoietic stem cell treatment will give some idea of how things can go wrong. http://tinyurl.com/stellas-blog Just one person's experience, of course, but makes for sobering reading.
Re: "http://tinyurl.com/stellas-blog "Very scary! About 12 years ago when I was working at the Royal Free Hospital I did due diligence on autologous bone marrow transplantation (BMT) in MS. The Royal Free had a BMT programme for treating other autoimmune diseases. It did not take me long to realise that the procedure was too risky and the results not clear cut. In my opnion, with the emergence of mitoxantrone, natalizumab, alemtuzumab and rituximab as effective treatments for MS there is little reason to consider BMT in the vast majority of MSers with highly-active disease.