Blood. 2003 Jul 3
Burt RK, Cohen BA, Russell E, Spero K, Joshi A, Oyama Y, Karpus WJ, Luo K, Jovanovic B, Traynor AE, Karlin K, Stefoski DH, Burns WH.
Northwestern University, Chicago, IL, USA.
Twenty-one patients with rapidly progressive multiple sclerosis (MS) were treated on a phase I/II study of intense immune suppressive therapy and autologous hematopoietic stem cell (HSC) support with no one year mortality.
Following transplant, one patient had a confirmed acute attack of MS.
Neurologic progression defined by the expanded disability status scale (EDSS) did not increase in disability by 1.0 or more steps in any of 9 patients with a pre transplant EDSS </= 6.0.
In 8 of 12 patients with high pre transplant disability scores (EDSS > 6.0), progressive neurologic disability as defined by at least a 1 point increase in the EDSS has occurred and was manifested as gradual neurologic deterioration.
Two patients with a pre-transplant EDSS of 7.0 and 8.0 died from complications of progressive disease at 13 and 18 months following treatment.
Our experience suggests that intense immune suppression and hematopoietic stem cell transplantation (HSCT) is not effective for patients with progressive disease and high pre-transplant disability scores.
Further studies are necessary to determine the role of intense immune suppressive therapy and HSC support in ambulatory patients with less accumulated disability and more inflammatory disease activity.
In specific, more patients and longer follow-up would be required in patients with an EDSS </= 6.0 before drawing conclusions on this subgroup.