Multiple sclerosisWednesday, June 24, 2009, 08:30 - 10:00Autologous haematopoietic stem cell transplantation as a treatment modality for progressive multiple sclerosisY. Shevchenko, A. Novik, A. Kuznetsov, B. Afanasiev, I. Lisukov, O. Rykavicin, T. Ionova, V. Melnichenko, D. Fedorenko, K. Kurbatova, R. Ivanov, N. Osipova, A. Kulagin, S. Shamansky, R. Kruglina, A. Kartashov, G. Gorodokin (Moscow, St. Petersburg, Novosibirsk, RU; New Jersey, US)
High dose immunosuppression and autologous haematopoietic stem cell transplantation (AHSCT) is a new and promising therapy for multiple sclerosis (MS) patients. In accordance with Guidelines of AHSCT in MS (Comi et al, 2000) to assess risk/benefit ratio of AHSCT in MS it is mandatory to collect further data. We aimed to study outcomes in progressive MS patients after early (EDSS 1.5-3.0), conventional (3.5-6.5) and salvage (7.0-8.0) AHSCT.
94 patients with MS (secondary progressive – 59, primary progressive – 25, progressive-relapsing – 10) were included in this study (mean age - 33.7, range: 17-54; male/female – 43/51). BEAM (n=49) or BEAM-modified (n=45) conditioning was used. Seventy six patients underwent conventional, 11 patients – early and 7 patients – salvage AHSCT. Median EDSS at base-line was 5.5 (range 1.5 – 8.0). The mean follow-up duration was 20 months (range 6-114 months). Neurological evaluation was performed at baseline, at discharge, at 3, 6, 9, 12 months, and every 6 months thereafter; MRI - at baseline, at 6, 12 months, and later once a year.
Transplantation procedure was well tolerated by the patients with no transplant-related deaths. The efficacy analysis was performed in 61 patients who had had follow-up for at least 9 months. At 6 months post transplant the following distribution of patients according to clinical response was observed: 31 patients achieved an objective improvement of neurological symptoms; 30 patients had disease stabilization. Among the patients after conventional AHSCT there were 48% patients with improvement and 52% with stabilization; after early AHSCT – 71% with improvement and 29% with stabilization; after salvage AHSCT – 33% with improvement and 67% with stabilization. In 1 year post-transplant 2 patients relapsed (both - conventional AHSCT); in 2 years post-transplant 3 patients had disease progression (all conventional AHSCT). All other patients experienced either improvement or stabilization throughout the follow-up.
MRI analysis before AHSCT revealed Gd+ lesions in 27 out of 75 patients. No active, new or enlarging lesions were registered after AHSCT in patients without disease progression.
This study provides ample evidence in support of safety and benefits of AHSCT in progressive MS patients. The data obtained points to the feasibility of early, conventional, and salvage AHSCT in progressive MS patients.
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