Autologous stem cell transplantation as rescue therapy in malignant forms of multiple sclerosis.
Malignant forms of multiple sclerosis (MS) represent a limited group of very aggressive demyelinating diseases, which rapidly progress to severe disability leading often to life-threatening conditions. On these clinical entities, currently available therapies for MS are not very effective. Recently, it has been demonstrated that intense immunosuppression followed by autologous stem cell transplantation (ASCT) can affect the clinical course of individuals with severe MS and completely abrogate the inflammatory activity detected by magnetic resonance imaging (MRI). We report on the treatment with intense immune ablation followed by ASCT of three patients with malignant MS whose clinical course indicated a dramatically poor prognosis. This procedure succeeded in halting the rapidly worsening course of disease. The effect was long lasting, as demonstrated by a sustained efficacy over a two-year period in two subjects and 12 months in the third case. In addition, a striking effect on inflammation-related MRI findings was obtained. These results support a role for intense immunosuppression followed by ASCT as treatment in rapidly evolving malignant MS cases unresponsive to conventional therapies.
Mancardi
Giovanni Luigi
GL
Department of Neurosciences, Ophthalmology and Genetics, University of Genoa, Genoa, Italy. glmancardi@neurologia.unige.it
Murialdo
Alessandra
A
Rossi
Paolo
P
Gualandi
Francesca
F
Martino
Gianvito
G
Marmont
Alberto
A
Ciceri
Fabio
F
Schenone
Angelo
A
Parodi
Roberto Carlo
RC
Capello
Elisabetta
E
Comi
Giancarlo
G
Uccelli
Antonio
A
D000293:Adolescent; D000328:Adult; D005260:Female; D006801:Humans; D007165:Immunosuppression Therapy / Q000379:methods*; D008279:Magnetic Resonance Imaging; D008297:Male; D020528:Multiple Sclerosis, Chronic Progressive / Q000276:immunology / Q000473:pathology / Q000628:therapy*; D011379:Prognosis; D012720:Severity of Illness Index; D033581:Stem Cell Transplantation; D014182:Transplantation, Autologous