(Abst.) Autologous hematopoietic stem cell transplantaion in MS: A meta-analysis

Neurology. 2017 Apr 28.

Autologous hematopoietic stem cell transplantation in multiple sclerosis: A meta-analysis

Sormani MP1, Muraro PA2, Schiavetti I2, Signori A2, Laroni A2, Saccardi R2, Mancardi GL2.

Author information

1
From the Departments of Health Sciences (M.P.S., I.S., A.S.) and Neuroscience, Rehabilitation, Ophthalmology, Genetics, and Maternal and Child Health (A.L., G.L.M.), University of Genoa, Italy; Division of Brain Sciences (P.A.M.), Imperial College, London, UK; IRCCS Azienda Ospedaliera Universitaria San Martino-IST (A.L., G.L.M.), Genoa; and Cell Therapy and Transfusion Medicine Unit (R.S.), Careggi University Hospital, Firenze, Italy. mariapia.sormani@unige.it.
2
From the Departments of Health Sciences (M.P.S., I.S., A.S.) and Neuroscience, Rehabilitation, Ophthalmology, Genetics, and Maternal and Child Health (A.L., G.L.M.), University of Genoa, Italy; Division of Brain Sciences (P.A.M.), Imperial College, London, UK; IRCCS Azienda Ospedaliera Universitaria San Martino-IST (A.L., G.L.M.), Genoa; and Cell Therapy and Transfusion Medicine Unit (R.S.), Careggi University Hospital, Firenze, Italy.

OBJECTIVE:

To summarize the evidence on immunoablative therapy followed by autologous hematopoietic stem cell transplantation (aHSCT) to manage severe and treatment-refractory multiple sclerosis (MS).

METHODS:

We collected all the published studies of aHSCT in any form of MS from 1995 to 2016, carefully excluding reports that were updated in subsequent studies. Endpoints were transplant-related mortality (TRM), rate of disease progression, and no evidence of disease activity (NEDA) status. A weighted metaregression based on a Poisson model was run, assessing whether there were study-specific characteristics with an effect on TRM and progression.

RESULTS:

Fifteen studies including 764 transplanted patients were pooled in the meta-analysis. The pooled estimate of TRM was 2.1% (95% confidence interval [CI] 1.3%-3.4%). TRM was higher in older studies (p = 0.014) and in studies with a lower proportion of patients with relapsing-remitting MS (RRMS) (p = 0.028). A higher baseline Expanded Disability Status Scale (p = 0.013) was also significantly associated with a higher TRM. Pooled rate of progression was 17.1% at 2 years (95% CI 9.7%-24.5%) and 23.3% (95% CI 16.3%-31.8%) at 5 years. Lower 2-year progression rate was significantly associated with higher proportions of patients with RRMS (p = 0.004). The pooled proportion of NEDA patients at 2 years was 83% (range 70%-92%) and at 5 years was 67% (range 59%-70%).

CONCLUSIONS:

The emerging evidence on this therapeutic approach in MS indicates that the largest benefit/risk profile form this therapeutic approach can be obtained in patients with aggressive MS with a relapsing-remitting course and who have not yet accumulated a high level of disability.