STUDY: Long-term follow-up more than 10 years after HSCT: a monocentric experience

[ActiveMSers]

Journal of Neurology

February 2018, Volume 265, Issue 2, pp 410–416

Long-term follow-up more than 10 years after HSCT: a monocentric experience

Authors
Jessica Frau, Margherita Carai, Giancarlo Coghe, Giuseppe Fenu, Lorena Lorefice, Giorgio La Nasa, Elena Mamusa, Adriana Vacca, Maria Giovanna Marrosu, Eleonora Cocco

Abstract

Background
Autologous hematopoietic stem cell transplantation (aHSCT) is used in aggressive relapsing and progressive multiple sclerosis (MS). The multicentre studies and case series reported have relatively short follow-up.

Aim
To evaluate long-term effect and safety of HSCT in MS.

Materials and methods
Patients referred to the MS centre of Cagliari and undergoing HSCT were included. Variations in relapses and EDSS before and after HSCT were evaluated by Wilcoxon test. A descriptive analysis was made for other clinical data.

Results
Nine patients (female 6, males 3; 5 relapsing–remitting, 2 secondary progressive, 1 primary progressive, and 1 progressive relapsing) performed HSCT (1999–2006). The median follow-up was 11 years (11–18). Eight patients underwent aHSCT, seven using a low intensity conditioning regimen, and one an intermediate intensity. The primary progressive underwent allogeneic HSCT, due to onco hematological disease. The relapses number decreased in the 2 years following the procedure compared to the two preceding years (p = 0.041). New relapses or disease progressions were observed after a range of 7 (low intensity regimen)–118 (intermediate intensity) months. At last follow-up, the EDSS was stable in two patients, improved in two, and worse in five (maximum 2 EDSS in one patient). Six patients showed new lesions, and seven gadolinium-enhancing on brain MRI after a mean of 23.3 and 19.8 months, respectively. Two serious adverse events were reported: melanoma, and progressive multifocal leukoencephalopathy.

Conclusions and discussion
Our results confirm in a long follow-up the efficacy of HSCT in reducing relapses and disability progression. The risk/benefit profile is better for intermediate intensity regimens.