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

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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.

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Author information

Authors and Affiliations

  1. Department of Medical Sciences and Public Health, Multiple Sclerosis Center, ATS Sardegna, Ospedale Binaghi, University of Cagliari, Via Is Guadazzonis 2, 09126, Cagliari, Italy

    Jessica Frau, Margherita Carai, Giancarlo Coghe, Giuseppe Fenu, Lorena Lorefice, Elena Mamusa, Maria Giovanna Marrosu & Eleonora Cocco

  2. Department of Medical Sciences and Public Health, Bone Marrow Transplant Center, University of Cagliari, Via Is Guadazzonis 2, 09126, Cagliari, Italy

    Giorgio La Nasa & Adriana Vacca

Authors
  1. Jessica Frau
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  2. Margherita Carai
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  3. Giancarlo Coghe
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  4. Giuseppe Fenu
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  5. Lorena Lorefice
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  6. Giorgio La Nasa
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  7. Elena Mamusa
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  9. Maria Giovanna Marrosu
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  10. Eleonora Cocco
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Corresponding author

Correspondence to Jessica Frau.

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Conflicts of interest

The author(s) declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article.

Ethical standards

All the subjects signed written informed consent for the use of their data at the moment of MS diagnosis or their first visit to the MS centre. The data were collected from the medical records by an MS neurologist. The ethics committee of the University of Cagliari approved the study.

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Frau, J., Carai, M., Coghe, G. et al. Long-term follow-up more than 10 years after HSCT: a monocentric experience. J Neurol 265, 410–416 (2018). https://doi.org/10.1007/s00415-017-8718-2

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  • DOI: https://doi.org/10.1007/s00415-017-8718-2

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