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. 2024 Aug:88:105745.
doi: 10.1016/j.msard.2024.105745. Epub 2024 Jun 26.

Spinal cord size as promising biomarker of disability outcomes after hematopoietic stem cell transplantation in multiple sclerosis

Alice Mariottini  1 , Emily H Stack  2 , Govind Nair  2 , Chiara Nozzoli  3 , Tianxia Wu  2 , Leonardo Marchi  4 , Riccardo Boncompagni  3 , Anna Maria Repice  5 , Enrico Fainardi  6 , Francesca Di Pasquale  7 , Edoardo Carlesi  8 , Riccardo Saccardi  3 , Steven Jacobson  2 , Luca Massacesi  9
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Free article

Spinal cord size as promising biomarker of disability outcomes after hematopoietic stem cell transplantation in multiple sclerosis

Alice Mariottini et al. Mult Scler Relat Disord. 2024 Aug.
Free article

Abstract

Background: Biomarkers predictive of disability outcomes in individual multiple sclerosis (MS) patients undergoing autologous haematopoietic stem cell transplantation (AHSCT) are currently lacking. As correlations between spinal cord atrophy and clinical disability in MS were previously described, in this study spinal cord size was investigated in MS patients treated with AHSCT, exploring whether baseline spinal cord volume may predict disability progression after AHSCT.

Methods: relapsing-remitting (RR-) and secondary-progressive (SP-) MS patients treated with AHSCT (BEAM/ATG regimen) at a single academic centre in Florence, who performed at least two standardized brain magnetic resonance imaging (MRIs) scans (acquired between one-year pre-AHSCT to 5 years after AHSCT) were included. Cervical spinal cord atrophy was estimated as upper cervical spinal cord cross-sectional area (SCCSA). Brain volume loss (BVL) was analysed at the same timepoints.

Results: Eleven (8 RR-; 3 SP-) MS patients were included. Over a median follow-up of 66 (range 37 - 100) months, no relapses nor brain MRI activity were observed; disability progressed in 2 cases (both SP-MS). Baseline SCCSA was associated with EDSS change between pre- and one-year post-AHSCT. Compared to patients who stabilized, patients who progressed after AHSCT tended to have lower SCCSA at C4 level at baseline and year 1 after AHSCT. Longitudinal changes in SCCSA or BVL did not correlate with EDSS change.

Conclusions: Baseline pre-AHSCT SCCSA, but not its longitudinal changes nor BVL, predicted EDSS change within the two years following AHSCT. SCCSA may represent a biomarker of treatment response and a promising screening tool for assessing patient eligibility for high-impact treatments such as AHSCT.

Keywords: Atrophy; Disability; Magnetic resonance imaging; Multiple sclerosis; Spinal cord; Transplant; hematopoietic stem cell transplantation.

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Conflict of interest statement

Declaration of competing interest Dr. Mariottini reports no conflicts of interest relevant to this paper; she discloses speaker's honoraria and non-financial support from Sanofi, Viatris, Genzyme, Biogen, Novartis, and Janssen, outside the submitted work. Ms. Stack, Dr. Nair, Dr. Nozzoli, Dr. Wu, Dr. Marchi, Dr. Boncompagni, Dr. Repice, Prof. Fainardi, Dr. Di Pasquale, and Dr. Carlesi have no conflicts to disclose relevant to the paper. Dr. Saccardi discloses personal fee from Sanofi. Dr. Jacobson has no conflicts to disclose relevant to the paper. Prof. Massacesi has no conflicts to disclose relevant to the paper; he reports personal fees and non-financial support from Biogen, Novartis, Merck Serono, Teva, Sanofi, and Janssen, outside the submitted work.

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