Autologous hematopoietic stem cell transplantation stabilizes retinal atrophy in multiple sclerosis

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ID: 326886
2026
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Abstract
Abstract Autologous hematopoietic stem cell transplantation (aHSCT) is an effective treatment for aggressive multiple sclerosis refractory to disease-modifying therapies. Yet, its impact on neurodegeneration remains underexplored, and few predictive biomarkers for clinical outcomes after autologous hematopoietic stem cell transplantation exist. We investigated whether autologous hematopoietic stem cell transplantation attenuates retinal neurodegeneration and whether optical coherence tomography-derived retinal measures predict disability progression after transplantation. In this single-center longitudinal cohort study, optical coherence tomography was performed in people with multiple sclerosis treated with autologous hematopoietic stem cell transplantation and non-transplanted controls with relapsing-remitting multiple sclerosis. Retinal layer atrophy rates pre-transplantation and up to 36 months post-transplantation were estimated using linear mixed-effects models and post-transplantation rates were compared with those of non-transplanted controls. Cumulative link mixed models were used to assess whether baseline retinal layer thickness predicted clinical progression after transplantation. The autologous hematopoietic stem cell transplantation cohort included 39 participants (23/39 [59%] female), comprising 23 with relapsing-remitting (15/23 [65%] female), 8 with secondary progressive (5/8 [62.5%] female), and 8 with primary progressive multiple sclerosis (3/8 [37.5%] female). The relapsing-remitting multiple sclerosis control cohort on disease-modifying treatment included 48 participants (31/48 [65%] female). In relapsing-remitting multiple sclerosis, autologous hematopoietic stem cell transplantation reduced thinning of the ganglion-cell/inner-plexiform layer by 0.65 µm/year (95% CI 0.16–1.15, p=0.010), temporal-quadrant peripapillary retinal-nerve-fiber layer by 1.22 µm/year (95% CI 0.63–1.81, p<0.001), and papillomacular bundle peripapillary retinal-nerve-fiber layer by 1.59 µm/year (95% CI 0.89–2.29, p<0.001). Post-transplantation atrophy rates of the global, temporal, and papillomacular-bundle peripapillary retinal nerve fiber layer were lower in the aHSCT cohort than in the control cohort by 0.275 µm/year (95% CI 0.048–0.502, p=0.014), 0.294 µm/year (95% CI 0.118–0.470, p=0.001), and 0.200 µm/year (95% CI 0.043–0.357, p=0.015), respectively. Higher baseline inner nuclear layer thickness predicted better disability outcomes after transplantation in both relapsing and progressive multiple sclerosis in exploratory statistical models (p<0.001). In conclusion, retinal optical coherence tomography demonstrated reduced retinal atrophy rates in relapsing-remitting multiple sclerosis after autologous hematopoietic stem cell transplantation. Higher inner nuclear layer thickness before transplantation predicted more favorable neurological outcomes, indicating that the inner nuclear layer, a retinal layer linked to inflammatory multiple sclerosis activity, may help identify patients most likely to benefit from autologous hematopoietic stem cell transplantation.
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Authors Jay R T Zoellin, Dario E Mattle, Daniel Agostino, Fabienne C. Fierz, Josefine Ruder, Angela Zaugg, Marina Herwerth, Michael Weller, Shiv Saidha, Sven Schippling, Ilijas Jelcic, Patrick Roth, Roland Martin, Veronika Kana
Journal Brain communications
Year 2026
DOI
10.1093/braincomms/fcag336
URL
https://academic.oup.com/braincomms/advance-article-pdf/doi/10.1093/braincomms/fcag336/70896072/fcag336.pdf
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