Journal of Movement Disorders

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Hematopoietic Stem Cell Transplantation for CSF1R-Related Disorder: A Longitudinal Study of Efficacy and Safety: Tomasz Chmiela, Kamila Żur-Wyrozumska, Patrycja Mensah-Glanowska, Audrey J. Strongosky, Erik H. Middlebrooks, Ernesto Ayala, Zbigniew K. Wszolek; Received January 6, 2026 Accepted February 27, 2026 Published online February 27, 2026; DOI: https://doi.org/10.14802/jmd.26005 [Epub ahead of print]

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Objective
This longitudinal study evaluated the long-term efficacy and safety of hematopoietic stem cell transplantation (HSCT) in slowing the progression of CSF1R-related disorder (CSF1R-RD).
Methods
Six symptomatic patients (mean follow-up, 6.6 years) were compared with six matched, untreated controls. The CSF1R Clinical Severity Score (CCSS), Montreal Cognitive Assessment, and Sundal radiological score were used for evaluation.
Results
Post-HSCT clinical progression slowed significantly from 14.1 to 3.7 CCSS/year, compared with 15.2 CCSS/year in the control group (p<0.01). Cognitive decline was substantially reduced (-1.5 points/year vs. -7.6 points/year), and radiological deterioration slowed (0.4 per year vs. 3.9 per year). Notably, during the observation period, all HSCT patients survived, whereas 50% of the patients in the control group died. No serious transplant-related complications were observed.
Conclusion
HSCT is a potent disease-modifying therapy for CSF1R-RD that drastically improves survival and slows deterioration. These findings underscore the necessity of early intervention during the symptomatic phase to maximize the preservation of quality of life for affected individuals.

Citations

Citations to this article as recorded by

The path to clinical application of human microglia transplantation for the treatment of CSF1R-related disorder
Tomasz Chmiela, Robert C. Spitale, Zbigniew K. Wszolek
Expert Review of Neurotherapeutics.2026; : 1. CrossRef

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