Hematopoietic Stem Cell Transplantation for CSF1R-Related Disorder: A Longitudinal Study of Efficacy and Safety

Journal of Movement Disorders 2026 Open access

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Authors: Tomasz Tomasz, Kamila Żur-Wyrozumska, Patrycja Mensah-Glanowska, Audrey Strongosky, Erik H. Middlebrooks, Ernesto Ayala, Zbigniew K. Wszolek
DOI: 10.14802/jmd.26005
Cited by: 2
References: 20

Abstract

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

Fields of Study & Subjects

Acute Myeloid Leukemia Research Hematopoietic Stem Cell Transplantation Eosinophilic Disorders and Syndromes

Publication details

Journal / source
Journal of Movement Disorders
Publisher
The Korean Movement Disorder Society
Volume
19
Issue
3
Pages
322–326
Publication date
27 February 2026
Language
EN