An analysis of bone marrow burden scores in a retrospective analysis of adult patients with type 1 Gaucher disease
• Bone marrow burden (BMB) scores modestly correlated with disease severity markers. • BMB scores remained mainly consistent over time despite treatment. • BMB scores did not correlate with pain or physical functioning. • Less frequent BMB monitoring may be possible in long-term treated patients. • Additional studies are needed to evaluate the utility of BMB scores in established GD1.
Authors
- Marie‐Claude Miron (ORCID: https://orcid.org/0000-0002-6425-4785)
- Rakesh Mohankumar
- Graeme Nimmo
- Dominick Amato (ORCID: https://orcid.org/0000-0002-1803-5442)
- Orla Drumm
Institutions
- University Health Network (CA)
- Toronto Western Hospital (CA)
- Women's College Hospital (CA)
- Centre Hospitalier Universitaire Sainte-Justine (CA)
- Sinai Health System (CA)
Publication Details
- Journal
- Blood Cells Molecules and Diseases
- Published
- 2025-05-08
- DOI
- https://doi.org/10.1016/j.bcmd.2025.102933
- Citations
- 1
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.16