ReviewOrphanet journal of rare diseases2026
Paget's disease of bone: pathogenesis, ocular manifestations, and therapeutic strategies.
Review in Orphanet journal of rare diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Authors and funding
2 authors.
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Abstract
objectivePaget's disease of bone (PDB) is a chronic, focal metabolic bone disorder characterized by increased bone destruction and disordered bone formation, and is a rare disease in Asian populations. This review aims to systematically summarize the pathogenesis, ocular manifestations, and treatment strategies for PDB, with the goal of enhancing the understanding of its multi-system involvement and exploring more effective clinical management approaches.
methodsA systematic search of electronic databases, including PubMed, was conducted using Boolean operators to combine keywords such as "Paget's disease of bone", "osteitis deformans", "pathogenesis", "disease mechanism", "ocular", "oculus", "ophthalmic", "eye", "treatment", "pharmacotherapy", "medication", etc. Following an initial screening and the exclusion of duplicate records, relevant articles were selected for comprehensive analysis. The level of evidence was assessed.
results68 articles were included. This review elaborates on the roles of genetic and environmental factors in PDB and details its diverse ocular manifestations, including ptosis, cataract, macular disease, angioid streaks, optic neuropathy, and orbital disease. It also summarizes advances in current pharmacotherapy, primarily based on bisphosphonates, and other potential treatment strategies.
conclusionAbnormal bone remodeling, driven by a combination of genetic susceptibility and environmental factors, constitutes the common pathological basis for both systemic skeletal lesions and specific ocular complications in PDB. Management should be based on multidisciplinary collaboration, integrating pharmacological intervention with individualized ocular care to control disease progression and prevent visual impairment.
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