title: Ocular changes in the mucopolysaccharidoses after bone marrow transplantation. A preliminary report.
abstract: Metabolic correction and physiologic response were evaluated after bone marrow transplantation in mucopolysaccharidosis. Eleven patients were prospectively evaluated to determine the effect of bone marrow transplantation on the progressive ocular manifestations of these disorders. Follow-up of 0.6 to 2.8 years after successful donor stem cell engraftment showed that some patients had slow clearing of the corneal clouding, reduction of intracytoplasmic inclusions in the conjunctiva, resolution of optic nerve edema, and stabilized or improved retinal function as determined by electroretinography. These preliminary results suggest that early bone marrow transplantation may alter some of the progressive ophthalmic characteristics of the mucopolysaccharidoses. Long-term follow-up is necessary to determine if these early alterations in the ocular features are predictive of a prolonged functional improvement in the visual status.
authors.affiliation: Department of Ophthalmology, University of Minnesota, Minneapolis.
authors.forename: C G
authors.initials: CG
authors.lastname: Summers
authors.forename: R L
authors.initials: RL
authors.lastname: Purple
authors.forename: W
authors.initials: W
authors.lastname: Krivit
authors.forename: R
authors.initials: R
authors.lastname: Pineda
authors.forename: G T
authors.initials: GT
authors.lastname: Copland
authors.forename: N K
authors.initials: NK
authors.lastname: Ramsay
authors.forename: J H
authors.initials: JH
authors.lastname: Kersey
authors.forename: C B
authors.initials: CB
authors.lastname: Whitley
mesh_terms: D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D003228:Conjunctiva / Q000503:physiopathology; D003318:Corneal Opacity / Q000503:physiopathology; D004487:Edema / Q000628:therapy; D004596:Electroretinography; D005128:Eye Diseases / Q000209:etiology*; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D007429:Intraocular Pressure; D009083:Mucopolysaccharidoses / Q000150:complications*; D009900:Optic Nerve / Q000503:physiopathology; D011446:Prospective Studies; D013997:Time Factors