Ocular Changes in the Mucopolysaccharidoses after Bone Marrow Transplantation: A Preliminary Report

C. Gail Summers, Richard L. Purple, William Krivit, Roberto Pineda, Gary T. Copland, Norma K.C. Ramsay, John H. Kersey, Chester B. Whitley

Research output: Contribution to journalArticlepeer-review

47 Scopus citations

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. Longterm 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.

Original languageEnglish (US)
Pages (from-to)977-985
Number of pages9
JournalOphthalmology
Volume96
Issue number7
DOIs
StatePublished - 1989

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