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 language||English (US)|
|Number of pages||9|
|State||Published - 1989|
Bibliographical noteFunding Information:
Supported in part by an unrestricted grant to the Department of Ophthalmology from Research to Prevent Blindness, Inc, New York, New York; grants CA21737 and DK39891 from the National Institutes of Health; grant JWW 18-87 from the Minnesota Medical Foundation; and grant 10429 from the Graduate School, University of Minnesota.