Ocular Findings after Bone Marrow Transplantation in a Pediatric Population Donny W. Suh, MD, Mark S. Ruttum, MD, Bradley J. Stuckenschneider, MD, William F. Mieler, MD, Jane D. Kivlin, MD Purpose: With the increasing use of bone marrow transplantation (BMT) in children to treat diseases of the hematopoietic system, ocular complications have been recognized in greater numbers. The authors performed a retrospective study to determine the prevalence and types of ocular abnormalities in a pediatric population following BMT. Design: Retrospective noncomparative study. Participants: One hundred and four consecutive pediatric patients with aplastic anemia and various hema- tologic malignancies who required BMT. Intervention: Bone marrow transplantation. Main Outcome Measures: Visual acuity and slit-lamp biomicroscopic and funduscopic examinations. Results: Ocular changes developed in 51% of patients. The most frequent findings included dry eye syndrome (12.5%), cataract (23.0%), and posterior segment complications (13.5%). A final visual acuity of 20/40 or better was achieved in 95.7% of eyes. Conclusions: Pediatric patients can develop severe and potentially vision-threatening complications follow- ing BMT. Despite the high incidence of anterior and posterior segment abnormalities, ocular symptoms were generally mild and the majority of patients retained excellent visual function. Factors associated with ocular complications included the underlying disease, total body irradiation, systemic chemotherapy, graft-versus-host disease, and immunosuppression. Familiarity with these risk factors and potential complications in pediatric patients is important for successful treatment. Ophthalmology 1999;106:1564 –1570 Bone marrow transplantation (BMT) is an increasingly suc- cessful treatment for severe aplastic anemia, for a variety of hematologic malignancies when chemotherapy has failed, and for a rapidly expanding list of other diseases. 1 Bone marrow transplantation has resulted in a marked improvement in long- term survival in many of these patients, especially in the pediatric population. 1 Although transplant preparative regi- mens have become increasingly complicated, with numerous protocols in use, most protocols consist of chemotherapy and total body irradiation (TBI) to suppress the patient’s immune system and eradicate the underlying neoplasm, followed by transplantation of donor bone marrow to restore hematologic function. 2,3 Patients with acute lymphocytic leukemia (ALL) additionally receive prophylactic cranial irradiation during their initial induction therapy, often many months before BMT, to eradicate central nervous system disease. The eyes are not shielded during irradiation because they are a potential site of leukemia relapse. Three types of BMTs are performed: (1) allogeneic, in which donor marrow is harvested from a human leukocyte antigen-compatible donor; (2) syngeneic, in which donor marrow is harvested from an identical twin; and (3) autol- ogous, in which the patient’s own marrow is harvested prior to induction therapy and later restored after chemotherapy and TBI have been completed. 3 Patients receiving allogeneic bone marrow transplants, the most commonly performed type, are predisposed to graft-versus-host disease (GVHD) because the donor graft mounts an immunologic response against the immunocom- promised host. 4 Graft-versus-host disease may involve nu- merous organ systems, most frequently the skin, liver, and gastrointestinal tract, and in its most severe form may be life-threatening. 4 Its treatment may require large doses of systemic corticosteroids. Ocular complications are common in GVHD and include diffuse or pseudomembranous con- junctivitis, dry eye syndrome (DES), cataract, retinal vas- culitis, retinal hemorrhage, and optic neuropathy. 2,5–7 This study describes the ocular complications in a group of pediatric patients who underwent BMT at Children’s Hospital of Wisconsin during a 15-year period. Patients and Methods A retrospective study was performed of all patients who were less than 18 years of age at the time of BMT at Children’s Hospital of Wisconsin from October 1981 through August 1996. Originally received: July 8, 1998. Revision accepted: May 5, 1999. Manuscript no. 98370. From The Eye Institute, Medical College of Wisconsin, Milwaukee, Wis- consin. Presented in part as a poster at the American Academy of Ophthalmology annual meeting, New Orleans, Louisiana, November 1998. Supported in part by an unrestricted grant from Research to Prevent Blindness, Inc., New York, New York. Address correspondence to Mark S. Ruttum, MD, The Eye Institute, 925 N. 87th Street, Milwaukee, WI 53226-4812. 1564