Introduction: The common etiology of central nervous system (CNS) complications after allogeneic hematopoietic stem cell transplantation (allo-HSCT) includes CNS infection, metabolic abnormalities, drug toxicity, cerebrovascular events, Epstein-Barr virus-associated posttransplant lymphoproliferative diseases, and hematologic CNS relapse of leukemia. Although graft-versus-host disease (GVHD) is a major complication of allo-HSCT, its CNS involvement is exceedingly rare.

Case Presentation: In this report, we describe a patient who exhibited acute myeloid leukemia with t(8;21) (q22;q22) and who suddenly lost visual acuity ~1 year after receipt of allo-HSCT. Given the observation of negative cerebrospinal fluid findings, cyclosporine-related encephalopathy, intracranial hemorrhage, CNS infection, leukemia recurrence, and tumors were excluded. He was diagnosed with both CNS and pulmonary GVHD. After steroid treatment, the lesions gradually reduced in images acquired via cranial and pulmonary computed tomography.

Conclusions: CNS-GVHD is a rare, serious complication of allo-HSCT that is difficult to diagnose. Biopsy and autopsy may identify the CNS as the target of GVHD in some patients. Treatment is mainly based on the use of immunosuppressive drugs, including high doses of steroids. Early diagnosis and treatment can improve disease outcome.

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