Immune-Mediated Demyelinating Central Nervous System Graft-Versus-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation

Central nervous system graft-versus-host disease (CNS GVHD) is a rare and challenging complication of allogeneic hematopoietic stem cell transplantation (HSCT) that can mimic infection, malignancy, or primary demyelinating disorders. We describe a 21-year-old man with high-risk Philadelphia chromosome–like B-cell acute lymphoblastic leukemia underwent matched unrelated donor allogeneic HSCT after achieving measurable residual disease negativity. His post-transplant course was complicated by hepatic and cutaneous GVHD. Sixteen months after transplantation, he developed progressive bilateral lower-extremity weakness, gait ataxia, and blurred vision. Brain MRI demonstrated multifocal subcortical lesions, while spinal MRI revealed enhancing intramedullary lesions at multiple levels. Orbital MRI showed bilateral optic neuritis. Extensive infectious, malignant, and autoimmune evaluations were unrevealing. Given his prior GVHD, multifocal demyelinating pattern, exclusion of alternative etiologies, and response to immunosuppression, probable demyelinating CNS GVHD was diagnosed. He was treated with corticosteroids, IVIG, plasma exchange, and rituximab, followed by maintenance IVIG and prednisone, with substantial clinical and radiographic improvement. CNS GVHD should be considered in HSCT recipients with new multifocal neurologic deficits and demyelinating lesions, particularly with antecedent systemic GVHD. Early recognition and immunosuppressive treatment may improve neurologic outcomes.