Successful use of ruxolitinib in steroid-refractory chronic GvHD affecting central nervous system: a case report

Chronic graft-versus-host disease (cGvHD) is a major complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT). Beyond its classical manifestations, cGvHD may involve additional organs, including the central nervous system (CNS), where involvement remains poorly characterized, and therapeutic options are undefined, particularly in steroid-refractory cases. We report the case of a 50-year-old woman who underwent allo-HSCT for myelodysplastic syndrome and subsequently developed classical acute followed by chronic GvHD-overlap syndrome. At 2 years and 9 months post-transplant, she presented with progressive spastic paraparesis, and brain MRI revealed multifocal hyperintense white-matter lesions. A diagnosis of possible CNS-cGvHD was established. Due to poor response and intolerance to prolonged corticosteroid therapy, ruxolitinib was initiated at 10 mg/day and subsequently increased to 20 mg/day, with concomitant tapering of prednisone and immunoglobulin supplementation. Over 12 months, the patient demonstrated both clinical and radiological improvement, including significant functional recovery and marked regression of CNS lesions. This case suggests that ruxolitinib may represent a potential therapeutic option for atypical CNS involvement in cGvHD. Although clinical improvement likely reflected combined treatments, the neurological and radiological response suggests meaningful contribution from JAK inhibition. Further studies are needed to better define the role of ruxolitinib in this rare manifestation of cGvHD.