Case Presentation: 18-Year Old Patient With Moderate Chronic Graft-Versus-Host Disease Case Presentation
Patient Case:
Background
An 18-year-old male with relapsed acute myeloid leukemia (AML) underwent a haploidentical peripheral blood stem cell transplant from his 40-year-old father following myeloablative conditioning with busulfan and fludarabine. GVHD prophylaxis consisted of posttransplant cyclophosphamide, tacrolimus, and mycophenolate mofetil.
Transplant Course and Early Posttransplant Period
During the early posttransplant period, the patient developed moderate hepatic veno-occlusive disease (VOD), which resolved completely with a course of defibrotide. Neutrophil engraftment occurred on day 21. On day 42, he developed stage 2, grade 1 acute GVHD involving approximately 35% of body surface area (BSA), characterized by an erythematous maculopapular skin rash. This was managed with topical corticosteroids, leading to complete resolution by day 60.
Mycophenolate mofetil was discontinued on day 35. A tacrolimus taper was initiated on day 100. However, by day 125, during the taper, the patient developed a recurrent erythematous maculopapular rash involving 50% to 60% of BSA, affecting the face, torso, and upper extremities. He also reported oral pain and ocular discomfort. Examination revealed lichenoid mucosal changes with ulcerations in the oral cavity, conjunctival erythema, and dry eye symptoms. Laboratory evaluation demonstrated normal complete blood cell count and electrolyte levels, but liver function tests were notable for an alanine aminotransferase level 4 times the upper limit of normal and a total bilirubin level of 1.2 mg/dL, both of which had been within normal range 2 weeks earlier. Pulmonary function tests were stable from baseline.
Presentation of Chronic GVHD
The clinical and laboratory findings were consistent with moderate chronic GVHD involving the skin, oral mucosa, eyes, and liver. Tacrolimus was increased to therapeutic levels. The patient was started on systemic corticosteroids (prednisone, 1 mg/kg daily), cyclosporine ophthalmic drops, and dexamethasone mouthwash.
Initial Management and Treatment Response
Within 2 weeks, tacrolimus levels reached the therapeutic range. The patient reported resolution of oral pain and healing of mucosal ulcerations. Ocular symptoms improved, and he was using lubricating eye drops only 1 to 2 times daily. The skin rash showed partial improvement, decreasing to approximately 15% BSA, localized primarily to the upper extremities. The rash had evolved from a bright erythematous appearance to a more violaceous hue. Liver function tests remained largely unchanged, though the total bilirubin level had increased to 3.2 mg/dL.
Follow-up and Ongoing Management
Given the worsening of liver function and the change in appearance of the skin rash, additional therapy was warranted. Ruxolitinib was added to the regimen at the recommended starting dose of 10 mg twice daily. Though improvement in liver function was observed within 2 weeks, the skin rash persisted and became more sclerotic in nature. Third-line treatment was therefore considered.
