Case Presentation: A 66-Year-Old Woman With Chronic GVHD After Allogeneic Transplant for IDH2-Mutated AML
Patient Case:
Background
A 66-year-old woman with IDH2-mutated acute myeloid leukemia (AML) achieved first complete remission (CR1) following induction chemotherapy (7+3 and 5+2 regimens) and a single cycle of cytarabine consolidation. Minimal residual disease (MRD) was undetectable by flow cytometry prior to hematopoietic cell transplantation. Her comorbidities included moderate to severe aortic stenosis with preserved ejection fraction and no clinical signs or symptoms of heart failure, as well as osteoporosis managed with weekly alendronate therapy. Her baseline performance status was excellent.
Transplant Course and Early Posttransplant Period
The patient underwent a fully human leukocyte antigen–matched unrelated donor peripheral blood stem cell transplant (PBSCT) using reduced-intensity conditioning with fludarabine and melphalan (100 mg/m²). Graft-vs-host disease (GVHD) prophylaxis consisted of posttransplant cyclophosphamide (40 mg/kg on days 3 and 4), tacrolimus, and mycophenolate mofetil. The early posttransplant course was uncomplicated. She developed stage 1, grade 1 acute cutaneous GVHD, which was managed with topical corticosteroids alone. Tacrolimus was tapered and discontinued by month 6. She experienced 2 mild upper respiratory infections during this period, both of which resolved in less than 1 week without sequelae. She received seasonal influenza and COVID-19 vaccinations approximately 6 months after transplantation.
Presentation of Chronic GVHD
At 7 months posttransplant, the patient developed progressive stiffness affecting the shoulders, trunk, and lower extremities. She reported difficulty with activities such as dressing and bending to put on footwear. There was no associated rash. Serum creatine kinase and aldolase levels were within reference range. She also reported mild dry eyes, requiring artificial tears 1 to 2 times daily, although examination showed no signs of conjunctival dryness. Oral symptoms included sensitivity to toothpaste and acidic foods, without overt lichenoid or ulcerative lesions. However, detailed oral examination revealed a reticular pattern on the bilateral buccal mucosa. Musculoskeletal examination identified reduced range of motion in multiple joints and signs of soft tissue inflammation, consistent with myofascial involvement.
Initial Management and Treatment Response
Prednisone was initiated at 0.5 mg/kg daily. After 2 weeks, the patient reported persistent stiffness and dryness, with minor improvements in appetite and sleep. Ruxolitinib was initiated at 10 mg twice daily, and prednisone was tapered to 20 mg daily. Over the subsequent 2 weeks, she experienced clinical improvement in joint mobility, ocular symptoms, and oral dryness. Prednisone was gradually tapered to 15 mg daily and subsequently to 10 mg daily.
Follow-up and Ongoing Management
The patient was co-managed through monthly visits at a tertiary transplant center and biweekly visits with her local physician. Follow-up included comprehensive physical examinations and symptom assessments, with a focus on musculoskeletal and mucosal GVHD manifestations. After 3 months of therapy, she achieved a partial response by National Institutes of Health consensus criteria, with approximately 80% of baseline mobility restored and substantial improvement in systemic symptoms. However, she reported persistent fatigue and a subjective sense of not having returned to her pretransplant quality of life.
Given that ruxolitinib had been introduced after just 2 weeks of corticosteroids—now a common approach—its early use may have influenced the trajectory of response or contributed to residual symptoms. At 4 months of therapy, she reported a plateau in symptom improvement. A shared decision-making approach was employed to discuss treatment goals, and third-line therapy options were reviewed in light of the chronic nature of GVHD and the challenges in achieving durable complete remission.
