Overview: A 42-Year-Old Woman With Moderate Steroid-Refractory Chronic GVHD

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Yi-Bin Chen, MD, discusses the work-up, diagnosis, and treatment of a 42-year-old woman with moderate steroid-refractory chronic graft-versus-host disease (GVHD).

Case: A 42-Year-Old Woman With Moderate Steroid-Refractory Chronic Graft-Versus-Host Disease

Initial Presentation

  • A 42-year-old woman previously underwent myeloablative conditioning and an allogeneic PBSC transplant for acute myeloid leukemia
  • Received tacrolimus and methotrexate for GVHD prophylaxis
  • At 11 months post-transplant, patient developed moderately dry and painful eyes, mild muscle and joint pain, mild erythematous rash on face and arms

First-line Treatment

  1. Patient received eye drops, topical steroid cream for rash, and was started on oral prednisone at 0.5 mg/kg/day
  2. After 4 weeks of treatment, eye dryness resolved, but the patient’s skin rash and muscle/joint pain worsened; steroid dose was increased to 1 mg/kg/day
  3. Skin rash and muscle/joint pain continued despite increased steroid dose

Second-line Treatment

Patient is now receiving ruxolitinib 10 mg orally twice daily, alongside 1 mg/kg/day steroids

Yi-Bin Chen, MD: Today we have a case of a 42-year-old woman who previously underwent a myeloablative unrelated donor allogeneic peripheral blood stem cell transplant for a diagnosis of acute myeloid leukemia. She received conventional tacrolimus and methotrexate for graft-versus-host disease prevention.

She did not experience any acute graft-versus-host disease, but about 11 months after transplant, the patient presented with moderately dry and painful eyes, mild muscle and joint pains, and a mild erythematous scaly rash on her face and her arms. At that point, a diagnosis of chronic graft-versus-host disease was made, and the patient was initially started on some steroid eyedrops for her dry eyes, some topical steroid cream for her rash, and oral prednisone at 0.5mg/kg daily as initial first-line therapy for chronic graft-versus-host disease.

After 4 weeks of treatment, the symptoms of her ocular chronic graft-versus-host disease had pretty much resolved, but the patient's skin rash, as well as muscle and joint pains, had worsened. The steroid dose was increased to 1mg/kg daily of oral prednisone. Despite this increase, the skin rash and the muscle and joint pains continued to worsen. At this point, a diagnosis of steroid-refractory chronic graft-versus-host disease was made, and the patient was started on ruxolitinib 10mg orally, twice daily, along with her 1mg/kg of oral prednisone.


This case is fairly representative of many cases that I see in my clinic, and I'm sure my colleagues too, for those of us who follow patients longitudinally after allergenic transplantation.

Transcript edited for clarity.

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