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Graft-vs.-Host Disease Resource Center


Treatment of Ocular and Skin GVHD Following Allogeneic Stem Cell Transplant

Last Updated: Wednesday, May 17, 2023


A 56-year-old African American female patient with a history of myelodysplastic syndrome (MDS) transformed to acute myeloid leukemia (AML). She underwent haploidentical allogeneic stem cell transplant (allo-HSCT) in first remission using her daughter as the donor. Both the patient and donor were cytomegalovirus (CMV) IgG negative and had O positive blood types.

The conditioning regimen included total-body irradiation (TBI) of 200 cGy, fludarabine, and melphalan with anti-thymocyte globulin (ATG) for GVHD prophylaxis. Unfortunately, she developed acute GVHD of the skin approximately 30 days post-transplant and required a course of prednisone which was successfully tapered over 3 months, at approximately 120 days after her allo-HSCT. During this period, the patient intermittently required topical triamcinolone cream for discoid hyperpigmented lesions on the skin.

The patient also developed ocular symptoms with conjunctival erythema and dryness requiring more than three administrations of topical eye drops (preservative-free and moisturizing) per day. She also developed severe itching and photophobia of the eyes.


The patient was referred to the ophthalmology care team. Their exam confirmed suspected diagnosis of chronic ocular GvHD, with keratoconjunctivitis sicca. They established care and helped to co-manage the patient.


The patient was given cyclosporine ophthalmic eye drops twice daily; symptoms waxed and waned but did not improve overall. She was also prescribed loteprednol etabonate ophthalmic suspension 0.5% by the ophthalmologist. Symptoms temporarily improved but returned after each course. Low-dose steroids were also administered multiple times but attempts at discontinuing them resulted in repeated flares of not only dry, erythematous eyes and photophobia, but also itching and hyperpigmentation of the upper and lower eyelids.

The patient was started on 10 mg of ruxolitinib twice a day due to steroid-dependent chronic GVHD.

Labs were monitored throughout the patient’s course. Baseline CBC prior to the start of ruxolitinib was as follows:

  • WBC count 7.1 x 109/L
  • Hemoglobin 11.4 g/dL
  • Platelets 184 x109/L
  • Absolute neutrophil count (ANC) 4.1 x 109/L

After approximately 1 month on ruxolitinib, the patient’s symptoms had improved significantly, and she was able to discontinue topical therapies and eye drops. Ophthalmologic exam confirmed improvement in keratoconjunctivitis sicca. In addition, the skin flares also reduced to nearly resolved.

Repeat labs 2 months into treatment with ruxolitinib were as follows:

  • WBC count 8.9 x 109/L
  • Hemoglobin 9.4 g/dL
  • Platelets 239 x109/L
  • ANC 5.1 x 109/L

The patient’s ruxolitinib dose was reduced to 5 mg twice a day after another month due to the development of anemia. This helped to stabilize her blood counts, without a flare in symptoms.


Ocular GvHD is fairly common in patients who undergo allo-HSCT. It affects 10% of individuals with acute GvHD and over 50% of patients with chronic GVHD.1

For both acute and chronic GVHD after allo-HSCT, steroids are the most common first-line therapy, but they are associated with serious adverse events.2

To assess ocular GVHD, clinicians use the National Institutes of Health (NIH) scoring system for chronic GVHD. In the past, an invasive, uncomfortable examination in the outpatient setting (Schirmer’s test) was part of the scoring, but now the NIH Consensus scoring rather includes symptoms and eye drop requirements.

For ocular symptoms, the scoring is as follows:

  • 0: No dry eye symptoms
  • 1: Mild dry eye symptoms not affecting activities of daily living (ADL) with 3 or less eye lubricant drops required per day
  • 2: Moderate dry eye symptoms partially affecting ADL, requiring lubricant eye drops >3x per day or punctal plugs, without new vision impairment due to keratoconjunctivitis sicca (KCS)
  • 3: Severe dry eye symptoms significantly affecting ADL, such as special eyewear to relieve pain, or unable to work due to ocular symptoms, or loss of vision due to KCS

The patient was considered for systemic therapy due to her score 2 GVHD of the eyes plus intermittent skin involvement requiring ongoing topical therapy but with worsening ocular symptoms. The addition of ruxolitinib improved the patient’s score from 2 to 1. Ibrutinib was also considered second-line systemic treatment option due to U.S. Food and Drug Administration (FDA) approval, but the clinician opted against this due to the patient’s poorly controlled hypertension, which is a common adverse reaction. Belumosidil can be considered if ruxolitinib fails as a third-line option.

Additional management options include autologous serum eye drops made from the patient’s blood or insertion of punctal plugs to block the tears from leaving the ocular area. In addition, scleral lenses that help to block the ocular surface from air may be considered, although they require specialist fitting and follow-up. Additional systemic options include other immunosuppressive therapies for GVHD.



  1. Kezic JM, Wiffen S, Degli-Esposti M. Keeping an 'eye' on ocular GVHD. Clin Exp Optom. 2022 Mar;105(2):135-142. doi: 10.1080/08164622.2021.1971047. Epub 2021 Sep 19.
  2. Bell EJ, Yu J, Bhatt V, et al. Healthcare resource utilization and costs of steroid-associated complications in patients with graft-versus-host disease. Transplant Cell Ther. 2022 Apr 26:S2666-6367(22)01231-3. doi: 10.1016/j.jtct.2022.04.014. Epub ahead of print.


Test your knowledge on treatment of oclular and skin GvHD after allogenic stem cell transplant

Last Updated: Wednesday, May 17, 2023
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