Effect of ruxolitinib on the oral mucosa of patients with steroid-refractory chronic Graft-versus-Host disease and oral involvement

Abstract

Background: Chronic Graft-versus-Host Disease (cGVHD) can impact quality of life, especially in patients with oral involvement. Half of the patients with cGVHD do not respond to first-line therapy with corticosteroids and calcineurin inhibitors. Ruxolitinib is effective in steroid-refractory (SR)-cGVHD cases, but the long-term effects of ruxolitinib on the oral mucosa are unknown.

Objective(s): This study aims to assess the effect of ruxolitinib on the oral mucosa of SR-cGVHD patients with oral involvement.

Materials and methods: An observational longitudinal patient study was conducted in 53 patients with SR-cGVHD and oral involvement who were treated with ruxolitinib. The baseline condition of the oral mucosa was compared to its condition at 4 and 12 weeks after starting ruxolitinib.

Results: The overall response was 81% (43/53), with a complete response in 53% (28/53) and partial response in 28% (15/53) after 12 weeks (p < 0.001). Men and patients concurrently using immunosuppressive therapy responded better than women (p = 0.005) and patients with ruxolitinib monotherapy (p = 0.02), respectively. At a longer follow-up (median 20 months), oral symptoms were comparable to the 12-week symptoms (p = 0.78), regardless of ruxolitinib use (p = 0.83).

Conclusion: Ruxolitinib treatment of SR-cGVHD patients with oral involvement was associated with a significant response of the oral manifestations at 12 weeks.

Clinical relevance: The oral mucosa of SR-cGVHD patients is likely to improve after 4 and 12 weeks of ruxolitinib treatment. Symptom severity at baseline does not affect the response of the oral mucosa.

Keywords: Graft vs host disease; INCB018424; Immunosuppression; Mouth mucosa.

Fig. 1

Flowchart showing the patient selection

Fig. 2

Presentation of the score distribution on the NIH Mouth Staging Score at baseline (N = 53), at 4 weeks (N = 51), at 12 weeks (N = 49) and at follow-up (N = 48). Scores range from 0 to 3, representing no symptoms (score 0), mild symptoms without limitation of oral intake (score 1), moderate symptoms with partial limitation of oral intake (score 2) and severe symptoms with limitation of oral intake (score 3). Error bars represent the standard error of mean

Fig. 3

Score distribution of the NIH Mouth Staging Score of patients using ruxolitinib at follow-up (N = 29) and patients not using ruxolitinib at follow-up (N = 19). Scores range from 0 to 3, representing no symptoms (score 0), mild symptoms without limitation of oral intake (score 1), moderate symptoms with partial limitation of oral intake (score 2) and severe symptoms with limitation of oral intake (score 3). Error bars represent the standard error of mean