Tacrolimus ointment in the treatment of chronic cutaneous graft-vs-host disease - A case series of 18 patients

Background: Tacrolimus (formerly FK 506) is an immunosuppressive drug that works by inhibiting calcineurin, a calcium-dependent protein phosphatase re quired for immune function. Tacrolimus has been shown to be effective topic ally in atopic dermatitis and systemically in psoriasis and graft-vs-host d isease (GVHD). However, its efficacy in treating cutaneous GVHD when applie d topically has not been reported. Objective: To assess the therapeutic efficacy of 0.1% tacrolimus ointment o n chronic cutaneous GVHD in patients with symptoms refractory to systemic c orticosteroid therapy. Results: Tacrolimus ointment effectively treated pruritus and/or erythema i n 13 (72%) of 18 patients with chronic GVHD. Responding patients had a rapi d effect within several hours to days. Effectiveness was measured by means of patient report, results of physical examination, side-by-side comparison s of tacrolimus vs a vehicle control, and temporal flares of the cutaneous symptoms of the disease in the context of stopping tacrolimus ointment ther apy. Because of the progression of GVHD and in 2 cases, loss of drug effica cy, all patients eventually went on to receive more aggressive treatment, i ncluding increases in steroid dosage, psoralen-UV-A therapy, and extracorpo real photopheresis. Conclusions: This case series suggests that tacrolimus ointment has efficac y in treating the erythema and pruritus of steroid-refractory, chronic cuta neous GVHD in most patients, The rapid response of tacrolimus ointment may provide a useful therapeutic bridge to systemic therapies that have slower onset, such as psoralen-UV-A therapy or extracorporeal photopheresis.