Background: Etanercept is a recombinant human soluble tumor necrosis factor-alpha (TNF-Ձ) receptor fusion protein that inhibits TNF-Ձ, a major mediator in the pathogenesis of graft-versus-host disease (GVHD). The purpose of our study was to evaluate the safety and efficacy of etanercept therapy in children with steroid-refractory acute GVHD (aGVHD) (n=5) and chronic GVHD (cGVHD) (n=3).
Methods: Five males and 3 females were enrolled and their median age was 14.4 years (range, 2.1∼18.8). Etanercept 0.4 mg/kg per dose (maximum dose, 25 mg) was given subcutaneously twice weekly for 4 weeks followed by 0.4 mg/kg per dose (maximum dose, 25 mg) weekly for 4 weeks. At the time of initiation of etanercept, 5 patients had aGVHD grade III to IV (III=4, IV=1) and 3 patients had moderate to severe cGVHD (moderate=1, severe=2).
Results: Overall, 6 of 8 patients (75%) responded to the treatment with etanercept, including 5 patients with aGVHD [n=3 complete response (CR), n=2 partial response (PR)] and 1 patient with cGVHD [n=1 PR, n=2 no response (NR)]. Clinical responses were most commonly seen in patients with refractory gut aGVHD. CMV reactivation occurred in 2 patients, bacterial infection in 1 patient, and fungal infection in 1 patient.
Conclusion: Our preliminary data indicate that etanercept is well tolerated and can induce a high response rate in patients with steroid refractory aGVHD, particularly in the setting of intestinal involvement. (Korean J Hematol 2009;44:212-219.)

Keywords: Hematopoietic stem cell transplantation, Graft-versus-host disease, Etanercept