Etanercept for steroid-refractory acute graft-versus-host disease: A single center experience

De Jong, Cornelis N.; Saes, Lotte; Klerk, Clara P. W.; Van der Klift, Marjolein; Cornelissen, Jan J.; Broers, Annoek E. C.

We performed a retrospective analysis in a small cohort of patients treated with etanercept for grade III SR-aGVHD of the gut. Second-line treatment with etanercept resulted in an encouraging overall response rate of 53%. Nevertheless, eventually all treated patients died, most importantly due to progression of GVHD and opportunistic infections. Our results compare well to a recent prospective study published by Van Groningen et al. concerning 21 patients with SR-aGVHD treated with a combination of etanercept and inolimomab, an IL-2 receptor antibody [13] . This cohort was strikingly similar to ours with respect to age, aGvHD severity and proportion of patients with gut involvement. Despite an ORR of 48%, OS was only 10% at a median of 55 days, due to progressive GVHD, serious infections and relapse of underlying disease. The pattern of a promising initial response in about 50% of patients, followed by non-relapse mortality (NRM) in patients due to either progressive GVHD or the occurrence of serious infections, in particular, compares well to our findings. Wolff et al. prospectively studied the use of etanercept (16mg/m 2 ) in combination with dacluzimab (1mg/kg), an IL-2 receptor antibody, in 21 patients with SR-aGVHD (9). This cohort was younger with a median age of 44 years, and included fewer patients with high-stage involvement of the gut. Although a promising ORR of 67% was observed, survival was disappointing as only 4 out of 21 patients survived.