Extracorporeal photochemotherapy in the treatment of severe steroid-refractory acute graft-versus-host disease: a pilot study

Extracorporeal exposure of peripheral blood mononuclear cells to the photos ensitizing agent 8-methoxypsoralen and UV-A radiation has been shown to be effective in the treatment of selected diseases mediated by T cells, reject ion after solid organ transplantation, and chronic graft-versus-host diseas e (GVHD). We present 21 patients with a median age of 38 years who develope d steroid-refractory acute GVHD grades II to IV after stem cell grafting fr om sibling or unrelated donors and were referred to extracorporeal photoche motherapy (ECP). Three months after initiation of ECP 60% of patients achie ved a complete resolution of GVHD manifestations. Complete responses were o btained in 100% of patients with grade II, 67% of patients with grade III, and 12% of patients with grade IV acute GVHD. Three months after start of E CP complete responses were achieved in 60% of patients with cutaneous, 67% with liver, and none with gut involvement. Adverse events observed during E CP included a decrease in peripheral blood cell counts in the early phase a fter stem cell transplantation (SCT), Currently, 57% of patients are alive at a median observation time of 25 months after SCT. Probability of surviva l at 4 years after SCT is 91% in patients with complete response to ECP com pared to 11% in patients not responding completely. Our findings suggest th at ECP is an effective adjunct therapy for acute steroid-refractory GVHD wi th cutaneous and liver involvement. However, in patients with acute GVHD gr ade IV or gut involvement other therapeutic options are warranted. (Blood. 2000;96:2426-2431) (C) 2000 by The American Society of Hematology.