Graft vs host disease (GVHD) is the most severe complication of allogeneic hematopoietic cell transplantation. Conventional immunosuppressive therapy increases morbidity and mortality without improving survival. Extracorporeal photopheresis (ECP) has been introduced as an alternative treatment in steroid‐dependent and steroid‐refractory disease.

We studied the safety and efficacy of ECP as a second‐ or third‐line treatment in GVHD.

ECP was administered in 21 patients with grade III‐IV acute GVHD and 88 patients with extensive chronic GVHD, without ECP‐related adverse events. Eight patients receiving four or less ECP sessions were not further analyzed. The majority of acute GVHD patients (84%) presented partial (15) or complete (1) response to ECP. Immunosuppression was reduced in 10 of 19 patients and ceased in 1 of 19 patients. One‐year cumulative incidence (CI) of transplant‐related mortality (TRM) (17.6%) was associated with the lack of response to ECP and steroid refractoriness. With a follow‐up of 17.5 (1.8‐58.3) months, 1‐year overall survival (OS) (52.5%) was independently associated with a higher number of ECP sessions. Regarding chronic GVHD, complete response was achieved in 35 patients, whereas partial response in 25 patients, leading to an overall response rate of 73%. Cutaneous sclerosis manifestations were associated with higher response rates. With a follow‐up of 68.1 (5.4‐283.1) months, 5‐year CI of TRM (24.1%) was associated only with a number of ECP sessions. The 5‐year OS (64.5%) was independently associated with number of ECP sessions and cutaneous manifestations.

Our findings suggest that ECP is safe and effective for GVHD and should be considered early in the course of GVHD, before irreversible end‐organ damage has been established.