Introduction: Graft-versus-host disease (GVHD) remains the most common cause of morbidity and transplant-related mortality after allogeneic hematopoietic stem cell transplantation, despite significant advances in prophylaxis and treatment. Extracorporeal photopheresis (ECP) is an immunomodulatory therapy for which cutaneous T cell lymphoma is currently the only FDA-approved indication; it is, however, increasingly used as a second-line therapy for acute and chronic GVHD patients who are steroid-refractory or -dependent. Evidence suggests overall response rates of 60-80%, with better response rates among patients with cutaneous manifestations. This retrospective analysis reports our experience using ECP to treat adults with acute and chronic GVHD over a 5-year period.

Methods: We identified 67 patients (age 21-69, 70% males) who underwent ECP therapy for GVHD between November 1, 2010 and June 2, 2016. Eleven patients were excluded based on exclusion criteria of fewer than 8 procedures, any part of ECP course performed at an outside institution, and inadequate documentation regarding GVHD response. Among the remaining 56 patients, 6 underwent more than one course of ECP, resulting in a total of 62 courses of ECP included in final analysis. Charts were reviewed for each of these 62 courses to obtain relevant demographic and transplant-related data, type of GVHD, and organs involved. Response was assessed separately for each involved organ using strict response criteria and was censored on the date of last ECP procedure. Hematology notes were reviewed for noted worsening or improvement of GVHD, and a response was counted only if it was maintained throughout the remainder of the ECP course, including the date of last procedure.

Results: Results are summarized in Table 1. Tapering of immunosuppressive therapy by end of ECP course, defined as steroid tapering by at least 50% from baseline or steroid withdrawal and tapering of other immunosuppressive therapy, was successful in 43 out of 62 (69%) ECP courses. Overall response rate, defined as complete or partial response, was highest for skin GVHD (73%) and lowest for lung GVHD (27%). However, 6 out of 11 (54%) patients with lung involvement experienced no progression of disease, which may be considered a successful response in these difficult-to-manage patients. Twenty-two out of 62 courses did have a new immunosuppressive agent, such as mycophenylate mofetil, tacrolimus, or rituximab, introduced at the start of or during the ECP course, and 11 of these 22 courses achieved a response that may not reflect therapeutic effect of ECP alone. Four patients developed new progressive liver or lung involvement by GVHD even as other involved organs in these patients, such as skin, showed complete response.

Conclusion: Response rates to ECP at our institution are similar to those reported in the literature; it is a useful therapeutic option in patients with acute or chronic GVHD, particularly those with skin involvement. ECP allows successful tapering or withdrawal of immunosuppressive therapy in the majority of patients, even in those who are steroid-refractory or steroid-dependent.


Sarode:CSL Behring: Consultancy, Honoraria.

Author notes


Asterisk with author names denotes non-ASH members.