Extracorporeal Photopheresis for Chronic Graft-Versus-Host Disease According to 2014 NIH Consensus Criteria

Chronic graft-versus-host disease (cGVHD) remains a major cause of morbidity and mortality following allogeneic hematopoietic stem cell transplantation. Many cGVHD patients require prolonged systemic immunosuppression with corticosteroids, which carry significant adverse effects, and alternative therapies are often required. Extracorporeal photopheresis (ECP) has historically shown promise as a cGVHD treatment, but recent efficacy data have been limited and inconsistent. To evaluate the efficacy of ECP as a treatment for cGVHD patients according to the 2014 NIH Consensus Criteria. We retrospectively analyzed 53 patients treated with ECP for cGVHD at our center from 2010 to 2024. ORR was 51%, with 3 CRs (5.7%) and 24 PRs (45.3%). Highest organ-specific responses were seen in the gastrointestinal tract (70%) and liver (69.2%), and lowest in joints/fascia (15.4%) and lung (21.4%). A total of 23 (43.4%) patients experienced at least one treatment-related complication (TRC), with a median time to first TRC of 29.3 weeks (IQR = 7.4-55.2). The estimated 1- and 3-year OS was 79.2% (95% CI = 65.7%-87.9%) and 70.1% (95% CI = 55.0%-80.9%), respectively. The estimated 1- and 3-year FFS was 75.5% (95% CI = 61.5%-84.9%) and 43.1% (95% CI = 28.6%-56.9%), respectively. Of the 37 patients who were on corticosteroids at the start of ECP, 22 (59.5%) were able to decrease their daily dose by ≥ 50% by the time of ECP discontinuation. Multivariable analyses revealed that patients on tacrolimus and other non-steroid immunosuppressive treatments (ISTs) had better outcomes in clinical benefit, steroid-sparing, overall survival, and failure-free survival. ECP was a safe and effective treatment for cGVHD in our population and showed promising efficacy when used in combination with tacrolimus or other ISTs.