Extracorporeal Photopheresis Shows Promising Efficacy in Steroid-Refractory cGvHD
Extracorporeal photopheresis (ECP) is a safe and effective second-line treatment option for patients with steroid-refractory chronic graft-versus-host disease (SR-cGvHD), with sustained survival benefits and clinically meaningful response rates, according to study results published in Transplantation and Cellular Therapy.
“The objective of this [systematic literature review/meta-analysis] was to evaluate evidence on the efficacy and safety of ECP for the treatment of cGvHD, by synthesizing reported estimates to quantify clinically relevant endpoints for ECP,” explained Zachariah DeFilipp, Massachusetts General Hospital in Boston, Massachusetts, and coauthors.
The researchers evaluated 47 studies involving 2361 patients with SR-cGvHD treated with ECP, an immunomodulatory therapy that alters cytokine production and boosts T regulatory cell function without direct immunosuppression. Most studies focused on inline ECP systems (eg, CELLEX or UVAR-XTS), and nearly 40% of them employed standardized NIH criteria to define cGvHD and measure treatment outcomes.
Among the 24 studies included in the quantitative meta-analyses, 12-month overall survival (OS) was 84% (95% CI, 77.3–88.9; 14 studies, 704 patients), while 5-year OS was 58% (95% CI, 35.5–77.6; 8 studies, 431 patients). Failure-free survival (FFS) at 12 months was 60.8% (95% CI, 38.9–79.0; 4 studies, 169 patients). Overall response rate (ORR) improved with time: pooled ORR was 45.3% at 3–4 months and 58.2% at 6–8 months, though study-level responses varied widely. Subgroup analysis showed no significant difference between studies using NIH vs non-NIH assessment criteria.
Skin-specific responses, one of the most frequently reported organ outcomes, were 34.9% at 2–3 months and 54.2% at 4–6 months. Liver and lung responses, though reported, varied too greatly across studies for pooled analysis. Safety reporting was limited and inconsistent; infection rates were documented in only nine studies, and quality-of-life data were sparse.
“This systematic review and [meta-analysis], conducted according to PRISMA guidelines, indicated that ECP results in favorable outcomes, both long- and short- term, in cGvHD, including OS, FFS and ORR,” concluded the study authors.
Reference
DeFilipp Z, Fox L, Holderried TAW, et al. Systematic review and meta-analysis of extracorporeal photopheresis for the treatment of steroid-refractory chronic graft-versus-host disease. Transplant Cell Ther. 2025;31(2):76.e1-76.e13. doi:10.1016/j.jtct.2024.11.004
