A new study in the journal Pharmaceuticals (Basel) suggests that extracorporeal photopheresis (ECP) is a safe treatment approach for children who have steroid-refractory chronic graft vs host disease (cGVHD), while enabling high response rates but with further immunosuppressive treatment required for some patients.
The study was a retrospective analysis of pediatric patients with steroid-refractory cGVHD treated at the RM Gorbacheva Research Institute at I. Pavlov University in St. Petersburg, Russian Federation. Patients were given ECP in second or third lines of therapy. It was administered every 2 months for 2 days at a time or longer depending on response, and with the option to continue other treatments.
A total of 42 patients were evaluated in this analysis, of whom 76% had acute leukemia. ECP was the second line of therapy for 43% of patients, and it was the third line of therapy for 57% of patients. The rate of complete response was 17%, and 57% of patients had a partial response. Overall responses rates specific to organs included 75% for the skin, 73% for mucous membranes, 80% in the liver, 80% in the gut, 67% in the joints, and 22% in the lungs.
The 5-year nonrelapse mortality rate was 14% (95% CI, 5-28). The median follow-up duration was 774 days, at which the 5-year overall survival was 57% (95% CI, 39-72). The 5-year progression-free survival rate was 56% (95% CI, 37-72), and the 5-year failure-free survival rate was 30% (95% CI, 16-46). Insufficient clinical effect was reported in 43% of patients who needed additional immunosuppressive therapy following ECP.
The authors of the study indicated that clinically significant complications attributed to ECP were not observed. Most common adverse effects were reported to be catheter associated, in 5 cases, and these did not require discontinuation of ECP.
The study authors consider ECP to remain an important approach for pediatric patients with steroid-refractory cGVHD, owing to safety and high response rates. The authors noted the failure-free survival rate is suboptimal with this treatment, with many patients also requiring immunosuppressive therapy. “This means that ECP should not be regarded as a game changer but as an important approach that can help to relieve symptoms in most of the patients at least transiently,” the authors wrote in their report.
Kozlov A, Estrina M, Paina O, et al. Extracorporeal photopheresis in children with chronic graft-versus-host disease. Pharmaceuticals (Basel). 2021;14(8):808. doi:10.3390/ph14080808