Extracorporeal Photopheresis in Children with Chronic Graft-Versus-Host Disease

Area of Science:

• Hematology
• Immunology
• Pediatric Oncology

Background:

• Chronic graft-versus-host disease (cGVHD) is a significant complication following allogeneic hematopoietic stem cell transplantation (allo-HSCT).
• cGVHD severely impacts patient survival and quality of life, particularly in pediatric populations.
• Steroid-refractory cGVHD presents a therapeutic challenge, necessitating alternative treatment strategies.

Purpose of the Study:

• To evaluate the efficacy and safety of extracorporeal photopheresis (ECP) in children diagnosed with steroid-refractory cGVHD.
• To assess treatment response rates across various organs affected by cGVHD.
• To determine the long-term survival outcomes and non-relapse mortality in pediatric patients receiving ECP.

Main Methods:

• Retrospective analysis of 42 pediatric patients with steroid-refractory cGVHD who underwent ECP therapy.
• ECP administered as second or third-line treatment, with a bimonthly schedule initially.
• Concurrent therapies were allowed if deemed necessary; treatment response was monitored by organ involvement.

Main Results:

• An overall response rate of 74% (complete response: 17%, partial response: 57%) was observed.
• High response rates were noted in skin (75%), mucous membranes (73%), liver (80%), and gut (80%).
• Five-year survival rates were 57% overall, 56% progression-free, and 30% failure-free, with 14% non-relapse mortality.

Conclusions:

• Extracorporeal photopheresis (ECP) is an important and safe therapeutic option for pediatric patients with cGVHD.
• ECP offers significant clinical benefits and favorable survival outcomes in steroid-refractory cGVHD.
• The procedure was well-tolerated, with no clinically significant complications attributed to ECP.