Photopheresis in pediatric graft-versus-host disease after allogeneic marrow transplantation: clinical practice guidelines based on field experience and review of the literature

Abstract

Background: Extracorporeal photochemotherapy (ECP) gives positive results in the management of graft-versus-host disease (GVHD), but in children, specific difficulties can outweigh this benefit. These difficulties must be taken into consideration when establishing a standardized reproducible procedure for implementation under a quality management plan.

Study design and methods: Twenty-seven children underwent ECP for severe acute GVHD (aGVHD) or chronic GVHD (cGVHD) after allogeneic marrow transplantation. Data were collected prospectively, with particular emphasis placed on technical, biologic, immunologic, clinical, and long-term follow-up issues.

Results: The 27 children underwent a total of 750 sessions. Mononuclear cells were collected on a commercially available apheresis system (COBE Spectra, Gambro BCT). Overall survival was 73 percent, and ECP led to significant improvement in 21 of the 27 patients (11 with complete response and 10 with partial response, i.e., >50% of organ involvement). Tolerance was good overall, the main limiting factors being vascular access and the psychological impact of repeated apheresis procedures. Children weighing less than 25 kg were not more susceptible to side effects.

Conclusion: A specifically pediatric-dedicated and -experienced team faces only limited difficulties when treating children with GVHD by ECP. Overall, ECP is efficient and well tolerated. Our experience was therefore pooled together with available pediatric data to establish clinical practice guidelines. These guidelines consider ECP as a first-line therapy in Grade IV aGVHD (in association with conventional pharmacologic approaches) and limited cGVHD and as a second-line therapy in steroid-resistant Grades II to III aGVHD and extensive cGVHD.