Extracorporeal photopheresis (ECP) has become an oft-used therapy for the treatment of acute and chronic graft versus host disease (aGVHD and cGVHD, respectively) after hematopoietic stem cell transplantation (HSCT). ECP is not inherently immunosuppressive and has few side effects, making it a tempting therapeutic option for the treatment of steroid refractory GVHD; issues of vascular access present practical obstacles to its use in children and young adults. We describe our experience using ECP in children after HSCT at our center. We performed 20 courses of ECP in 17 children (age 1.6 – 18 yrs) between January 2010 and December 2012. ECP restarted after a hiatus of at least 4 weeks is counted as a new course. Primary diagnoses of our patients (pts) were malignant disease in 13 pts and non-malignant disease in four. Nine courses of ECP were performed for aGVHD, 6 for cGVHD , and 5 for pts with overlap syndrome (OvS). Most pts had ECP initiated as second-line therapy; in 2 cases ECP was initiated as first-line therapy due to underlying infectious complications where steroid-avoidance was imperative. Pts underwent a median of 9 treatments (range 2-90). Vascular access for ECP included Permacath ® in 9 courses, Hickman® catheter in 2 courses, temporary arterial line in 2 courses, temporary peripheral (venous) lines in 1 course, and a combination of the above in 6 courses. ECP was discontinued in 1 pt due to issues of vascular access.
We have observed 10 (50%) complete responses (CR), 3 (15%) partial responses (PR), and 6 (30%) mixed responses (MR). 14 pts (70%) could be weaned from their initial steroid dose. Among pts with aGVHD, CR occurred after 6/9 (67%) courses and steroid dose could be tapered in 5 cases. In pts with cGVHD, CR was seen in only one pt, with PR and MR in 3 and 2 pts, respectively. Steroids could be tapered in 5 (83%) pts. Among 5 pts with OvS, 3 achieved CR, 1 had an MR and one pt was resistant. Steroids were tapered in 4 pts. There were 10 deaths in the cohort, 3 from GVHD . In addition, 1 pt with CR in all 3 initially involved organs developed progressive bronchiolitis obliterans and died of respiratory failure, and 1 pt with severe steroid myopathy died of culture-negative sepsis. Three pts died of sepsis, one of fulminant hepatic failure (aspergillus niger and Penicillium species) and 1 of disease relapse.
Our results support the use of ECP as a feasible, effective treatment for GVHD in children after HSCT.
Full conference title:
- EBMT 39th (2013)