Extracorporeal photopheresis in children with graft‐versus‐host disease

Abstract

Acute and chronic graft-versus-host disease (GVHD) are major complications after allogeneic hematopoietic stem cell transplantation. Systemic corticosteroid is the first line of therapy but only half of the patients will respond. The management of steroid-refractory or steroid-dependent GVHD is challenging. Intensification of immunosuppression has been the main strategy but the response rate is not satisfactory. Furthermore, the incidence of treatment-related toxicity and opportunistic infection is unacceptably high. Extracorporel photopheresis (ECP) has been used in the management of refractory GVHD. Retrospective analysis of the experience in adult patients showed activity in both acute and chronic GVHD. The procedure was well tolerated with minimal changes in the hematologic and biochemical parameters. However the machine currently approved is designed for patients over 40 kg of body weight. Significant fluid shift and venous access are major concerns when ECP is performed in children. Various modifications of the ECP procedure have been tried to manage patients with low body weight. Experience with ECP in children is limited but preliminary data also showed favorable response in children with resistant GVHD. Further investigations are needed to refine the optimal schedule, duration, and treatment technique for pediatric patients.

Analysis of Clinical Outcome and Survival in Pediatric Patients Undergoing Extracorporeal Photopheresis for the Treatment of Steroid-refractory GVHD

“…This is in accordance to earlier published reports by others. [5][6][7][8][9][10][11][12] Most patients had not had adverse effects. Fungal infections developed in patients who were on treatment with high-dose steroids, probably the main cause of them.…”

“…6 However, there are few published studies analyzing clinical outcome in pediatric patients undergoing ECP as steroidrefractory GVHD treatment. [7][8][9][10][11][12] This long-term follow-up study was designed to analyze clinical outcome in a group of pediatric patients who underwent ECP for treatment of steroid-refractory acute and chronic GVHD.…”

Extracorporeal photochemotherapy in graft‐versus‐host disease: a longitudinal study on factors influencing the response and survival in pediatric patients

“…Very recently, it has been reported that the proportion of immature CD19+CD21-B lymphocytes may be correlated with ECP response in cGVHD even though this is a report on a small number of patients. 13 In the pediatric setting, many authors report a response rate to ECP in steroid-resistant aGVHD ranging from 50% to 100% (depending on the organs involved) 3,[14][15][16] and in cGVHD from 33% to 93%. 10,16 In aGVHD steroid-resistant patients, the probability of survival at 4 years is significantly increased in complete responders to ECP (59%), while it decreases dramatically to 11% for nonresponders.…”

“…13 In the pediatric setting, many authors report a response rate to ECP in steroid-resistant aGVHD ranging from 50% to 100% (depending on the organs involved) 3,[14][15][16] and in cGVHD from 33% to 93%. 10,16 In aGVHD steroid-resistant patients, the probability of survival at 4 years is significantly increased in complete responders to ECP (59%), while it decreases dramatically to 11% for nonresponders. 16 In cGVHD refractory patients, a significantly higher 5-year overall survival rate was observed in ECP responders compared to nonresponders (96% vs. 58%).…”

Acute graft-versus-host disease in children

“…Another approach is to put the mononuclear cell suspension in the UVAR XTS machine for processing (both two-step procedures). 32 Our group has been able to use the standard procedure with the UVAR Table 3 Approaches to post-HSCT GVHD prophylaxis and treatment in children XTS in children down to 20 kg. We follow an algorithm demanding a specific hematocrit before ECP, depending on the child's weight, and normal saline is given pre-ECP depending on expected extracorporeal volume.…”