Abstract
Introduction Acute graft-versus-host disease (aGVHD) is major cause of morbidity and mortality following hematopoietic cell transplantation (HCT). Despite advances in the understanding the pathophysiology of aGVHD and new immunosuppressive agents, steroids remain the first line of treatment. Second-line treatment for steroid-refractory patients is still controversial. Extracorporeal photopheresis (ECP) has been associated with an overall response of 70-80% in adult patients with steroid-refractory aGVHD, yet data on the efficacy of this therapy is scarce in children. Method Retrospective case series of pediatric patients with steroid-refractory aGVHD who were treated with ECP at Sirio-Libanes Hospital from Jul'15 to Feb'19. Results Ten patients were included. Median age was 8.5 years (0.4-19), and six were male. The underlying diseases were: acute myeloid leukemia (2 patients), acute lymphocytic leukemia (5), Blackfan-Diamond anemia (1), myelodysplastic syndrome (1), and severe combined immunodeficiency (1). Median follow-up time was 382 days (113-1561). Myeloablative conditioning regimen was performed in 9 patients, and reduced intensity conditioning in 1 patient. There were 3 haploidentical, 2 10/10-HLA-matched, 3 9/10-HLA-mismatched and 2 HLA-mismatched cord blood donor HCTs. Stem cell source was peripheral blood in 3 patients, umbilical cord in 2, and bone marrow in 5 patients. All patients engrafted, and the median number of neutrophil recovery was 15.5 days (13-27). Maximum grades of aGVHD were: grade II (2 patients), III (2), and IV (6). Pre-ECP aGVHD therapy beyond steroids and calcineurin inhibitors was: etanercept (8 patients), MMF (8), rituximab (2), ruxolitinib (6) and ATG (4), MTX(1), Sirolimus(2), mesenchymal stem cell(4), basiliximab(3) (patients may have received more than one). ECP was used as third line in 2 patients, and as fourth line or beyond in the remaining cases. Concomitant immunosuppressive agents at the time of ECP initiation were continued. The median time of ECP initiation was D+30 (22-82). The ECP equipment used was Therakos XT in 8 patients and Therakos Cellex in 2 patients. The initial ECP schedule consisted of 2 sessions/week over 4 weeks, followed by 1 session/week over 3-6 weeks, and then tapered down to biweekly and monthly according to clinical improvement. ECP was withdrawn only after complete clinical improvement or death. Four deaths occurred, three associated with GVHD progression and/or infection, and one due to disease relapse. Four patients attained six patients attained complete response. Conclusion In this small case series, ECP led to complete remission of steroid-refractory aGVHD in 6/10 patients. Although clinical improvement may have also been related to concomitant immunosuppressive therapy, ECP seems to be safe and efficacious for steroid aGVHD in pediatric patients and should possibly be used as second-line.
Published Version
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