title: Treatment of Acute Graft-versus-Host Disease in Childhood with Extracorporeal Photochemotherapy/Photopheresis: The Padova Experience.
abstract: Acute graft-versus-host disease (aGVHD) is the major cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation. Systemic steroid treatment represents the first-line therapy for aGVHD and is associated with a response rate of 30% to 60%. Steroid-resistant patients have a poor prognosis with high transplantation-related mortality (TRM). Several second-line therapies have been proposed for the management of unresponsive aGVHD, without proven beneficial effects on patients' outcome or overall long-term survival. For these reasons, extracorporeal photochemotherapy/photopheresis (ECP), a cell-based approach to control GVHD that spares generalized immunosuppression, seems to be promising. In this study, we report the outcome of 72 consecutive pediatric patients treated with ECP between 1997 and 2013 for aGVHD. Among them, 21 patients had steroid-resistant aGVHD, 42 had steroid-dependent aGVHD, and 9 did not receive steroid as first-line therapy because of clinical contraindications. A complete response was obtained in 72% of patients, a partial response was observed in 11%, and there was no response in 17% of patients. At day +180, TRM was 4% in the whole cohort; TRM was 3% and 20% among responders and nonresponders to ECP, respectively (P < .0001). The 5-year overall survival was 71%, showing a difference between responders and nonresponders of 78% and 30%, respectively (P = .0004). The 5-year time to progression of primary disease was 81%, without any significant difference between the 2 groups. Moreover, the 5-year progression-free survival of primary disease was 72%, with a significant difference (P = .0007) between responders (79%) and nonresponders (30%) to ECP. In conclusion, this study demonstrates that ECP is highly effective in aGVHD without a negative impact on primary disease.
authors.affiliation: Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy. Electronic address: elisabetta.calore@unipd.it.
authors.forename: Elisabetta
authors.initials: E
authors.lastname: Calore
authors.affiliation: Department of Transfusion Medicine, Therapeutic Apheresis Unit, University Hospital of Padova, Italy.
authors.forename: Piero
authors.initials: P
authors.lastname: Marson
authors.affiliation: Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy.
authors.forename: Marta
authors.initials: M
authors.lastname: Pillon
authors.affiliation: Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy.
authors.forename: Manuela
authors.initials: M
authors.lastname: Tumino
authors.affiliation: Department of Transfusion Medicine, Therapeutic Apheresis Unit, University Hospital of Padova, Italy.
authors.forename: Tiziana
authors.initials: T
authors.lastname: Tison
authors.affiliation: Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy.
authors.forename: Chiara
authors.initials: C
authors.lastname: Mainardi
authors.affiliation: Department of Transfusion Medicine, Therapeutic Apheresis Unit, University Hospital of Padova, Italy.
authors.forename: Giustina
authors.initials: G
authors.lastname: De Silvestro
authors.affiliation: Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy.
authors.forename: Sara
authors.initials: S
authors.lastname: Rossin
authors.affiliation: Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy.
authors.forename: Genny
authors.initials: G
authors.lastname: Franceschetto
authors.affiliation: Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy.
authors.forename: Elisa
authors.initials: E
authors.lastname: Carraro
authors.affiliation: Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy.
authors.forename: Matilde
authors.initials: M
authors.lastname: Pescarin
authors.affiliation: Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy.
authors.forename: Stefania
authors.initials: S
authors.lastname: Varotto
authors.affiliation: Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy.
authors.forename: Roberta
authors.initials: R
authors.lastname: Destro
authors.affiliation: Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy.
authors.forename: Maria Vittoria
authors.initials: MV
authors.lastname: Gazzola
authors.affiliation: Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy.
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Basso
authors.affiliation: Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy.
authors.forename: Chiara
authors.initials: C
authors.lastname: Messina
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D005260:Female; D006086:Graft vs Host Disease / Q000276:immunology / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D019337:Hematologic Neoplasms / Q000276:immunology / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006650:Histocompatibility Testing; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000627:therapeutic use; D017893:Photopheresis; D012189:Retrospective Studies; D013256:Steroids / Q000627:therapeutic use*; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D061349:Unrelated Donors
chemical_list: D019653:Myeloablative Agonists; D013256:Steroids
keywords: Acute graft-versus-host disease; Children; Extracorporeal photochemotherapy; Hematopoietic stem cell transplantation