Treatment of Acute Graft-versus-Host Disease in Childhood with Extracorporeal Photochemotherapy/Photopheresis: The Padova Experience

Elisabetta Calore; Piero Marson; Marta Pillon; Manuela Tumino; Tiziana Tison; Chiara Mainardi; Giustina De Silvestro; Sara Rossin; Genny Franceschetto; Elisa Carraro; Matilde Pescarin; Stefania Varotto; Roberta Destro; Maria Vittoria Gazzola; Giuseppe Basso; Chiara Messina

doi:10.1016/j.bbmt.2015.07.007

Treatment of Acute Graft-versus-Host Disease in Childhood with Extracorporeal Photochemotherapy/Photopheresis: The Padova Experience

Biol Blood Marrow Transplant. 2015 Nov;21(11):1963-72. doi: 10.1016/j.bbmt.2015.07.007. Epub 2015 Jul 14.

Authors

Elisabetta Calore¹, Piero Marson², Marta Pillon³, Manuela Tumino³, Tiziana Tison², Chiara Mainardi³, Giustina De Silvestro², Sara Rossin³, Genny Franceschetto³, Elisa Carraro³, Matilde Pescarin³, Stefania Varotto³, Roberta Destro³, Maria Vittoria Gazzola³, Giuseppe Basso³, Chiara Messina³

Affiliations

¹ Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy. Electronic address: elisabetta.calore@unipd.it.
² Department of Transfusion Medicine, Therapeutic Apheresis Unit, University Hospital of Padova, Italy.
³ Clinic of Pediatric Hemato-Oncology, Department of Women's and Children's Health, University Hospital of Padova, Italy.

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.

Keywords: Acute graft-versus-host disease; Children; Extracorporeal photochemotherapy; Hematopoietic stem cell transplantation.

MeSH terms

Acute Disease
Adolescent
Adult
Child
Child, Preschool
Female
Graft vs Host Disease / immunology
Graft vs Host Disease / mortality
Graft vs Host Disease / pathology
Graft vs Host Disease / therapy*
Hematologic Neoplasms / immunology
Hematologic Neoplasms / mortality
Hematologic Neoplasms / pathology
Hematologic Neoplasms / therapy*
Hematopoietic Stem Cell Transplantation*
Histocompatibility Testing
Humans
Infant
Male
Middle Aged
Myeloablative Agonists / therapeutic use
Photopheresis*
Retrospective Studies
Steroids / therapeutic use*
Survival Analysis
Transplantation Conditioning / methods*
Transplantation, Homologous
Unrelated Donors

Substances

Myeloablative Agonists
Steroids