Single-Agent Post-Transplantation Cyclophosphamide as Graft-versus-Host Disease Prophylaxis after Human Leukocyte Antigen-Matched Related Bone Marrow Transplantation for Pediatric and Young Adult Patients with Hematologic Malignancies

Elad Jacoby, Allen Chen, David M. Loeb, Christopher J. Gamper, Elias Zambidis, Nicolas J. Llosa, Jeffrey Huo, Kenneth R. Cooke, Rick Jones, Ephraim Fuchs, Leo Luznik, Heather J. Symons

Research output: Contribution to journalArticle

18 Citations (Scopus)

Abstract

High-dose cyclophosphamide given after HLA-matched related and unrelated allogeneic bone marrow transplantation (BMT) for patients with hematologic malignancies is effective single-agent graft-versus-host disease (GVHD) prophylaxis in adults. Data describing outcomes for pediatric and young adult patients have not been reported. Between the years 2007 and 2013, 29 pediatric and young adult patients ages ≤21 years of age treated at our institution for high-risk hematologic malignancies underwent myeloablative HLA-matched related T cell-replete BMT. Eleven patients received post-transplantation cyclophosphamide (PTCy) as single-agent GVHD prophylaxis and were followed prospectively. Eighteen patients received calcineurin inhibitor (CNI)-based standard GVHD prophylaxis and were studied retrospectively as a control group. No acute GVHD (aGVHD) developed in patients receiving PTCy, whereas patients receiving CNI-based GVHD prophylaxis had cumulative incidences of grades II to IV and grades III and IV aGVHD of 27% and 5%, respectively. No patients receiving PTCy developed chronic GHVD, compared to 1 in the control group. Two-year overall survival was similar between the 2 groups (54% PTCy versus 58% CNI-based prophylaxis), as was event-free survival (42% PTCy versus 47% CNI-based). The 5-year cumulative incidence of relapse was 58% for PTCy and 42% for CNI-based GVHD prophylaxis (. P = .45). These results suggest that PTCy is a safe and efficacious method of GVHD prophylaxis after an HLA-matched related BMT in the pediatric and young adult population that affords patients to be off all post-transplantation immunosuppression on day +5.

Original languageEnglish (US)
Pages (from-to)112-118
Number of pages7
JournalBiology of Blood and Marrow Transplantation
Volume22
Issue number1
DOIs
StatePublished - Jan 1 2016
Externally publishedYes

Fingerprint

Graft vs Host Disease
Hematologic Neoplasms
HLA Antigens
Bone Marrow Transplantation
Cyclophosphamide
Young Adult
Transplantation
Pediatrics
Control Groups
Incidence
Homologous Transplantation
Immunosuppression
Disease-Free Survival
Calcineurin Inhibitors
T-Lymphocytes
Recurrence
Survival

Keywords

  • Cyclophosphamide
  • Graft-versus-host disease prophylaxis
  • Hematologic malignancies
  • Pediatric bone marrow transplantation

ASJC Scopus subject areas

  • Hematology
  • Transplantation

Cite this

Single-Agent Post-Transplantation Cyclophosphamide as Graft-versus-Host Disease Prophylaxis after Human Leukocyte Antigen-Matched Related Bone Marrow Transplantation for Pediatric and Young Adult Patients with Hematologic Malignancies. / Jacoby, Elad; Chen, Allen; Loeb, David M.; Gamper, Christopher J.; Zambidis, Elias; Llosa, Nicolas J.; Huo, Jeffrey; Cooke, Kenneth R.; Jones, Rick; Fuchs, Ephraim; Luznik, Leo; Symons, Heather J.

In: Biology of Blood and Marrow Transplantation, Vol. 22, No. 1, 01.01.2016, p. 112-118.

Research output: Contribution to journalArticle

Jacoby, Elad ; Chen, Allen ; Loeb, David M. ; Gamper, Christopher J. ; Zambidis, Elias ; Llosa, Nicolas J. ; Huo, Jeffrey ; Cooke, Kenneth R. ; Jones, Rick ; Fuchs, Ephraim ; Luznik, Leo ; Symons, Heather J. / Single-Agent Post-Transplantation Cyclophosphamide as Graft-versus-Host Disease Prophylaxis after Human Leukocyte Antigen-Matched Related Bone Marrow Transplantation for Pediatric and Young Adult Patients with Hematologic Malignancies. In: Biology of Blood and Marrow Transplantation. 2016 ; Vol. 22, No. 1. pp. 112-118.
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