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.
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.
Division of Pediatric Oncology, Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, Maryland; Pediatric Oncology Branch, National Institutes of Health, Bethesda, Maryland.
Elad
E
Jacoby
Division of Pediatric Oncology, Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, Maryland.
Allen
A
Chen
Division of Pediatric Oncology, Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, Maryland.
David M
DM
Loeb
Division of Pediatric Oncology, Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, Maryland.
Christopher J
CJ
Gamper
Division of Pediatric Oncology, Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, Maryland.
Elias
E
Zambidis
Division of Pediatric Oncology, Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, Maryland.
Nicolas J
NJ
Llosa
Division of Pediatric Oncology, Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, Maryland.
Jeffrey
J
Huo
Division of Pediatric Oncology, Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, Maryland.
Kenneth R
KR
Cooke
The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Johns Hopkins University School of Medicine, Baltimore, Maryland.
Rick
R
Jones
The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Johns Hopkins University School of Medicine, Baltimore, Maryland.
Ephraim
E
Fuchs
The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Johns Hopkins University School of Medicine, Baltimore, Maryland.
Leo
L
Luznik
Division of Pediatric Oncology, Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, Maryland. Electronic address: Hsymons2@jhmi.edu.
Heather J
HJ
Symons
D000293:Adolescent; D000328:Adult; D064591:Allografts; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology / Q000517:prevention & control*; D019337:Hematologic Neoplasms / Q000628:therapy*; D006801:Humans; D007223:Infant; D008297:Male; D014019:Tissue Donors; D055815:Young Adult
D003520:Cyclophosphamide
Cyclophosphamide; Graft-versus-host disease prophylaxis; Hematologic malignancies; Pediatric bone marrow transplantation