title: OCTET-CY: a phase II study to investigate the efficacy of post-transplant cyclophosphamide as sole graft-versus-host prophylaxis after allogeneic peripheral blood stem cell transplantation.
abstract: OBJECTIVE
  Post-transplant cyclophosphamide is increasingly used as graft-versus-host disease (GvHD) prophylaxis in the setting of bone marrow transplantation. No data have been published on the use of single-agent GvHD prophylaxis with post-transplant cyclophosphamide in the setting of peripheral blood stem cell transplantation (PBSCT).
  
  
  METHODS
  In a phase II trial, 11 patients with myeloma or lymphoma underwent conditioning with fludarabine and busulfan followed by T-replete PBSCT and application of 50 mg/kg/d of cyclophosphamide on day+3 and +4 without other concurrent immunosuppression (IS).
  
  
  RESULTS
  Median time to leukocyte, neutrophil, and platelet engraftment was 18, 21, and 18 d. The incidence of grade II-IV and grade III-IV GvHD was 45% and 27%, with a non-relapse mortality (NRM) of 36% at one and 2 yr. After median follow-up of 927 d, overall and relapse-free survival was 64% and 34%. Three patients did not require any further systemic IS until day+100 and thereafter. Analysis of immune reconstitution demonstrated rapid T- and NK-cell recovery. B- and CD3+/CD161+NK/T-cell recovery was superior in patients not receiving additional IS.
  
  
  CONCLUSIONS
  Post-transplant cyclophosphamide as sole IS in PBSCT is feasible and allows rapid immune recovery. Increased rates of severe acute GvHD explain the observed NRM and may advise a temporary combination partner such as mTor-inhibitors in the PBSCT setting.
authors.affiliation: Stem Cell Transplantation Program, Department I of Internal Medicine, Center for Integrated Oncology CIO KölnBonn, University Hospital of Cologne, Cologne, Germany.|Cologne Interventional Immunology, Department I of Internal Medicine, University Hospital of Cologne, Cologne, Germany.
authors.forename: Udo
authors.initials: U
authors.lastname: Holtick
authors.affiliation: Stem Cell Transplantation Program, Department I of Internal Medicine, Center for Integrated Oncology CIO KölnBonn, University Hospital of Cologne, Cologne, Germany.
authors.forename: Jens-Markus
authors.initials: JM
authors.lastname: Chemnitz
authors.affiliation: Stem Cell Transplantation Program, Department I of Internal Medicine, Center for Integrated Oncology CIO KölnBonn, University Hospital of Cologne, Cologne, Germany.|Cologne Interventional Immunology, Department I of Internal Medicine, University Hospital of Cologne, Cologne, Germany.
authors.forename: Alexander
authors.initials: A
authors.lastname: Shimabukuro-Vornhagen
authors.affiliation: Stem Cell Transplantation Program, Department I of Internal Medicine, Center for Integrated Oncology CIO KölnBonn, University Hospital of Cologne, Cologne, Germany.|Cologne Interventional Immunology, Department I of Internal Medicine, University Hospital of Cologne, Cologne, Germany.
authors.forename: Sebastian
authors.initials: S
authors.lastname: Theurich
authors.affiliation: Stem Cell Transplantation Program, Department I of Internal Medicine, Center for Integrated Oncology CIO KölnBonn, University Hospital of Cologne, Cologne, Germany.|Cologne Interventional Immunology, Department I of Internal Medicine, University Hospital of Cologne, Cologne, Germany.
authors.forename: Geothy
authors.initials: G
authors.lastname: Chakupurakal
authors.affiliation: Stem Cell Transplantation Program, Department I of Internal Medicine, Center for Integrated Oncology CIO KölnBonn, University Hospital of Cologne, Cologne, Germany.
authors.forename: Anke
authors.initials: A
authors.lastname: Krause
authors.affiliation: Cologne Interventional Immunology, Department I of Internal Medicine, University Hospital of Cologne, Cologne, Germany.
authors.forename: Anne
authors.initials: A
authors.lastname: Fiedler
authors.affiliation: Sidney Kimmel Comprehensive Cancer Center and Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
authors.forename: Leo
authors.initials: L
authors.lastname: Luznik
authors.affiliation: Institute of Medical Statistics, Informatics and Epidemiology, University of Cologne, Cologne, Germany.
authors.forename: Martin
authors.initials: M
authors.lastname: Hellmich
authors.affiliation: Medical Clinic III for Oncology, Haematology and Rheumatology, Center for Integrated Oncology CIO KölnBonn, University Hospital Bonn, Bonn, Germany.
authors.forename: Dominik
authors.initials: D
authors.lastname: Wolf
authors.affiliation: Stem Cell Transplantation Program, Department I of Internal Medicine, Center for Integrated Oncology CIO KölnBonn, University Hospital of Cologne, Cologne, Germany.
authors.forename: Michael
authors.initials: M
authors.lastname: Hallek
authors.affiliation: Stem Cell Transplantation Program, Department I of Internal Medicine, Center for Integrated Oncology CIO KölnBonn, University Hospital of Cologne, Cologne, Germany.|Cologne Interventional Immunology, Department I of Internal Medicine, University Hospital of Cologne, Cologne, Germany.
authors.forename: Michael
authors.initials: M
authors.lastname: von Bergwelt-Baildon
authors.affiliation: Stem Cell Transplantation Program, Department I of Internal Medicine, Center for Integrated Oncology CIO KölnBonn, University Hospital of Cologne, Cologne, Germany.
authors.forename: Christof
authors.initials: C
authors.lastname: Scheid
mesh_terms: D000328:Adult; D064591:Allografts; D003520:Cyclophosphamide / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D006086:Graft vs Host Disease / Q000097:blood / Q000209:etiology / Q000517:prevention & control*; D019337:Hematologic Neoplasms / Q000097:blood / Q000628:therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D036102:Peripheral Blood Stem Cell Transplantation
chemical_list: D003520:Cyclophosphamide
keywords: allogeneic hematopoietic stem cell transplantation; graft-versus-host disease; post-transplant cyclophosphamide