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Graft-versus-Host Disease Prophylaxis with Post- Transplantation Cyclophosphamide in Chronic Myeloid Leukemia Patients Undergoing Allogeneic Hematopoietic Cell Transplantation from an Unrelated or Mismatched Related Donor: A Comparative Study from the Chronic Malignancies Working Party of the EBMT (CMWP-EBMT)

Transplantation and Cellular Therapy, vol. 30, pp. 93.e1–93.e12

Abstract

Outcomes following allogeneic hematopoietic cell transplantation (allo-HCT) for chronic myeloid leukemia (CML) with post-transplantation cyclophosphamide (PTCy) using an unrelated donor (UD) or a mismatched related donor (MMRD) remain unknown. We report a retrospective comparison of PTCy-based allo-HCT from a UD, non-PTCy allo-HCT from a UD, and PTCy allo-HCT from an MMRD. Inclusion criteria were adult patients with CML undergoing first allo-HCT between 2012 and 2019 from a UD with either PTCy or non-PTCy graft-versus-host disease (GVHD) prophylaxis or from an MMRD using PTCy. The primary endpoint was GVHD-free/relapse-free survival (GRFS). A total of 1341 patients were included (82% in the non-PTCy UD cohort). With a median follow-up of 34.9 months, the 3-year GRFS was 43% in the non-PTCy cohort, 37% in the PTCy-UD cohort, and 39% PTCy-MMRD cohort (P = .15). Multivariable analyses revealed no significant differences among the 3 cohorts in terms of overall survival (OS), progression-free survival, RI, and nonrelapse mortality. Factors independently associated with worse OS in the overall cohort were Karnofsky Performance Status <90 (hazard ratio [HR], 1.86; 95% confidence interval [CI], 1.41 to 2.45; P < .001), older age (HR, 1.24, 95% CI, 1.11 to 1.38; P < .001), and disease stage (compared to chronic phase [CP] 1): blast phase (HR, 2.25; 95% CI, 1.60 to 3.16; P < .001), accelerated phase (HR, 1.63; 95% CI, 1.05 to 2.54; P = .03), and CP >2 (HR, 1.58; 95% CI, 1.15 to 2.17; P = .005). These results suggest that allo-HCT in patients with CML using either a UD or an MMRD with PTCy-based GVHD prophylaxis are feasible transplantation, platforms and that the disease stage at allo-HCT remains a major prognostic factor, highlighting the importance of closely monitoring CML patients and proposing transplantation when indicated when still in CP1.

Authors 26

  1. Guillermo Ortí corresponding

    Vall d'Hebron Hospital Universitari · Vall d'Hebron Institute of Oncology

    Affiliation as printed

    Department of Hematology, Vall d`Hebron University Hospital, Vall d'Hebron Institute of Oncology, Barcelona, Spain. Electronic address: gorti@vhio.net

    Department of Hematology, Vall d`Hebron University Hospital, Vall d'Hebron Institute of Oncology, Barcelona, Spain

  2. European Society for Blood and Marrow Transplantation

    Affiliation as printed

    EBMT Statistical Unit, Leiden, the Netherlands

  3. European Society for Blood and Marrow Transplantation

    Affiliation as printed

    EBMT Leiden Study Unit, Leiden, the Netherlands

  4. First Pavlov State Medical University of St. Petersburg

    Affiliation as printed

    RM Gorbacheva Research Institute, Pavlov University, Petersburg, Russian Federation

  5. University of Nottingham

    Affiliation as printed

    Nottingham University, Nottingham, United Kingdom

  6. Imperial College London

    Affiliation as printed

    Imperial College, London, United Kingdom

  7. Instytut Hematologii i Transfuzjologi

    Affiliation as printed

    Institute of Hematology and Transfusion Medicine, Warsaw, Poland

  8. Charité - Universitätsmedizin Berlin

    Affiliation as printed

    Charité-Universitätsmedizin Berlin, Berlin, Germany

    Charité–Universitätsmedizin Berlin, Berlin, Germany

  9. Universität Hamburg · University Medical Center Hamburg-Eppendorf

    Affiliation as printed

    University Hospital Eppendorf, Hamburg, Germany

  10. Vilnius University

    Affiliation as printed

    Vilnius University Hospital, Vilnius, Lithuania

  11. Uppsala University Hospital

    Affiliation as printed

    University Hospital, Uppsala, Sweden

  12. Affiliation as printed

    Northern Centre for Bone Marrow Transplantation, Newcastle Upon Tyne, United Kingdom

  13. The Christie Hospital · The Christie NHS Foundation Trust

    Affiliation as printed

    Christie NHS Trust Hospital, Manchester, United Kingdom

  14. Ospedale Policlinico San Martino

    Affiliation as printed

    IRCCS Ospedale Policlinico San Martino, Genova, Italy

  15. Affiliation as printed

    Programme de Transplantation & Therapie Cellulaire, Marseille, France

  16. King Faisal Specialist Hospital & Research Centre

    Affiliation as printed

    King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia

  17. Instituto de Investigación Biomédica de Salamanca · Centro de Investigación Biomédica en Red de Cáncer

    Affiliation as printed

    Hematology Department, Hospital Universitario de Salamanca, IBSAL, CIBERONC, Salamanca, Spain

  18. G. Papanikolaou General Hospital

    Affiliation as printed

    George Papanicolaou General Hospital, Thessaloniki, Greece

  19. University of Liège · Centre Hospitalier Universitaire de Liège

    Affiliation as printed

    University of Liege and CHU of Liege, Liege, Belgium

  20. Wroclaw Medical University

    Affiliation as printed

    Wroclaw Medical University, Wroclaw, Poland

  21. Azienda Ospedaliera San Camillo-Forlanini

    Affiliation as printed

    Ospedale S. Camillo-Forlanini, Rome, Italy

  22. Guy's and St Thomas' NHS Foundation Trust

    Affiliation as printed

    Guy's and St.Thomas' NHS Foundation Trust, London, United Kingdom

  23. Trinity College Dublin · St. James's Hospital

    Affiliation as printed

    St. James's Hospital, Trinity College, Dublin, Ireland

  24. University College Hospital

    Affiliation as printed

    University College Hospital, London, United Kingdom

  25. University of Geneva · University of Liège · University Hospital of Geneva · Centre Hospitalier Universitaire de Liège

    Affiliation as printed

    Hematology Division and Faculty of Medicine, Hôpitaux Universitaires de Genève, University of Geneva, Geneva, Switzerland

    University of Liege and CHU of Liege, Liege, Belgium

  26. Inserm · Université de Lille · Centre Hospitalier Universitaire de Lille · Institute for Translational Research in Inflammation

    Affiliation as printed

    CHU de Lille, Univ Lille, INSERM U1286, Infinite, 59000, Lille, France

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References 37