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Pilot study using post-transplant cyclophosphamide (PTCy), tacrolimus and mycophenolate GVHD prophylaxis for older patients receiving 10/10 HLA-matched unrelated donor hematopoietic stem cell transplantation

Bone Marrow Transplantation volume 54, pages 601–606 (2019)Cite this article

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Abstract

Allogeneic SCT for older patients remains challenging at least in part due to graft-versus-host disease (GVHD) and higher non-relapse mortality (NRM). We conducted a prospective pilot study primarily for older patients undergoing matched unrelated donor (MUD) SCT using a reduced-intensity (RIC) melphalan-based conditioning and post-transplant cyclophosphamide (PTCy)-based GVHD prophylaxis with tacrolimus and mycophenolate mofetil. Twenty-two patients (median age 64, IQR 58, 66) underwent RIC MUD SCT for high-risk hematological malignancies including AML/MDS (73%), CML/MPD (18%), and other (10%). Two (9%) patients had early death; the rest (100%) engrafted. After a median follow-up of 17 months, 11 patients were alive and disease-free with an estimated 2-year progression-free (PFS) and overall (OS) survival of 48%. The cumulative incidences of grades 2–4 and 3–4 acute GVHD (aGVHD) at day + 100 and 2-years were 32 and 4%, and 59 and 24%, respectively. No cases of chronic GVHD (cGVHD) were noted. However, late acute GVHD was observed in 6 (27%) patients. In conclusion, RIC MUD SCT with melphalan-based conditioning and PTCy-based GVHD-based prophylaxis for older patients appears effective in controlling relapse. While cGVHD was not seen and early aGVHD appears controllable, a significant proportion developed late aGVHD responsible for higher NRM seen in these patients.

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Author contributions

MVS, collected data, wrote, reviewed and approved the manuscript; RMS analyzed the data and interpreted the results, reviewed and approved the manuscript; GR, JC, DS collected data, reviewed and approved the manuscript; AA, BO, PK, MQ, SP, CH, CH, IFK, URP, and REC. enrolled and treated patients, edited, and approved the manuscript; SOC conceived the project, contributed in study design, enrolled and treated the patients, wrote, edited, and approved the manuscript.

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Authors and Affiliations

  1. Department of Stem Cell Transplantation and Cellular Therapy, the University of Texas M.D. Anderson Cancer Center, Houston, TX, USA

    Mithun Vinod Shah, Rima M. Saliba, Gabriela Rondon, Julianne Chen, Doris Soebbing, Ioana Rus, Amin Alousi, Betul Oran, Partow Kebriaei, Muzaffar Qazilbash, Simrit Parmar, Chitra Hosing, Issa F. Khouri, Uday R. Popat, Richard E. Champlin & Stefan O. Ciurea

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  1. Mithun Vinod Shah
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  2. Rima M. Saliba
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Correspondence to Stefan O. Ciurea.

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Shah, M.V., Saliba, R.M., Rondon, G. et al. Pilot study using post-transplant cyclophosphamide (PTCy), tacrolimus and mycophenolate GVHD prophylaxis for older patients receiving 10/10 HLA-matched unrelated donor hematopoietic stem cell transplantation. Bone Marrow Transplant 54, 601–606 (2019). https://doi.org/10.1038/s41409-018-0367-2

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