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Long-term follow-up of a prospective randomized trial comparing CYA and MTX with CYA and mycophenolate mofetil for GVHD prophylaxis in myeloablative sibling donor hematopoietic cell transplantation

Bone Marrow Transplantation volume 48, pages 1578–1580 (2013)Cite this article

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To determine the efficacy of a calcineurin inhibitor in combination with mycophenolate mofetil (MMF) in myeloablative hematopoietic cell transplantation (HCT), we previously conducted a prospective randomized trial comparing CYA and MMF with the standard CYA and MTX for GVHD prophylaxis in myeloablative matched related donor transplants. The results of this trial have been reported and demonstrated faster hematopoietic recovery, decreased mucositis, similar incidence of acute (a)GVHD and comparable survival between MMF and MTX.1 The study was prematurely closed due to the reduced toxicity of MMF. We now update these data with longer follow-up, and report survival, relapse, infection and GVHD outcomes observed in this study.

Patient and disease characteristics were as previously reported1 (Table 1). Briefly, 40 patients were enrolled, with 21 randomized to receive MMF and 19 receiving MTX. All received a myeloablative HCT from a 6/6 HLA-matched related donor, with BM as the hematopoietic cell source. The preparative regimen consisted of BU (16 mg/kg orally) and CY (120 mg/kg). All patients received 300 mg/m2 CYA intravenously daily from day −1, and then orally, adjusted to maintain a therapeutic trough level of 200. Patients randomized to MTX received a dose of 5 mg/m2 intravenously on days +1, +3, +6 and +11. Patients randomized to MMF received 500 mg three times daily from day +1 until day +100.

Table 1 Patient demographics
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Acknowledgements

We thank the transplantation team at the Cleveland Clinic Taussig Cancer Institute for their contributions, and all the patients for their participation in clinical trials.

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Authors and Affiliations

  1. Department of Hematologic Oncology and Blood Disorders, Bone Marrow Transplant Program, Cleveland Clinic Taussig Cancer Institute, Cleveland, OH, USA

    B K Hamilton, B Bolwell, M Kalaycio, R Sobecks, B Pohlman, S Andresen, R Dean, H Duong & B Hill

  2. Quantitative Health Sciences, Cleveland Clinic Lerner Research Institute, Cleveland, OH, USA

    L Rybicki

  3. Pediatric Hematology Oncology, Cleveland Clinic, Cleveland, OH, USA

    R Hanna

  4. Levine Cancer Institute, Carolinas HealthCare System, Charlotte, NC, USA

    E Copelan

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  1. B K Hamilton
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  2. B Bolwell
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Correspondence to B K Hamilton.

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Hamilton, B., Bolwell, B., Kalaycio, M. et al. Long-term follow-up of a prospective randomized trial comparing CYA and MTX with CYA and mycophenolate mofetil for GVHD prophylaxis in myeloablative sibling donor hematopoietic cell transplantation. Bone Marrow Transplant 48, 1578–1580 (2013). https://doi.org/10.1038/bmt.2013.89

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