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Graft-Versus-Host Disease

Mycophenolate mofetil for the management of steroid-refractory acute graft vs host disease

Bone Marrow Transplantation volume 45, pages 919–924 (2010)Cite this article

Abstract

Acute graft vs host disease (aGVHD) is a significant obstacle to successful allogeneic hematopoietic cell transplantation, as only 30–40% of those with aGVHD show complete response to front-line glucocorticoids. The role of mycophenolate mofetil (MMF) as salvage therapy in steroid-refractory aGVHD remains incompletely defined. Here, we examine outcomes of 27 patients with refractory aGVHD treated with MMF as second-line therapy. Seven (26%) patients achieved complete remission (CR) of steroid-refractory aGVHD with only the addition of MMF as salvage therapy. CR of aGVHD differed by overall grade at salvage (grade I, 1/3; grade II, 5/12; grade III 0/5; grade IV, 1/7) with odds ratio for CR in grade I/II vs III/IV of 7.3 (95% CI: 0.7–72.6, P=0.09). Overall survival (OS) at 3 years was 40%. Overall aGVHD grade at salvage (hazard ratio (HR) grade I/II vs III/IV 0.18 (95% CI: 0.06–0.57), P=0.003) and achievement of CR (HR 0.12 (95% CI: 0.04–0.39), P=0.0004) were significant predictors of OS. MMF was overall well tolerated, with only two patients requiring discontinuation for myelosuppression. MMF shows activity in the salvage of steroid-refractory, grades I–II aGVHD.

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Acknowledgements

We acknowledge support from grants 3 P30-CA7692 and 5 K12-CA087989 from the NCI, and 5 K30-RR022270 from the National Center for Research Resources, National Institutes of Health.

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Authors and Affiliations

  1. Department of Blood and Marrow Transplantation, H. Lee Moffitt Cancer Center and Research Institute, University of South Florida, Tampa, FL, USA

    J Pidala, J Perkins, T Field, H Fernandez, L Perez, E Ayala, M Kharfan-Dabaja & C Anasetti

  2. Department of Biostatistics, H. Lee Moffitt Cancer Center and Research Institute, University of South Florida, Tampa, FL, USA

    J Kim

  3. Department of Oncological Sciences, University of South Florida, Tampa, FL, USA

    J Pidala, J Kim, J Perkins, T Field, H Fernandez, L Perez, E Ayala, M Kharfan-Dabaja & C Anasetti

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Pidala, J., Kim, J., Perkins, J. et al. Mycophenolate mofetil for the management of steroid-refractory acute graft vs host disease. Bone Marrow Transplant 45, 919–924 (2010). https://doi.org/10.1038/bmt.2009.252

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