title: Mycophenolate mofetil for the management of steroid-refractory acute graft vs host disease.
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.
authors.affiliation: Department of Blood and Marrow Transplantation, H. Lee Moffitt Cancer Center and Research Institute, University of South Florida, Tampa, FL 33612-9416, USA. joseph.pidala@moffitt.org
authors.forename: J
authors.initials: J
authors.lastname: Pidala
authors.forename: J
authors.initials: J
authors.lastname: Kim
authors.forename: J
authors.initials: J
authors.lastname: Perkins
authors.forename: T
authors.initials: T
authors.lastname: Field
authors.forename: H
authors.initials: H
authors.lastname: Fernandez
authors.forename: L
authors.initials: L
authors.lastname: Perez
authors.forename: E
authors.initials: E
authors.lastname: Ayala
authors.forename: M
authors.initials: M
authors.lastname: Kharfan-Dabaja
authors.forename: C
authors.initials: C
authors.lastname: Anasetti
mesh_terms: D000208:Acute Disease; D000328:Adult; D004351:Drug Resistance; D005938:Glucocorticoids / Q000627:therapeutic use*; D006086:Graft vs Host Disease / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D020714:Maximum Tolerated Dose; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000031:analogs & derivatives* / Q000627:therapeutic use; D012074:Remission Induction; D016879:Salvage Therapy; D016019:Survival Analysis; D014019:Tissue Donors; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D005938:Glucocorticoids; D009173:Mycophenolic Acid