title: Pentostatin as rescue therapy for glucocorticoid-refractory acute and chronic graft-versus-host disease.
abstract: BACKGROUND
  Despite scientific advances in hematopoietic cell allografting, glucocorticoid-refractory acute (aGVHD) and chronic graft-versus-host disease (cGVHD) represent major sources of transplant-related morbidity and mortality. We aimed to characterize the activity of pentostatin as rescue therapy for refractory GVHD.
  
  
  METHODS
  In a retrospective analysis, we examined the activity of pentostatin as rescue therapy of glucocorticoid-refractory acute and chronic GVHD.
  
  
  RESULTS
  In 12 patients with advanced (overall aGVHD grade III/IV in 8/12) refractory aGVHD, overall response (ORR) was achieved in 6/12, and complete remission (CR) of aGVHD in 4/12 allowing additional rescue immunosuppressive agents. Median overall survival (OS) was 1.4 months (95% CI: 0.26-2.4). Causes of death included refractory aGVHD and infection. In 18 patients with refractory cGVHD (12/18 with severe cGVHD), pentostatin induced CR in 1/18, and partial response (PR) in 9/18. Activity was observed in all affected organs. The median decrease in glucocorticoid therapy over 24 months after pentostatin initiation for refractory cGVHD was 38% (range=0-100%). Median OS was 5 months (95% CI: 1.6 - NR).
  
  
  CONCLUSIONS
  Allowing for the utilization of additional immune suppressive agents, this series suggests the activity of pentostatin as rescue therapy of refractory GVHD.
authors.affiliation: Department of Blood and Marrow Transplantation, Moffitt Cancer Center and Research Institute, University of South Florida, Tampa, FL, USA. joseph.pidala@moffitt.org
authors.forename: Joseph
authors.initials: J
authors.lastname: Pidala
authors.forename: Jongphil
authors.initials: J
authors.lastname: Kim
authors.forename: Jaime
authors.initials: J
authors.lastname: Roman-Diaz
authors.forename: Jamie
authors.initials: J
authors.lastname: Shapiro
authors.forename: Taiga
authors.initials: T
authors.lastname: Nishihori
authors.forename: Ryan
authors.initials: R
authors.lastname: Bookout
authors.forename: Claudio
authors.initials: C
authors.lastname: Anasetti
authors.forename: Mohamed A
authors.initials: MA
authors.lastname: Kharfan-Dabaja
mesh_terms: D000208:Acute Disease; D058892:Adenosine Deaminase Inhibitors / Q000627:therapeutic use*; D002908:Chronic Disease; D004351:Drug Resistance; D005938:Glucocorticoids / Q000627:therapeutic use; D006086:Graft vs Host Disease / Q000188:drug therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D015649:Pentostatin / Q000627:therapeutic use*; D012189:Retrospective Studies; D016879:Salvage Therapy / Q000379:methods*; D016019:Survival Analysis; D016896:Treatment Outcome
chemical_list: D058892:Adenosine Deaminase Inhibitors; D005938:Glucocorticoids; D015649:Pentostatin