TY - JOUR
T1 - Pentostatin therapy for steroid-refractory acute graft versus host disease
T2 - Identifying those who may benefit
AU - Ragon, Brittany Knick
AU - Mehta, Rohtesh S.
AU - Gulbis, Alison M.
AU - Saliba, Rima M.
AU - Chen, Julianne
AU - Rondon, Gabriela
AU - Popat, Uday R.
AU - Nieto, Yago
AU - Oran, Betul
AU - Olson, Amanda L.
AU - Patel, Krina
AU - Hosing, Chitra M.
AU - Qazilbash, Muzaffar H.
AU - Shah, Nina
AU - Kebriaei, Partow
AU - Shpall, Elizabeth J.
AU - Champlin, Richard E.
AU - Alousi, Amin M.
N1 - Publisher Copyright:
© 2017 The Author(s) 2017, under exclusive licence to Macmillan Publishers Limited, part of Springer Nature.
PY - 2018/3/1
Y1 - 2018/3/1
N2 - We report outcomes of 60 patients with steroid-refractory (SR)-aGVHD treated with pentostatin. Almost half (47%) of patients had grade 4 GVHD - 22% had stage 3-4 liver GVHD and 51% had stage 3-4 lower gastrointestinal tract (LGI) GVHD. Patients received a median of 3 courses (range, 1-9) of pentostatin. Day 28 overall response rate (ORR) was 33% (n = 20) (complete response 18% (n = 11), partial response 15% (n = 9)). Non-relapse mortality was 72% (95% confidence interval (CI) 61-84%) and overall survival (OS) was 21% (95% CI 12-32%) at 18 months. On univariate analysis, age >60 years (HR 1.9, 95% CI 1.01-3.7, p = 0.045) and presence of liver GVHD (HR 1.9, 95% CI 1.9, 95% CI 1.5-3.3, p = 0.03) were significant predictors of poor OS while patients with LGI GVHD had superior OS than those without (HR 0.4, 95% CI 0.2-0.8, p = 0.01). On stratified analysis, patients <60 years with isolated LGI GVHD had the best outcomes with an ORR of 48% and OS of 42% at 18 months. Among older patients, OS was 14% in those with isolated LGI aGVHD and 0% in others. Pentostatin remains a viable treatment option for SR-aGVHD, especially in patients 60 years or younger with isolated LGI involvement.
AB - We report outcomes of 60 patients with steroid-refractory (SR)-aGVHD treated with pentostatin. Almost half (47%) of patients had grade 4 GVHD - 22% had stage 3-4 liver GVHD and 51% had stage 3-4 lower gastrointestinal tract (LGI) GVHD. Patients received a median of 3 courses (range, 1-9) of pentostatin. Day 28 overall response rate (ORR) was 33% (n = 20) (complete response 18% (n = 11), partial response 15% (n = 9)). Non-relapse mortality was 72% (95% confidence interval (CI) 61-84%) and overall survival (OS) was 21% (95% CI 12-32%) at 18 months. On univariate analysis, age >60 years (HR 1.9, 95% CI 1.01-3.7, p = 0.045) and presence of liver GVHD (HR 1.9, 95% CI 1.9, 95% CI 1.5-3.3, p = 0.03) were significant predictors of poor OS while patients with LGI GVHD had superior OS than those without (HR 0.4, 95% CI 0.2-0.8, p = 0.01). On stratified analysis, patients <60 years with isolated LGI GVHD had the best outcomes with an ORR of 48% and OS of 42% at 18 months. Among older patients, OS was 14% in those with isolated LGI aGVHD and 0% in others. Pentostatin remains a viable treatment option for SR-aGVHD, especially in patients 60 years or younger with isolated LGI involvement.
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U2 - 10.1038/s41409-017-0034-z
DO - 10.1038/s41409-017-0034-z
M3 - Article
C2 - 29269797
AN - SCOPUS:85038639501
SN - 0268-3369
VL - 53
SP - 315
EP - 325
JO - Bone marrow transplantation
JF - Bone marrow transplantation
IS - 3
ER -