TY - JOUR
T1 - Vedolizumab for Steroid Refractory Lower Gastrointestinal Tract Graft-Versus-Host Disease
AU - Mehta, Rohtesh S.
AU - Saliba, Rima M.
AU - Jan, Anna
AU - Shigle, Terri Lynn
AU - Wang, Emily
AU - Nieto, Yago
AU - Ciurea, Stefan O.
AU - Oran, Betul
AU - Im, Jin
AU - Olson, Amanda
AU - Marin, David
AU - Qazilbash, Muzaffar
AU - Khouri, Issa
AU - Rondon, Gabriela
AU - Anderlini, Paolo
AU - Rezvani, Katayoun
AU - Popat, Uday
AU - Kebriaei, Partow
AU - Shpall, Elizabeth
AU - Champlin, Richard
AU - Alousi, Amin
N1 - Publisher Copyright:
© 2020 The American Society for Transplantation and Cellular Therapy
PY - 2021/3
Y1 - 2021/3
N2 - Steroid-refractory (SR) lower gastrointestinal (LGI) acute graft-versus-host disease (aGVHD) has poor prognosis, and novel drugs are needed. We describe outcomes of patients with SR-LGI aGVHD treated with vedolizumab. The primary objective was to determine overall response rate (ORR) at days 14, 28, and 56. Secondary outcomes included overall survival (OS), non-relapse mortality and toxicities. Twenty patients, median age 46 years (range, 23-71), were included. All but 2 patients (90%) had grade 3 to 4 aGVHD (45% stage 4, 40% stage 3 LGI). Median time to vedolizumab was 21 days (range, 5-1031) and 13 days (range, 0-533) after diagnosis of LGI aGVHD and SR-LGI aGVHD, respectively. It was given as ≥3rd line (median 3; range 2-6) in 75% after failure of steroids, and additional treatments including ruxolitinib (n = 12) and others. Median follow-up was 17 months (range, 10-34). The days 14, 28 and 56 ORRs were 45% (9/20; complete response [CR] 25%), 35% (7/20; CR 20%), and 25% (5/20; CR 20%), respectively. Among ruxolitinib failures, it was 50% (6/12; CR 25%), 50% (6/12; CR 25%) and 25% (3/12; CR 16.7%), respectively. Fifteen patients died (14 GVHD, 1 leukemia relapse). The actuarial 6-month OS was 35% (95% confidence interval 16-55). No progressive multifocal leukoencephalopathy or infusion reaction occurred. Forty-four infection events (22 viral, 18 bacterial, and 4 fungal) were noted in 16 patients. Vedolizumab was well tolerated and demonstrated potential efficacy even after ruxolitinib failure for SR-LGI aGVHD. Yet the responses were suboptimal, and its use requires further investigation.
AB - Steroid-refractory (SR) lower gastrointestinal (LGI) acute graft-versus-host disease (aGVHD) has poor prognosis, and novel drugs are needed. We describe outcomes of patients with SR-LGI aGVHD treated with vedolizumab. The primary objective was to determine overall response rate (ORR) at days 14, 28, and 56. Secondary outcomes included overall survival (OS), non-relapse mortality and toxicities. Twenty patients, median age 46 years (range, 23-71), were included. All but 2 patients (90%) had grade 3 to 4 aGVHD (45% stage 4, 40% stage 3 LGI). Median time to vedolizumab was 21 days (range, 5-1031) and 13 days (range, 0-533) after diagnosis of LGI aGVHD and SR-LGI aGVHD, respectively. It was given as ≥3rd line (median 3; range 2-6) in 75% after failure of steroids, and additional treatments including ruxolitinib (n = 12) and others. Median follow-up was 17 months (range, 10-34). The days 14, 28 and 56 ORRs were 45% (9/20; complete response [CR] 25%), 35% (7/20; CR 20%), and 25% (5/20; CR 20%), respectively. Among ruxolitinib failures, it was 50% (6/12; CR 25%), 50% (6/12; CR 25%) and 25% (3/12; CR 16.7%), respectively. Fifteen patients died (14 GVHD, 1 leukemia relapse). The actuarial 6-month OS was 35% (95% confidence interval 16-55). No progressive multifocal leukoencephalopathy or infusion reaction occurred. Forty-four infection events (22 viral, 18 bacterial, and 4 fungal) were noted in 16 patients. Vedolizumab was well tolerated and demonstrated potential efficacy even after ruxolitinib failure for SR-LGI aGVHD. Yet the responses were suboptimal, and its use requires further investigation.
KW - Allogeneic hematopoietic stem cell transplantation
KW - GVHD
KW - Gastrointestinal tract
KW - Ruxolitinib
KW - Steroid-refractory GVHD
KW - Vedolizumab
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UR - http://www.scopus.com/inward/citedby.url?scp=85101158882&partnerID=8YFLogxK
U2 - 10.1016/j.jtct.2020.12.011
DO - 10.1016/j.jtct.2020.12.011
M3 - Article
C2 - 33781539
AN - SCOPUS:85101158882
SN - 2666-6367
VL - 27
SP - 272.e1-272.e5
JO - Transplantation and Cellular Therapy
JF - Transplantation and Cellular Therapy
IS - 3
ER -