Experimental Pharmaceuticals for Steroid-Refractory Acute Graft-versus-Host Disease

References

• Kanate AS, Majhail NS, Savani BN, et al. Indications for hematopoietic cell transplantation and immune effector cell therapy: guidelines from the American society for transplantation and cellular therapy. Biol Blood Marrow Transplant. 2020;26(7):1247–1256. doi:10.1016/j.bbmt.2020.03.002
   PubMed Web of Science ®Google Scholar
• Majhail NS, Farnia SH, Carpenter PA, et al. Indications for autologous and allogeneic hematopoietic cell transplantation: guidelines from the American society for blood and marrow transplantation. Biol Blood Marrow Transplant. 2015;21(11):1863–1869. doi:10.1016/j.bbmt.2015.07.032
   PubMed Web of Science ®Google Scholar
• D’Souza A, Fretham C. Current uses and outcomes of hematopoietic cell transplantation (HCT): CIBMTR summary slides. 2018. Available from: https://www.cibmtr.org. Accessed January 20, 2020.
   Google Scholar
• Przepiorka D, Weisdorf D, Martin P, et al. 1994 consensus conference on acute GVHD grading. Bone Marrow Transplant. 1995;15(6):825–828.
   PubMed Web of Science ®Google Scholar
• Martin PJ, Rizzo JD, Wingard JR, et al. First and second-line systemic treatment of acute graft-versus-host disease: recommendations of the American society of blood and marrow transplantation. Biol Blood Marrow Transplant. 2012;18(8):1150–1163. doi:10.1016/j.bbmt.2012.04.005
   PubMed Web of Science ®Google Scholar
• MacMillan ML, Robin M, Harris AC, et al. A refined risk score for acute graft-versus-host disease that predicts response to initial therapy, survival, and transplant-related mortality. Biol Blood Marrow Transplant. 2015;21(4):761–767. doi:10.1016/j.bbmt.2015.01.001
   PubMed Web of Science ®Google Scholar
• Zeiser R, Blazar BR. Acute graft-versus-host disease — biologic process, prevention, and therapy. N Engl J Med. 2017;377(22):2167–2179. doi:10.1056/NEJMra1609337
   PubMed Web of Science ®Google Scholar
• Holler E, Butzhammer P, Schmid K, et al. Metagenomic analysis of the stool microbiome in patients receiving allogeneic stem cell transplantation: loss of diversity is associated with use of systemic antibiotics and more pronounced in gastrointestinal graft-versus-host disease. (1523-6536 (Electronic)).
   Google Scholar
• Schoemans HA-O, Lee SJ, Ferrara JL, et al. EBMT-NIH-CIBMTR Task Force position statement on standardized terminology & guidance for graft-versus-host disease assessment. (1476-5365 (Electronic)).
   Google Scholar
• Harris AC, Young R, Devine S, et al. International, multicenter standardization of acute graft-versus-host disease clinical data collection: a report from the mount sinai acute GVHD international consortium. Biol Blood Marrow Transplant. 2016;22(1):4–10. doi:10.1016/j.bbmt.2015.09.001
   PubMed Web of Science ®Google Scholar
• Levine JE, Braun TM, Harris AC, et al. A prognostic score for acute graft-versus-host disease based on biomarkers: a multicentre study. (2352-3026 (Electronic)).
   Google Scholar
• Martin PJ. How I treat steroid-refractory acute graft-versus-host disease. Blood. 2020;135(19):1630–1638. doi:10.1182/blood.2019000960
   Web of Science ®Google Scholar
• Hoda D, Pidala J, Salgado-Vila N, et al. Sirolimus for treatment of steroid-refractory acute graft-versus-host disease. Bone Marrow Transplant. 2010;45(8):1347–1351. doi:10.1038/bmt.2009.343
   PubMed Web of Science ®Google Scholar
• Pidala J, Kim J, Perkins J, et al. Mycophenolate mofetil for the management of steroid-refractory acute graft vs host disease. Bone Marrow Transplant. 2010;45(5):919–924. doi:10.1038/bmt.2009.252
   PubMed Web of Science ®Google Scholar
• Hsu B, May R, Carrum G, Krance R, Przepiorka D. Use of antithymocyte globulin for treatment of steroid-refractory acute graft-versus-host disease: an international practice survey. Bone Marrow Transplant. 2001;28(10):945–950. doi:10.1038/sj.bmt.1703269
   PubMed Web of Science ®Google Scholar
• Ragon BK, Mehta RS, Gulbis AM, et al. Pentostatin therapy for steroid-refractory acute graft versus host disease: identifying those who may benefit. Bone Marrow Transplant. 2018;53(3):315–325. doi:10.1038/s41409-017-0034-z
   PubMed Web of Science ®Google Scholar
• Yalniz FF, Hefazi M, McCullough K, et al. Safety and efficacy of infliximab therapy in the setting of steroid-refractory acute graft-versus-host disease. Biol Blood Marrow Transplant. 2017;23(9):1478–1484. doi:10.1016/j.bbmt.2017.05.001
   PubMed Web of Science ®Google Scholar
• Drobyski WR, Pasquini M, Kovatovic K, et al. Tocilizumab for the treatment of steroid refractory graft-versus-host disease. Biol Blood Marrow Transplant. 2011;17(12):1862–1868. doi:10.1016/j.bbmt.2011.07.001
   PubMed Web of Science ®Google Scholar
• Socié G, Vigouroux S, Yakoub-Agha I, et al. A phase 3 randomized trial comparing inolimomab vs usual care in steroid-resistant acute GVHD. Blood. 2017;129(5):643–649. doi:10.1182/blood-2016-09-738625
   Web of Science ®Google Scholar
• Abu-Dalle I, Reljic T, Nishihori T, et al. Extracorporeal photopheresis in steroid-refractory acute or chronic graft-versus-host disease: results of a systematic review of prospective studies. Biol Blood Marrow Transplant. 2014;20(11):1677–1686. doi:10.1016/j.bbmt.2014.05.017
   PubMed Web of Science ®Google Scholar
• Yang LPH, Keating GM. Ruxolitinib. Drugs. 2012;72(16):2117–2127. doi:10.2165/11209340-000000000-00000
   PubMed Web of Science ®Google Scholar
• Quintás-Cardama A, Vaddi K, Liu P, et al. Preclinical characterization of the selective JAK1/2 inhibitor INCB018424: therapeutic implications for the treatment of myeloproliferative neoplasms. Blood. 2010;115(15):3109–3117. doi:10.1182/blood-2009-04-214957
   PubMed Web of Science ®Google Scholar
• Spoerl S, Mathew NR, Bscheider M, et al. Activity of therapeutic JAK 1/2 blockade in graft-versus-host disease. Blood. 2014;123(24):3832–3842.
   PubMed Web of Science ®Google Scholar
• Choi J, Cooper ML, Alahmari B, et al. Pharmacologic blockade of JAK1/JAK2 reduces GvHD and preserves the graft-versus-leukemia effect. PLoS One. 2014;9(10):e109799. doi:10.1371/journal.pone.0109799
   PubMed Web of Science ®Google Scholar
• Zeiser R, Burchert A, Lengerke C, et al. Ruxolitinib in corticosteroid-refractory graft-versus-host disease after allogeneic stem cell transplantation: a multicenter survey. Leukemia. 2015;29(10):2062–2068. doi:10.1038/leu.2015.212
   PubMed Web of Science ®Google Scholar
• Jagasia M, Zeiser R, Arbushites M, Delaite P, Gadbaw B, Bubnoff N. Ruxolitinib for the treatment of patients with steroid-refractory GVHD: an introduction to the REACH trials. Immunotherapy. 2018;10(5):391–402. doi:10.2217/imt-2017-0156
   Web of Science ®Google Scholar
• Jagasia M, Perales M-A, Schroeder MA, et al. Ruxolitinib for the treatment of steroid-refractory acute GVHD (REACH1): a multicenter, open-label Phase 2 trial. Blood. 2020;135(20):1739–1749. doi:10.1182/blood.2020004823
   PubMed Web of Science ®Google Scholar
• Zeiser R, von Bubnoff N, Butler J, et al. Ruxolitinib for glucocorticoid-refractory acute graft-versus-host disease. N Engl J Med. 2020;382(19):1800–1810. doi:10.1056/NEJMoa1917635
   PubMed Web of Science ®Google Scholar
• Zeiser R, Niederwieser D, Mohty M, et al. Ruxolitinib versus best available therapy in patients with steroid-refractory acute graft-versus-host disease: overall response rate by baseline characteristics in the randomized phase 3 REACH2 trial. EHA Annual Meeting. 2020.
   Google Scholar
• Abedin S, McKenna E, Chhabra S, et al. Efficacy, toxicity, and infectious complications in ruxolitinib-treated patients with corticosteroid-refractory graft-versus-host disease after hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2019;25(8):1689–1694. doi:10.1016/j.bbmt.2019.04.003
   PubMed Web of Science ®Google Scholar
• Gavriilaki E, Bousiou Z, Chatzikonstantinou T, et al. Transplant-associated thrombotic microangiopathy is independently associated with ruxolitinib administration in patients with graft-versus-host-disease. EBMT. 2020;2020.
   Google Scholar
• Schroeder MA, Khoury HJ, Jagasia M, et al. A Phase 1 trial of itacitinib, a selective JAK1 inhibitor, in patients with acute graft-versus-host disease. Blood Adv. 2020;4(8):1656–1669. doi:10.1182/bloodadvances.2019001043
   PubMed Web of Science ®Google Scholar
• Groth C, van Groningen LFJ, Matos TR, et al. Phase I/II trial of a combination of anti-CD3/CD7 immunotoxins for steroid-refractory acute graft-versus-host disease. Biol Blood Marrow Transplant. 2019;25(4):712–719. doi:10.1016/j.bbmt.2018.10.020
   PubMed Web of Science ®Google Scholar
• Groningen LFJ, Groth C, Bremmers MEJ, et al. Results from an expanded access program of anti-CD3/CD7 immunotoxin combination (T-guard®) for the treatment of steroid-refractory acute Gvhd. Blood. 2019;134(Supplement_1):4553. doi:10.1182/blood-2019-122923
   Web of Science ®Google Scholar
• Bacigalupo A, Angelucci E, Raiola AM, et al. Treatment of steroid resistant acute graft versus host disease with an anti-CD26 monoclonal antibody—Begelomab. Bone Marrow Transplant. 2020;55(8):1580–1587. doi:10.1038/s41409-020-0855-z
   PubMed Web of Science ®Google Scholar
• Magenau JM, Goldstein SC, Peltier D, et al. α1-Antitrypsin infusion for treatment of steroid-resistant acute graft-versus-host disease. Blood. 2018;131(12):1372–1379. doi:10.1182/blood-2017-11-815746
   PubMed Web of Science ®Google Scholar
• S HM A, Holtan SG, Anand S, et al. Neihulizumab (ABGN-168H) in patients with steroid-refractory acute graft-versus-host-disease (SR-aGVHD): preliminary results of a phase I study. Paper presented at: EHA Annual Meeting; June 12, 2020.
   Google Scholar
• J MP K, Prockop SE, Burke E, Segal K. Aggregate results of remestemcel-L treatment for steroid-refractory acute graft-versus-host disease in pediatric patients. Orlando, FL:TCT; 2020.
   Google Scholar
• Kakihana K, Fujioka Y, Suda W, et al. Fecal microbiota transplantation for patients with steroid-resistant acute graft-versus-host disease of the gut. Blood. 2016;128(16):2083–2088. doi:10.1182/blood-2016-05-717652
   PubMed Web of Science ®Google Scholar
• van Lier YF, Davids M, Haverkate NJE, et al. Donor fecal microbiota transplantation ameliorates intestinal graft-versus-host disease in allogeneic hematopoietic cell transplant recipients. Sci Transl Med. 2020;12(556):eaaz8926. doi:10.1126/scitranslmed.aaz8926
   PubMed Web of Science ®Google Scholar
• Zhao Y, Li X, Zhou Y, et al. Fecal microbiota transplantation for grade IV steroid refractory GI-GvHD: interim results a non-randomized, open-label, phase 1 clinical study. Research Square; 2020.
   Google Scholar
• Benito AI, Furlong T, Martin PJ, et al. Sirolimus (rapamycin) for the treatment of steroid-refractory acute graft-versus-host disease1. Transplantation. 2001;72(12):1924–1929. doi:10.1097/00007890-200112270-00010
   PubMed Web of Science ®Google Scholar
• Bolaños-Meade J, Jacobsohn DA, Margolis J, et al. Pentostatin in steroid-refractory acute graft-versus-host disease. J Clin Oncol. 2005;23(12):2661–2668. doi:10.1200/JCO.2005.06.130
   Web of Science ®Google Scholar
• Macmillan ML, Couriel D, Weisdorf DJ, et al. A phase 2/3 multicenter randomized clinical trial of ABX-CBL versus ATG as secondary therapy for steroid-resistant acute graft-versus-host disease. Blood. 2007;109:2657–2662. doi:10.1182/blood-2006-08-013995
   PubMed Web of Science ®Google Scholar
• Yucebay F, Matthews C, Puto M, et al. Tocilizumab as first-line therapy for steroid-refractory acute graft-versus-host-disease: analysis of a single-center experience. Leuk Lymphoma. 2019;60(9):2223–2229. doi:10.1080/10428194.2019.1573996
   PubMed Web of Science ®Google Scholar
• Jagasia M, Greinix H, Robin M, et al. Extracorporeal photopheresis versus anticytokine therapy as a second-line treatment for steroid-refractory acute GVHD: a multicenter comparative analysis. Biol Blood Marrow Transplant. 2013;19(7):1129–1133. doi:10.1016/j.bbmt.2013.04.018
   PubMed Web of Science ®Google Scholar