1. Richardson, PG, Carreras, E, Iacobelli, M, Nejadnik, B. The use of defibrotide in blood and marrow transplantation. Blood Adv. 2018;2(12):1495–1509. doi:10.1182/bloodadvances.2017008375.
Google Scholar | Crossref | Medline2. Corbacioglu, S, Cesaro, S, Faraci, M, et al. Defibrotide for prophylaxis of hepatic veno-occlusive disease in paediatric haemopoietic stem-cell transplantation: an open-label, phase 3, randomised controlled trial. Lancet. 2012;379(9823):1301–1309. doi:10.1016/S0140-6736(11)61938-7.
Google Scholar | Crossref | Medline | ISI3. Tekgündüz, E, Kaya, AH, Bozdağ, SC, et al. Does defibrotide prophylaxis decrease the risk of acute graft versus host disease following allogeneic hematopoietic cell transplantation? Transfus Apher Sci. 2016;54(1):30–34. doi:10.1016/j.transci.2016.01.009.
Google Scholar | Crossref | Medline | ISI4. Hill, GR, Ferrara, JL. The primacy of the gastrointestinal tract as a target organ of acute graft-versus-host disease: rationale for the use of cytokine shields in allogeneic bone marrow transplantation. Blood. 2000;95(9):2754–2759.
Google Scholar | Crossref | Medline5. Hill, GR, Koyama, M. Cytokines and costimulation in acute graft-versus-host disease. Blood. 2020;136(4):418–428. doi:10.1182/blood.2019000952.
Google Scholar | Crossref | Medline6. Socié, G, Blazar, BR. Acute graft-versus-host disease: from the bench to the bedside. Blood. 2009;114(20):4327–4336. doi:10.1182/blood-2009-06-204669.
Google Scholar | Crossref | Medline7. Zeiser, R, Blazar, BR. Acute Graft-versus-Host Disease—Biologic Process, Prevention, and Therapy. http://dx.doi.org.ressources-electroniques.univ-lille.fr/10.1056/NEJMra1609337.
Google Scholar8. Przepiorka, D, Weisdorf, D, Martin, P, et al. 1994 Consensus conference on acute GVHD grading. Bone Marrow Transplant. 1995;15(6):825–828.
Google Scholar9. Carreras, E, Diaz-Ricart, M. The role of the endothelium in the short-term complications of hematopoietic SCT. Bone Marrow Transplant. 2011;46(12):1495–1502. doi:10.1038/bmt.2011.65.
Google Scholar | Crossref | Medline10. Coppell, JA, Richardson, PG, Soiffer, R, et al. Hepatic veno-occlusive disease following stem cell transplantation: incidence, clinical course, and outcome. Biol Blood Marrow Transplant. 2010;16(2):157–168. doi:10.1016/j.bbmt.2009.08.024.
Google Scholar | Crossref | Medline | ISI11. Bonifazi, F, Barbato, F, Ravaioli, F, et al. Diagnosis and treatment of VOD/SOS after allogeneic hematopoietic stem cell transplantation. Front Immunol. 2020;11:489. doi:10.3389/fimmu.2020.00489.
Google Scholar | Crossref | Medline12. Carreras, E . Vascular endothelial syndromes after HCT: 2020 update. Bone Marrow Transplant. 2020;55(10):1885–1887. doi:10.1038/s41409-020-0852-2.
Google Scholar | Crossref | Medline13. Tichelli, A, Gratwohl, A. Vascular endothelium as “novel” target of graft-versus-host disease. Best Pract Res Clin Haematol. 2008;21(2):139–148. doi:10.1016/j.beha.2008.02.002.
Google Scholar | Crossref | Medline | ISI14. Nomura, S, Ishii, K, Fujita, S, Nakaya, A, Satake, A, Ito, T. Associations between acute GVHD-related biomarkers and endothelial cell activation after allogeneic hematopoietic stem cell transplantation. Transpl Immunol. 2017;43–44:27-32. doi:10.1016/j.trim.2017.06.004.
Google Scholar | Crossref15. Aziz, MT, Kakadiya, PP, Kush, SM, Weigel, K, Lowe, DK. Defibrotide: an oligonucleotide for sinusoidal obstruction syndrome. Ann Pharmacother. 2018;52(2):166–174. doi:10.1177/1060028017732586.
Google Scholar | SAGE Journals | ISI16. European Medicines Agency . EU/3/04/212: orphan designation for the treatment of hepatic veno-occlusive disease. Published September 17, 2018. Accessed October 20, 2021. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu304212.
Google Scholar17. Martinez-Sanchez, J, Hamelmann, H, Palomo, M, et al. Acute graft-vs.-host disease-associated endothelial activation in vitro is prevented by defibrotide. Front Immunol. 2019;10:2339. doi:10.3389/fimmu.2019.02339
Google Scholar | Crossref | Medline18. García-Bernal, D, Palomo, M, Martínez, CM, et al. Defibrotide inhibits donor leucocyte-endothelial interactions and protects against acute graft-versus-host disease. J Cell Mol Med. 2020;24(14):8031–8044. doi:10.1111/jcmm.15434.
Google Scholar | Crossref | Medline19. European Medicines Agency. EU/3/13/1201: public summary of opinion on orphan designation Defibrotide for the prevention of graft-versus-host disease . Published November 28, 2013. Accessed October 20, 2021. https://www.ema.europa.eu/en/documents/orphan-designation/eu/3/13/1201-public-summary-opinion-orphan-designation-defibrotide-prevention-graft-versus-host-disease_en.pdf
Google Scholar20. Mohty, M, Malard, F, Abecassis, M, et al. Revised diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in adult patients: a new classification from the European Society for Blood and Marrow Transplantation. Bone Marrow Transplant. 2016;51(7):906–912. doi:10.1038/bmt.2016.130.
Google Scholar | Crossref | Medline21. Jones, RJ, Lee, KS, Beschorner, WE, et al. Venoocclusive disease of the liver following bone marrow transplantation. Transplantation. 1987;44(6):778–783. doi:10.1097/00007890-198712000-00011.
Google Scholar | Crossref | Medline22. Yakoub-Agha, I . [Fourth annual series of workshops of the SFGM-TC to harmonize practices in allogeneic stem cell transplantation]. Pathol Biol (Paris). 2014;62(4):179. doi:10.1016/j.patbio.2014.05.001.
Google Scholar | Crossref23. Chao, N . How I treat sinusoidal obstruction syndrome. Blood. 2014;123(26):4023–4026. doi:10.1182/blood-2014-03-551630.
Google Scholar | Crossref | Medline24. Spyridonidis, A, Labopin, M, Savani, BN, et al. Redefining and measuring transplant conditioning intensity in current era: a study in acute myeloid leukemia patients. Bone Marrow Transplant. 2020;55(6):1114–1125. doi:10.1038/s41409-020-0803-y.
Google Scholar | Crossref | Medline25. Dignan, FL, Wynn, RF, Hadzic, N, et al. BCSH/BSBMT guideline: diagnosis and management of veno-occlusive disease (sinusoidal obstruction syndrome) following haematopoietic stem cell transplantation. Br J Haematol. 2013;163(4):444–457. doi:10.1111/bjh.12558.
Google Scholar | Crossref | Medline | ISI26. Common Terminology Criteria for Adverse Events (CTCAE v50) . Accessed December 20, 2021. https://evs.nci.nih.gov/ftp1/CTCAE/About.html.
Google Scholar27. Flowers, MED, Inamoto, Y, Carpenter, PA, et al. Comparative analysis of risk factors for acute graft-versus-host disease and for chronic graft-versus-host disease according to National Institutes of Health consensus criteria. Blood. 2011;117(11):3214–3219. doi:10.1182/blood-2010-08-302109.
Google Scholar | Crossref | Medline28. Sorror, ML, Storer, B, Storb, RF. Validation of the hematopoietic cell transplantation-specific comorbidity index (HCT-CI) in single and multiple institutions: limitations and inferences. Biol Blood Marrow Transplant. 2009;15(6):757–758. doi:10.1016/j.bbmt.2009.02.007.
Google Scholar | Crossref | Medline29. Kumar, S, Deleve, LD, Kamath, PS, Tefferi, A. Hepatic veno-occlusive disease (sinusoidal obstruction syndrome) after hematopoietic stem cell transplantation. Mayo Clin Proc. 2003;78(5):589–598. doi:10.4065/78.5.589.
Google Scholar | Crossref | Medline30. Jagasia, M, Arora, M, Flowers, MED, et al. Risk factors for acute GVHD and survival after hematopoietic cell transplantation. Blood. 2012;119(1):296–307. doi:10.1182/blood-2011-06-364265.
Google Scholar | Crossref | Medline31. Steelandt, J, Bocquet, F, Cordonnier, AL, De Courtivron, C, Fusier, I, Paubel, P. Defibrotide in severe sinusoidal obstruction syndrome: medicine and economic issues. Biol Blood Marrow Transplant. 2017;23(2):347–356. doi:10.1016/j.bbmt.2016.11.020.
Google Scholar | Crossref | Medline32. Corbacioglu, S, Jabbour, EJ, Mohty, M. Risk factors for development of and progression of hepatic veno-occlusive disease/sinusoidal obstruction syndrome. Biol Blood Marrow Transplant. 2019;25(7):1271–1280. doi:10.1016/j.bbmt.2019.02.018.
Google Scholar | Crossref | Medline33. Jazz Pharmaceuticals. A Phase 2, prospective, randomized, open-label study on the efficacy of defibrotide added to standard of care immunoprophylaxis for the prevention of acute graft-versus-host-disease in adult and pediatric patients after allogeneic hematopoietic stem cell transplant . clinicaltrials.gov. Published 2021. Accessed October 14, 2021. https://clinicaltrials.gov/ct2/show/results/NCT03339297.
Google Scholar