Management of Allogeneic Stem Cell Transplant Recipients with Hepatic Veno-Occlusive Disease
DOI:
https://doi.org/10.15379/2408-9877.2017.04.01.01Keywords:
Hepatic VOD/SOS, Endothelial damage, Transplant-related complications, DefibrotideAbstract
Hepatic veno-occlusive disease (VOD) or sinusoidal obstruction syndrome (SOS) is one the major limiting factor for the successful outcome of patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT), with a reported mortality rate of up to 50%. VOD/SOS is thought to result from an endothelial damage and occurs with a highly variable incidence ranging from 8% to 14%. Management of patients with VOD/SOS is based on both prevention and treatment, which rely on non-pharmacological approaches, for instance the control of additional risk factors, and pharmacologic treatments.
Herein we provide a review of the current understanding for the management of patients with VOD/SOS after allogeneic HSCT.
References
Jones, RJ, Lee, KS, Beschorner, WE, et al. Venocclusive disease of the liver following bone marrow transplantation. Transplantation, 1987; 44: 778-783. https://doi.org/10.1097/00007890-198712000-00011
McDonald GB, Sharma P, Matthews DE at. Al. Venocclusive disease of the liver after bone marrow transplantation: diagnosis, incidence and predisposing factors. Hepatology 1984; 4: 116-122. https://doi.org/10.1002/hep.1840040121
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. BMT 2016, 1-7.
Chao N. How I treat sinusoidal obstruction syndrome. Blood 2014; 123: 4023-4026. https://doi.org/10.1182/blood-2014-03-551630
McDonald GB, Hinds MS, Fisher ID et al. veno-occlusive disease of the liver and multiorgan failure after bone marrow transplantation: a cohort study of 355 patients. Ann Int Med 1993; 118: 255-267. https://doi.org/10.7326/0003-4819-118-4-199302150-00003
Carreras E. I how manage sinusoidal obstruction syndrome after haematopoietic cell transplantation. Br J Haematol 2015; 168: 481-491. https://doi.org/10.1111/bjh.13215
Ferra` C, de Sanjose´ S, Lastra CF et al. Pentoxifylline, ciprofloxacin and prednisone failed to prevent transplant-related toxicities in bone marrow transplant recipients and were associated with an increased incidence of infectious complications. Bone marrow Transplant 1997; 20: 1075-1080. https://doi.org/10.1038/sj.bmt.1701023
Haussmann U, Fischer J, Eber S et al. Hepatic veno-occlusive disease in pediatric stem cell transplantation: impact of pre-emptive antithrombin III replacement and combined antithrombin III/defibrotide therapy. Haematologica 2006; 91: 795-800
Attal M, Huguet F, Rubie H et al. Prevention of Hepatic Veno-occlusive Disease After Bone Marrow Transplantation by Continuous Infusion of Low-Dose Heparin: A Prospective, Randomized Trial. Blood 1992; 79: 2834-2840.
Or R, Nagler A, Shpilberg O, Elad S, et al. Low molecular weight heparin for the prevention of veno-occlusive disease of the liver in bone marrow transplantation patients. Transplantation 1996; 61: 1067-1071. https://doi.org/10.1097/00007890-199604150-00014
Marsa-Vila L, Gorin NC, Laporte JP et al. Prophylactic heparin does not prevent liver veno-occlusive disease following autologous bone marrow transplantation. Eur J Haematol 1991; 47: 346-354. https://doi.org/10.1111/j.1600-0609.1991.tb01859.x
Imran H, Tleyjeh IM, Zirakzadeh A et al. Use of prophylactic anticoagulation and the risk of hepatic veno-occlusive disease in patients undergoing hematopoietic stem cell transplantation: a systematic review and meta-analysis. Bone Marrow Transplantation 2006; 37: 677-686. https://doi.org/10.1038/sj.bmt.1705297
Song JS, Seo JJ, Moon HN et al. Prophylactic Low-dose Heparin or Prostaglandin E1 may Prevent Severe Veno-occlusive Disease of the Liver after Allogeneic Hematopoietic Stem Cell Transplantation in Korean Children. J Korean Med Sci 2006; 21: 897-903. https://doi.org/10.3346/jkms.2006.21.5.897
Tay J, Tinmouth A, Fergusson D et al. Systematic Review of Controlled Clinical Trials on the Use of Ursodeoxycholic Acid for the Prevention of Hepatic Veno-occlusive Disease in Hematopoietic Stem Cell Transplantation. Biology of Blood and Marrow Transplantation 2007; 13: 206-217. https://doi.org/10.1016/j.bbmt.2006.09.012
Chalandon Y, Roosnek E, Mermillod B et al. Prevention of veno-occlusive disease with defibrotide after allogeneic stem cell transplantation. Biol Blood Marrow Transplant 2004; 10: 347-54. https://doi.org/10.1016/j.bbmt.2004.01.002
Dignan F, Gujral D, Ethell M, et al. Prophylactic defibrotide in allogeneic stem cell transplantation: minimal morbidity and zero mortality from veno-occlusive disease. Bone Marrow Transplant 2007; 40: 79-82. https://doi.org/10.1038/sj.bmt.1705696
Bonini A, Imovilli A, Ghirarduzzi A, et al. Defibrotide low-dose continuous infusion after allogenic stem cell transplantation as prophylaxis for veno-occlusive disease of the liver. Blood (ASH annual meeting abstract) 2010; 116: abstract 3483.
Park M, Park HJ, Eom H-S, et al. Effects of prophylactic defibrotide for veno-occlusive disease in haematopoietic stem cell transplantation. Blood (ASH annual meeting abstract) 2011; 118: abstract 4517.
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: 1301-09. https://doi.org/10.1016/S0140-6736(11)61938-7
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. British Journal of Haematology, 2013; 163: 444-457. https://doi.org/10.1111/bjh.12558
Mohty M, Malard F, Abecassis M et al. Sinusoidal obstruction syndrome/veno-occlusive disease: current situation and perspectives—a position statement from the European Society for Blood and Marrow Transplantation (EBMT). Bone Marrow Transplantation 2015; 50: 781-789. https://doi.org/10.1038/bmt.2015.52
Bearman SI, Lee JL, Baro´ AE, Mc Donald GB. Treatment of Hepatic Venocclusive Disease With Recombinant Human Tissue Plasminogen Activator and Heparin in 42 Marrow Transplant Patients. Blood 1997; 89: 1501-1506.
Barkholt L, Remberger M, Hassan Z et al. A prospective randomized study using N-acetyl-L-cysteine for early liver toxicity after allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplantation 2008; 41:
-790. https://doi.org/10.1038/sj.bmt.1705969
Al Beihany A, Al Omar H, Sahovic E et al. Successful treatment of hepatic veno-occlusive disease after myeloablative allogeneic hematopoietic stem cell transplantation by early administration of a short course of methylprednisolone. Bone Marrow Transplantation 2008; 41: 287-291. https://doi.org/10.1038/sj.bmt.1705896
Richardson PG, Riches ML, Kernan NA, et al. Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure. Blood 2016; 127: 1656-1665. https://doi.org/10.1182/blood-2015-10-676924
Strouse C, Richardson P, Prentice G et al. Defibrotide for Treatment of Severe Veno-Occlusive Disease in Pediatrics and Adults: An Exploratory Analysis Using Data from the Center for International Blood and Marrow Transplant Research. Biol Blood Marrow Transplant 2016; 22:
-1312. https://doi.org/10.1016/j.bbmt.2016.04.011
Triplett BM, Kuttab HI, Kang G, Leung W. Escalation to High-Dose Defibrotide in Patients with Hepatic Veno-Occlusive Disease. Biol Blood Marrow Transplant 2015, 21: 2148-2153. https://doi.org/10.1016/j.bbmt.2015.08.013
Richardson PG, Soiffer RJ, Antin JH et al. Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease and Multiorgan Failure after Stem Cell Transplantation: A Multicenter, Randomized, Dose-Finding Trial. Biol Blood Marrow Transplant 2010; 16:1005-1017. https://doi.org/10.1016/j.bbmt.2010.02.009
Grupp SA, Smith AR, Triplett BM et al., Timing of Initiation of Defibrotide Post-Diagnosis of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome Post-Hematopoietic Stem Cell Transplantation: exploratory age-group analysis from an expanded access study. Blood 2016; 128: Abstracts 66.
Richardson PG, Smith AR, Triplett BM, et al. Early Initiation of Defibrotide in Patients with Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome Following Hematopoietic Stem Cell Transplantation Improves Day +100 Survival. ASH 2015. Poster Abs.
31. Ohashi K, Tanabe J, Watanabe R et al. The Japanese Multicenter Open Randomized Trial of Ursodeoxycholic Acid Prophylaxis for Hepatic Veno-Occlusive Disease After Stem Cell Transplantation. American Journal of Hematology 2000; 64: 32-38. https://doi.org/10.1002/(SICI)1096-8652(200005)64:1<32::AID-AJH6>3.0.CO;2-N
Ruutu T, Eriksson B, Remes K et al. Ursodeoxycholic acid for the prevention of hepatic complications in allogeneic stem cell transplantation. Blood. 2002; 100: 1977-1983. https://doi.org/10.1182/blood-2001-12-0159
Park SH, Lee MH, Lee HA et al. Randomized trial of heparin plus ursodiol vs heparin alone to prevent hepatic veno-occlusive disease after hematopoietic stem cell transplantation. Bone Marrow Transplantation 2002; 29:
-143. https://doi.org/10.1038/sj.bmt.1703342
Sucak GT, Ak ZS, Yag M et al. Treatment of Sinusoidal Obstruction Syndrome With Defibrotide: A Single-Center Experience. Transplantation Proceedings 2007; 39: 1558-1563. https://doi.org/10.1016/j.transproceed.2007.01.075
Corbacioglu S, Greil J, Peters C et al. Defibrotide in the treatment of children with veno-occlusive disease (VOD): a retrospective multicentre study demonstrates therapeutic efficacy upon early intervention. Bone Marrow Transplantation 2004; 33: 189-195. https://doi.org/10.1038/sj.bmt.1704329
Corbacioglu S, Carreras E, Mohty M et al. Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease: Final Results From the International Compassionate-Use Program. Biol Blood Marrow Transplant 2016,22: 1874-1882. https://doi.org/10.1016/j.bbmt.2016.07.001
Richardson PG, Smith AR, Triplett BM et al. Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) Post-Hematopoietic Stem Cell Transplantation (HSCT) in Patients with Acute Leukemias: A Subgroup Analysis from the Defibrotide Expanded-Access program. Blood 2016, 128: Abstracts 3412.
Downloads
Published
Issue
Section
License
Policy for Journals/Articles with Open Access
Authors who publish with this journal agree to the following terms:- Authors retain copyright and grant the journal right of first publication with the work simultaneously licensed under a Creative Commons Attribution License that allows others to share the work with an acknowledgement of the work's authorship and initial publication in this journal.
- Authors are permitted and encouraged to post links to their work online (e.g., in institutional repositories or on their website) prior to and during the submission process, as it can lead to productive exchanges, as well as earlier and greater citation of published work
Policy for Journals / Manuscript with Paid Access
Authors who publish with this journal agree to the following terms:- Publisher retain copyright .
- Authors are permitted and encouraged to post links to their work online (e.g., in institutional repositories or on their website) prior to and during the submission process, as it can lead to productive exchanges, as well as earlier and greater citation of published work .