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Drug Profile

Defibrotide for children and adults with hepatic veno-occlusive disease post hematopoietic cell transplantation

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Pages 885-898 | Received 09 Jun 2017, Accepted 18 Aug 2017, Published online: 04 Sep 2017

References

  • Wallhult E, Kenyon M, Liptrott S, et al. Management of veno-occlusive disease: the multidisciplinary approach to care. Eur J Haematol. 2017;98(4):322–329.
  • 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.
  • Corbacioglu S, Carreras E, Ansari M, et al. Diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in pediatric patients: a new classification from the European Society For Blood and Marrow Transplantation. Bone Marrow Transplant. published online July 31 2017. doi: 10.1038/bmt.2017.161.
  • Dalle JH, Giralt SA. Hepatic veno-occlusive disease after hematopoietic stem cell transplantation: risk factors and stratification, prophylaxis, and treatment. Biol Blood Marrow Transplant. 2016;22(3):400–409.
  • 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.
  • Dvorak CC, Nejadnik B, Cao Z, et al. Hospital cost associated with veno-occlusive disease (VOD) in patients with hematopoietic stem cell transplant (HSCT) [ASH abstract 4452]. Biol Blood Marrow Transplant. 2016;22(3 Suppl):S275.
  • Barker CC, Butzner JD, Anderson RA, et al. Incidence, survival and risk factors for the development of veno-occlusive disease in pediatric hematopoietic stem cell transplant recipients. Bone Marrow Transplant. 2003;32(1):79–87.
  • Corbacioglu S, Kernan N, Lehmann L, et al. Defibrotide for the treatment of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation. Expert Rev Hematol. 2012;5(3):291–302.
  • Carreras E, Diaz-Beya M, Rosinol L, et al. The incidence of veno-occlusive disease following allogeneic hematopoietic stem cell transplantation has diminished and the outcome improved over the last decade. Biol Blood Marrow Transplant. 2011;17(11):1713–1720.
  • Tsirigotis PD, Resnick IB, Avni B, et al. Incidence and risk factors for moderate-to-severe veno-occlusive disease of the liver after allogeneic stem cell transplantation using a reduced intensity conditioning regimen. Bone Marrow Transplant. 2014;49(11):1389–1392.
  • Corbacioglu S, Honig M, Lahr G, et al. Stem cell transplantation in children with infantile osteopetrosis is associated with a high incidence of VOD, which could be prevented with defibrotide. Bone Marrow Transplant. 2006;38(8):547–553.
  • Schulz AS, Classen CF, Mihatsch WA, et al. HLA-haploidentical blood progenitor cell transplantation in osteopetrosis. Blood. 2002;99(9):3458–3460.
  • Cesaro S, Spiller M, Sartori MT, et al. Veno-occlusive disease in pediatric patients affected by Wilms tumor. Pediatr Blood Cancer. 2011;57(2):258–261.
  • Elli M, Pinarli FG, Dagdemir A, et al. Veno-occlusive disease of the liver in a child after chemotherapy for brain tumor. Pediatr Blood Cancer. 2006;46(4):521–523.
  • Sulis ML, Bessmertny O, Granowetter L, et al. Veno-occlusive disease in pediatric patients receiving actinomycin D and vincristine only for the treatment of rhabdomyosarcoma. J Pediatr Hematol Oncol. 2004;26(12):843–846.
  • Czauderna P, Katski K, Kowalczyk J, et al. Venoocclusive liver disease (VOD) as a complication of Wilms’ tumour management in the series of consecutive 206 patients. Eur J Pediatr Surg. 2000;10(5):300–303.
  • Jones RJ, Lee KS, Beschorner WE, et al. Venoocclusive disease of the liver following bone marrow transplantation. Transplantation. 1987;44(6):778–783.
  • McDonald GB, Hinds MS, Fisher LD, et al. Veno-occlusive disease of the liver and multiorgan failure after bone marrow transplantation: a cohort study of 355 patients. Ann Intern Med. 1993;118(4):255–267.
  • 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 Transplant. 2015;50(6):781–789.
  • Rio B, Andreu G, Nicod A, et al. Thrombocytopenia in venocclusive disease after bone marrow transplantation or chemotherapy. Blood. 1986;67(6):1773–1776.
  • Bearman SI. The syndrome of hepatic veno-occlusive disease after marrow transplantation. Blood. 1995;85(11):3005–3020.
  • 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.
  • 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.
  • Johnson DB, Savani BN. How can we reduce hepatic veno-occlusive disease-related deaths after allogeneic stem cell transplantation? Exp Hematol. 2012;40(7):513–517.
  • Hod E, Schwartz J. Platelet transfusion refractoriness. Br J Haematol. 2008;142(3):348–360.
  • 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.
  • Carreras E. How I manage sinusoidal obstruction syndrome after haematopoietic cell transplantation. Br J Haematol. 2015;168(4):481–491.
  • Cheuk DK, Chiang AK, Ha SY, et al. Interventions for prophylaxis of hepatic veno-occlusive disease in people undergoing haematopoietic stem cell transplantation. Cochrane Database Syst Rev. 2015;(5):CD009311.
  • 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 Transplant. 2006;37(7):677–686.
  • 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(11):2834–2840.
  • Or R, Nagler A, Shpilberg O, et al. Low molecular weight heparin for the prevention of veno-occlusive disease of the liver in bone marrow transplantation patients. Transplantation. 1996;61(7):1067–1071.
  • 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(5):346–354.
  • Ruutu T, Eriksson B, Remes K, et al. Ursodeoxycholic acid for the prevention of hepatic complications in allogeneic stem cell transplantation. Blood. 2002;100(6):1977–1983.
  • 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. Biol Blood Marrow Transplant. 2007;13(2):206–217.
  • Tsochatzis EA, Feudjo M, Rigamonti C, et al. Ursodeoxycholic acid improves bilirubin but not albumin in primary biliary cirrhosis: further evidence for nonefficacy. Biomed Res Int. 2013;2013:139763.
  • 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 Transplant. 2004;33(2):189–195.
  • Grupp SA, Smith AR, Triplett BM, et al. An exploratory post hoc analysis of survival by age and time to defibrotide initiation after diagnosis of hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) in patients receiving hematopoietic stem cell transplantation (HSCT) Poster session presented at: 5th Annual American Society of Pediatric Hematology/Oncology–Pediatric Blood and Marrow Transplant Consortium (ASPHO-PBMTC) Educational Meeting; 2017 Apr 26; Montreal, Quebec, Canada. Poster 1007.
  • Defitelio® (defibrotide) [EU SPC]. Summary of product characteristics (Europe). Villa Guardia, 22079 Italy: Gentium Srl; 2016.
  • Defitelio® (defibrotide sodium) [US PI]. Prescribing information (United States). Palo Alto, CA 94304: Jazz Pharmaceuticals, Inc; 2016.
  • Bianchi G, Barone D, Lanzarotti E, et al. Defibrotide, a single-stranded polydeoxyribonucleotide acting as an adenosine receptor agonist. Eur J Pharmacol. 1993;238(2–3):327–334.
  • Pescador R, Capuzzi L, Mantovani M, et al. Defibrotide: properties and clinical use of an old/new drug. Vascul Pharmacol. 2013;59(1–2):1–10.
  • Bracht F, Schror K. Isolation and identification of aptamers from defibrotide that act as thrombin antagonists in vitro. Biochem Biophys Res Commun. 1994;200(2):933–937.
  • Palomo M, Diaz-Ricart M, Rovira M, et al. Defibrotide prevents the activation of macrovascular and microvascular endothelia caused by soluble factors released to blood by autologous hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2011;17(4):497–506.
  • Benimetskaya L, Wu S, Voskresenskiy AM, et al. Angiogenesis alteration by defibrotide: implications for its mechanism of action in severe hepatic veno-occlusive disease. Blood. 2008;112(10):4343–4352.
  • Echart CL, Graziadio B, Somaini S, et al. The fibrinolytic mechanism of defibrotide: effect of defibrotide on plasmin activity. Blood Coagul Fibrinolysis. 2009;20(8):627–634.
  • Falanga A, Vignoli A, Marchetti M, et al. Defibrotide reduces procoagulant activity and increases fibrinolytic properties of endothelial cells. Leukemia. 2003;17(8):1636–1642.
  • Palomo M, Mir E, Rovira M, et al. What is going on between defibrotide and endothelial cells? Snapshots reveal the hot spots of their romance. Blood. 2016;127(13):1719–1727.
  • Schroder H. Defibrotide protects endothelial cells, but not L929 tumour cells, from tumour necrosis factor-alpha-mediated cytotoxicity. J Pharm Pharmacol. 1995;47(3):250–252.
  • Scalia R, Kochilas L, Campbell B, et al. Effects of defibrotide on leukocyte-endothelial cell interaction in the rat mesenteric vascular bed: role of P-selectin. Methods Find Exp Clin Pharmacol. 1996;18(10):669–676.
  • Eissner G, Multhoff G, Gerbitz A, et al. Fludarabine induces apoptosis, activation, and allogenicity in human endothelial and epithelial cells: protective effect of defibrotide. Blood. 2002;100(1):334–340.
  • Nunziata A, Mariani MF, Cascone M, et al. Increased plasma t-PA activity and urinary PGE2 after repeated oral administration of defibrotide to the rat and the mouse. Thromb Res. 1991;64(2):279–284.
  • Zhou Q, Chu X, Ruan C. Defibrotide stimulates expression of thrombomodulin in human endothelial cells. Thromb Haemost. 1994;71(4):507–510.
  • Coccheri S, Biagi G, Legnani C, et al. Acute effects of defibrotide, an experimental antithrombotic agent, on fibrinolysis and blood prostanoids in man. Eur J Clin Pharmacol. 1988;35(2):151–156.
  • Palmer KJ, Goa KL. Defibrotide. A review of its pharmacodynamic and pharmacokinetic properties, and therapeutic use in vascular disorders. Drugs. 1993;45(2):259–294.
  • Tocchetti P, Tudone E, Marier J-F, et al. Pharmacokinetic profile of defibrotide in patients with renal impairment. Drug Des Devel Ther. 2016;10:2631–2641.
  • Richardson PG, Elias AD, Krishnan A, et al. Treatment of severe veno-occlusive disease with defibrotide: compassionate use results in response without significant toxicity in a high-risk population. Blood. 1998;92(3):737–744.
  • Chopra R, Eaton JD, Grassi A, et al. Defibrotide for the treatment of hepatic veno-occlusive disease: results of the European compassionate-use study. Br J Haematol. 2000;111(4):1122–1129.
  • Richardson PG, Murakami C, Jin Z, et al. Multi-institutional use of defibrotide in 88 patients after stem cell transplantation with severe veno-occlusive disease and multisystem organ failure: response without significant toxicity in a high-risk population and factors predictive of outcome. Blood. 2002;100(13):4337–4343.
  • 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(7):1005–1017.
  • 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(7):1306–1312.
  • 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(13):1656–1665.
  • 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(10):1874–1882.
  • Richardson PG, Smith AR, Triplett BM, et al. Defibrotide for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome: interim results from a treatment IND study. Biol Blood Marrow Transplant. 2017;23(6):997–1004.
  • Kernan NA, Richardson PG, Triplett BM, et al. Efficacy and safety of defibrotide to treat hepatic veno-occlusive disease/sinusoidal obstruction syndrome after primary chemotherapy: a post hoc analysis of final data. J Clin Oncol. 2017;35(suppl):abstr 10513.
  • Richardson PG, Smith AR, Triplett BM, et al. Earlier defibrotide initiation post-diagnosis of veno-occlusive disease/sinusoidal obstruction syndrome improves Day +100 survival following haematopoietic stem cell transplantation. Br J Haematol. 2017. cited 2017 Jun 9. DOI:10.1111/bjh.14727.
  • 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(5):347–354.
  • Chalandon Y, Simonetta F, Dantin C, et al. Efficient prophylaxis with defibrotide for sinusoidal obstruction syndrome (SOS) after allogeneic hematopoietic stem cell transplantation (HSCT). Blood. 2016;128:2204.
  • 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(1):79–82.
  • Qureshi A, Marshall L, Lancaster D. Defibrotide in the prevention and treatment of veno-occlusive disease in autologous and allogeneic stem cell transplantation in children. Pediatr Blood Cancer. 2008;50(4):831–832.
  • Zhang L, Wang Y, Huang H. Defibrotide for the prevention of hepatic veno-occlusive disease after hematopoietic stem cell transplantation: a systematic review. Clin Transplant. 2012;26(4):511–519.
  • Richardson PG, Corbacioglu S, Ho VT, et al. Drug safety evaluation of defibrotide. Expert Opin Drug Saf. 2013;12(1):123–136.

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