Defibrotide for the management of sinusoidal obstruction syndrome in patients who undergo haemopoietic stem cell transplantation

Cancer Treatment Reviews

Volumn 50, Issue , 2016, Pages 200-204

  Coutsouvelis, John ^a,b   Avery, Sharon ^c   Dooley, Michael ^a,b   Kirkpatrick, Carl ^b   Spencer, Andrew ^c  

^a Pharmacy Department   (Australia)

^b MONASH UNIVERSITY   (Australia)

^c ALFRED HOSPITAL   (Australia)

Author keywords

Defibrotide; Haemopoietic stem cell transplantation; Sinusoidal obstruction syndrome; Veno occlusive disease

Indexed keywords

DEFIBROTIDE; PLACEBO; TINZAPARIN; URSODEOXYCHOLIC ACID; ANTITHROMBOCYTIC AGENT; POLYDEOXYRIBONUCLEOTIDE;

BLEEDING; DISEASE SEVERITY; DRUG DOSE COMPARISON; DRUG DOSE ESCALATION; DRUG DOSE INCREASE; DRUG EFFICACY; DRUG SAFETY; ENDOTHELIUM CELL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HYPOTENSION; LIVER BIOPSY; LIVER VENOOCCLUSIVE DISEASE; MORTALITY RATE; MULTIPLE ORGAN FAILURE; OVERALL SURVIVAL; REVIEW; THALASSEMIA MAJOR; ADULT; CHILD; DISEASE MANAGEMENT; HEPATIC VENO-OCCLUSIVE DISEASE;

ADULT; CHILD; DISEASE MANAGEMENT; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEPATIC VENO-OCCLUSIVE DISEASE; HUMANS; PLATELET AGGREGATION INHIBITORS; POLYDEOXYRIBONUCLEOTIDES;

EID: 84990176997     PISSN: 03057372     EISSN: 15321967     Source Type: Journal    
DOI: 10.1016/j.ctrv.2016.09.014     Document Type: Review

References (38)

1. [1] Carreras, E., How I manage sinusoidal obstruction syndrome after haematopoietic cell transplantation. Br J Haematol 168:4 (2015), 481–491.
2. [2] Dignan, F.L., Wynn, R.F., Hadzic, N., Karani, J., Quaglia, A., Pagliuca, A., et al. BCSH/BSBMT guideline: diagnosis and management of veno-occlusive disease (sinusoidal obstruction syndrome) following haematopoietic stem cell transplantation. Br J Haematol 163:4 (2013), 444–457.
3. [3] Senzolo, M., Germani, G., Cholongitas, E., Burra, P., Burroughs, A.K., Veno occlusive disease: update on clinical management. World J Gastroenterol 13:29 (2007), 3918–3924.
4. [4] Carreras, E., Bertz, H., Arcese, W., Vernant, J.-P., Tomas, J.-F., Hagglund, H., et al. Incidence and outcome of hepatic veno-occlusive disease after blood or marrow transplantation: a prospective cohort study of the European Group for Blood and Marrow Transplantation. Blood 92:10 (1998), 3599–3604.
5. [5] Carreras, E., Diaz-Beya, M., Rosinol, L., Martinez, C., Fernandez-Aviles, F., Rovira, M., 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 17:11 (2011), 1713–1720.
6. [6] Ho, V.T., Revta, C., Richardson, P.G., Hepatic veno-occlusive disease after hematopoietic stem cell transplantation: update on defibrotide and other current investigational therapies. Bone Marrow Transplant 41:3 (2008), 229–237.
7. [7] McDonald, G.B., Sharma, P., Matthews, D.E., Shulman, H.M., Thomas, E.D., Venocclusive disease of the liver after bone marrow transplantation: diagnosis, incidence, and predisposing factors. Hepatology 4:1 (1984), 116–122.
8. [8] Jones, R.J., Lee, K.S., Beschorner, W.E., Vogel, V.G., Grochow, L.B., Braine, H.G., et al. Venoocclusive disease of the liver following bone marrow transplantation. Transplantation 44:6 (1987), 778–783.
9. [9] Ramasamy, K., Lim, Z.Y., Pagliuca, A., Grundy, R., Devereux, S., Ho, A.Y., et al. Incidence and management of hepatic venoocclusive disease in 237 patients undergoing reduced-intensity conditioning (RIC) haematopoietic stem cell transplantation (HSCT). Bone Marrow Transplant 38:12 (2006), 823–824.
10. [10] Coppell, J.A., Richardson, P.G., Soiffer, R., Martin, P.L., Kernan, N.A., Chen, A., et al. Hepatic veno-occlusive disease following stem cell transplantation: incidence, clinical course, and outcome. Biol Blood Marrow Transplant 16:2 (2010), 157–168.
11. [11] Pescador, R., Capuzzi, L., Mantovani, M., Fulgenzi, A., Ferrero, M.E., Defibrotide: properties and clinical use of an old/new drug. Vascul Pharmacol 59:1–2 (2013), 1–10.
12. [12] Carmona, A., Diaz-Ricart, M., Palomo, M., Molina, P., Pino, M., Rovira, M., et al. Distinct deleterious effects of cyclosporine and tacrolimus and combined tacrolimus-sirolimus on endothelial cells: protective effect of defibrotide. Biol Blood Marrow Transplant 19:10 (2013), 1439–1445.
13. [13] Palomo, M., Diaz-Ricart, M., Rovira, M., Escolar, G., Carreras, E., 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 17:4 (2011), 497–506.
14. [14] Corbacioglu, S., Cesaro, S., Faraci, M., Valteau-Couanet, D., Gruhn, B., Rovelli, A., 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 379:9823 (2012), 1301–1309.
15. [15] Cappelli, B., Chiesa, R., Evangelio, C., Biffi, A., Roccia, T., Frugnoli, I., et al. Absence of VOD in paediatric thalassaemic HSCT recipients using defibrotide prophylaxis and intravenous Busulphan. Br J Haematol 147:4 (2009), 554–560.
16. [16] Gray, J.C., Marshall, L., Qureshi, A., Ridwana, R., Sankpal, S., Ethell, M., et al. Defibrotide as prophylaxis for veno-occlusive disease in children undergoing allogenic and autologous stem cell transplantation. Bone Marrow Transplant, 41, 2008, S298.
17. [17] 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 50:4 (2008), 831–832.
18. [18] Versluys, B., Bhattacharaya, R., Steward, C., Cornish, J., Oakhill, A., Goulden, N., Prophylaxis with defibrotide prevents veno-occlusive disease in stem cell transplantation after gemtuzumab ozogamicin exposure. Blood, 103(5), 2004, 1968.
19. [19] Cheuk, D.K.L., Chiang, A.K.S., Ha, S.Y., Chan, G.C.F., Interventions for prophylaxis of hepaticveno-occlusive disease in people undergoing haematopoietic stem cell transplantation. Cochrane Database Syst Rev(5), 2015, CD009311.
20. [20] 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 26:4 (2012), 511–519.
21. [21] Richardson, P.G., Soiffer, R.J., Antin, J.H., Uno, H., Jin, Z., Kurtzberg, J., 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 16:7 (2010), 1005–1017.
22. [22] Mohty, M., Malard, F., Abecassis, M., Aerts, E., Alaskar, A.S., Aljurf, 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 50:6 (2015), 781–789.
23. [23] Bulley, S.R., Strahm, B., Doyle, J., Dupuis, L.L., Defibrotide for the treatment of hepatic veno-occlusive disease in children. Pediatr Blood Cancer 48:7 (2007), 700–704.
24. [24] Corbacioglu, S., Carreras, E., Mohty, M., Pagliuca, A., Ballabio, M., Hume, R., et al. Defibrotide for the treatment of hepatic veno-occlusive disease: an update from the international compassionate use program in 710 patients. Biol Blood Marrow Transplant, 21(2), 2015, S108.
25. [25] Richardson, P.G., Smith, A.R., Triplett, B.M., Kernan, N.A., Grupp, S.A., Arai, S., et al. Updated results from the ongoing US treatment IND study using defibrotide for patients with hepatic veno-occlusive disease. Biol Blood Marrow Transplant 21:2 (2015), S109–S110.
26. [26] Cefalo, M.G., Maurizi, P., Arlotta, A., Scalzone, M., Attina, G., Ruggiero, A., et al. Hepatic veno-occlusive disease: a chemotherapy-related toxicity in children with malignancies. Pediatr Drugs 12:5 (2010), 277–284.
27. [27] Hassan, Z., Optimal approach to prevent veno-occlusive disease following hematopoietic stem cell transplantation in children. Pediatr Transplant 14:6 (2010), 683–687.
28. [28] Del Toro, G., Defibrotide in sinusoidal obstruction syndrome: mounting evidence in pediatric patients (Commentary on Qureshi et al., page 831). Pediatr Blood Cancer 50:4 (2008), 735–736.
29. [29] Tsakiras, D.A., Prophylaxis and treatment of heaptic veno-occlusive disease after pediatric stem cell transplantation. Haematologica, 91(6), 2006, 726B.
30. [30] Dignan, F., Gujral, D., Ethell, M., Evans, S., Treleaven, J., Morgan, G., et al. Prophylactic defibrotide in allogeneic stem cell transplantation: minimal morbidity and zero mortality from veno-occlusive disease. Bone Marrow Transplant 40:1 (2007), 79–82.
31. [31] Park, M., Park, H.J., Eom, H.S., Kwon, Y.J., Park, J.A., Lim, Y.J., et al. Safety and effects of prophylactic defibrotide for sinusoidal obstruction syndrome in hematopoietic stem cell transplantation. Ann Transplant 18 (2013), 36–42.
32. [32] Richardson, P., Elias, A.D., Krishnan, A., Wheeler, C., Nath, R., Hoppensteadt, D., et al. Treatment of severe veno-occlusive disease with defibrotide: compassionate use results in response without significant toxicity in a high-risk population. Blood 92:3 (1998), 737–744.
33. [33] Richardson, P.G., Murakami, C., Jin, Z., Warren, D., Momtaz, P., Hoppensteadt, D., 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 100:13 (2002), 4337–4343.
34. [34] Richardson, P., Riches, M.L., Keman, N.A., Brochstein, J., Mineishi, S., Termuhlen, A.M., et al. Phase 3 trial ofdefibrotide for the treatment of severe veno-occlusive disease and multi-organ failure. Blood 127:13 (2016), 1656–1665.
35. [35] Richardson, P.G., Kernan, N.A., Grupp, S.A., Martin, P.L., Soiffer, R.J., Martin, R., et al. Defibrotide for the treatment of severe hepatic veno-occlusive disease: an analysis of clinical benefit as determined by number needed to treat (NNT) to achieve complete response and to improve survival. Biol Blood Marrow Transplant, 21(2), 2015, S110.
36. [36] Sucak, G.T., Aki, Z.S., Yagci, M., Yegin, Z.A., Ozkurt, Z.N., Haznedar, R., Treatment of sinusoidal obstruction syndrome with defibrotide: a single-center experience. Transplant Proc 39:5 (2007), 1558–1563.
37. [37] Chopra, R., Eaton, J.D., Grassi, A., Potter, M., Shaw, B., Salat, C., et al. Defibrotide for the treatment of hepatic veno-occlusive disease: results of the European compassionate-use study. Br J Haematol 111 (2000), 1122–1129.
38. [38] Dalle, J.H., Giralt, S.A., Hepatic veno-occlusive disease after hematopoietic stem cell transplantation: risk factors and stratification, prophylaxis, and treatment. Biol Blood Marrow Transplant 22:3 (2016), 400–409.