Defibrotide for prophylaxis of hepatic veno-occlusive disease in paediatric haemopoietic stem-cell transplantation: An open-label, phase 3, randomised controlled trial

The Lancet

Volumn 379, Issue 9823, 2012, Pages 1301-1309

  Corbacioglu, Selim ^a   Cesaro, Simone ^b   Faraci, Maura ^c   Valteau Couanet, Dominique ^d   Gruhn, Bernd ^e   Rovelli, Attilio ^f   Boelens, Jaap J ^g   Hewitt, Annette ^h   Schrum, Johanna ⁱ   Schulz, Ansgar S ^j   Müller, Ingo ^k   Stein, Jerry ^l   Wynn, Robert ^m   Greil, Johann ⁿ   Sykora, Karl Walter ^o   Matthes Martin, Susanne ^p   Führer, Monika ^q   O'Meara, Anne ^r   Toporski, Jacek ^s   Sedlacek, Petr ^t   Schlegel, Paul G ^u   Ehlert, Karoline ^v   Fasth, Anders ^w   Winiarski, Jacek ^x   Arvidson, Johan ^y   Mauz Körholz, Christine ^z   Ozsahin, Hulya ^aa   Schrauder, Andre ^ab   Bader, Peter ^ac   Massaro, Joseph ^ad   D'Agostino, Ralph ^ad   Hoyle, Margaret ^ae   Iacobelli, Massimo ^ae   Debatin, Klaus Michael ^j   Peters, Christina ^p   Dini, Giorgio ^c   more..

^a UNIVERSITY HOSPITAL REGENSBURG   (Germany)

^b UNIVERSITY OF PADOVA   (Italy)

^c G GASLINI INSTITUTE   (Italy)

^d INSTITUT GUSTAVE ROUSSY   (France)

^e JENA UNIVERSITY HOSPITAL   (Germany)

^f UNIVERSITY OF MILANO BICOCCA   (Italy)

^g UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^h GREAT ORMOND STREET HOSPITAL   (United Kingdom)

ⁱ UNIVERSITY OF HAMBURG   (Germany)

^j UNIVERSITY HOSPITAL ULM   (Germany)

^k UNIVERSITY HOSPITAL TÜBINGEN   (Germany)

^l SCHNEIDER CHILDREN S MEDICAL CENTER OF ISRAEL   (Israel)

^m ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

ⁿ UNIVERSITY HOSPITAL HEIDELBERG   (Germany)

^o HANNOVER MEDICAL SCHOOL   (Germany)

^p ST ANNA CHILDREN S HOSPITAL   (Austria)

^q LUDWIG MAXIMILIANS UNIVERSITY   (Germany)

^r OUR LADY S CHILDREN S HOSPITAL   (Ireland)

^s LUND UNIVERSITY HOSPITAL   (Sweden)

^t UNIVERSITY HOSPITAL MOTOL   (Czech Republic)

^u UNIVERSITY HOSPITAL WÜRZBURG   (Germany)

^v UNIVERSITY HOSPITAL MÜNSTER   (Germany)

^w UNIVERSITY OF GOTHENBURG   (Sweden)

^x KAROLINSKA INSTITUTE   (Sweden)

^y UNIVERSITY CHILDREN S HOSPITAL   (Sweden)

^z MARTIN LUTHER UNIVERSITY HALLE WITTENBERG   (Germany)

^aa Hopital des Enfants Hopitaux universitaires de Geneve   (Switzerland)

^ab UNIVERSITY HOSPITAL SCHLESWIG HOLSTEIN   (Germany)

^ac UNIVERSITY HOSPITAL FRANKFURT   (Germany)

^ad BOSTON UNIVERSITY   (United States)

^ae Gentium SpA   (Italy)

more..

Author keywords

[No Author keywords available]

Indexed keywords

DEFIBROTIDE;

ABDOMINAL PAIN; ACUTE GRAFT VERSUS HOST DISEASE; ADOLESCENT; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTICLE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; BLEEDING; BLOOD CLOTTING DISORDER; BLOOD DISEASE; BLOODY DIARRHEA; CHILD; CHRONIC GRAFT VERSUS HOST DISEASE; CONTROLLED STUDY; DRUG EFFICACY; DRUG FATALITY; DRUG SAFETY; EPISTAXIS; FEMALE; GASTROINTESTINAL HEMORRHAGE; HEMATEMESIS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOTHORAX; HIGH RISK PATIENT; HUMAN; INFANT; LIVER VENOOCCLUSIVE DISEASE; LUNG HEMORRHAGE; LYMPHATIC SYSTEM DISEASE; MAJOR CLINICAL STUDY; MALE; MEDIASTINUM DISEASE; MICROANGIOPATHY; MULTICENTER STUDY; MYELOABLATIVE CONDITIONING; NAUSEA; ORAL BLEEDING; PARTIAL THROMBOPLASTIN TIME; PHASE 3 CLINICAL TRIAL; PRESCHOOL CHILD; PRIORITY JOURNAL; PROTHROMBIN TIME; RANDOMIZED CONTROLLED TRIAL; RESPIRATORY TRACT DISEASE; RISK FACTOR; SCHOOL CHILD; SIDE EFFECT; THORAX DISEASE; UPPER GASTROINTESTINAL BLEEDING; VASCULAR DISEASE; VOMITING;

EID: 84859432625     PISSN: 01406736     EISSN: 1474547X     Source Type: Journal    
DOI: 10.1016/S0140-6736(11)61938-7     Document Type: Article

References (39)

1. Copelan EA. Hematopoietic stem-cell transplantation. N Engl J Med 2006; 354: 1813-26.
2. McDonald GB, Sharma P, Matthews DE, Shulman HM, Thomas ED. Venocclusive disease of the liver after bone marrow transplantation: diagnosis, incidence, and predisposing factors. Hepatology 1984; 4: 116-22. (Pubitemid 14185436)
3. Richardson P, Guinan E. The pathology, diagnosis, and treatment of hepatic veno-occlusive disease: current status and novel approaches. Br J Haematol 1999; 107: 485-93.
4. 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: 255-67. (Pubitemid 23046384)
5. 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: 157-68.
6. Horn B, Reiss U, Matthay K, McMillan A, Cowan M. Veno-occlusive disease of the liver in children with solid tumors undergoing autologous hematopoietic progenitor cell transplantation: a high incidence in patients with neuroblastoma. Bone Marrow Transplant 2002; 29: 409-15. (Pubitemid 34257715)
7. Lucarelli G, Clift RA, Galimberti M, et al. Marrow transplantation for patients with thalassemia: results in class 3 patients. Blood 1996; 87: 2082-88. (Pubitemid 26068926)
8. Lucarelli G, Galimberti M, Polchi P, et al. Bone marrow transplantation in patients with thalassemia. N Engl J Med 1990; 322: 417-21. (Pubitemid 20059509)
9. Barker CC, Butzner JD, Anderson RA, Brant R, Sauve RS. Incidence, survival and risk factors for the development of veno-occlusive disease in pediatric hematopoietic stem cell transplant recipients. Bone Marrow Transplant 2003; 32: 79-87. (Pubitemid 36850284)
10. Ringdén O, Ruutu T, Remberger M, et al. A randomized trial comparing busulfan vs total body irradiation in allogeneic marrow transplant recipients with hematological malignancies. Transplant Proc 1994; 26: 1831-32. (Pubitemid 24211809)
11. McKoy JM, Angelotta C, Bennett CL, et al. Gemtuzumab ozogamicin- associated sinusoidal obstructive syndrome (SOS): an overview from the research on adverse drug events and reports (RADAR) project. Leuk Res 2007; 31: 599-604. (Pubitemid 46578145)
12. Cesaro S, Pillon M, Talenti E, et al. A prospective survey on incidence, risk factors and therapy of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation. Haematologica 2005; 90: 1396-404. (Pubitemid 41503656)
13. Reiss U, Cowan M, McMillan A, Horn B. Hepatic venoocclusive disease in blood and bone marrow transplantation in children and young adults: incidence, risk factors, and outcome in a cohort of 241 patients. J Pediatr Hematol Oncol 2002; 24: 746-50. (Pubitemid 35448741)
14. Corbacioglu S, Hönig 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: 547-53. (Pubitemid 44509547)
15. Ouachée-Chardin M, Elie C, de Saint Basile G, et al. Hematopoietic stem cell transplantation in hemophagocytic lymphohistiocytosis: a single-center report of 48 patients. Pediatrics 2006; 117: e743-50. (Pubitemid 46071479)
16. Schulz AS, Classen CF, Mihatsch WA, et al. HLA-haploidentical blood progenitor cell transplantation in osteopetrosis. Blood 2002; 99: 3458-60. (Pubitemid 34525331)
17. Carreras E, Bertz H, Arcese W, et al, and the European Group for Blood and Marrow Transplantation Chronic Leukemia Working Party. 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 1998; 92: 3599-604. (Pubitemid 28525185)
18. 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: 334-40. (Pubitemid 35177465)
19. Mitsiades CS, Rouleau C, Echart C, et al. Preclinical studies in support of defibrotide for the treatment of multiple myeloma and other neoplasias. Clin Cancer Res 2009; 15: 1210-21.
20. Ostrovsky O, Shimoni A, Rand A, Vlodavsky I, Nagler A. Genetic variations in the heparanase gene (HPSE) associate with increased risk of GVHD following allogeneic stem cell transplantation: Effect of discrepancy between recipients and donors. Blood 2010; 115: 2319-28.
21. Echart CL, Graziadio B, Somaini S, et al. The fibrinolytic mechanism of defibrotide: Effect of defibrotide on plasmin activity. Blood Coagul Fibrinolysis 2009; 20: 627-34.
22. Falanga A, Vignoli A, Marchetti M, Barbui T. Defibrotide reduces procoagulant activity and increases fibrinolytic properties of endothelial cells. Leukemia 2003; 17: 1636-42. (Pubitemid 36986967)
23. 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: 1122-29. (Pubitemid 32104928)
24. 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: 189-95. (Pubitemid 38180932)
25. 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: 4337-43. (Pubitemid 35429671)
26. 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: 1005-17.
27. Richardson P, Tomblyn M, Kernan N, et al. Defibrotide (DF) in the treatment of severe hepatic veno-occlusive disease (VOD) with multi-organ failure (MOF) following stem cell transplantation (SCT): results of a phase 3 study utilizing a historical control. ASH Annual Meeting Abstracts 2009; 114: 654.
28. 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. (Pubitemid 38519573)
29. 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. (Pubitemid 47018417)
30. 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: 831-32. (Pubitemid 351354794)
31. Rowlings PA, Przepiorka D, Klein JP, et al. IBMTR Severity Index for grading acute graft-versus-host disease: retrospective comparison with Glucksberg grade. Br J Haematol 1997; 97: 855-64. (Pubitemid 27321388)
32. Ruutu T, Barosi G, Benjamin RJ, et al, and the European Group for Blood and Marrow Transplantation, and the European LeukemiaNet. Diagnostic criteria for hematopoietic stem cell transplant-associated microangiopathy: results of a consensus process by an International Working Group. Haematologica 2007; 92: 95-100. (Pubitemid 46232661)
33. Vassal G, Koscielny S, Challine D, et al. Busulfan disposition and hepatic veno-occlusive disease in children undergoing bone marrow transplantation. Cancer Chemother Pharmacol 1996; 37: 247-53.
34. Tai BC, Machin D, White I, Gebski V, and the EOI (The European Osteosarcoma Intergroup). Competing risks analysis of patients with osteosarcoma: a comparison of four different approaches. Stat Med 2001; 20: 661-84. (Pubitemid 32177843)
35. Aalen OO, Johansen S. An empirical transition matrix for non-homogeneous Markov chains based on censored observations. Scand J Stat 1978; 5: 141-50.
36. Satagopan JM, Ben-Porat L, Berwick M, Robson M, Kutler D, Auerbach AD. A note on competing risks in survival data analysis. Br J Cancer 2004; 91: 1229-35. (Pubitemid 39424779)
37. Jones RJ, Lee KS, Beschorner WE, et al. Venoocclusive disease of the liver following bone marrow transplantation. Transplantation 1987; 44: 778-83. (Pubitemid 18034278)
38. Carreras E, Grañena A, Navasa M, et al. On the reliability of clinical criteria for the diagnosis of hepatic veno-occlusive disease. Ann Hematol 1993; 66: 77-80. (Pubitemid 23079672)
39. Ertault-Daneshpouy M, Leboeuf C, Lemann M, et al. Pericapillary hemorrhage as criterion of severe human digestive graft-versus-host disease. Blood 2004; 103: 4681-84. (Pubitemid 38746004)