Hepatic veno-occlusive disease

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For placebo or observational studies in this condition, please visit this page.
Also known as sinusoidal obstructive syndrome (SOS)
Last updated on 2024-07-23:2 regimens on this page
2 variants on this page
=Prophylaxis=
==Defibrotide monotherapy==
===Regimen===
Study 	Dates of enrollment 	Evidence 	Comparator 	Comparative Efficacy
Corbacioglu et al. 2012 (VOD-DF)	2006-2009 	Phase 3 (E-esc)	Observation 	Seems to have lower incidence of VOD by day +30 (primary endpoint)
Grupp et al. 2023 (HARMONY) 	2017-01-11 to 2020-10-20	Phase 3 (E-esc)	Best supportive care	Did not meet primary endpoint of VOD-free survival by day +30
====Supportive therapy====
*Defibrotide (Defitelio) 6.25 mg/kg IV over 2 hours every 6 hours
Begins on first day of preparative regimen, continues for at least 14 days or until day +30
===References===
#VOD-DF: Corbacioglu S, Cesaro S, Faraci M, Valteau-Couanet D, Gruhn B, Rovelli A, Boelens JJ, Hewitt A, Schrum J, Schulz AS, Müller I, Stein J, Wynn R, Greil J, Sykora KW, Matthes-Martin S, Führer M, O'Meara A, Toporski J, Sedlacek P, Schlegel PG, Ehlert K, Fasth A, Winiarski J, Arvidson J, Mauz-Körholz C, Ozsahin H, Schrauder A, Bader P, Massaro J, D'Agostino R, Hoyle M, Iacobelli M, Debatin KM, Peters C, Dini G. Defibrotide for prophylaxis of hepatic veno-occlusive disease in paediatric haemopoietic stem-cell transplantation: an open-label, phase 3, randomised controlled trial. Lancet. 2012 Apr 7;379(9823):1301-9. Epub 2012 Feb 23. link to original article dosing details in manuscript have been reviewed by our editors EuroPMC NCT00272948
#HARMONY: Grupp SA, Corbacioglu S, Kang HJ, Teshima T, Khaw SL, Locatelli F, Maertens J, Stelljes M, Stepensky P, Lopez P, Amber V, Pagliuca A, Richardson PG, Mohty M. Defibrotide plus best standard of care compared with best standard of care alone for the prevention of sinusoidal obstruction syndrome (HARMONY): a randomised, multicentre, phase 3 trial. Lancet Haematol. 2023 May;10(5):e333-e345. Epub 2023 Mar 28. Erratum in: Lancet Haematol. 2023 Jul;10(7):e490. link to original article EuroPMC NCT02851407
=Treatment=
==Defibrotide monotherapy==
===Regimen===
Study 	Dates of enrollment	Evidence 	Comparator 	Comparative Efficacy
Richardson et al. 2016 (DFCI 2005-01)	2006-2008 	Phase 3 (E-esc)	"32 historical controls"	Seems to have higher survival at day +100 (primary endpoint)
Note: If after 21 days signs and symptoms of VOD have not resolved, give until VOD is resolved, up to a maximum of 60 days.
====Supportive therapy====
*Defibrotide (Defitelio) 6.25 mg/kg IV over 2 hours every 6 hours
21- to 60-day course
===References===
#DFCI 2005-01: Richardson PG, Riches ML, Kernan NA, Brochstein JA, Mineishi S, Termuhlen AM, Arai S, Grupp SA, Guinan EC, Martin PL, Steinbach G, Krishnan A, Nemecek ER, Giralt S, Rodriguez T, Duerst R, Doyle J, Antin JH, Smith A, Lehmann L, Champlin R, Gillio A, Bajwa R, D'Agostino RB Sr, Massaro J, Warren D, Miloslavsky M, Hume RL, Iacobelli M, Nejadnik B, Hannah AL, Soiffer RJ. Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure. Blood. 2016 Mar 31;127(13):1656-65. Epub 2016 Jan 29. link to original article dosing details in manuscript have been reviewed by our editors link to PMC article EuroPMC NCT00358501
Category:Hepatic veno-occlusive disease regimens
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