Hepatic veno-occlusive disease - null regimens

The purpose of this page is to provide references to "null therapy" such as placebo and observation. These references provide further insight into the historical development of the treatment landscape. See the main VOD/SOS page for regimens that include active treatment.

Prophylaxis

Observation

Regimen

Study	Dates of enrollment	Evidence	Comparator	Comparative Efficacy
Corbacioglu et al. 2012 (VOD-DF)	2006-2009	Phase 3 (C)	Defibrotide	Seems to have higher incidence of VOD by day +30 (primary endpoint)
Grupp et al. 2023 (HARMONY)	2017-01-11 to 2020-10-20	Phase 3 (C)	Defibrotide	Did not meet primary endpoint of VOD-free survival by day +30

No active treatment.

References

1. 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 PubMed NCT00272948
2. 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 PubMed NCT02851407