m and 176 pts in control arm. 40% reduction in VOD within 30 days after SCT, p=0.0539 (ITT population). In per protocol population (164 treatment and 169 control), 40% reduction in the rate of incidence of VOD within 30 days after SCT, p=0.0366. (4)
31/03/2009 16:56:25
Nov 08: An interim review of the P3 trial by the Data Safety Monitoring Board (DSMB) suggests that the sample size needs to be increased to 160 pt in the treatment arm (currently 102) and 80 in the historical control arm in order to be 80% powered to detect a p value of 0.01, which is required by the FDA. Increased recruitment to the treatment arm is unlikely. The DSMB did not raise any safety issues or recommend that the trail is stopped due to futility (3)
13/01/2009 10:46:58
Evidence Based eva luations
AWMSG http://www.awmsg.org/awmsgonline/app/appraisalinfo/508
SMC http://www.scottishmedicines.org.uk/SMC_Advice/Advice/967_14_defibrotide_Defitelio/defibrotide_Defitelio
LNDG http://www.medicinesresources.nhs.uk/upload/Defibrotide.pdf
NHSC/NIHR http://www.hsc.nihr.ac.uk/topics/defibrotide-defitelio-for-hepatic-veno-occlusive-d/
EMA doc http://www.ema.europa.eu/ema/pages/includes/document/open_document.jsp?webContentId=WC500146652
References
Available only to registered users
Category
BNF Category: Peripheral vasodilators and related drugs (02.06.04)
Pharmacology: Cytokine modulator
Epidemiology: Hepatic VOD is caused by toxic injury leading to hepatic vein obstruction and is associated with the pre-conditioning chemotherapy regimen for HSCT. Up to 17% of patients who receive HSCT develop VOD and about 33% progress to severe VOD. Without treatment 80% of those with severe VOD die within 100 days of HSCT.
Indication: Veno-occlusive disorders
Additional Details: hepatic VOD, treatment and prevention in HSCT
Method(s) of Administration
Intravenous infusion
Company Information
Name: Jazz Pharmaceuticals
US Name: Gentium
Further Information
Anticipated commissioning route (England) NHSE
High cost drug list? Yes
Tariff Not routinely commissioned by NHSE - IFR approval [28]
Implications Available only to registered users
