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Efficacy of Defitelio was evaluated in 689 patients1,2
Studies included a broad range of adult and pediatric patients with VOD with renal or pulmonary dysfunction following HSCT1-3
Renal or pulmonary dysfunctionc
of days on
every 6 hours
transplant, n (%)
study entry, n (%)
Defitelio was proven to improve survival at Day +100 consistently across 3 studies1,2
Survival at Day +100 post HSCT with Defitelio across studies in adult and pediatric patients1,2
EXPECTED DAY +100 SURVIVAL WITH SUPPORTIVE CARE
21% TO 31%
Based on published reports and analyses of patient-level data for individuals with hepatic VOD with renal or pulmonary dysfunction who received supportive care or interventions other than Defitelio.1
Delays in Defitelio initiation were associated with increased mortality at Day +1002
In an exploratory post hoc analysis from Study 32
- Analysis of Study 3 indicated that increased mortality at Day +100 was associated with longer delays in Defitelio administration following a diagnosis of VOD with renal or pulmonary dysfunction (confirmed by the Cochran-Armitage trend test; P<0.001)
- Analysis based on adult and pediatric patients (n=512) with a diagnosis of VOD with renal or pulmonary dysfunction
- The reason for initiation delay following diagnosis was not assessed in this expanded access study
aA diagnosis of VOD was made using Baltimore criteria (bilirubin ≥2 mg/dL and at least 2 of the following: hepatomegaly, ascites, and weight gain >5% by Day +21 post HSCT).1
bA diagnosis of VOD was made using Baltimore criteria (bilirubin ≥2 mg/dL and at least 2 of the following: ascites, weight gain >5% from baseline, hepatomegaly, or right upper quadrant pain) by Day +35 post HSCT.5
cA diagnosis of VOD was made using Baltimore criteria (bilirubin ≥2 mg/dL and at least 2 of the following: hepatomegaly, ascites, and weight gain >5%) by Day +35 post HSCT or modified Seattle criteria (presentation by Day 20 post HSCT of at least 2 of the following: bilirubin >2 mg/dL, hepatomegaly or right upper quadrant pain, and weight gain >2% of baseline).2,6,7
dDuration of treatment from first dose to last dose is presented because days without treatment were not captured for the expanded access study.
Defitelio® (defibrotide sodium) is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT).
IMPORTANT SAFETY INFORMATION
Defitelio is contraindicated in the following conditions:
- Concomitant administration with systemic anticoagulant or fibrinolytic therapy
- Known hypersensitivity to Defitelio or to any of its excipients
Warnings and Precautions
Defitelio may increase the risk of bleeding in patients with VOD after HSCT. Do not initiate Defitelio in patients with active bleeding. Monitor patients on Defitelio for signs of bleeding. If bleeding occurs, withhold or discontinue Defitelio.
Concomitant systemic anticoagulant or fibrinolytic therapy may increase the risk of bleeding and should be discontinued prior to Defitelio treatment. Consider delaying Defitelio administration until the effects of the anticoagulant have abated.
Hypersensitivity reactions including rash, urticaria, and angioedema have occurred in less than 2% of patients treated with Defitelio. One case of an anaphylactic reaction was reported in a patient who had previously received Defitelio. Monitor patients for hypersensitivity reactions, especially if there is a history of previous exposure. If a severe hypersensitivity reaction occurs, discontinue Defitelio, treat according to the standard of care, and monitor until symptoms resolve.
Most Common Adverse Reactions
The most common adverse reactions (incidence ≥10% and independent of causality) with Defitelio treatment were hypotension, diarrhea, vomiting, nausea, and epistaxis.Please see full Prescribing Information.
HSCT=hematopoietic stem-cell transplantation; VOD=veno-occlusive disease.
References: 1. Defitelio [package insert]. Palo Alto, CA: Jazz Pharmaceuticals. 2. Kernan NA, Grupp S, Smith AR, et al. Final results from a defibrotide treatment-IND study for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome. Br J Haematol. 2018;181(6):816-827. 3. Data on file. DEF-2018-022. Palo Alto, CA: Jazz Pharmaceuticals. 4. Richardson PG, Riches ML, Kernan NA, et al. Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure. Blood. 2016;127(13):1656-1665. 5. 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(7):1005-1017. 6. Jones RJ, Lee KS, Beschorner WE, et al. Venoocclusive disease of the liver following bone marrow transplantation. Transplantation. 1987;44(6):778-783. 7. 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(4):255-267.