Treatment with Defitelio®

Treatment with Defitelio®

Defitelio® met the primary endpoint of OS at Day +100 after HSCT* in the T-IND study:1

  • 49.5% (95% CI 45.0%, 53.8%) in patients with VOD with MOD (N=512)1
  • 43.0% (95% CI 32.5%, 53.0%) in adults with late-onset VOD with MOD (N=88)1

Note, VOD with MOD is also known as very severe VOD.2

* The open-label, single-arm T-IND study was designed to provide expanded access to Defitelio® and assess safety and survival in patients with hepatic VOD, with or without MOD, post-HSCT or post-non-transplant associated chemotherapy alone, treated with Defitelio®. The primary efficacy measure in the T-IND study was survival at Day +100 after HSCT or initiation of primary chemotherapy.

This treatment journey is designed by Jazz Pharmaceuticals for illustrative purposes.

Explore our Resources page for expert-led educational videos on treating severe VOD with Defitelio®.

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Licence and funding

Defitelio® is licensed and funded for the treatment of both classical and late-onset severe VOD.3,4

Defitelio® is indicated for the treatment of severe VOD in HSCT therapy in adults and in adolescents, children and infants over 1 month of age.2

A recent study found that:5

  • Late-onset VOD accounts for 30% of all adult VOD cases
  • Survival rates were similar for classical and late-onset VOD

The authors concluded that Defitelio® should be considered early for the treatment of severe VOD, regardless of time of onset.5

Defitelio® is only recommended to be available as a treatment option through routine commissioning for severe VOD following HSCT within the criteria set out in this document.4

The BlueTeq form has been updated as of April 2021 to reflect this change to the NHSE commissioning document

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Treat early

Treat early to improve outcomes.1,6

In this open-label, single-arm, expanded access T-IND study Defitelio® met its primary endpoint.1

Early initiation of Defitelio® significantly improved OS at Day +100 of HSCT compared with delayed treatment.1

Post-hoc analysis of the MOD subgroup data from the expanded access T-IND programme (N=512).1

In patients diagnosed with VOD with MOD who were treated with Defitelio® (n=351), the Day +100 OS rate according to treatment initiation was:


(>2 days after diagnosis)6



(<2 days after diagnosis)6


Difference 25.6% (95% CI 13.8%, 36.9%)

Please note, these data are retrospective.

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Resolution of symptoms

Treat with Defitelio® until complete resolution of symptoms.3

Defitelio® should be administered for a minimum of 21 days and continued until the symptoms and signs of severe VOD resolve.3

  • The efficacy of Defitelio® as documented by clinical trials is based on a treatment duration of 21 and 21.5 days1,7
  • The median time to CR with Defitelio® was 24.5 days (range 7–123)8
    • Pooled data from expanded access, Phase 2, and Phase 3 studies

* 102 patients received Defitelio® 25 mg/kg/day vs. 32 patients who had received a historical control.

Kaplan-Meier estimates for time-to-event analysis by Day +100 post-HSCT.

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Tolerability information

Defitelio® is generally well tolerated with manageable toxicity.9

  • The overall incidence of adverse events was consistent across multiple other Defitelio® studies3,9
  • There was no difference in the frequency, nature or severity of adverse reactions in adults and children over 1 month of age3
  • Interim efficacy and safety results from the DEFIFrance study are consistent with prior registry studies and support the utility of Defitelio® for treating severe/very severe VOD post-HSCT in a real-world setting*10

* The DEFIFrance study is ongoing; final data are expected in 2021.

Safety profile in the T-IND study

Summary of AEs from the T-IND final analysis:1

  • Age, MOD status, and transplant type were associated with some apparent variations in safety profiles
  • TRAEs occurred in 21% of patients in the overall population (210/1,000)
  • TRAEs led to discontinuation in 12.4% of patients (124/1,000) and death in 2.8% of patients (28/1,000)
  • TRAEs occurring in ≥2% of patients were pulmonary haemorrhage (4.6%), gastrointestinal haemorrhage (3%), epistaxis (2.3%), and hypotension (2%)


Summary of TEAEs.1

Safety profile in the Summary of Product Characteristics

The safety evaluation of Defitelio® is based on the safety pooled data set, which included patients who received 25 mg/kg of Defitelio® for the treatment of VOD, from the following clinical studies:3

  • Phase 3 pivotal treatment study
  • Treatment-IND study
  • Dose-finding study
  • Controlled randomised prophylaxis study

For full information on the methodology used to create the table below, please see section 4.8 of the SmPC.

Tabulated list of adverse reactions, by system organ class and frequency.

Frequencies are defined as:

  • Very common: ≥1/10
  • Common: ≥1/100 to <1/10
  • Uncommon: ≥1/1,000 to <1/100

For full information on the undesirable effects associated with Defitelio®, please see section 4.8 of the SmPC.


Adverse events should be reported. Reporting forms and information for the UK can be found at:

For Ireland, reporting forms and information can be found at:

Adverse events should also be reported to Jazz Pharmaceuticals at:

Defitelio® is indicated for the treatment of severe hepatic veno-occlusive disease (VOD) also known as sinusoidal obstruction syndrome (SOS) in haematopoietic stem-cell transplantation (HSCT) therapy. It is indicated in adults and in adolescents, children and infants over 1 month of age.