Defibrotide for hepatic veno-occlusive disease
Ludwig Boltzmann Institut fuer Health Technology Assessment (LBI-HTA)
Record ID 32015000952
English
Authors' objectives:
This systematic review evaluates the following research questions:
- Is defibrotide for the prophylaxis of veno-occlusive disease (VOD) for patients undergoing haematopoietic stem cell transplantation (HSCT) in comparison to no prophylaxis, placebo or other experimental options in adult and paediatric patients more effective and safe concerning overall and disease-specific mortality, incidence of VOD, quality of life and adverse events?
- Is defibrotide for the treatment of severe veno-occlusive disease (VOD) after oncologic therapy or after haematopoietic stem cell transplantation (HSCT) in comparison to no treatment, placebo or other experimental
options in adult and paediatric patients more effective and safe concerning overall and disease-specific mortality, resolution of VOD, quality of life and adverse events?
Authors' recommendations:
Defibrotide for VOD prophylaxis:
Overall, the strength of evidence for the effectiveness and safety of defibrotide for the prophylaxis of VOD was very low to moderate.
Even though there are indications that the prophylaxis of VOD with defibrotide reduces the incidence of VOD, a life-threatening disease, at least in children at high risk and few adverse events are associated with this therapy, an inclusion in the catalogue of benefits for an off-label indication cannot be recommended.
A re-evaluation is recommended when the licensing status of the drug changes.
Defibrotide for VOD treatment:
Overall, the strength of evidence for the effectiveness and safety of defibrotide for the treatment of VOD is very low.
The current evidence is not sufficient to evaluate the effectiveness and safety of defibrotide for the treatment of VOD. Since VOD is a rare and life-threatening disease with no other therapeutic options available, defibrotide offers a new therapeutic approach for patients with severe VOD and meets an area of high unmet clinical need. Therefore, an inclusion in the catalogue of benefits is recommended with restrictions.
The proper assessment of risk factors and, whenever possible, the avoidance of factors contributing to the development VOD have to be ensured. Data on outcomes, foremost on long-term safety, should be collected in a prospective patient registry. Re-evaluation is recommended once these data become available. Thus, EMA website surveillance is also recommended to identify any change in the licensing status. Risk-sharing agreements are also indicated.
Details
Project Status:
Completed
Year Published:
2015
URL for published report:
http://eprints.hta.lbg.ac.at/1061/1/DSD_84.pdf
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
Austria
MeSH Terms
- Fibrinolytic Agents
- Polydeoxyribonucleotides
Contact
Organisation Name:
Ludwig Boltzmann Institute for Health Technology Assessment
Contact Address:
Ludwig Boltzmann Institute for fuer Health Technology Assessment (LBI-HTA), Garnisongasse 7/rechte Stiege Mezzanin (Top 20), 1090 Vienna, Austria. Tel: +43 1 236 8119 - 0 Fax: +43 1 236 8119 - 99
Contact Name:
tarquin.mittermayr@aihta.at
Contact Email:
office@aihta.at
Copyright:
Ludwig Boltzmann Institut fuer Health Technology Assessment (LBI-HTA)
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.