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Can defibrotide be given safely for VOD in patients with refractory thrombocytopenia to platelet transfusions?

1 Answer
Mednet MemberInvited Expert
Pediatric Hematology/Oncology · Kapiolani Medical Center for Women & Children
Answered on

The short answer is "yes."

The slightly longer answer is: “Yes, and in patients with veno-occlusive disease (VOD), the use of defibrotide is potentially life-saving (Richardson et al., PMID 26825712).” In the cited study, which led to its FDA approval for this indication, there was no significant difference in the occurrence of hemorrhagic adverse events between those receiving the drug vs in the historical control group (including pulmonary alveolar [11.8% and 15.6%] and gastrointestinal bleeding [7.8% and 9.4%]), but a pronounced survival benefit was seen with its use.

The detailed answer is: Refractory thrombocytopenia is a common feature of VOD and has been proposed as a diagnostic criteria (Cairo et al., PMID 32133623). However, at prese...

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