Defibrotide: Redefining the treatment for severe veno-occlusive disease (sVOD)

At the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), held in Valencia, Spain, Paul Richardson, MD, from Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, discusses the clinical development and recent FDA approval of the novel polydisperse oligonucleotide defibrotide for the treatment of patients with severe veno-occlusive disease (sVOD) following a hematopoietic stem cell transplant (HSCT). In three retrospective studies, defibrotide was associated with a survival benefit over what would have been expected. During EBMT the overriding message regarding VOD was “treat early”, because if you wait the efficacy of drug diminishes as the patient gets sicker. Prof. Paul Richardson also discusses the safety profile of defibrotide, and how effective it has been in Pediatrics, and the new treatment guidelines created by EBMT.

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