Sergio A. Giralt, MD, Chief of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center, New York, told The ASCO Post that defibrotide could be helpful in high-risk patients and applauded the investigators for completing the trial to prove it.

“Veno-occlusive disease/sinusoidal obstruction syndrome is extremely rare, and initially there was lots of pushback about doing a traditional randomized clinical trial in patients because their outcomes were usually horrible. The ideal trial design, therefore, was debated,” he said.

“Finally, and much to Dr. Richardson’s credit, they were able to do the study, which hopefully will lead to having this drug on the market,” he said.

First Choice Is Prevention

Although defibrotide could help save lives, Dr. Giralt emphasized that the best way to deal with severe veno-occlusive disease/sinusoidal obstruction syndrome is to prevent it in the first place.

“We need to identify risk factors, modulate the preoperative regimen, and—while this won’t be the indication for this drug—think about using defibrotide prophylaxis for patients at high risk,” he suggested. ■

Disclosure: Dr. Giralt has received honoraria from Millennium and Celgene for consulting and speakers bureau activities. He also has served on advisory boards for Jazz Pharmaceuticals, the maker of defibrotide.