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Defibrotide: real-world management of veno-occlusive disease/sinusoidal obstructive syndrome after stem cell

Mary Nauffal1, Haesook T Kim2, Paul G Richardson3

  • 1Department of Pharmacy, Memorial Sloan-Kettering Cancer Center, New York, NY.

Blood Advances
|October 19, 2021
PubMed

Insights

Defibrotide effectively treats severe hepatic veno-occlusive disease or sinusoidal obstructive syndrome (VOD/SOS) after hematopoietic stem cell transplantation (HSCT). Prompt treatment initiation and early diagnosis are crucial for successful VOD/SOS management and improved patient survival.

Area of Science:

  • Hematology
  • Transplantation Medicine
  • Hepatology

Background:

  • Hepatic veno-occlusive disease or sinusoidal obstructive syndrome (VOD/SOS) is a severe complication following hematopoietic stem cell transplantation (HSCT).
  • Defibrotide is the sole FDA-approved therapy for severe VOD/SOS post-HSCT.

Purpose of the Study:

  • To evaluate the efficacy and safety of commercially available defibrotide in managing VOD/SOS at a single center.
  • To analyze treatment outcomes based on VOD/SOS severity and HSCT conditioning regimens.

Main Methods:

  • Retrospective analysis of 28 VOD/SOS cases diagnosed between March 2016 and June 2019.
  • VOD/SOS diagnosis based on European Society of Blood and Marrow Transplantation criteria.
  • Assessment of defibrotide initiation timing, response rates, survival, and adverse events.

Main Results:

  • Complete VOD/SOS resolution was observed in 75% of patients.
  • Day 100 survival was 64% overall and 53% for very severe VOD/SOS.
  • Similar outcomes were noted for myeloablative and reduced-intensity conditioning HSCT.
  • Mild adverse events included hematuria and epistaxis; severe hemorrhage occurred in 7%.

Conclusions:

  • Defibrotide demonstrates significant efficacy in treating VOD/SOS post-HSCT.
  • Early diagnosis and prompt, uninterrupted defibrotide administration are vital for optimal outcomes.
  • Defibrotide is a safe and effective treatment option for VOD/SOS, with manageable adverse events.

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