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Hepatic sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) is a major complication following hematopoietic stem cell transplantation, resulting from immune and chemical toxicity in the sinusoidal endothelium and hepatocellular damage. In the most severe cases, multiorgan dysfunction occurs, so it is essential to promptly identify patients at greater risk of SOS/VOD and to adopt prophylactic strategies. : This study aims to systematize the impact of different approaches as primary prophylaxes against SOS/VOD in patients undergoing hematopoietic stem cell transplantation (HSCT).

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The benefits of prophylactic defibrotide: Are the tides turning?

Pediatr Blood Cancer

January 2025

Pediatric Hematology Oncology and Stem Cell Transplant, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, Indiana, USA.

Article Synopsis
  • - Veno-occlusive disease (VOD) is a serious condition that can arise after stem cell transplants (SCT), with various factors influencing its development, and the effectiveness of prophylactic defibrotide (DF) is still being evaluated.
  • - The study examined 58 pediatric SCT patients to compare VOD incidence, severity, and survival outcomes between those who received prophylactic DF and those who did not.
  • - While results weren't statistically significant, patients who developed VOD after not receiving prophylactic DF had worse disease severity and lower one-year survival rates compared to those who received DF.
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Article Synopsis
  • - Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a serious complication after hematopoietic cell transplantation (HCT), and early treatment can improve patient outcomes, but diagnosis is difficult due to overlapping symptoms with other issues post-transplant.
  • - The study aimed to validate "VODCheck," a biomarker-based test designed to predict VOD/SOS risk using three key biomarkers: hyaluronan (HA), angiopoietin-2 (ANG-2), and thrombomodulin (TM).
  • - Results showed that VODCheck accurately predicted VOD/SOS risk at multiple time points post-HCT, demonstrating its effectiveness as an independent risk predictor and providing valuable insights for better
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Cyclophosphamide and Thiotepa Increases Risk of Transplant-Associated Thrombotic Microangiopathy.

Transplant Cell Ther

September 2024

Division of Pediatric Allergy, Immunology, and Bone Marrow Transplant, University of California San Francisco, Benioff Children's Hospitals, San Francisco, California.

Transplant associated thrombotic microangiopathy (TA-TMA) is a complication of hematopoietic cell transplant (HCT) associated with endothelial injury resulting in severe end organ damage, acute and long-term morbidity, and mortality. Myeloablative conditioning is a known risk factor, though specific causative agents have not been identified. We hypothesized that the combination of cyclophosphamide and thiotepa (CY + TT) is particularly toxic to the endothelium, placing patients at elevated risk for TA-TMA.

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