Defibrotide for prophylaxis of sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) in pediatric high-risk patients: consensus guidelines from the European Society for Blood and Marrow Transplantation (EBMT).
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2026-04
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SOS/VOD is a life-threatening complication of hematopoietic stem cell transplantation, especially in children, with incidences reaching up to 15-20%. Despite efforts, SOS/VOD remains unpredictable with significant morbidity and mortality. High-risk criteria are clearly defined, and the pediatric EBMT diagnostic criteria have improved sensitivity, reducing treatment delays and enhancing outcomes. A meta-analysis combining retrospective and prospective studies found a risk ratio of 0.30 for SOS/VOD with defibrotide (DF) prophylaxis. Additionally, two prospective trials were conducted: the pediatric prevention trial (NCT00272948) and the Harmony Trial (NCT02851407), involving adults and children, with primary outcomes of incidence and SOS/VOD-free survival, respectively. The trials produced conflicting results regarding the effectiveness of prophylactic DF. Despite significant limitations of the Harmony trial, a direct healthcare professional communication (DHPC) from the European Medicines Agency (EMA) advised against prophylactic DF. This recommendation has serious consequences for children, especially infants, who are among the most vulnerable groups receiving HSCT. Therefore, a panel of experts issued guidelines for children at high risk for SOS/VOD, in which DF prophylaxis is considered justified. These guidelines include a weighted scoring system based on all relevant high-risk criteria to predict SOS/VOD, supporting decisions regarding the use of prophylactic DF in children.
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Corbacioglu, Selim, Rajinder Bajwa, Ali Bülent Antmen, Adriana Balduzzi, Jaap Jan Boelens, Francesca Bonifazi, Simone Cesaro, Fabio Ciceri, et al. (2026). Defibrotide for prophylaxis of sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) in pediatric high-risk patients: consensus guidelines from the European Society for Blood and Marrow Transplantation (EBMT). Bone marrow transplantation, 61(4). pp. 417–425. 10.1038/s41409-025-02793-x Retrieved from https://hdl.handle.net/10161/34733.
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Kris Mahadeo
As a committed life-long learner, I am awed by the continued advancements in the field of pediatric transplant and cellular therapy (PTCT). Following completion of my PTCT fellowship training at Duke, I have developed a career dedicated to expanding indications for PTCT and improving associated outcomes. I have a special interest in the mitigation of endotheliopathies and continue to lead global harmonization efforts related to management of toxicities of therapy. As the Division Chief of PTCT at Duke, I am committed to the ensuring our patients receive state of the art care with access to innovative research protocols that change the field. We are committed to training the next generation of physicians in our field with a commitment to global partnerships to improve care for patients across the world.
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