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) - Scorecard - MDSpire
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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)
Clinical Scorecard: Guidelines from the European Society for Blood and Marrow Transplantation (EBMT) on the Use of Defibrotide to Prevent Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease in High-Risk Pediatric Patients
Hematopoietic stem cell transplantation centers and pediatric oncology units
Key Highlights
SOS/VOD incidence in children remains high (15-20%, up to 60% historically) compared to adults (<10%).
Anicteric SOS/VOD (without hyperbilirubinemia) occurs in up to 30% of patients, complicating diagnosis.
Defibrotide prophylaxis reduces endothelial damage and lowers SOS/VOD fatality in high-risk pediatric patients.
Guideline-Based Recommendations
Diagnosis
Use pediatric-specific EBMT (pEBMT) criteria for early and sensitive diagnosis, including transfusion-refractory thrombocytopenia (tRT) as an early marker.
Recognize limitations of Seattle and Baltimore criteria, especially the bilirubin threshold (≥2 mg/dL) which may miss anicteric cases.
Monitor for clinical signs such as painful hepatomegaly, fluid retention, ascites, and tRT.
Management
Initiate defibrotide prophylaxis in high-risk pediatric patients to prevent SOS/VOD.
Employ early therapeutic intervention upon diagnosis per pEBMT criteria regardless of bilirubin levels.
Consider defibrotide as a treatment to reduce endothelial damage and improve survival.
Monitoring & Follow-up
Regularly assess platelet transfusion requirements to detect tRT as an early sign of SOS/VOD.
Monitor liver function tests, including bilirubin, but do not rely solely on hyperbilirubinemia for diagnosis.
Observe clinical symptoms such as hepatomegaly, ascites, and weight gain.
Risks
High risk of SOS/VOD in pediatric HSCT patients due to unmodifiable factors like pre-existing liver disease and conditioning regimens.
Delayed diagnosis due to nonspecific symptoms and variable diagnostic criteria may increase morbidity and mortality.
Anicteric SOS/VOD cases may be underdiagnosed if relying on hyperbilirubinemia.
Patient & Prescribing Data
High-risk pediatric patients undergoing HSCT or chemotherapy with elevated SOS/VOD risk
Defibrotide prophylaxis significantly reduces incidence and severity of SOS/VOD by protecting endothelial integrity; early initiation based on sensitive criteria improves outcomes.
Clinical Best Practices
Adopt pEBMT diagnostic criteria incorporating transfusion-refractory thrombocytopenia for early detection.
Implement defibrotide prophylaxis in identified high-risk pediatric patients prior to SOS/VOD onset.
Maintain high clinical vigilance for anicteric SOS/VOD presentations to avoid missed diagnoses.
Use a multidisciplinary approach involving transplant and pediatric oncology teams for monitoring and management.
by Selim Corbacioglu, Rajinder Bajwa, Ali Bülent Antmen, Adriana Balduzzi, Jaap Jan Boelens, Francesca Bonifazi, Simone Cesaro, Fabio Ciceri, Antonio Colecchia, Fiona Dignan, Katharina Kleinschmidt, Kris M. Mahadeo, Antonio Pagliuca, Petr Sedlacek, Peter J. Shaw, Jerry Stein, Zofia Szmit, Francesco Tambaro, Elif Ince, Marta Verna, Akif Yesilipek, Marco Zecca, Paul G. Richardson, Mohamad Mohty, Krzysztof Kalwak
