"Pre-emptive strike"-the case for early treatment of hepatic sinusoidal obstruction syndrome with defibrotide
Revathi Rajagopal1,2, Marianne B Phillips1,3,4, Nicholas G Gottardo1,3,4
1Department of Hematology and Oncology, Princess Margaret Hospital for Children, Perth, Australia.
Insights
Hepatic sinusoidal obstruction syndrome (HSOS) in children is hard to detect. Early signs like low platelets are key indicators, and prompt defibrotide treatment is effective.
Area of Science:
- Pediatric Oncology
- Hematology
- Hepatology
Background:
- Hepatic sinusoidal obstruction syndrome (HSOS) is a serious complication, often seen post-hematopoietic stem cell transplantation.
- Recognizing early HSOS signs in children outside the transplant setting is clinically challenging.
- Unexplained thrombocytopenia and transfusion-refractory platelets are critical early indicators.
Observation:
- A pediatric case of primitive neuroectodermal tumor (right 11th rib) is presented.
- The child developed HSOS following focal radiotherapy and actinomycin-D treatment.
- The case highlights HSOS development in a non-transplant pediatric oncology scenario.
Findings:
- The European Society for Blood and Marrow Transplantation proposed diagnostic criteria for pediatric HSOS.
- These criteria emphasize early recognition of consumptive and transfusion-refractory thrombocytopenia.
- The described case aligns with these early HSOS indicators.
Implications:
- Prompt identification of HSOS in pediatric patients is crucial for timely intervention.
- Pre-emptive treatment with defibrotide shows efficacy in managing HSOS.
- This case underscores the importance of recognizing "bellwether" signs for improved pediatric HSOS outcomes.
Abstract:
The initial signs of hepatic sinusoidal obstruction syndrome (HSOS) can be challenging to recognize in children, especially outside the hematopoietic stem cell transplantation setting. To assist clinicians to promptly identify HSOS, the European Society for Blood and Marrow Transplantation has proposed pediatric HSOS diagnostic criteria which emphasize unexplained consumptive and transfusion-refractory thrombocytopenia. To highlight the importance of these "bellwether" early signs of HSOS and the efficacy of pre-emptive treatment with defibrotide, we describe the case of a child with a right 11th rib primitive neuroectodermal tumor who developed HSOS following focal radiotherapy and actinomycin-D treatment.
