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The prophylaxis of hepatic veno-occlusive disease/sinusoidal obstruction syndrome with defibrotide after hematopoietic stem cell transplantation in children: single center experience

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SCHOOL OF MEDICINE
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Karagün, Barbaros S.
Akbaş, Tuana
Şaşmaz, İlgen
Antmen, Bülent

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Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is one of the most severe and life-threatening complications after hematopoietic stem cell transplantation (HSCT). Defibrotide (DF) is approved for adult and pediatric patients with VOD/SOS with renal or pulmonary dysfunction after HSCT in the United States, and for severe VOD/SOS post-HSCT in patients above 1 month of age in the European Union. Several studies have examined whether DF prophylaxis can reduce the incidence of VOD/SOS in high-risk patients. A total of 334 pediatric allogeneic HSCT were included in this study. All patients received DF at the dose of 25 mg/kg/d, from the first day of the conditioning regimen to the 30th day after transplantation for VOD/SOS prophylaxis. Seventeen patients (5.08%) developed VOD/SOS; 4 of these had moderate, while 13 had mild VOD/SOS. None of the patients were developed severe or very severe VOD/SOS. In conclusion, we showed that prophylactic intervention with DF lowered the incidence of VOD/SOS in high-risk pediatric patients.

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Lippincott Williams and Wilkins (LWW)

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Oncology, Hematology, Pediatrics

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Journal of Pediatric Hematology Oncology

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10.1097/MPH.0000000000002379

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