Hepatic Veno-occlusive Disease Following Hematopoietic Stem Cell Transplantation in Children: Retrospective Analysis in a Single Institution / 대한소아혈액종양학회지

ABSTRACT

PURPOSE: Hepatic veno-occlusive disease (VOD) is a life-threatening complication occurring early after stem cell transplantation (SCT). Early diagnosis and effective treatment has not been established in severe VOD. Because there are few reports on VOD in Korean children, we evaluated the clinical characteristics of VOD following SCT in children. METHODS: We retrospectively reviewed the chart of all patients (n=116) receiving SCTs in CNUH Pediatric BMT center between May, 1991 and June, 2004. RESULTS: VOD developed in 11 patients (9.5%) (median age, 9.8 years; range, 2 to 13.9). Underlying diagnoses were ALL (n=3), severe aplastic anemia (n=3), AML (n=2), acute biphenotypic leukemia (n=1), neuroblastoma (n=1), and myelodysplastic syndrome (n=1). The median day of onset of VOD was D+9 (range, D-3 to D+19). VOD was classified as moderate in 5 and severe in 6 cases. Maximum level of serum total bilirubin was 2.9 mg/dL (range, 2.1 to 9.2) in moderate VOD and 7.3 mg/dL in severe VOD (range, 2.0 to 24.2) at D+18 (range, D-5 to D+59). We successfully treated VOD with various combinations including tPA and heparin (2/5, 40%), ursodeoxycholic acid (2/5, 40%), N-acetylcysteine (3/5, 60%), and defibrotide (1/2, 50%). All of 5 patients with moderate VOD survived at D+100 (range, 5.5+ to 66.6+ months). Five of 6 (83%) patients with severe VOD died within first 19 day from complications of VOD. CONCLUSION: This retrospective study showed that the incidence of VOD was 9.5%, and the mortality of severe VOD was still high which would necessitate early diagnosis, effective prevention and treatment.