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J Pediatr Hematol Oncol. 2002 Dec;24(9):746-50.

Hepatic venoocclusive disease in blood and bone marrow transplantation in children and young adults: incidence, risk factors, and outcome in a cohort of 241 patients.

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  • 1Department of Hematology/Oncology, Children's Hospital Oakland, Oakland, CA, USA.



To describe recent characteristics of incidence, risk factors, treatment, and outcome of venoocclusive disease (VOD) in children and young adults undergoing blood and bone marrow transplantation (BMT).


All children and young adults (n = 241) undergoing first myeloablative transplant at the UCSF Pediatric BMT unit between 1992 and 2000 were included. Retrospective chart review was done. Descriptive statistics and univariate and multivariate analyses of risk factors are presented.


Venoocclusive disease developed in 65 patients (27%); it was severe in 13/65 patients (20%). Matched unrelated donor transplantation, advanced-stage malignancies, and transplantation in the recent period (1998-2000) were identified as significant risk factors for VOD in univariate and multivariate analyses. Heparin prophylaxis did not decrease the incidence of VOD. Venoocclusive disease was diagnosed at a median day 8 after BMT. Five of 13 patients with severe VOD (38%) survived for more than 1 year after BMT, even after renal and respiratory failure and high total bilirubin levels up to 35 mg/dL. Nine of the 13 patients received fibrinolytic treatment with tissue plasminogen activator, anti-thrombin 3, or defibrotide. The survival rate at day 100 after BMT for children with VOD was 77%; it was 94% for those without VOD.


The persistently high incidence of VOD, its significant impact on posttransplant survival, and the demonstration of recovery from even severe VOD underscore the importance of early diagnosis and initiation of specific therapy. The use of Bearman's model of prediction of severity of VOD and the application of fibrinolytic drugs when adequate are highly recommended.

[PubMed - indexed for MEDLINE]
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