Risk factors for hepatic veno-occlusive disease: a retrospective unicentric study in 116 children autografted after a high-dose BU-thiotepa regimen

Abstract

At our Institute, during the last decade, the incidence of hepatic veno-occlusive disease (HVOD) appears to be on the increase among pediatric patients treated with BUthiotepa (BU-TTP)-conditioning regimen. We thus performed a retrospective analysis to identify the risk factors for HVOD, which could explain such a change. In total, 116 patients treated at Institut Gustave Roussy, between May 1998 and December 2005 were eligible for this study having received BU-TTP as their first high-dose chemotherapy regimen, followed by autologous hematopoietic SCT (AHSCT). According to McDonald's clinical criteria, HVOD was diagnosed in 31% of these children. Demographic, clinical, biological and therapeutic parameters were evaluated in uni-and multivariate analyses that showed a significant correlation between previous carboplatin therapy and risk of developing post transplant HVOD (P ¼ 0.028). Comparable results were found for etoposide (P ¼ 0.048). In addition, a correlation between HVOD and risk of post transplant death was linked to its association with other types of organ failure (P ¼ 0.029). This study demonstrates that previous VPCARBO administration in conventional chemotherapy significantly increases the risk of HVOD among brain tumor patients later consolidated with BU-TTP followed by AHSCT.

Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease after Autologous or Allogeneic Hematopoietic Stem Cell Transplantation in Children: a retrospective study of the Italian Hematology-Oncology Association–Hematopoietic Stem Cell Transplantation Group

“…However, considering the very low number of patients affected by osteopetrosis in this cohort, no conclusions can be drawn. Conversely, our data confirmed that females, children younger than 2 years of age, and a Bu-based conditioning regimen represent risk factors for SOS/VOD development, as already reported in other studies [1,12,[14][15][16]22]. No information about the Bu route of administration are collected in the AIEOP registry, so no information on the role of either oral or i.v.…”

“…Based on the data reported to the AIEOP registry and after interrogating the centers participating into the study, all patients meeting the criteria for SOS/VOD had the new pediatric EBMT criteria applied, and all were shown to have severe or very severe disease. Remarkably, the incidence of severe and very severe SOS/VOD reported in this study is lower than the incidence of overall SOS/VOD reported in other pediatric studies (13.2% to 30%) [15,16]. It has been reported that the incidence of severe SOS/ VOD was 20% in children and 48% in adults [17,18].…”

Autologous Hematopoetic Stem Cell Transplantation for Refractory Crohn Disease

“…One patient randomized to HSCT died 20 days after the start of conditioning, with postmortem evidence of sinusoidal obstructive syndrome. 27,28 He was taking no drugs at trial entry and had no antecedent risk factors for sinusoidal obstructive syndrome. 27,28 He had been thought to have intraperitoneal sepsis and underwent (negative) laparotomy 2 days before death, when his liver appeared normal and results of liver function blood tests were normal.…”

Advancements in the treatment of pediatric acute leukemia and brain tumor - continuous efforts for 100% cure

“…One example is the prevention of veno-occlusive disease (VOD) with lipo-prostaglandin E1 (PGE1). Incidence of VOD ranges from 11% to 31% of pediatric SCT, but the mortality rate has been reported to be up to 50%17,18,19,20). Because of the high mortality rate, the prevention of VOD is critical.…”