1. Sinusoidal obstruction syndrome/veno-occlusive disease after high-dose intravenous busulfan/melphalan conditioning therapy in high-risk Ewing Sarcoma
- Author
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Anna Paioli, Sivlia Cammelli, Emanuela Palmerini, Elisa Carretta, Alessandra Longhi, Piero Picci, Massimo Eraldo Abate, Fabio Piscaglia, Stefano Ferrari, Marilena Cesari, Abate, Massimo Eraldo, Paioli, Anna, Cammelli, Silvia, Cesari, Marilena, Longhi, Alessandra, Palmerini, Emanuela, Ferrari, Stefano, Carretta, Elisa, Picci, Piero, and Piscaglia, Fabio
- Subjects
Adult ,Male ,Melphalan ,medicine.medical_specialty ,Transplantation Conditioning ,Adolescent ,Hepatic Veno-Occlusive Disease ,Sarcoma, Ewing ,Defibrotide ,Transplantation, Autologous ,Gastroenterology ,Young Adult ,03 medical and health sciences ,Polydeoxyribonucleotides ,0302 clinical medicine ,Autologous stem-cell transplantation ,Risk Factors ,Internal medicine ,Humans ,Medicine ,Cumulative incidence ,Child ,Busulfan ,Survival analysis ,Transplantation ,Univariate analysis ,Radiotherapy ,business.industry ,Hematopoietic Stem Cell Transplantation ,Hematology ,Survival Analysis ,Treatment Outcome ,Child, Preschool ,030220 oncology & carcinogenesis ,Female ,business ,030215 immunology ,medicine.drug - Abstract
This mono-institutional observational study was conducted to determine incidence, severity, risk factors, and outcome of sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) in high-risk Ewing sarcoma (ES) patients treated with intravenous busulfan and melphalan (BU-MEL) followed by autologous stem cell transplantation (ASCT). During the past 10 years, 75 consecutive ES patients resulted evaluable for the analysis. After diagnosis of SOS/VOD, defibrotide therapy was started as soon as the medication was available. The variables analyzed as potential risk factors were: gender, patient's age at diagnosis, primary tumor site, disease stage, and prior radiation therapy (RT) given, focusing on RT liver exposure. The median age at diagnosis was 18.8 years. Five patients developed moderate to severe SOS/VOD (cumulative incidence, 6.67%). None of 32 pediatric patients (â¤17 years) developed SOS/VOD (p = 0.0674). In univariate analysis, prior RT liver exposure resulted statistically significant (p = 0.0496). There was one death due to severe SOS/VOD. This study reports the largest series of high-risk ES patients treated with intravenous BU-MEL before ASCT. The incidence of SOS/VOD was lower when compared with other studies that used oral busulfan. Any prior RT liver exposure should be avoided. Earlier defibrotide treatment confirms to be effective.
- Published
- 2018