biostudies-literatureDuncan CPfizerNCI NIH HHSHealth Resources and Services AdministrationOffice of Naval Researche29067https://www.ebi.ac.uk/biostudies/studies/S-EPMC8324076biostudies-literatureUnknown68(8)<h4>Background</h4>Gemtuzumab ozogamicin (GO) administered before allogeneic hematopoietic cell transplantation (alloHCT) has been linked to an increased risk of hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS).<h4>Procedure</h4>This retrospective analysis examined VOD/SOS risk and clinical outcomes in pediatric patients with acute myeloid leukemia who received myeloablative alloHCT in 2008-2011 with (n = 148) and without (n = 348; controls) prior GO exposure and were reported to the Center for International Blood and Marrow Transplant Research.<h4>Results</h4>Cumulative incidences (95% confidence interval [CI]) of VOD/SOS and severe VOD/SOS, respectively, at 100 days were 16% (11-23%) and 8% (4-13%) for GO-exposed patients and 10% (7-13%) and 3% (2-5%) for controls. With a median follow-up of approximately 7 years, the 5-year adjusted overall survival probability (95% CI) after alloHCT was 51% (43-58%) and 55% (50-60%) for GO-exposed patients and controls, respectively; three (4%) and one (<1%) deaths were attributed to VOD/SOS. In multivariate analyses, GO exposure was observed to be associated with an increased risk of VOD/SOS at 100 days, but was not associated with overall survival, disease-free survival, relapse, or nonrelapse mortality.<h4>Conclusions</h4>Results suggest that GO treatment prior to alloHCT in pediatric patients may increase the risk of VOD/SOS but not death.Pediatric blood & cancerVeno-occlusive disease risk in pediatric patients with acute myeloid leukemia treated with gemtuzumab ozogamicin before allogeneic hematopoietic cell transplantation.PMC8324076N00014‐20‐1‐2705U24 CA076518HHSH250201700006CHHSH250201700007CN00014‐20‐1‐2832Zhang MJSt Martin ALoberiza FRSaber WDuncan CPerez WSChirnomas DSteinert PHoang CJfalseVeno-occlusive disease risk in pediatric patients with acute myeloid leukemia treated with gemtuzumab ozogamicin before allogeneic hematopoietic cell transplantation.<h4>Background</h4>Gemtuzumab ozogamicin (GO) administered before allogeneic hematopoietic cell transplantation (alloHCT) has been linked to an increased risk of hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS).<h4>Procedure</h4>This retrospective analysis examined VOD/SOS risk and clinical outcomes in pediatric patients with acute myeloid leukemia who received myeloablative alloHCT in 2008-2011 with (n = 148) and without (n = 348; controls) prior GO exposure and were reported to the Center for International Blood and Marrow Transplant Research.<h4>Results</h4>Cumulative incidences (95% confidence interval [CI]) of VOD/SOS and severe VOD/SOS, respectively, at 100 days were 16% (11-23%) and 8% (4-13%) for GO-exposed patients and 10% (7-13%) and 3% (2-5%) for controls. With a median follow-up of approximately 7 years, the 5-year adjusted overall survival probability (95% CI) after alloHCT was 51% (43-58%) and 55% (50-60%) for GO-exposed patients and controls, respectively; three (4%) and one (<1%) deaths were attributed to VOD/SOS. In multivariate analyses, GO exposure was observed to be associated with an increased risk of VOD/SOS at 100 days, but was not associated with overall survival, disease-free survival, relapse, or nonrelapse mortality.<h4>Conclusions</h4>Results suggest that GO treatment prior to alloHCT in pediatric patients may increase the risk of VOD/SOS but not death.2021-01-01T00:00:00Z2021 Aug2024-11-09T10:52:40.97Z2024-11-09T10:52:40.97ZS-EPMC83240763387189210.1002/pbc.29067