Veno-occlusive disease risk in pediatric patients with acute myeloid leukemia treated with gemtuzumab ozogamicin before allogeneic hematopoietic cell transplantation. Pediatr Blood Cancer 2021 Aug;68(8):e29067
Date
04/20/2021Pubmed ID
33871892Pubmed Central ID
PMC8324076DOI
10.1002/pbc.29067Scopus ID
2-s2.0-85104540510 (requires institutional sign-in at Scopus site) 2 CitationsAbstract
BACKGROUND: 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).
PROCEDURE: 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.
RESULTS: 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.
CONCLUSIONS: Results suggest that GO treatment prior to alloHCT in pediatric patients may increase the risk of VOD/SOS but not death.
Author List
Duncan C, St Martin A, Pérez WS, Steinert P, Zhang MJ, Chirnomas D, Hoang CJ, Loberiza FR Jr, Saber WAuthors
Wael Saber MD, MS Professor in the Medicine department at Medical College of WisconsinPatricia Steinert PhD Associate Professor in the Medicine department at Medical College of Wisconsin
Mei-Jie Zhang PhD Professor in the Institute for Health and Equity department at Medical College of Wisconsin
MESH terms used to index this publication - Major topics in bold
ChildHematopoietic Stem Cell Transplantation
Hepatic Veno-Occlusive Disease
Humans
Leukemia, Myeloid, Acute
Retrospective Studies