Medical College of Wisconsin
CTSICores SearchResearch InformaticsREDCap

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

04/20/2021

Pubmed ID

33871892

Pubmed Central ID

PMC8324076

DOI

10.1002/pbc.29067

Scopus ID

2-s2.0-85104540510 (requires institutional sign-in at Scopus site)   2 Citations

Abstract

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 W

Authors

Wael Saber MD, MS Professor in the Medicine department at Medical College of Wisconsin
Patricia 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

Child
Hematopoietic Stem Cell Transplantation
Hepatic Veno-Occlusive Disease
Humans
Leukemia, Myeloid, Acute
Retrospective Studies