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Risk Score for the Development of Veno-Occlusive Disease after Allogeneic Hematopoietic Cell Transplant. Biol Blood Marrow Transplant 2018 Oct;24(10):2072-2080

Date

06/22/2018

Pubmed ID

29928989

Pubmed Central ID

PMC6239945

DOI

10.1016/j.bbmt.2018.06.013

Scopus ID

2-s2.0-85054166744 (requires institutional sign-in at Scopus site)   54 Citations

Abstract

A risk score identifying patients at high risk for veno-occlusive disease (VOD) may aid efforts to study preventive strategies for this uncommon complication of hematopoietic cell transplantation (HCT). Patients receiving a first allogeneic HCT between 2008 and 2013 as reported to the Center for International Blood and Marrow Transplant Research (N = 13,097) were randomly divided into training and validation sets. Independent prognostic factors for development of VOD by day +100 after HCT were identified with a multivariate logistic regression model. A risk score was constructed in the training set using the significant factors and confirmed in the validation set. Baseline characteristics of the training and validation sets were balanced. In total, 637 patients (4.9%) developed VOD by day +100. Younger age, positive hepatitis B/C serology, lower Karnofsky performance scale score, use of sirolimus, disease, disease status at transplant, and conditioning regimen were independent prognostic factors. Myeloablative conditioning regimens were associated with higher risk of VOD. Busulfan-based myeloablative conditioning regimens guided by pharmacokinetic monitoring were associated with higher risk than those without pharmacokinetic monitoring. Patients were stratified into 4 distinct, statistically significantly different groups by their risk score percentile. This pretransplant risk score successfully stratified allogeneic HCT patients by risk of developing VOD, was validated in an independent set, and demonstrated strong discriminatory ability to identify a high-risk cohort.

Author List

Strouse C, Zhang Y, Zhang MJ, DiGilio A, Pasquini M, Horowitz MM, Lee S, Ho V, Ramanathan M, Chinratanalab W, Loren A, Burns LJ, Artz A, Villa KF, Saber W

Authors

Mary M. Horowitz MD, MS Professor in the Medicine department at Medical College of Wisconsin
Marcelo C. Pasquini MD, MS Professor in the Medicine department at Medical College of Wisconsin
Wael Saber MD, MS 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

Adolescent
Adult
Aged
Aged, 80 and over
Allografts
Busulfan
Child
Child, Preschool
Female
Hematopoietic Stem Cell Transplantation
Humans
Infant
Male
Middle Aged
Risk Factors
Transplantation Conditioning
Vascular Diseases