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Biol Blood Marrow Transplant. 2018 Oct;24(10):2072-2080. doi: 10.1016/j.bbmt.2018.06.013. Epub 2018 Jun 19.

Risk Score for the Development of Veno-Occlusive Disease after Allogeneic Hematopoietic Cell Transplant.

Author information

Division of Hematology, Oncology, and Bone Marrow Transplantation, University of Iowa, Iowa City, Iowa. Electronic address:
Division of Biostatistics, Institute for Health and Society, Medical College of Wisconsin, Milwaukee, Wisconsin.
Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, Wisconsin.
Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.
Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Department of Medicine, University of Massachusetts Memorial Medical Center, Worchester, Massachusetts.
Department of Medicine, Vanderbilt University Medical Center, Nashville, Tennessee.
Division of Hematology/Oncology, Abramson Cancer Center, University of Pennsylvania, Philadelphia, Pennsylvania.
National Marrow Donor Program, University of Minnesota, Minneapolis, Minnesota.
Section of Hematology/Oncology, Department of Medicine, University of Chicago, Chicago, Illinois.
Jazz Pharmaceuticals, Palo Alto, California.


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.


Busulfan; Conditioning regimen; Hematopoietic cell transplantation; Veno-occlusive disease

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