TY - JOUR
T1 - An integrative scoring system for survival prediction following umbilical cord blood transplantation in acute leukemia
AU - Shouval, Roni
AU - Ruggeri, Annalisa
AU - Labopin, Myriam
AU - Mohty, Mohamad
AU - Sanz, Guillermo
AU - Michel, Gerard
AU - Kuball, Jürgen
AU - Chevallier, Patrice
AU - Al-Seraihy, Amal
AU - Milpied, Noel Jean
AU - De Heredia, Cristina Diaz
AU - Arcese, William
AU - Blaise, Didier
AU - Rocha, Vanderson
AU - Fein, Joshua
AU - Unger, Ron
AU - Baron, Frederic
AU - Bader, Peter
AU - Gluckman, Eliane
AU - Nagler, Arnon
N1 - Funding Information:
This study was supported by The Varda and Boaz Dotan Research Center in Hemato-Oncology affiliated with the CBRC of Tel Aviv University and The Shalvi Foundation for the Support of Medical Research.
Funding Information:
J. Kuball is a consultant/advisory board member for Gadeta and reports receiving commercial research grants from Gadeta, Miltenyi, and Novartis. No potential conflicts of interest were disclosed by the other authors.
Publisher Copyright:
©2017 AACR.
PY - 2017/11/1
Y1 - 2017/11/1
N2 - Purpose: Survival of acute leukemia (AL) patients following umbilical cord blood transplantation (UCBT) is dependent on an array of individual features. Integrative models for risk assessment are lacking. We sought to develop a scoring system for prediction of overall survival (OS) and leukemia-free survival (LFS) at 2 years following UCBT in AL patients. Experimental Design: The study cohort included 3,140 pediatric and adult AL UCBT patients from the European Society of Blood and Marrow Transplantation and Eurocord registries. Patients received single or double cord blood units. The dataset was geographically split into a derivation (n = 2,362, 65%) and validation set (n = 778, 35%). Top predictors of OS were identified using the Random Survival Forest algorithm and introduced into a Cox regression model, which served for the construction of the UCBT risk score. Results: The score includes nine variables: disease status, diagnosis, cell dose, age, center experience, cytomegalovirus serostatus, degree of HLA mismatch, previous autograft, and anti-thymocyte globulin administration. Over the validation set an increasing score was associated with decreasing probabilities for 2 years OS and LFS, ranging from 70.21% [68.89–70.71, 95% confidence interval (CI)] and 64.76% (64.33–65.86, 95% CI) to 14.78% (10.91–17.41) and 18.11% (14.40–22.30), respectively. It stratified patients into six distinct risk groups. The score's discrimination (AUC) over multiple imputations of the validation set was 68.76 (68.19–69.04, range) and 65.78 (65.20–66.28) for 2 years OS and LFS, respectively. Conclusions: The UCBT score is a simple tool for risk stratification of AL patients undergoing UCBT. Widespread application of the score will require further independent validation.
AB - Purpose: Survival of acute leukemia (AL) patients following umbilical cord blood transplantation (UCBT) is dependent on an array of individual features. Integrative models for risk assessment are lacking. We sought to develop a scoring system for prediction of overall survival (OS) and leukemia-free survival (LFS) at 2 years following UCBT in AL patients. Experimental Design: The study cohort included 3,140 pediatric and adult AL UCBT patients from the European Society of Blood and Marrow Transplantation and Eurocord registries. Patients received single or double cord blood units. The dataset was geographically split into a derivation (n = 2,362, 65%) and validation set (n = 778, 35%). Top predictors of OS were identified using the Random Survival Forest algorithm and introduced into a Cox regression model, which served for the construction of the UCBT risk score. Results: The score includes nine variables: disease status, diagnosis, cell dose, age, center experience, cytomegalovirus serostatus, degree of HLA mismatch, previous autograft, and anti-thymocyte globulin administration. Over the validation set an increasing score was associated with decreasing probabilities for 2 years OS and LFS, ranging from 70.21% [68.89–70.71, 95% confidence interval (CI)] and 64.76% (64.33–65.86, 95% CI) to 14.78% (10.91–17.41) and 18.11% (14.40–22.30), respectively. It stratified patients into six distinct risk groups. The score's discrimination (AUC) over multiple imputations of the validation set was 68.76 (68.19–69.04, range) and 65.78 (65.20–66.28) for 2 years OS and LFS, respectively. Conclusions: The UCBT score is a simple tool for risk stratification of AL patients undergoing UCBT. Widespread application of the score will require further independent validation.
UR - http://www.scopus.com/inward/record.url?scp=85033595794&partnerID=8YFLogxK
U2 - 10.1158/1078-0432.CCR-17-0489
DO - 10.1158/1078-0432.CCR-17-0489
M3 - Article
C2 - 28754820
AN - SCOPUS:85033595794
SN - 1078-0432
VL - 23
SP - 6478
EP - 6486
JO - Clinical Cancer Research
JF - Clinical Cancer Research
IS - 21
ER -