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A risk score based on real-world data to predict early death in acute promyelocytic leukemia

Österroos, Albin ; Maia, Tânia ; Eriksson, Anna ; Jädersten, Martin ; Lazarevic, Vladimir LU ; Wennström, Lovisa ; Antunovic, Petar ; Cammenga, Jörg LU ; Deneberg, Stefan and Lorenz, Fryderyk , et al. (2022) In Haematologica 107(7). p.1528-1537
Abstract

With increasingly effective treatments, early death (ED) has become the predominant reason for therapeutic failure in patients with acute promyelocytic leukemia (APL). To better prevent ED, patients with high-risk of ED must be identified. Our aim was to develop a score that predicts the risk of ED in a real-life setting. We used APL patients in the population-based Swedish AML Registry (n=301) and a Portuguese hospital-based registry (n=129) as training and validation cohorts, respectively. The cohorts were comparable with respect to age (median, 54 and 53 years) and ED rate (19.6% and 18.6%). The score was developed by logistic regression analyses, risk-per-quantile assessment and scoring based on ridge regression coefficients from... (More)

With increasingly effective treatments, early death (ED) has become the predominant reason for therapeutic failure in patients with acute promyelocytic leukemia (APL). To better prevent ED, patients with high-risk of ED must be identified. Our aim was to develop a score that predicts the risk of ED in a real-life setting. We used APL patients in the population-based Swedish AML Registry (n=301) and a Portuguese hospital-based registry (n=129) as training and validation cohorts, respectively. The cohorts were comparable with respect to age (median, 54 and 53 years) and ED rate (19.6% and 18.6%). The score was developed by logistic regression analyses, risk-per-quantile assessment and scoring based on ridge regression coefficients from multivariable penalized logistic regression analysis. White blood cell count, platelet count and age were selected by this approach as the most significant variables for predicting ED. The score identified low-, high- and very high-risk patients with ED risks of 4.8%, 20.2% and 50.9% respectively in the training cohort and with 6.7%, 25.0% and 36.0% as corresponding values for the validation cohort. The score identified an increased risk of ED already at sub-normal and normal white blood cell counts and, consequently, it was better at predicting ED risk than the Sanz score (AUROC 0.77 vs. 0.64). In summary, we here present an externally validated and population-based risk score to predict ED risk in a real-world setting, identifying patients with the most urgent need of aggressive ED prevention. The results also suggest that increased vigilance for ED is already necessary at sub-normal/normal white blood cell counts.

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organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Haematologica
volume
107
issue
7
pages
1528 - 1537
publisher
Ferrata Storti Foundation
external identifiers
  • scopus:85133348990
  • pmid:35081688
ISSN
0390-6078
DOI
10.3324/haematol.2021.280093
language
English
LU publication?
yes
additional info
Funding Information: The Swedish AML Registry is supported by the Swedish Association of Local Authorities and Regions, Region Skåne, Regionalt Cancercentrum Syd, and the Swedish Cancer Society. Publisher Copyright: © 2022 Ferrata Storti Foundation
id
4c4b2a38-be26-4935-b91b-39252f6e9813
date added to LUP
2022-07-17 12:35:11
date last changed
2022-09-14 03:24:45
@article{4c4b2a38-be26-4935-b91b-39252f6e9813,
  abstract     = {{<p>With increasingly effective treatments, early death (ED) has become the predominant reason for therapeutic failure in patients with acute promyelocytic leukemia (APL). To better prevent ED, patients with high-risk of ED must be identified. Our aim was to develop a score that predicts the risk of ED in a real-life setting. We used APL patients in the population-based Swedish AML Registry (n=301) and a Portuguese hospital-based registry (n=129) as training and validation cohorts, respectively. The cohorts were comparable with respect to age (median, 54 and 53 years) and ED rate (19.6% and 18.6%). The score was developed by logistic regression analyses, risk-per-quantile assessment and scoring based on ridge regression coefficients from multivariable penalized logistic regression analysis. White blood cell count, platelet count and age were selected by this approach as the most significant variables for predicting ED. The score identified low-, high- and very high-risk patients with ED risks of 4.8%, 20.2% and 50.9% respectively in the training cohort and with 6.7%, 25.0% and 36.0% as corresponding values for the validation cohort. The score identified an increased risk of ED already at sub-normal and normal white blood cell counts and, consequently, it was better at predicting ED risk than the Sanz score (AUROC 0.77 vs. 0.64). In summary, we here present an externally validated and population-based risk score to predict ED risk in a real-world setting, identifying patients with the most urgent need of aggressive ED prevention. The results also suggest that increased vigilance for ED is already necessary at sub-normal/normal white blood cell counts.</p>}},
  author       = {{Österroos, Albin and Maia, Tânia and Eriksson, Anna and Jädersten, Martin and Lazarevic, Vladimir and Wennström, Lovisa and Antunovic, Petar and Cammenga, Jörg and Deneberg, Stefan and Lorenz, Fryderyk and Möllgård, Lars and Uggla, Bertil and Ölander, Emma and Aguiar, Eliana and Trigo, Fernanda and Höglund, Martin and Juliusson, Gunnar and Lehmann, Sören}},
  issn         = {{0390-6078}},
  language     = {{eng}},
  number       = {{7}},
  pages        = {{1528--1537}},
  publisher    = {{Ferrata Storti Foundation}},
  series       = {{Haematologica}},
  title        = {{A risk score based on real-world data to predict early death in acute promyelocytic leukemia}},
  url          = {{http://dx.doi.org/10.3324/haematol.2021.280093}},
  doi          = {{10.3324/haematol.2021.280093}},
  volume       = {{107}},
  year         = {{2022}},
}