Prediction of complete remission and survival in acute myeloid leukemia using supervised machine learning

Research output: Contribution to journalResearch articleContributedpeer-review

Contributors

  • Jan-Niklas Eckardt - , Department of internal Medicine I, University Hospital Carl Gustav Carus Dresden (Author)
  • Christoph Röllig - , Department of internal Medicine I, University Hospital Carl Gustav Carus Dresden (Author)
  • Klaus Metzeler - , University Hospital Leipzig (Author)
  • Michael Kramer - , University Hospital Carl Gustav Carus Dresden (Author)
  • Sebastian Stasik - , Department of internal Medicine I, University Hospital Carl Gustav Carus Dresden (Author)
  • Julia-Annabell Georgi - , Department of internal Medicine I, University Hospital Carl Gustav Carus Dresden (Author)
  • Peter Heisig - , Institute of Software and Multimedia Technology, Chair of Software Technology (Author)
  • Karsten Spiekermann - , University Hospital (Author)
  • Utz Krug - , Hospital Leverkusen (Author)
  • Jan Braess - , Barmherzige Brüder Hospital Regensburg (Author)
  • Dennis Görlich - , Münster University of Applied Sciences (Author)
  • Cristina M Sauerland - , Münster University of Applied Sciences (Author)
  • Bernhard Woermann - , Aarhus University (Author)
  • Tobias Herold - , University Hospital (Author)
  • Wolfgang E Berdel - , University Hospital Münster (Author)
  • Wolfgang Hiddemann - , University Hospital (Author)
  • Frank Kroschinsky - , Department of internal Medicine I, University Hospital Carl Gustav Carus Dresden (Author)
  • Johannes Schetelig - , Department of internal Medicine I, University Hospital Carl Gustav Carus Dresden (Author)
  • Uwe Platzbecker - , University Hospital Leipzig (Author)
  • Carsten Müller-Tidow - , University Hospital Heidelberg (Author)
  • Tim Sauer - , University Hospital Heidelberg (Author)
  • Hubert Serve - , Goethe University Frankfurt a.M. (Author)
  • Claudia Baldus - , University Hospital Schleswig-Holstein Campus Kiel (Author)
  • Kerstin Schäfer-Eckart - , Paracelsus Medical University Nuremberg (Author)
  • Martin Kaufmann - , Robert Bosch Krankenhaus Stuttgart (Author)
  • Stefan Krause - , University Hospital at the Friedrich-Alexander University Erlangen-Nürnberg (Author)
  • Mathias Hänel - , University Hospital Essen (Author)
  • Christoph Schliemann - , University Hospital Münster (Author)
  • Maher Hanoun - , University Hospital Essen (Author)
  • Christian Thiede - , Department of internal Medicine I, University Hospital Carl Gustav Carus Dresden (Author)
  • Martin Bornhäuser - , University Cancer Centre, Department of internal Medicine I, National Center for Tumor Diseases (Partners: UKD, MFD, HZDR, DKFZ), University Hospital Carl Gustav Carus Dresden (Author)
  • Karsten Wendt - , University Hospital Leipzig (Author)
  • Jan Moritz Middeke - , Department of internal Medicine I, University Hospital Carl Gustav Carus Dresden (Author)

Abstract

Achievement of complete remission signifies a crucial milestone in the therapy of acute myeloid leukemia (AML) while refractory disease is associated with dismal outcomes. Hence, accurately identifying patients at risk is essential to tailor treatment concepts individually to disease biology. We used nine machine learning (ML) models to predict complete remission and 2-year overall survival in a large multicenter cohort of 1,383 AML patients who received intensive induction therapy. Clinical, laboratory, cytogenetic and molecular genetic data were incorporated and our results were validated on an external multicenter cohort. Our ML models autonomously selected predictive features including established markers of favorable or adverse risk as well as identifying markers of so-far controversial relevance. De novo AML, extramedullary AML, double-mutated CEBPA, mutations of CEBPA-bZIP, NPM1, FLT3-ITD, ASXL1, RUNX1, SF3B1, IKZF1, TP53, and U2AF1, t(8;21), inv(16)/t(16;16), del(5)/del(5q), del(17)/del(17p), normal or complex karyotypes, age and hemoglobin concentration at initial diagnosis were statistically significant markers predictive of complete remission, while t(8;21), del(5)/del(5q), inv(16)/t(16;16), del(17)/del(17p), double-mutated CEBPA, CEBPA-bZIP, NPM1, FLT3-ITD, DNMT3A, SF3B1, U2AF1, and TP53 mutations, age, white blood cell count, peripheral blast count, serum lactate dehydrogenase level and hemoglobin concentration at initial diagnosis as well as extramedullary manifestations were predictive for 2-year overall survival. For prediction of complete remission and 2-year overall survival areas under the receiver operating characteristic curves ranged between 0.77-0.86 and between 0.63-0.74, respectively in our test set, and between 0.71-0.80 and 0.65-0.75 in the external validation cohort. We demonstrated the feasibility of ML for risk stratification in AML as a model disease for hematologic neoplasms, using a scalable and reusable ML framework. Our study illustrates the clinical applicability of ML as a decision support system in hematology.

Details

Original languageEnglish
Pages (from-to)690-704
Number of pages15
JournalHaematologica
Volume108
Issue number3
Publication statusPublished - 1 Mar 2023
Peer-reviewedYes

External IDs

PubMedCentral PMC9973482
Scopus 85135451258

Keywords

Keywords

  • Humans, Prognosis, Splicing Factor U2AF/genetics, Nucleophosmin, Leukemia, Myeloid, Acute/diagnosis, Mutation, Supervised Machine Learning, Hemoglobins/genetics, fms-Like Tyrosine Kinase 3/genetics