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

Publikation: Beitrag in FachzeitschriftForschungsartikelBeigetragenBegutachtung

Beitragende

  • Jan-Niklas Eckardt - , Medizinische Klinik und Poliklinik I, Universitätsklinikum Carl Gustav Carus Dresden (Autor:in)
  • Christoph Röllig - , Medizinische Klinik und Poliklinik I, Universitätsklinikum Carl Gustav Carus Dresden (Autor:in)
  • Klaus Metzeler - , Universitätsklinikum Leipzig (Autor:in)
  • Michael Kramer - , Universitätsklinikum Carl Gustav Carus Dresden (Autor:in)
  • Sebastian Stasik - , Medizinische Klinik und Poliklinik I, Universitätsklinikum Carl Gustav Carus Dresden (Autor:in)
  • Julia-Annabell Georgi - , Medizinische Klinik und Poliklinik I, Universitätsklinikum Carl Gustav Carus Dresden (Autor:in)
  • Peter Heisig - , Institut für Software- und Multimediatechnik (SMT), Professur für Softwaretechnologie (Autor:in)
  • Karsten Spiekermann - , University Hospital (Autor:in)
  • Utz Krug - , Hospital Leverkusen (Autor:in)
  • Jan Braess - , Krankenhaus Barmherzige Brüder (Autor:in)
  • Dennis Görlich - , Münster University of Applied Sciences (Autor:in)
  • Cristina M Sauerland - , Münster University of Applied Sciences (Autor:in)
  • Bernhard Woermann - , Universität Aarhus (Autor:in)
  • Tobias Herold - , University Hospital (Autor:in)
  • Wolfgang E Berdel - , Universitätsklinikum Münster (Autor:in)
  • Wolfgang Hiddemann - , University Hospital (Autor:in)
  • Frank Kroschinsky - , Medizinische Klinik und Poliklinik I, Universitätsklinikum Carl Gustav Carus Dresden (Autor:in)
  • Johannes Schetelig - , Medizinische Klinik und Poliklinik I, Universitätsklinikum Carl Gustav Carus Dresden (Autor:in)
  • Uwe Platzbecker - , Universitätsklinikum Leipzig (Autor:in)
  • Carsten Müller-Tidow - , Universitätsklinikum Heidelberg (Autor:in)
  • Tim Sauer - , Universitätsklinikum Heidelberg (Autor:in)
  • Hubert Serve - , Johann Wolfgang Goethe-Universität Frankfurt am Main (Autor:in)
  • Claudia Baldus - , Universitätsklinikum Schleswig-Holstein Campus Kiel (Autor:in)
  • Kerstin Schäfer-Eckart - , Paracelsus Medizinische Privatuniversität Nürnberg (Autor:in)
  • Martin Kaufmann - , Robert Bosch Krankenhaus Stuttgart (Autor:in)
  • Stefan Krause - , Universitätsklinikum der Friedrich-Alexander-Universität Erlangen-Nürnberg (Autor:in)
  • Mathias Hänel - , Universitätsklinikum Essen (Autor:in)
  • Christoph Schliemann - , Universitätsklinikum Münster (Autor:in)
  • Maher Hanoun - , Universitätsklinikum Essen (Autor:in)
  • Christian Thiede - , Medizinische Klinik und Poliklinik I, Universitätsklinikum Carl Gustav Carus Dresden (Autor:in)
  • Martin Bornhäuser - , Universitäts KrebsCentrum Dresden, Medizinische Klinik und Poliklinik I, Nationales Centrum für Tumorerkrankungen (Partner: UKD, MFD, HZDR, DKFZ), Universitätsklinikum Carl Gustav Carus Dresden (Autor:in)
  • Karsten Wendt - , Universitätsklinikum Leipzig (Autor:in)
  • Jan Moritz Middeke - , Medizinische Klinik und Poliklinik I, Universitätsklinikum Carl Gustav Carus Dresden (Autor:in)

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

OriginalspracheEnglisch
Seiten (von - bis)690-704
Seitenumfang15
FachzeitschriftHaematologica
Jahrgang108
Ausgabenummer3
PublikationsstatusVeröffentlicht - 1 März 2023
Peer-Review-StatusJa

Externe IDs

PubMedCentral PMC9973482
Scopus 85135451258

Schlagworte

Schlagwörter

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