Precision Medicine in Hematology 2021: Definitions, Tools, Perspectives, and Open Questions
Peter Valent,
Alberto Orfao,
Stefan Kubicek,
Philipp Staber,
Torsten Haferlach,
Michael Deininger,
Karoline Kollmann,
Thomas Lion,
Irene Virgolini,
Georg Winter,
Oliver Hantschel,
Lukas Kenner,
Johannes Zuber,
Florian Grebien,
Richard Moriggl,
Gregor Hoermann,
Olivier Hermine,
Michael Andreeff,
Christoph Bock,
Tariq Mughal,
Stefan N. Constantinescu,
Robert Kralovics,
Veronika Sexl,
Radek Skoda,
Giulio Superti-Furga,
Ulrich Jäger
Affiliations
Peter Valent
1 Department of Medicine I, Division of Hematology & Hemostaseology, Medical University of Vienna, Austria
Alberto Orfao
3 Servicio Central de Citometria, Centro de Investigacion del Cancer (IBMCC; CSIC/USAL), IBSAL, CIBERONC and Department of Medicine, University of Salamanca, Spain
Stefan Kubicek
4 CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria
Philipp Staber
1 Department of Medicine I, Division of Hematology & Hemostaseology, Medical University of Vienna, Austria
Torsten Haferlach
5 MLL Munich Leukemia Laboratory, Munich, Germany
Michael Deininger
6 Division of Hematology and Hematologic Malignancies, University of Utah, Huntsman Cancer Institute, Salt Lake City, Utah, USA
Karoline Kollmann
7 Institute of Pharmacology and Toxicology, University of Veterinary Medicine Vienna, Austria
Thomas Lion
2 Ludwig Boltzmann Institute for Hematology and Oncology, Medical University of Vienna, Austria
Irene Virgolini
9 Department of Nuclear Medicine, Medical University of Innsbruck, Austria
Georg Winter
4 CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria
Oliver Hantschel
10 Institute of Physiological Chemistry, Faculty of Medicine, Philipps-University of Marburg, Germany
Lukas Kenner
11 Pathology of Laboratory Animals, University of Veterinary Medicine, Vienna, Austria
Johannes Zuber
12 Research Institute of Molecular Pathology (IMP), Vienna, Austria
Florian Grebien
13 Institute for Medical Biochemistry, University of Veterinary Medicine Vienna, Austria
Richard Moriggl
14 Institute of Animal Breeding and Genetics, Unit for Functional Cancer Genomics, University of Veterinary Medicine Vienna, Austria
Gregor Hoermann
2 Ludwig Boltzmann Institute for Hematology and Oncology, Medical University of Vienna, Austria
Olivier Hermine
15 Imagine Institute Université Paris Descartes, Sorbonne, Paris Cité, Paris, France
Michael Andreeff
17 University of Texas MD Anderson Cancer Center, Houston, Texas, USA
Christoph Bock
4 CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria
Tariq Mughal
18 Division of Hematology & Oncology, Tufts University Medical Center, Boston, Massachusetts, USA
Stefan N. Constantinescu
19 de Duve Institute and Ludwig Cancer Research Brussels, Université catholique de Louvain, Brussels, Belgium
Robert Kralovics
4 CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria
Veronika Sexl
7 Institute of Pharmacology and Toxicology, University of Veterinary Medicine Vienna, Austria
Radek Skoda
20 Departement of Biomedicine, University of Basel, Switzerland.
Giulio Superti-Furga
4 CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria
Ulrich Jäger
1 Department of Medicine I, Division of Hematology & Hemostaseology, Medical University of Vienna, Austria
During the past few years, our understanding of molecular mechanisms and cellular interactions relevant to malignant blood cell disorders has improved substantially. New insights include a detailed knowledge about disease-initiating exogenous factors, endogenous (genetic, somatic, epigenetic) elicitors or facilitators of disease evolution, and drug actions and interactions that underlie efficacy and adverse event profiles in defined cohorts of patients. As a result, precision medicine and personalized medicine are rapidly growing new disciplines that support the clinician in making the correct diagnosis, in predicting outcomes, and in optimally selecting patients for interventional therapies. In addition, precision medicine tools are greatly facilitating the development of new drugs, therapeutic approaches, and new multiparametric prognostic scoring models. However, although the emerging roles of precision medicine and personalized medicine in hematology and oncology are clearly visible, several questions remain. For example, it remains unknown how precision medicine tools can be implemented in healthcare systems and whether all possible approaches are also affordable. In addition, there is a need to define terminologies and to relate these to specific and context-related tools and strategies in basic and applied science. To discuss these issues, a working conference was organized in September 2019. The outcomes of this conference are summarized herein and include a proposal for definitions, terminologies, and applications of precision and personalized medicine concepts and tools in hematologic neoplasms. We also provide proposals aimed at reducing costs, thereby making these applications affordable in daily practice.