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Precision medicine for cardiovascular disease

Learning lessons from cardiomyopathies

Präzisionsmedizin bei Herz-Kreislauf-Erkrankungen

Lehrreiche Erfahrungen durch Kardiomyopathien

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Abstract

Evidence-based medicine has considerably advanced the treatment of highly prevalent cardiovascular diseases. Its implementation was driven by multicenter interventional trials in treatment and placebo cohorts, propelling numerous biomedical innovations toward standard of care. While a uniform treatment can be effective in such disease cohorts (“one size fits all”), it neglects the genetic and phenotypic individuality of a single patient and his or her disease. Accordingly, a recent observation was made that several newer “mega” trials, demanding considerable resources for their execution, showed statistically significant differences in outcome, however, with small overall efficacies that render implementation in the clinics unlikely. To overcome this concerning development, new methods for individualized treatment of cardiovascular disease are required. Rarer conditions, such as distinct cardiomyopathies, may deliver the blueprint for a paradigm shift: deep and precise phenotyping of individual patients by a multimodal approach and development of targeted treatments for smaller groups (“one treatment for many”) or even for single patients (“one treatment of some”).

Zusammenfassung

Durch die evidenzbasierte Medizin kam es zu beträchtlichen Fortschritten in der Behandlung von Herz-Kreislauf-Erkrankungen mit hoher Prävalenz. Ihre Etablierung wurde durch multizentrische Interventionsstudien mit Therapie- und Placebokohorten gefördert, was dazu führte, dass zahlreiche biomedizinische Neuerungen in die Standardversorgung Aufnahme fanden. Während die gleiche Behandlung für alle in solchermaßen erkrankten Kohorten wirksam sein kann („Einheitsgröße“), werden die genetischen und phänotpyischen individuellen Merkmale des einzelnen Patienten und seiner Krankheit dabei vernachlässigt. Entsprechend wurde kürzlich beobachtet, dass einige neuere „Megastudien“, die beträchtliche Ressourcen für ihre Durchführung binden, statistisch signifikante Unterschiede im Ergebnis aufwiesen, jedoch mit geringer Gesamtwirksamkeit, welche die Etablierung im klinischen Alltag unwahrscheinlich macht. Um diese beunruhigende Entwicklung zu überwinden, sind neue Methoden für die individualisierte Behandlung von Herz-Kreislauf-Erkrankungen erforderlich. Seltenere Erkrankungen, wie bestimmte Kardiomyopathien, bieten möglicherweise eine Vorlage für einen Paradigmenwechsel: tiefe und präzise Phänotypisierung einzelner Patienten in einem multimodalen Ansatz und Entwicklung gezielter Therapie für kleinere Gruppen („eine Therapie für viele“) oder sogar für einzelne Patienten („eine Therapie für einige“).

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Acknowledgements

Our work is supported by grants from the DZHK (“Deutsches Zentrum für Herz-Kreislauf-Forschung”, German Centre for Cardiovascular Research), the German Ministry of Education and Research (caRNAtion, Promise), and the European Union (FP7 BestAgeing, ERA-CVD DETECTIN-HF).

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Authors and Affiliations

  1. Institute for Cardiomyopathies Heidelberg, Department of Medicine III, University of Heidelberg, Im Neuenheimer Feld 410, 69120, Heidelberg, Germany

    F. Sedaghat-Hamedani, H. A. Katus &  B. Meder

  2. DZHK (German Centre for Cardiovascular Research), Berlin, Germany

    F. Sedaghat-Hamedani, H. A. Katus &  B. Meder

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  1. F. Sedaghat-Hamedani
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  2. H. A. Katus
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  3. B. Meder
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Correspondence to B. Meder.

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F. Sedaghat-Hamedani, H. A. Katus, and B. Meder declare that they have no competing interests.

This article does not contain any studies with human participants or animals performed by any of the authors.

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Sedaghat-Hamedani, F., Katus, H.A. & Meder, B. Precision medicine for cardiovascular disease. Herz 43, 123–130 (2018). https://doi.org/10.1007/s00059-017-4667-x

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