Lukas Kazianka, Alexander Pichler, Christiane Agreiter, Johannes Rohrbeck, Christoph Kornauth, Edit Porpaczy, Christian Sillaber, Wolfgang R. Sperr, Karoline V. Gleixner, Alexander Hauswirth, Ulrich Jäger, Peter Valent, Constanze Jonak, Stefanie Porkert, Ruth Exner, Wolfgang Willenbacher, Dominik Wolf, Peter Neumeister, Katharina Prochazka, Alexander Deutsch, Richard Greil, Clemens Schmitt, Robin Ristl, Marius Mayerhoefer, Ingrid Simonitsch-Klupp, Tea Pemovska, Philipp B. Staber
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Comparing functional and genomic-based precision medicine in blood cancer patients
Tumor-agnostic precision medicine (PM) strategies promise to support treatment decisions in relapsed/refractory blood cancer patients. Genomic-based PM (gPM) and drug screening-based functional PM (fPM) currently represent the most prominent PM methodologies. In this study, we report the feasibility analysis of the first 55 patients enrolled in the multicentric, randomized controlled EXALT-2 trial (NCT04470947) comparing treatment recommendations of gPM, fPM, and physicians' choice (PC) head to head. In 54 patients (98%), the diagnostic workflow was successfully implemented, resulting in treatment recommendations for 42 patients (76%), of whom 29 (69%) received the suggested individualized treatments. Actionable targets were identified in 65% by gPM and 80% by fPM (64% microscopy-based, 86% flow cytometry-based fPM). The median time to report was shorter for fPM than for gPM testing. The two strategies revealed overlapping drug targets in 60% of cases. Both, gPM and fPM can efficiently be integrated into the clinical routine to guide therapy decisions for the majority of patients.
期刊介绍：
HemaSphere, as a publication, is dedicated to disseminating the outcomes of profoundly pertinent basic, translational, and clinical research endeavors within the field of hematology. The journal actively seeks robust studies that unveil novel discoveries with significant ramifications for hematology.
In addition to original research, HemaSphere features review articles and guideline articles that furnish lucid synopses and discussions of emerging developments, along with recommendations for patient care.
Positioned as the foremost resource in hematology, HemaSphere augments its offerings with specialized sections like HemaTopics and HemaPolicy. These segments engender insightful dialogues covering a spectrum of hematology-related topics, including digestible summaries of pivotal articles, updates on new therapies, deliberations on European policy matters, and other noteworthy news items within the field. Steering the course of HemaSphere are Editor in Chief Jan Cools and Deputy Editor in Chief Claire Harrison, alongside the guidance of an esteemed Editorial Board comprising international luminaries in both research and clinical realms, each representing diverse areas of hematologic expertise.
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