Comprehensive Genomic and Transcriptomic Analysis for Guiding Therapeutic Decisions in Patients with Rare Cancers.

Cancer Discovery
Peter HorakStefan Fröhling

Abstract

The clinical relevance of comprehensive molecular analysis in rare cancers is not established. We analyzed the molecular profiles and clinical outcomes of 1,310 patients (rare cancers, 75.5%) enrolled in a prospective observational study by the German Cancer Consortium that applies whole-genome/exome and RNA sequencing to inform the care of adults with incurable cancers. On the basis of 472 single and six composite biomarkers, a cross-institutional molecular tumor board provided evidence-based management recommendations, including diagnostic reevaluation, genetic counseling, and experimental treatment, in 88% of cases. Recommended therapies were administered in 362 of 1,138 patients (31.8%) and resulted in significantly improved overall response and disease control rates (23.9% and 55.3%) compared with previous therapies, translating into a progression-free survival ratio >1.3 in 35.7% of patients. These data demonstrate the benefit of molecular stratification in rare cancers and represent a resource that may promote clinical trial access and drug approvals in this underserved patient population. SIGNIFICANCE: Rare cancers are difficult to treat; in particular, molecular pathogenesis-oriented medical therapies are often lacking...Continue Reading

References

Oct 6, 2010·Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology·Daniel D Von HoffRobert Penny
Oct 29, 2011·European Journal of Cancer : Official Journal for European Organization for Research and Treatment of Cancer (EORTC) [and] European Association for Cancer Research (EACR)·Gemma GattaUNKNOWN RARECARE working group
May 12, 2012·Cancer Discovery·Nicolai J BirkbakAndrea L Richardson
Sep 12, 2012·Nature Reviews. Clinical Oncology·Javier Munoz, Razelle Kurzrock
Jun 2, 2015·The New England Journal of Medicine·Dung T LeLuis A Diaz
Aug 20, 2015·The New England Journal of Medicine·David M HymanJosé Baselga
Aug 26, 2015·Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology·Maria SchwaederleRazelle Kurzrock
Sep 18, 2015·Journal of the National Cancer Institute·Denis L JardimRazelle Kurzrock
May 4, 2016·Annals of Oncology : Official Journal of the European Society for Medical Oncology·J A Ledermann
Mar 14, 2017·Nature Medicine·Helen DaviesSerena Nik-Zainal
May 26, 2017·CA: a Cancer Journal for Clinicians·Carol E DeSantisAhmedin Jemal
Jun 10, 2017·International Journal of Cancer. Journal International Du Cancer·Peter HorakStefan Fröhling
Aug 8, 2017·Nature·Dan R RobinsonArul M Chinnaiyan
Jan 4, 2018·Nature Communications·Michael SchubertJulio Saez-Rodriguez
Feb 21, 2018·Trends in Cancer·Vivek Subbiah, Razelle Kurzrock
Mar 24, 2018·Science·Antoni Ribas, Jedd D Wolchok
May 29, 2018·Cancer Discovery·Christoph HeiningHanno Glimm
Aug 24, 2018·Annals of Oncology : Official Journal of the European Society for Medical Oncology·J MateoL Pusztai
Jan 16, 2019·Nature Genetics·Robert M SamsteinLuc G T Morris
Feb 15, 2019·Clinical Cancer Research : an Official Journal of the American Association for Cancer Research·Patrick G PiliéTimothy A Yap
Apr 23, 2019·International Journal of Cancer. Journal International Du Cancer·Jonas LeichsenringAlbrecht Stenzinger
Sep 6, 2019·Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology·R Wendel NaumannKathleen N Moore
Oct 1, 2019·The New England Journal of Medicine·Scott KopetzJosep Tabernero
Jan 11, 2020·Journal of the National Cancer Institute·Keith T FlahertyUNKNOWN NCI-MATCH Team
Jan 29, 2020·Annals of Oncology : Official Journal of the European Society for Medical Oncology·O TrédanUNKNOWN ProfiLER investigators
Feb 8, 2020·Cancer Discovery·Vivek SubbiahDavid M Hyman

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Citations

Sep 14, 2021·Molecular Oncology·Ulrik RingborgLaurence Zitvogel
Nov 3, 2021·Cancer Discovery·Alexander EggermontGiuseppe Curigliano

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