Retrospective Survival Studies

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Transcript Retrospective Survival Studies

BIOBANKING AND MOLECULAR PATHOBIOLOGY WG

MOLECULAR PATHOLOGY WG

Le Biobanche di Campioni Inclusi

Bari, 16/12/14 Giorgio Stanta, Dipartimento di Scienze Mediche Università di Trieste

ARCHIVE TISSUES: IMPROVING MOLECULAR MEDICINE RESEARCH AND CLINICAL PRACTICE

www.impactsnetwork.eu

G. Stanta

BIOBANKING AND MOLECULAR PATHOBIOLOGY WG

MOLECULAR PATHOLOGY WG

Technical working groups # DNA and RNA extraction SOPs and IQC rules in AT (OECI, ESP)- G. Stanta, A. Jung, S. Bonin # NGS in diagnostics and clinical research (ESP)- G. Hoefler, K. Kashofer # Proteomics SOPs in AT (ESP, OECI)- K. Becker # Preanalytical conditions in tissues (ESP, OECI)- M. Dietel, G. Bussolati, A. Sapino # Heterogeneity (inter-WGs of ESP - OECI) – ESP WGs chairman, G. Stanta # CEN–New ISO 15189 rules-DNA, RNA and proteins from tissues – K. Becker, P. Riegman, G. Stanta, K. Zatloukal Courses

BBMRI-ERIC CLINICAL BIOBANKS MEETING

Berlin 17/09/14 ARCHIVE TISSUES WG CLINICAL BIOBANKING WG

G. Stanta

MEDICO-BIOLOGICAL RESEARCH

BASIC RESEARCH ……………… TRANSLATIONAL RESEARCH > 10 years on average > devoted to future patients RETROSPECTIVE CLINICAL RESEARCH short time today’s patients -verification of clinical cases -efficacy of the new therapies -therapy response subgroups -intrinsic and acquired resistance BM -very long follow-up studies -performance evaluation -to establish costs and benefits Applied medicine and clinical research are today an integrated and indissoluble process

G. Stanta

CONCLUSIONS IN REVIEWS AND METANALYSIS……..

“ In conclusion, it is clearly evident from all these studies that, as with previous studies on gene profiling, most emerging miRNA signatures are not fully overlapping. These results might be explained by different specimens (frozen vs paraffin-embedded, micro- vs non-microdissected), experimental platforms used (quantitative PCR vs different miRNA array or in situ hybridization systems), tumour types, stage, and regimens as well as small sample size, ethnic differences in the study populations, lack of multivariate analysis and correction for multiple testing.

E Giovannetti et al Critical Reviews in Oncology/Hematology 81 (2012) 103–122

Gene expression signatures for predicting the outcome in stage II colorectal cancer meta-analysis

Takashi Akiyoshi et al “Recent approaches to identifying biomarkers for high-risk stage II colon cancer” Surg Today DOI 10.1007/s00595-012-0324-4 - 2012

Participating Organizations Organization Site Austrian BBMRI Node

www.bbmri.at

BBMRI-ERIC – Biobanking and Biomolecular Resources Research Infrastructure European Research Infrastructure Consortium Czech BBMRI Node

www.bbmri-eric.eu

www.recamo.cz/en/bbmri

EATRIS – European Infrastructure for Translational Medicine ECCO – European CanCer Organisation ECPC – European Cancer Patient Coalition EPAAC – European Partnership for Action Against Cancer

www.eatris.eu

www.ecco-org.eu

www.ecpc.eu

www.epaac.eu

ESP – European Society of Pathology EurocanPlatform French BBMRI Node German BBMRI Node German Society of Pathology Italian BBMRI Node Medical University of Graz OECI – Organisation of European Cancer Institutes Royal College Pathologists

www.esp-pathology.org

http://eurocanplatform.eu/ www.biobanques.eu

forthcoming www.dgp-berlin.de

www.bbmri-eric.it

www.meduni-graz.at

www.oeci.eu

www.rcpath.org

WHITE PAPER http://www.impactsnetwork.eu/Sections.aspx?section=170

Solutions

:

-RSS new study designs -Networks for accessibility of AT -Standardization of analysis methods

G. Stanta

Solutions

:

-RSS new study designs -Networks for accessibility of AT -Standardization of analysis methods

G. Stanta

RETROSPECTIVE SURVIVAL STUDIES (RSS)

STUDY DESIGN: WELL DEFINED AT THE BEGINNING OF THE STUDY CASES: POPULATION BASED or HIGH NUMBER UNSELECTED CASES COLLECTION OF CASES: WITHOUT FOLLOW-UP DATA MOLECULAR ANALYSES:

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ACCURATE MICRODISSECTION -DEFINED SOPs AND IQCs OUTCOMES: PROSPECTIVELY LIKE COLLECTED POSITIVE RESULTS NEGATIVE RESULTS ADDITIONAL STUDIES IN DIFFERENT POPULATION PROCESS OF VERIFICATION AND VALIDATION

G. Stanta

Solutions

:

-RSS new study designs -Networks for accessibility of AT -Standardization of analysis methods

G. Stanta

MODELS OF CLINICAL DATA AND TISSUE COLLECTION

Cancer Inst Pathology Pathology Network

Hospital Clinical Information

OECI MODEL

Archive of FFPE Tissues Hospital Clinical Information Archive of FFPE Tissues

BBMRI MODEL

Tumor Registry

Hospital

Pathology

Hospital Clinical Information Archive of FFPE Tissues

EPAAC MODEL

Regional Organiz.

Hospital Pathology

Hospital Clinical Information

AREA MODEL

Archive of FFPE Tissues

G. Stanta

SIAPEC

NIPAB Network of Italian Pathology Archive Biobanks Pathology Departments joined to develop a national network Tissue paraffin blocks stored for a minimum of 20 years by law.

It is estimated that in pathology archives over 300 million cases and around one billion tissue specimens are stored

How to organize the network?

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The network is set up as a virtual network of pathology archives (samples + associated clinical data) for clinical research.

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Participation in the network and in the projects is voluntary and collaborative (these materials are residues from clinical procedures with specific requirements).

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Clinical and follow-up information can be directly collected by pathologists because they also deal with the diagnostic procedures.

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Privacy is guaranteed by the professional secrecy of pathologists, who have the duty to pseudo-anonymize the cases.

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There can be different network models for different strategies that can be embedded in BBMRI-ERIC.

Solutions

:

-RSS new study designs -Networks for accessibility of AT -Standardization of analysis methods

G. Stanta

Clinical research irreproducibility

SOURCES OF VARIABILITY # Tissue and macromolecule pre-analytical preservation # Selection and standardization of analytical procedures # Heterogeneity at the clinical, morphological or molecular level Technological complexity Biological complexity BIOLOGICAL COMPLEXITY

G. Stanta

Clinical research irreproducibility

SOURCES OF VARIABILITY # Tissue and macromolecule pre-analytical preservation # Selection and standardization of analytical procedures # Heterogeneity at the clinical, morphological or molecular level Technological complexity Biological complexity BIOLOGICAL COMPLEXITY

G. Stanta

Pre-analytical Conditions in IHC

Outside the pathology lab Inside the pathology lab

G. Stanta

Pre-analytical Conditions in IHC

Outside the pathology lab Inside the pathology lab

Proteins during the preanalytical phase may be categorized into three groups: (1) predictable stable; (2) predictable unstable; (3) unpredictable.

RNA is always degraded and needs specific technical capabilities for extraction and degradation standardization.

CEN (the European Committee for Standardization) is developing preanalytic technical specifications on proteins, DNA and RNA in tissues to ISO 15189 which might become instrumental in 2015.

G. Stanta

Clinical research irreproducibility

SOURCES OF VARIABILITY # Tissue and macromolecule pre-analytical preservation # Selection and standardization of analytical procedures # Heterogeneity at the clinical, morphological or molecular level Technological complexity Biological complexity BIOLOGICAL COMPLEXITY

G. Stanta

Bonin S , Stanta G. Nucleic acids extraction methods in fixed and paraffin embedded tissues in cancer diagnostics. Exp Rev Mol. Diagn. 2013,13.

SOURCES OF VARIABILITY # Selection and standardization of analytical procedures

Serena Bonin, Falk Hlubek, Jean Benhattar, Carsten Denkert, Manfred Dietel, Pedro L. Fernandez, Gerald Höfler, Hannelore Kothmaier, Bozo Kruslin, Chiara Maria Mazzanti, Aurel Perren, Helmuth Popper, Aldo Scarpa, Paula Soares, Giorgio Stanta and Patricia JTA Groenen.”MULTICENTRE VALIDATION STUDY OF NUCLEIC ACIDS EXTRACTION FROM FFPE TISSUES” Virchow Arch 2009 “

Reverse transcription yield, indeed, can vary up to 100-fold depending on priming strategy, on the used enzyme, on the starting quantity of target RNA and even on the type of sequence that is going to be detected.

G. Stanta

Bonin S , Stanta G. Nucleic acids extraction methods in fixed and paraffin embedded tissues in cancer diagnostics. Exp Rev Mol. Diagn. 2013,13.

SOURCES OF VARIABILITY # Selection and standardization of analytical procedures

Serena Bonin, Falk Hlubek, Jean Benhattar, Carsten Denkert, Manfred Dietel, Pedro L. Fernandez, Gerald Höfler, Hannelore Kothmaier, Bozo Kruslin, Chiara Maria Mazzanti, Aurel Perren, Helmuth Popper, Aldo Scarpa, Paula Soares, Giorgio Stanta and Patricia JTA Groenen.”MULTICENTRE VALIDATION STUDY OF NUCLEIC ACIDS EXTRACTION FROM FFPE TISSUES” Virchow Arch 2009

SOPs-IQCs!!!

strategy, on the used enzyme, on the starting quantity of target RNA and even on the type of sequence that is going to be detected.

G. Stanta

Section Lysate

Proteomics in archive tissues

KF Becker

Clinical research irreproducibility

SOURCES OF VARIABILITY # Tissue and macromolecule pre-analytical preservation # Selection and standardization of analytical procedures # Heterogeneity at the clinical, morphological or molecular level Technological complexity Biological complexity BIOLOGICAL COMPLEXITY

G. Stanta

The complex problem of heterogeneity

MACROSCOPIC HETEROGENEITY

ETHNIC HETEROGENEITY CLINICAL HETEROGENEITY

MICROSCOPIC TISSUE HETEROGENEITY

HISTOLOGIC TISSUE COMPOSITION TISSUE REACTION

MOLECULAR HETEROGENEITY

GENETIC CLONAL EVOLUTION EPIGENETIC CLONAL EVOLUTION PHENOTYPIC PLASTICITY HOMO/HETERO-TYPIC INTERACTIONS

The complex problem of heterogeneity

MACROSCOPIC HETEROGENEITY

ETHNIC HETEROGENEITY CLINICAL HETEROGENEITY Design of the study

MICROSCOPIC TISSUE HETEROGENEITY

HISTOLOGIC TISSUE COMPOSITION TISSUE REACTION

MOLECULAR HETEROGENEITY

GENETIC CLONAL EVOLUTION EPIGENETIC CLONAL EVOLUTION PHENOTYPIC PLASTICITY HOMO/HETERO-TYPIC INTERACTIONS Micro-dissection

The complex problem of heterogeneity

MACROSCOPIC HETEROGENEITY

ETHNIC HETEROGENEITY CLINICAL HETEROGENEITY Design of the study

MICROSCOPIC TISSUE HETEROGENEITY

HISTOLOGIC TISSUE COMPOSITION TISSUE REACTION

MOLECULAR HETEROGENEITY

GENETIC CLONAL EVOLUTION EPIGENETIC CLONAL EVOLUTION PHENOTYPIC PLASTICITY HOMO/HETERO-TYPIC INTERACTIONS Micro-dissection NGS In situ methods

MICROSCOPIC TISSUE HETEROGENEITY

T.Centre

T. Invasion Hlubek et al.,Int J Cancer, 2007 F Elloumi et al BMC Medical Genomics 4:54;2011

G. Stanta

TMA ARRAYER MICRODISSECTION GENE - EXPRESSION QUANTITATIVE ANALYSIS - Ct Gene β-Actin mRNA CDK2 mRNA Sample Coring 1 Coring 2 Coring 1 Coring 2 1 2

21.48

28.45

21.64

28.22

29.43

32.92

29.16

32.92

3 4

23.71

28.84

23.72

28.75

32.32

33.29

31.99

33.29

5

28.08

28.36

33.24

33.24

#Treatment after coring 50°C for 30 min plus 60°C for 10 min (especially for 5mm cores) #Expected RNA yield from 5 sections (1cm 2 ), 5 μm thick: 5 - 25 μg (related to tissue type and extraction method

)

# 1 TM A #1

Gene β-Actin mRNA Sample Tissues Coring 1

23.01* 21.64

2

28.48

28.22

CDK2 mRNA Tissues Coring

30.11

29.16

33.13

32.92

3 4

24.53

29.72

23.72

28.75

31.76

33.25

31.99

33.29

5

29.15

28.36

33.56

33.24

*Cts after real time amplification of 10 ng of cDNA after reverse transcription with random hexamers - not standardized Cts G. Stanta

“Garbage in garbage out” “Standard in standard out”

G. Stanta