leukemia: An ERIC project within HARMONY Lesley Ann Sutton Uppsala - - PowerPoint PPT Presentation

‘Bench to Bedside’ Project

43rd ERIC GENERAL ASSEMBLY

Recurrent gene mutations in chronic lymphocytic leukemia:

An ERIC project within HARMONY

Lesley Ann Sutton Uppsala Universitet Sweden

HARMONY: A European network of excellence ― A total of 51 partners:

• 44 participants (10 European countries)
• 7 pharmaceutical companies (EFPIA)

The Consortium

― 64 associated members (22 countries) ― capture, integrate, analyze and harmonize Big Data from high- quality multidisciplinary sources for the purpose of acquiring valuable knowledge across the spectrum of hematologic malignancies (HMs)

Mission statement

― rapidly capture the current status of treatment of HMs; ― identify areas of utmost need to improve patient management; ― involve the patient perspective ― better characterization of HMs to improve treatment strategies; ― identify novel treatment targets and drugs; ― foster the design of innovative clinical and pre-clinical studies; ― provide novel resources and algorithms to more rapidly advance innovative concepts of healthcare and patient HARMONY: A European network of excellence

Aims of HARMONY

Success of HARMONY: specific objectives to be reached Define meaningful and harmonized clinical endpoints and

• utcomes in HMs to facilitate clinical decision-making and allow a

more rapid development and evaluation of MAPPs, while at the same time taking into account patients' needs. Improve access and management of Big Data series from HM patients through the development of a data platform that harmonizes data capture and provides a repository of representative relevant HM data

Success of HARMONY: specific objectives to be reached Identify specific biomarkers, which better define outcome

• parameters. Through better understanding of predictive and/or

prognostic biomarkers HARMONY aims to contribute to the development of specific treatments. ― Provide tools for analyzing complex data sets comprised

• f different layers of information so that molecular and

clinical data can be linked to predict clinical outcomes

Recurrent gene mutations in CLL: An ERIC project in HARMONY

Rationale

• Many recurrent gene mutations exist in CLL
• Variable and low frequency (<10% each)
• Correlate with distinct disease and clinical outcomes

Prognostic or predictive capacity of gene mutations? Could particular gene mutation(s) aid in clinical decision- making, including therapy selection and response prediction?

2) Clinical data: Gender

Date of birth Date of diagnosis Date of treatment initiation Treatment received (first‐line) Date of last follow‐up Binet/Rai stage at diagnosis IGHV gene mutational status FISH aberrations (11q-,13q-,+12, 17p-)

1) Gene mutations:

>25 centers ≥200 cases/center 100% ≥90% > 4000 cases

Recurrent gene mutations in CLL: An ERIC project in HARMONY

 Evaluate the mutational status several recurrently mutated genes in a large and well-annotated (both molecular parameters and clinical characteristics) series of CLL cases.  Assess the prognostic impact and clinical relevance of recurrent gene mutations.  Identify distinct patterns of associations between recurrent mutations with

• ther clinicobiological features in CLL

 Perform robust validation of recently proposed prognostication models that incorporate both cytogenetic and molecular lesions prognostic indices.  Building models which combine several clinical and genetic parameters will also be important to evaluate how to create training and validation cohorts in this era of ‘big data’.

Recurrent gene mutations in CLL: An ERIC project in HARMONY

Specific project goals

EHA22: HARMONY COMMUNICATION & DISSEMINATION ACTIVITIES

HARMONY session Enabling Better and Faster Treatment for Patients with Hematological Malignancies. Date and Time: Saturday, 24 June, From 14:45 – 15:45 hrs. Venue and Room: Congress center IFEMA, Madrid, Room N115 Objectives: Introduction of the HARMONY Alliance HARMONY Booth Let us connect and build our HARMONY community Date and Time: Thurs 22nd - Sat 24th June 09:00 - 16:30, Sun 25th June, 09:00 – 13:30 Venue and Room: Congress center IFEMA, Madrid, Booth number 800