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

‘Bench to Bedside’ Project Recurrent gene mutations in chronic lymphocytic leukemia: An ERIC project within HARMONY Lesley Ann Sutton Uppsala Universitet Sweden 43 rd ERIC GENERAL ASSEMBLY

HARMONY: A European network of excellence Mission statement ― 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) The Consortium ― A total of 51 partners: - 44 participants (10 European countries) - 7 pharmaceutical companies (EFPIA) ― 64 associated members (22 countries)

HARMONY: A European network of excellence Aims of HARMONY ― 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

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

Success of HARMONY: specific objectives to be reached ― Provide tools for analyzing complex data sets comprised of different layers of information so that molecular and clinical data can be linked to predict clinical outcomes 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.

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?

Recurrent gene mutations in CLL: An ERIC project in HARMONY 1) Gene mutations: ≥200 cases/center >25 centers > 4000 cases 2) Clinical data: Gender Date of birth Date of diagnosis 100% Date of treatment initiation Treatment received (first‐line) Date of last follow‐up Binet/Rai stage at diagnosis IGHV gene mutational status ≥90% FISH aberrations (11q-,13q-,+12, 17p-)

Recurrent gene mutations in CLL: An ERIC project in HARMONY Specific project goals  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 other 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’.

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 22 nd - Sat 24 th June 09:00 - 16:30, Sun 25 th June , 09:00 – 13:30 Venue and Room: Congress center IFEMA, Madrid, Booth number 800