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 highquality 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 decisionmaking, 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 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-) 100% 90%
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