Chronic lymphocytic leukemia: Improving survival and quality of life

Chronic lymphocytic leukemia (CLL) is the most common form of leukemia in Europe and it remains incurable. Targeted therapies have revolutionized the treatment of CLL. However, many patients develop resistance, have severe side effects, or relapse during treatment. To prevent ineffective treatment and reduce toxic effects, there is an unmet clinical need for tailoring optimal therapy to each patient. In CLL-OUTCOME, we aim to improve survival and health related quality of life (hrQoL) for patients with CLL; specifically, to increase treatment efficacy of targeted therapies and to reduce adverse events. The consortium has previously identified biomarkers for predicting clinical outcomes and developed a novel machine learning-based prognostic model based on these biomarkers as a clinical decision support tool in the ERA PerMed project CLL-CLUE. Here, we will validate this prognostic model in a feasibility study using samples and data from clinical studies. This will enable us to optimize the model input and individual patient outcomes for hrQoL and treatment efficacy in preparation for a prospective clinical trial. To facilitate implementation of the clinical decision support tool in the healthcare system, we will develop health economic models to assess the prognostic model retrospectively and prospectively, with emphasis on involving key stakeholders, including patient representatives and pharmaceutical companies with drug development for CLL, both in product development and when deciding trial endpoints. CLL-OUTCOME will be carried out as a collaborative effort including clinical, molecular, computational, and health economic research groups. It will benefit from the transnational consortium, including established access to patient samples and ongoing clinical trials from several European countries. The consortium is interdisciplinary, reflecting the complex process of developing and implementing new prognostic models for personalised medicine