1. Pharmacoepidemiology Of Multiple Myeloma Treatment Patterns, Safety And Effectiveness In Real Life Conditions Aurore Palmaro Thesis directors: Dr Maryse Lapeyre Mestre and Dr Fabien Despas Doctoral School Biologie Santé Biotechnologies. Start date: Nov 2013 UMR INSERM 1027, Equipe 6 Pharmaco-épidémiologie, évaluation de l'utilisation et du risque médicamenteux INTRODUCTION AXIS 1: EXPLORING PATTERNS OF USE CONCLUSION AND PERSPECTIVES REFERENCES AND SUPPORT Study design: population- based cohort study Population: newly diagnosed MM patients selected among Midi-Pyrénnées SNIIRAM Beneficiaries Exposure of interest: alkylants, imids, proteasome inhibitors Endpoints: combination used, duration and number of cycle, adherence to recommendations in terms of dose, periodicity and number of cycles and previous therapy Palmaro A, Despas F, Protin C, Hebraud B, Montatsruc JL, Laurent G, Lapeyre-Mestre M. Determinants of non-adherence with lenalidomide and thalidomide in multiple myeloma: a cohort study in outpatient settings.IXème Congrès de Physiologie, de Pharmacologie et de Thérapeutique, P2T, Poitiers, 22-24 avril 2014 (communication affichée) AXIS 2: CONCOMITANT DRUGS AND SAFETY Study design: nationwide population-based cohort study Population: newly diagnosed MM patients selected among SNIIRAM Beneficiaries Exposure of interest: Imids: thalidomide, lenalidomide and pomalidomide Endpoints: occurrence of selected events (thrombosis, liver injuries,..) AXIS 3: SURVIVAL IN REAL LIFE SETTINGS Study design: nationwide population-based cohort study Population: newly diagnosed MM patients selected among SNIIRAM Beneficiaries Exposure of interest: autologous or allogeneic stem cell transplantation, conventional chemotherapy, imids, proteasome inhibitors Endpoints: Overall Survival and Progression Free Survival at 12, 24 and 36 months Management and therapeutic outcomes of certain cancer have been considerably improved by new therapies. Some of these new therapies are proposed as oral formulations, and offer the opportunity to pursuit treatment in ambulatory care settings. These drugs are based on different treatment sequences and a longer duration of therapy compared to traditional chemotherapy. Theses aspects, combined with a prolongation of survival time, contribute to the idea that cancer could, in some way, be assimilated to a chronic disease. However, beyond their benefits, some of these new drugs are raising new issues, in relation with their long term administration, their potential for delayed effect or interactions with other drugs. Their administration outside hospital settings require considering patient adherence and compliance, exposure to other drugs, and management of adverse reactions. Even if acute effects are mainly described during phase III clinical trials, new toxicity data can arise as a result of exposure of patients with different comorbidities or exposed to a wide range of different drugs. Resulting events or interactions can affect the therapeutic outcomes of treated patients, including their survival. As a consequence, therapeutic outcomes (response to treatment, overall survival, etc.) and toxicity for targeted therapies need to be investigated in real life settings. These investigations will be implemented for multiple myeloma. The prognosis of patients suffering from this B-cell malignancy has been considerably improved by 3 agents, thalidomide, lenalidomide, and bortezomib. The overall objective will be to describe the patterns of use of drugs for multiple myeloma and their safety and effectiveness in a real life perspective This work is supported by the programme "Investissements d'avenir" ANR-11-PHUC-001 The research is expected to provide new evidence and to update data from clinical trials on safety profile and outcomes of therapies used in multiple myeloma. The findings could lead to identify patient profiles, risk factors or risk period that could support recommendations to adapt follow-up of patients treated with these therapies in ambulatory settings.