Building of a data bank of patients who have multiple Myeloma treated with medications not available in SUS to assess the therapeutics efficiency: retrospective and prospective study

• Grant number: 21/02038-1
• Support Opportunities: Scholarships in Brazil - Scientific Initiation
• Effective date (Start): July 01, 2021
• Effective date (End): June 30, 2022
• Field of knowledge: Health Sciences - Medicine - Medical Clinics
• Principal Investigator: Nelson Hamerschlak
• Grantee: Beatriz Peral Venet Ferreira
• Host Institution: Instituto Israelita de Ensino e Pesquisa Albert Einstein (IIEPAE). Sociedade Beneficente Israelita Brasileira Albert Einstein (SBIBAE). São Paulo , SP, Brazil

Abstract

Multiple myeloma is a malignant hematological disease with a higher incidence in the elderly. The disease is considered incurable. New drugs have become available in recent years and it is expected that the availability of new drugs will increase survival. Phase III pivotal studies, however, have focused on progression-free survival, not overall survival. This is an observational cohort study. There will be two cohorts: (1) a retrospective, from 2006 to 2020, and (2) a prospective, from 2021 to 2023. Data will be collected from patient records. All patients treated for multiple myeloma at HIAE will be included. The main objectives are to build a database of myeloma patients treated with drugs not available at SUS and to assess changes in risk of death over the years and try to correlate these changes with the availability of new medications. The hypothesis is that the availability of new drugs increases patient survival. Other objectives are to compare the results of eligible and ineligible patients for autologous bone marrow transplantation and to describe the clinical-epidemiological characteristics of the patients. The outcomes studied will be overall survival, progression-free survival, time to next treatment, response to induction regimen, quality of life, second transplant, and the number of cells collected for autologous transplantation. The survival curves will be analyzed by Kaplan-Meier curves and compared by log-rank test and Cox model analysis.