Real World Evidence- the Proportion of Myeloma Patients' Death Due to Early Progression

Introduction: Real-world (RW) studies provide valuable insights and complimentary data that may not be covered by clinical trials. In general, RW studies customarily collect data retrospectively, but it is important to have prospective RW studies that can contribute in a better way to reduce possible bias and qualify information. Besides that, RW data fully represent patients followed in a routine

practice and can better evidence the treatment accessibility impact. Here we analyzed data on patients in the Brazilian Multiple Myeloma Study Group (GBRAM) database platform who have since deceased. Methods: Patients diagnosed with MM after January 1, 2018 have been included prospectively. The eligibility criteria were: intent-to-treat (ITT) MM patients, aged over 18 years and under care in any healthcare system (private and public). The present study describes the decease data of the patients (pts) included to date. Analysis was performed with the JAMOVI project software v2.3. Results: A total of 1,992 pts were included, 1,570 (78.8%) of whom were treated in the public sector (PuS) and 422 (21.2%) in the non-public sector (NPuS). The median age of the total population at diagnosis was 63.6 (25 - 96) and 1,024 (51.4%) patients were male. A total of 452 (22.7%) deaths, of whom 354 (78.3%) and 98 (21.7%) came from the PuS and NPuS, respectively, were registered. Of these obits, 192 (42.4%) occurred in the first year, 122 (27%) during the second year and 138 (29.6%) over two years or more from diagnosis. Death was related to progression in 68 (35.5%), 54 (44%) and 78 (56.5%) pts in the first year, second and over the second year from diagnosis, respectively. The median age of those who died was 66.2 (33 - 96) and 240 (53.1%) pts were male. The ECOG performance was 0 for 56 (12.4%), 1 for 103 (22.8%), 2 for 53 (11.7%) and 3/4 for 122 (27%) patients. When the ECOG was evaluated by pt origin, it was 3/4 for 16% and 30% of NPuS and PuS, respectively. The ISS was 3 for 232 (51.3%) of the pts and it represents 39% and 55% of the NPuS and PuS pts, respectively. The MM IgG isotype represents 221 (48.9%) pts. Regarding the eligibility criteria for the ASCT for the deceased population, 220 (48.7%) and 216 (47.8%) were eligible and ineligible, respectively. Of the 452 deceased pts, two hundred and one patients (44.5%) died because of progression, the majority 174 (86.6%) being from the PuS ( p < 0.01). D iscussion: This prospective study shows RW data regarding deaths which occurred in a multiple myeloma Brazilian group series. The PuS pts had a worse performance than those in the NPuS and represent 75% of the cases. It became evident that the PuS had higher ECOG and ISS scores and the main cause of death was progression in the PuS. Conclusion: This study demonstrates the high discrepancy between PuS and NPuS regarding the risk of death due to progression. The treatment accessibility could impact on that and public health policies need an emergency review to allow for an egalitarian system.