Introduction: Novel drugs for multiple myeloma (MM), as IMIDs, new proteasome inhibitors, monoclonal antibodies, and CAR-T cells, have expanded the range of available therapies for this disease. Both transplant-eligible and ineligible patients have consistently benefited from these novel therapies. From 2015 to 2022, carfilzomib, daratumumab, elotuzumab, ixazomib, isatuximab, and pomalidomide have been approved in Brazil. This study aimed to evaluate the outcomes of MM patients with access to novel drugs approved in a developing country.
Methods: This prospective single center study included all newly diagnosed (ND) MM patients treated at Hospital Israelita Albert Einstein, Sao Paulo, Brazil, from 2019 to 2022. Overall (OS) and progression-free (PFS) survival curves were built with the Kaplan-Meier method. Cumulative incidence of progression and non-relapse mortality were built with the Gray method. Uni and multivariable analyses were carried out with Cox models. The impact of autologous HCT was measured with time-dependent Cox model.
Results: With a median follow-up of 1.6 years, 112 patients were included (19 had HCT). Median age was 67 y/o and most patients were International Staging System (ISS)-1 (32%) and ISS-2 (36%) and 17% were ISS-3. Respectively, 1-year OS and PFS were 83% and 66% and for the autologous HCT group,100% and 79%. Age (p = 0.03) and hemoglobin at diagnosis were associated with OS (p = 0.004). Both high ISS and autologous HCT were (not significantly) associated with lower OS, and improved OS (HR = 0.30), respectively. Only hemoglobin at diagnosis was associated with PFS (p = 0.009). One-year rates of progression and non-relapse mortality were 21% and 13%. Only lytic lesions were associated with relapse (p = 0.0008), while age (p = 0.05) and hemoglobin (p = 0.04) were independently associated with non-relapse mortality.
Conclusions: We have shown OS and PFS in patients treated with novel drugs for MM, comparable to those achieved in recent randomized trials. The leading cause of treatment failure was relapse. OS and PFS were 83% and 66% and in the HCT patients, 1-y OS was 100%. A recent real-world European study from high Human Development Index countries reported a 90% 1-y OS, which is similar to our results. A remarkable finding of our study is the adverse impact of anemia at diagnosis, with the risk of death 28% higher, mainly due to non-relapse mortality. However, anemia is not included in any of the International Staging Systems (ISS, R-ISS, R2-ISS). The relatively small number of patients in our study hampered some analyses, as the impact of high-dose chemotherapy. Nonetheless, the data was prospectively collected, and it reflects current practices in MM, including just-approved novel drugs. In summary, we achieved excellent outcomes in a recent cohort of patients with MM and have also shown the independent impact of anemia at diagnosis and the importance of such finding should be tested in a large cohort of patients.