DOES INCORPORATION MEAN ACCESS? REAL-WORLD USE OF CARFILZOMIB IN BRAZIL

@article{cysne2026does,
  title = {{DOES INCORPORATION MEAN ACCESS? REAL-WORLD USE OF CARFILZOMIB IN BRAZIL}},
  author = {Dalila Nunes CYSNE and Edvan CRUSOÉ and Glaciano RIBEIRO and João Tadeu SOUTO-FILHO and Fernando Viera PERICOLE and Milton ARANHA and Jayr SCHMIDT-FILHO and Victor José Ferreira CORRêA and Juliana LIMA and Abel Costa NETO and Danielle Ovigli LOPES and Emanuella SOUZA and Breno GUSMAO and Luiza SILVA and Eduardo Flavio RIBEIRO and Rafael CUNHA and Wolney BARRETO and Angelo MAIOLINO and Vania HUNGRIA},
  journal = {Hematology, Transfusion and Cell Therapy},
  year = {2026},
  month = oct,
  doi = {10.1016/j.htct.2026.107865},
  url = {https://doi.org/10.1016/j.htct.2026.107865},
  note = {CC BY},
}

TY  - JOUR
TI  - DOES INCORPORATION MEAN ACCESS? REAL-WORLD USE OF CARFILZOMIB IN BRAZIL
AU  - Dalila Nunes CYSNE
AU  - Edvan CRUSOÉ
AU  - Glaciano RIBEIRO
AU  - João Tadeu SOUTO-FILHO
AU  - Fernando Viera PERICOLE
AU  - Milton ARANHA
AU  - Jayr SCHMIDT-FILHO
AU  - Victor José Ferreira CORRêA
AU  - Juliana LIMA
AU  - Abel Costa NETO
AU  - Danielle Ovigli LOPES
AU  - Emanuella SOUZA
AU  - Breno GUSMAO
AU  - Luiza SILVA
AU  - Eduardo Flavio RIBEIRO
AU  - Rafael CUNHA
AU  - Wolney BARRETO
AU  - Angelo MAIOLINO
AU  - Vania HUNGRIA
PY  - 2026
DA  - 2026/10/23
JO  - Hematology, Transfusion and Cell Therapy
DO  - 10.1016/j.htct.2026.107865
UR  - https://doi.org/10.1016/j.htct.2026.107865
AB  - Background: Although novel therapies, including bispecific antibodies, antibody–drug conjugates, and CAR T-cell therapies, are expanding treatment options for relapsed/refractory multiple myeloma (RRMM), carfilzomib (CFZ)-based regimens remain an important therapeutic option, particularly in settings where access to newer agents is limited. CFZ was approved by the Brazilian Health Regulatory Agency (ANVISA) in 2016 and incorporated into the Brazilian Unified Health System (Sistema Único de Saúde [SUS]) in 2023. However, formal incorporation does not necessarily translate into effective access. This real-world multicenter study describes the clinical profile, survival outcomes, and patterns of use of CFZ-based regimens as second- and/or third-line within a registry comprising public and private centers. Methods: This retrospective, multicenter, observational real-world cohort study included RRMM patients diagnosed between 2018 and 2026, with data obtained from the prospective Brazilian Multiple Myeloma Group (GBRAM) redistry. Patients who received at least one cycle of a CFZ-based regimen as second- or third-line therapy were included. Demographic, clinical, and treatment variables were analyzed descriptively. Overall survival (OS) was estimated using the Kaplan–Meier method. Results: A total of 76 patients from 22 centers received a CFZ-based regimen as second- or third-line therapy and were included in this analysis. Although the registry included both public and private centers, all patients included in the present study were treated at private centers. The median age at diagnosis was 57 years (33–83), 48.7% of patients were male, and 55.3% were non-White. The most frequent isotype was IgG (k or L), reported in 36.8% of patients. ISS stage III disease was recorded in 34.2% and bone lesions in 81.6%. An ECOG of 3–4 was recorded in 7.9%, with data missing for 23.7%. Although 82.9% were considered transplant-eligible at diagnosis, only 57.9% underwent transplantation. CFZ was most frequently combined with an immunomodulatory drug (KRd or KTd): 39.2% in the second line and 41.7% in the third line. It was combined with an anti-CD38 monoclonal antibody (Dara-Kd or Isa-Kd) in 29.4% and 16.7%, respectively. CFZ plus dexamethasone alone (Kd) accounted for 23.5% in the second line and 16.7% in the third line, whereas combinations with alkylating agents or other chemotherapy accounted for 7.8% and 25%, respectively. Unequal group sizes and missing data may limit direct comparisons between regimens. The median follow-up was 22(0.7-87.6) months and the median OS was 56.5 (95%CI, 25.5-NR) for the overral cohort. Conclusion: In this cohort, all patients receiving carfilzomib-based therapy were treated in the private system, despite its formal incorporation into SUS and the reliance of most Brazilians on public healthcare. Thus, incorporation has not translated into measurable real-world access to CFZ in the public system within this registry. This is particularly relevant given Brazilian real-world data showing significant OS disparities between patients treated in public and private settings (Hungria et al., 2025). Limited access to novel therapies may contribute to these differences. Efforts are needed not only to incorporate new therapies into the public system, but also to ensure their timely availability. Expanding access to evidence-based therapies could reduce healthcare disparities and improve survival for Brazilian MM patients.
SN  - 2531-1379
SN  - 2531-1387
ER  -