Incorporating ibrutinib into the first-line treatment of patients with chronic lymphocytic leukemia in Brazil: supporting the clinical guideline development

Article type
Authors
da Silva Pereira Curado D1, do Nascimento A2, Machado-Rugolo J3, Millan Fachi M4, Montezuma T4, Barbosa W5, Tolentino Silva M6, Hellen Dantas de Oliveira K
1Department of Management and Incorporation of Health Technologies, Ministry of Health, Brasilia, Federal District, Brazil; Graduate Program in Public Health, University of Brasilia, Brasilia, Federal District, Brazil
2Department of Management and Incorporation of Health Technologies, Ministry of Health, Brasilia, Federal District, Brazil; Health Technology Assessment Division, Brazilian National Cancer Institute (INCA), Rio de Janeiro, Rio de Janeiro, Brazil
3Health Technology Assessment Center, Clinical Hospital of the Botucatu Medical School, Botucatu, São Paulo, Brazil; Pharmaceutical Science Program, University of Sorocaba, Sorocaba, São Paulo, Brazil
4Health Technology Assessment Unit, Hospital Alemão Oswaldo Cruz, São Paulo, São Paulo, Brazil
5Department of Management and Incorporation of Health Technologies, Ministry of Health, Brasilia, Federal District, Brazil; Faculty of Ceilandia, University of Brasilia, Brasilia, Federal District, Brazil
6Faculty of Health Sciences / Department of Public Health, University of Brasilia, Brasilia, Federal District, Brazil
Abstract
Background: Brazil does not have clinical guidelines for chronic lymphocytic leukemia (CLL), making it necessary for the medications to be formally incorporated into the Brazilian public health system (SUS). In 2023, rituximab associated with fludarabine and cyclophosphamide (FCR) was evaluated and recommended by the Brazilian HTA institution, the National Committee for Health Technology Incorporation (Conitec), for first-line treatment of CLL. Therefore, other therapeutic options, such as ibrutinib + rituximab, must be evaluated, to compose a future clinical guideline for CLL.
Objectives: evaluate the efficacy and safety of ibrutinib associated with rituximab (IR) in the first-line treatment of CLL compared to FCR.
Methods: a systematic review (SR) is being performed with searches in MEDLINE/PubMed, EMBASE, Cochrane Library and clinicaltrial.gov. Outcomes of interest are overall survival (OS), progression-free survival (PFS), quality of life and serious adverse events. The risk of bias was evaluated by RoB 2 tool and certainty of evidence will be evaluated with GRADE. A random effect model meta-analysis will be performed if it is possible. The SR protocol is registered in the PROSPERO database (CRD42023494868).
Results: Of the 1,678 references initially identified, three publications related to two RCTs were included. The initial findings suggest that, in terms of PFS, the IR regimen was significantly more effective compared to the FCR regimen across both studies, HRs 0.37 (95% CI: 0.27 to 0.51) and 0.44 (95% CI: 0.32 to 0.60). Regarding OS, the IR regimen outperformed the FCR regimen in one RCT (HR: 0.47 [95% CI: 0.25 to 0.89]), but not in another RCT (HR: 1.01 [95% CI: 0.61 to 1.68]). The global risk of bias was evaluated as some concern because of the lack of concealed allocation in both RCTs.
Conclusions: Both RCTs indicate gains in PFS when IR was used, with one of the RCTs suggesting significant benefits in OS. These advantageous findings of IR compared to FCR justify conducting an economic evaluation to determine the cost-effectiveness of the drug and subsequent assessment of Conitec. Therefore, if its incorporation into the SUS is viable, IR could be part of the national CLL guideline.