Effectiveness of palbociclib with aromatase inhibitors for the treatment of advanced breast cancer in an exposure retrospective cohort study: implications for clinical practice

Breast Cancer Research

Research

Mariane Cossito¹  Cláudia Furtado¹  Fábio Cardoso Borges²  Adriana Ramos²  Catarina Ramos²  Claudia Brito²  Alexandra Mayer²  Ana da Costa Miranda²  Catarina Bernardo²  António Lourenço³  Filipa Alves da Costa⁴  Diogo Martins-Branco⁵  Arlindo R. Ferreira⁶ 

[1] Direção de Avaliação de Tecnologias de Saúde, Autoridade Nacional do Medicamento e Produtos de Saúde, I.P. (INFARMED I.P.), Lisbon, Portugal;Registo Oncológico Nacional (RON), Instituto Português de Oncologia de Lisboa Francisco Gentil, EPE, Lisbon, Portugal;Registo Oncológico Nacional (RON), Instituto Português de Oncologia de Lisboa Francisco Gentil, EPE, Lisbon, Portugal;NOVA Medical School, Universidade Nova de Lisboa, Lisbon, Portugal;Registo Oncológico Nacional (RON), Instituto Português de Oncologia de Lisboa Francisco Gentil, EPE, Lisbon, Portugal;Research Institute for Medicines (iMED), Faculty of Pharmacy, University of Lisbon, Lisbon, Portugal;Serviço de Oncologia Médica, Instituto Português de Oncologia de Lisboa Francisco Gentil, EPE, Lisbon, Portugal;Academic Trials Promoting Team, Institute Jules Bordet, Rue Meylemeersch 90, 1070, Brussels, Belgium;Unidade de Mama, Centro Clínico Champalimaud, Fundação Champalimaud, Lisbon, Portugal;Católica Medical School, Universidade Católica Portuguesa, Lisbon, Portugal;

关键词: Palbociclib;    Effectiveness;    Safety;    Cancer registry;   

DOI  :  10.1186/s13058-023-01678-5

 received in 2022-10-03, accepted in 2023-06-23,  发布年份 2023

来源: Springer

【 摘 要 】

BackgroundNew drugs for locally advanced or metastatic breast cancer have led to clinical benefits, aside with increasing costs to healthcare systems. The current financing model for health technology assessment (HTA) privileges real-world data. As part of the ongoing HTA, this study aimed to evaluate the effectiveness of palbociclib with aromatase inhibitors (AI) and compare it with the efficacy reported in PALOMA-2.MethodsA population-based retrospective exposure cohort study was conducted including all patients initiating treatment in Portugal with palbociclib under early access use and registered in the National Oncology Registry. The primary outcome was progression free survival (PFS). Secondary outcomes considered included time to palbociclib failure (TPF), overall survival (OS), time to next treatment (TTNT), and proportion of patients discontinuing treatment due to  adverse events (AEs). The Kaplan–Meier method was used and median, 1- and 2-year survival rates were computed, with two-sided 95% confidence intervals (95%CI). STrengthening the Reporting of OBservational studies in Epidemiology (STROBE) guidelines for reporting observational studies were used.ResultsThere were 131 patients included. Median follow-up was 28.3 months (IQR: 22.7–35.2) and median duration of treatment was 17.5 months (IQR: 7.8–29.1). Median PFS was 19.5 months (95%CI 14.2–24.2), corresponding to a 1-year PFS rate of 67.9% (95%CI 59.2–75.2) and a 2-year PFS rate of 42.0% (95%CI 33.5–50.3). Sensitivity analysis showed median PFS would increase slightly when excluding those not initiating treatment with the recommended dose, raising to 19.8 months (95%CI 14.4–28.9). By considering only patients meeting PALOMA-2 criteria, we could observe a major difference in treatment outcomes, with a mean PFS of 28.8 months (95%CI 19.4–36.0). TPF was 19.8 months (95%CI 14.2–24.9). Median OS was not reached. Median TTNT was 22.5 months (95%CI 18.0–29.8). A total of 14 patients discontinued palbociclib because of AEs (10.7%).ConclusionsData suggest palbociclib with AI to have an effectiveness of 28.8 months, when used in patients with overlapping characteristics to those used in PALOMA-2. However, when used outside of these eligibility criteria, namely in patients with less favorable prognosis (e.g., presence of visceral disease), the benefits are inferior, even though still favorable.

【 授权许可】

CC BY   
© The Author(s) 2023

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