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Background: New 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. Methods: A 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. Results: There 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%). Conclusions: Data 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.

Funding Information: The authors acknowledge the RON network that cooperated in providing up-to-date information on cases diagnosed and treated with the drug of interest. Participating institutions: Centro Hospitalar Universitário de São João, Centro Hospitalar Universitário Lisboa Norte, Centro Hospitalar Universitário do Algarve, Hospital de Braga, Centro Hospitalar e Universitário de Coimbra, Centro Hospitalar de Trás-os-Montes e Alto Douro, Centro Hospitalar do Funchal, Centro Hospitalar de Vila Nova de Gaia/Espinho, Instituto Português de Oncologia de Coimbra Francisco Gentil, Centro Hospitalar Lisboa Ocidental, Hospital Garcia de Orta, Centro Hospitalar Universitário Lisboa Central, Hospital Distrital de Santarém, Centro Hospitalar de Entre o Douro e Vouga, Hospital da Senhora da Oliveira Guimarães, Centro Hospitalar de Setúbal, Centro Hospitalar e Universitário do Porto, Centro Hospitalar Tondela Viseu, Instituto Português de Oncologia de Lisboa Francisco Gentil, Hospital do Espírito Santo de Évora, Centro Hospitalar Barreiro Montijo, Hospital Beatriz Ângelo, Hospital do Santo Espírito da Ilha Terceira, Hospital do Divino Espírito Santo de Ponta Delgada, Hospital Pedro Hispano, Hospital Litoral Alentejano, Centro Hospitalar do Médio Ave, Hospital Distrital Caldas da Rainha, Centro Hospitalar Médio Tejo, Hospital Santa Luzia de Elvas, Hospital José Joaquim Fernandes Beja, Centro Hospitalar Universitário da Cova da Beira, Centro Hospitalar do Baixo Vouga, Hospital Professor Doutor Fernando Fonseca, Centro Clínico Champalimaud, Hospitais CUF, Hospitais da Luz, Hospitais dos Lusíadas). Publisher Copyright: © 2023, The Author(s). Background: New 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. Methods: A 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. Results: There 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%). Conclusions: Data 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. publishersversion published

Additional file 1: Table S1. Characterization of lines of therapy received prior to exposure to palbociclib and AI. Table S2. Sensitivity analysis for progression-free survival. Table S3. Subgroup analysis for progression-free survival.

Funding Information: We have read the journal's policy and the authors of this manuscript have the following competing interests: ARF reports travel grants from Roche and advisory board fees from Daiichi Sankyo, Gilead, Merck Sharp & Dohme, Novartis and Roche, outside the submitted work. DMB reports travel grants from LEO Farmacêuticos, Merck Sharp & Dohme, Ipsen, Janssen, Roche, and Novartis, advisory board fees from Janssen, Pfizer, Merck Sharp & Dohme, Angelini, AstraZeneca, and Novartis, and institutional grants from F. Hoffmann-La Roche, outside the submitted work. The other authors have declared that no competing interests exist. Funding Information: The authors acknowledge the RON network that cooperated in providing up-to-date information on cases diagnosed and treated with the drug of interest (participating institutions: Centro Hospitalar Universit?rio de S?o Jo?o, Centro Hospitalar Universit?rio Lisboa Norte, Centro Hospitalar Universit?rio do Algarve, Hospital de Braga, Centro Hospitalar e Universit?rio de Coimbra, Centro Hospitalar de Tr?s-os-Montes e Alto Douro, Hospital Central do Funchal, Centro Hospitalar de Vila Nova de Gaia/Espinho, Centro Hospitalar Lisboa Ocidental, Hospital Garcia de Orta, Centro Hospitalar Universit?rio Lisboa Central, Hospital Distrital de Santar?m, Centro Hospitalar de Entre o Douro e Vouga, Hospital da Senhora da Oliveira Guimar?es, Centro Hospitalar de Set?bal, Centro Hospitalar e Universit?rio do Porto, Centro Hospitalar Tondela Viseu, Hospital do Esp?rito Santo de ?vora, Centro Hospitalar Barreiro Montijo, Hospital Beatriz ?ngelo, Hospital do Santo Esp?rito da Ilha Terceira, Hospital do Divino Esp?rito Santo de Ponta Delgada, Hospital Pedro Hispano ? ULS Matosinhos, Hospital do Litoral Alentejano ? Santiago do Cac?m ? ULS Litoral Alentejano, Centro Hospitalar do Oeste, Centro Hospitalar M?dio Tejo, Hospital Jos? Joaquim Fernandes ? Beja ? ULS Baixo Alentejo, Centro Hospitalar Universit?rio da Cova da Beira, Centro Cl?nico Champalimaud, Hospitais CUF, Hospitais da Luz, Hospitais dos Lus?adas, Hospital Particular do Algarve). Publisher Copyright: © 2022 The Authors Background: Real-world (RW) data may provide valuable information on the effectiveness and safety of medicines, which is particularly relevant for clinicians, patients and third-party payers. Evidence on the effectiveness of palbociclib plus fulvestrant is scarce, which highlights the need of additional studies. The aim of this study was to evaluate the effectiveness of palbociclib plus fulvestrant in advanced breast cancer (ABC). Materials and methods: We conducted a population-based retrospective cohort study and cases of interest were identified through the Portuguese National Cancer Registry database and additional data sources. Patients aged≥18 years, diagnosed with ABC and exposed to palbociclib plus fulvestrant between May 31, 2017 and March 31, 2019 were included. Patients were followed-up until death or cut-off date (February 28, 2021). Primary outcome was rw-progression-free survival (rwPFS). Secondary outcomes were rw-overall survival (rwOS), rw-time to palbociclib failure (rwTPF) and rw-time to next treatment (rwTTNT). Results: A total of 210 patients were included. Median age was 58 years (range 29–83) and 99.05% were female. Median follow-up time was 23.22 months and, at cut-off date, treatment had been discontinued in 189 patients, mainly due to disease progression (n = 152). Median rwPFS was 7.43 months (95% confidence interval [CI] 6.28–9.05) and 2-year rwPFS was 16.65% (95%CI 11.97–22.00). Median rwOS was 24.70 months (95%CI 21.58–29.27), median rwTPF was 7.5 months (95%CI 6.51–9.08) and median rwTTNT was 11.74 months (95%CI 10.33–14.08). Conclusion: Palbociclib plus fulvestrant seems an effective treatment for ABC in real-world context. Compared to registrations studies, rwPFS and rwOS were shorter in real-life setting. publishersversion published

Funding Information: We would like to thank Andrea Bothwell who wrote the manuscript outline and first draft on behalf of Springer Healthcare Communications. We also thank Prof. Carina Silva (ESTEsL – Escola Superior de Tecnologias de Saúde de Lisboa) who performed the preliminary statistical analysis of this study. This medical writing assistance and statistical analysis was funded by CUF Oncologia. Funding Information: Diogo Alpuim Costa has received honoraria from the Portuguese Navy, CUF Oncologia, and NTT DATA, and has served as a speaker, advisory board member, or has received research or education funding from CUF Oncologia, AstraZeneca, Hoffmann-La Roche, Merck KGaA, Novartis, Pfizer, Uriage, Daiichi Sankyo, Gilead, Merck Sharp & Dohme, Nanobiotix, Puma Biotechnology Inc., Sanofi, and Seagen Inc. Margarida Brito has participated as advisory board member for Roche, Novartis, Merck Sharp & Dohme, and Pfizer. Mário Fontes-Sousa has served as a speaker or advisory board member for Bristol Myers Squibb, Daiichi Sankyo, Gilead, Lilly, Merck Sharp & Dohme, Novartis, Pfizer, Roche, and Servier. Diogo Martins-Branco received honoraria and advisory board fees from Janssen, Pfizer, Merck Sharp & Dohme, Angelini, AstraZeneca, and Novartis, meeting and travel grants from LEO Farmacêuticos, Merck Sharp & Dohme, Ipsen, Janssen, and Roche, and institutional grants from F. Hoffmann-La Roche Ltd. José Guilherme Gonçalves Nobre, João Paulo Fernandes, Marta Vaz Batista, Ana Simas, Carolina Sales, Helena Gouveia, Leonor Abreu Ribeiro, Andreia Coelho, Mariana Inácio, André Cruz, Mónica Mariano, Joana Savva-Bordalo, Ricardo Fernandes, André Oliveira, Andreia Chaves, Mafalda Sampaio-Alves, and Noémia Afonso have nothing to declare. Publisher Copyright: © 2022, The Author(s). Introduction: Cancer care providers have faced many challenges in delivering safe care for patients during the COVID-19 pandemic. This cross-sectional survey-based study investigated the impact of the pandemic on clinical practices of Portuguese medical oncologists caring for patients with breast cancer. Methods: An anonymous online survey comprising 42 questions gathered information regarding COVID-19 testing, treatment in (neo)adjuvant and metastatic settings, and other aspects of breast cancer management. Practices before and during the pandemic were compared, and potential differences in outcomes according to respondents’ regions, case volumes, and practice type were explored. Results: Of 129 respondents, 108 worked in the public health system, giving a representative national picture of the impact of the COVID-19 pandemic on breast cancer management. Seventy-one percent of respondents reported a reduction in visits for new cases of breast cancer, and there was a shift towards increased use of telemedicine. Clinical decision-making was largely unaffected in the most aggressive indications (i.e., triple-negative, HER2-positive, visceral crisis). The use of neoadjuvant therapy increased when access to surgery was difficult, whereas dose-dense regimens decreased, and cyclin-dependent kinase 4/6 inhibitor treatment decreased for less aggressive disease and increased for more aggressive disease. The use of oral formulations and metronomic chemotherapy regimens increased, and clinical trial participation decreased. Some differences by respondents’ region and case volume were noted. Conclusion: Medical oncologists in Portugal implemented many changes during the COVID-19 pandemic, most of which were logical and reasonable responses to the current healthcare emergency; however, the true impact on patient outcomes remains unknown. publishersversion published

Introduction There is growing concern about the aggressiveness of cancer care at the end of life (ACCEoL), defined as overly aggressive treatments that compromise the quality of life at its end. Recognising the most affected patients is a cornerstone to improve oncology care. Our aim is to identify factors associated with ACCEoL for patients with cancer dying in hospitals. Methods All adult patients with cancer who died in public hospitals in mainland Portugal (January 2010 to December 2015), identified from the hospital morbidity database. This database provided individual clinical and demographic data. We obtained hospital and region-level variables from a survey and National Statistics. The primary outcome is a composite ACCEoL measure of 16 indicators. We used multilevel random effects logistic regression modelling (p14 days in the hospital (43%, 42-43) and surgery (28%, 28-28) in the last 30 days. Older age (p