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Purpose: Immunotherapy is promising for lung cancer treatment, although at significant financial impact. The aim of this study was to evaluate the effectiveness and the efficacy-effectiveness gap of pembrolizumab in previously treated non-small cell lung cancer (NSCLC). Methods: A population-based ambispective cohort study was conducted. Cases of interest were identified through the National Cancer Registry database and additional data sources. Patients aged ≥18 years, diagnosed with NSCLC and exposed to pembrolizumab, between 23 June 2016 and 31 October 2018, as second or later lines of treatment for advanced disease were included. Patients were followed-up until death or cutoff date (30 April 2019). Primary outcome was overall survival (OS). Secondary outcomes were progression-free survival (PFS), event-free survival (EFS), and adverse events (AEs) leading to treatment discontinuation. The efficacyeffectiveness gap was evaluated comparing results with clinical trial data. Results: A total of 181 patients were included. Median age was 63 years (range 33-94); 74.6% were male. Median treatment duration was 5.6 months (interquartile range: 1.4-10.4) and, at cutoff date, treatment had been discontinued in 141 patients, mainly due to disease progression. Median OS was 13.0 months (95% confidence interval [CI] 9.3-15.9) and 1-year OS was 53.1% (95% CI 45.2%-60.3%). Median PFS was 5.6 months (95% CI 4.6-7.2), median EFS was 4.7 months (95% CI 3.2-6.0), and treatment was discontinued due to AE in 8.3% of cases (n = 15). The efficacy-effectiveness gap seems to favor pembrolizumab use in clinical practice. Conclusion: Real-world data suggest the performance of pembrolizumab to reflect the clinical trial outcomes in previously treated NSCLC.

Objectives To evaluate the effectiveness and safety of pembrolizumab use in advanced melanoma in a real‐life context; and to explore the existence of an efficacy‐effectiveness gap, comparing registry data with the reference clinical trial. Methods This study followed the guidelines for good pharmacoepidemology practice. An ambispective cohort was constituted, initiating the observation upon drug approval (17/07/2015) and following exposed patients until death or cut‐off date (15/11/2019). The primary outcome was overall survival (OS); secondary outcomes comprised progression‐free survival (PFS), overall response rate (ORR) and the occurrence of adverse events (AE). For all survival analyses, the Kaplan‐Meier estimator was used, considering a 95% confidence interval (CI), aside with one‐year survival rates. Results A total of 125 patients constituted the cohort, originating from 16 hospitals in Portugal. Median OS was estimated to be 16.9 months (CI95% 11.3‐25.5) and the probability of survival after 1 year was 57.5% (CI95% 48.4%‐65.6%). Median PFS was estimated to be 4.8 months (CI95% 3.9‐6.7) and the probability of remaining progression‐free after 1 year was 32.8% (CI95% 24.8‐41.1). ORR was 30.4% (CI95% 22.5%‐39.3%). AEs were experienced by 82% of patients, and 27% experienced AE≥ grade 3. Conclusions Our data suggest lower effectiveness in a real‐life context than the efficacy reported in the clinical trial. Safety data seems, however, quite comparable to KEYNOTE‐006.

Background Chronic lymphocytic leukaemia (CLL) is the most common leukaemia among adults in western countries. Considering the increasing incidence and prevalence of this condition, it is highly relevant to better characterise these patients in Portugal, where data is still scarce. Methods To determine incidence, clinical presentation, survival and second malignancies, a population-based historical cohort study was conducted. Cases of interest were identified through the South Region Cancer Registry database and additional data sources. Patients aged ≥18 years, with a confirmed diagnosis of CLL or small lymphocytic lymphoma between January 1st, 2013 and December 31st, 2014 were included. Patients were followed‐up until death or cut-off date (December 31st, 2019). Results A total of 496 patients were included and median follow-up time was 5.46 years. Crude incidence rates were 5.03 and 5.22 per 100,000 inhabitants for 2013 and 2014, respectively, and age-adjusted incidence rates were 3.18:100,000 European population for 2013 and 3.35:100,000 European population for 2014. Median age at diagnosis was 71 years and the male/female ratio was 1.40. The majority of patients had leukemic presentation of the disease (86.09%), was diagnosed in Binet stage A (75.58%) and did not present B symptoms (84.01%), anaemia (haemoglobin ≤10g/dL; 90.63%) nor thrombocytopenia (platelet count ≤100 000/μL; 91.73%). Five-year overall survival (OS) rate was 70.53% (95%CI 66.31–74.34) and age, lactate dehydrogenase, Binet stage and a ≥5 Charlson comorbidity index score were independently associated with OS. Standardised-incidence ratios for any second malignancy and cutaneous squamous cell carcinoma were 1.59 (95%CI 1.19–2.08) and 10.15 (95%CI 6.28–15.51), respectively. Conclusion Incidence, clinical presentation and survival of CLL Portuguese patients are similar to those reported for other western countries. The increased risk of second malignancies raises concerns and needs adequate clinical watchfulness.

An historical population-based cohort study was conducted aiming to estimate the incidence of cutaneous malignant melanoma in the South Region of Portugal between Jan 2016 and June 2017; to clinically characterize the diagnosed individuals; to describe instituted treatment; and to estimate survival outcomes. Data were extracted from a cancer registry (ROR-Sul) covering 4,800,000 inhabitants (46% of the Portuguese population) and included a total of 789 individuals meeting eligibility criteria. The crude incidence rate (18 months) of melanoma was 13.36/100,000 inhabitants and the Age-Standardized Incidence Rate per 100,000 World population was 9.65/100,000 inhabitants. The most common histological subtypes identified were superficial extension, followed by malignant melanoma and nodular melanoma. Most cases were diagnosed in stage I (50.39%), equally distributed by sex and with a median age of 65 years. During the study period, 174 recurrence events were recorded (23.45%) and recurrence-free survival rate was significantly lower in more advanced stages. Patients had a two-fold risk of recurrence/death when in presence of ulcerated tumors [adjusted hazard ratio (adj HR) = 2.28; 95% confidence interval (CI) 1.40–3.70]. Overall survival rate at 3-years was 80.54% (95% CI 77.58–83.15), higher than previous national reports, and considerably higher for individuals diagnosed at earlier stages (p < 0.001). We have also identified differential survival outcomes in stages II–III explained by the uptake of sentinel lymph node biopsy. The epidemiologic and clinical characteristics of malignant melanoma patients studied are consistent with international literature. The incidence and rates observed suggests additional public health campaigns are needed to modify behaviours of the Portuguese population and thus reduce their risk.