Small cell lung cancer treatment and survival in Portugal: A retrospective analysis from the I-O Optimise initiative.

OBJECTIVE: We aim to describe treatment patterns and overall survival (OS) among a Portuguese cohort of patients with small cell lung cancer (SCLC). METHODS: This study utilised a database held by IPO-Porto, Portugal's largest oncology hospital. Adult patients diagnosed with SCLC at IPO-Porto between January 2012 and June 2017, with follow-up to December 2017, were included. Patients were stratified into subgroups with limited disease (LD) or extensive disease (ED). Treatment analyses were performed from 2015 onwards. RESULTS: Overall, 227 patients diagnosed with SCLC (37 LD; 190 ED) were analysed. Median OS (interquartile range [IQR]) was 15.0 months (3.8-39.3) for LD-SCLC and 5.0 months (1.7-10.3) for ED-SCLC. Among 19 patients diagnosed with LD-SCLC from 2015 onwards, 12 (63.2%) received initial treatment with systemic anticancer therapy (SACT) ± radiotherapy; 6 (31.6%) received best supportive care (BSC). Among 89 patients with ED-SCLC, 57 (68.5%) received SACT ± palliative radiotherapy; 28 (31.5%) received BSC. For patients receiving platinum doublet chemotherapy (±radiotherapy), median OS (IQR) was not reached for LD-SCLC and 5.4 months (2.3-10.9) for ED-SCLC. CONCLUSION: This real-world data analysis from a large Portuguese oncology hospital demonstrates a high disease burden for patients diagnosed with SCLC, particularly those with ED, and highlights a need for more effective therapies.

Real-world treatment patterns and survival outcomes for advanced non-small cell lung cancer in the pre-immunotherapy era in Portugal: a retrospective analysis from the I-O Optimise initiative.

BACKGROUND: As part of the multinational I-O Optimise research initiative, this retrospective cohort study of patients with advanced non-small cell lung cancer (NSCLC) evaluated real-world treatment patterns and survival prior to immunotherapy reimbursement in Portugal. METHODS: This study utilized a database held by IPO-Porto, Portugal's largest oncology hospital. Adult patients diagnosed with stage IIIB or IV NSCLC from January 2012 to December 2016 at IPO-Porto, with follow-up to June 2017, were included. Treatment analyses were performed from 2015 onwards. Kaplan-Meier methods were used for overall survival (OS). Factors associated with OS and systemic anti-cancer therapy (SACT) treatment were assessed using multivariate statistical models. RESULTS: Of 1524 patients diagnosed with NSCLC at IPO-Porto, 1008 patients had advanced disease (stage IIIB: 10.1%, 154/1524, stage IV: 56.0%, 854/1524). For those with advanced disease, median age was 65 years (range: 21-92) and 75.6% (762/1008) were male. Median OS (interquartile range [IQR]) was 11.4 (5.2-26.9) months for stage IIIB and 6.3 (2.4-15.0) months for stage IV. Factors associated with decreased risk of death included female sex and epidermal growth factor receptor gene (EGFR)/anaplastic lymphoma kinase gene (ALK) mutations/rearrangements; factors associated with increased risk of death included older age and stage IV disease. Among patients diagnosed in 2015 or 2016, 75.8% (297/392) received ≥1 line of SACT. Platinum-based chemotherapy was the most common first-line therapy (non-squamous cell carcinoma [NSQ]: 72.9%; squamous cell carcinoma [SQ] 87.3%, 55/63; patients with EGFR/ALK mutations/rearrangements primarily received tyrosine kinase inhibitors). The likelihood of receiving SACT was lower in older patients and those diagnosed with stage IV disease. Patients not receiving SACT had poor survival outcomes (median OS [IQR]: NSQ, 1.8 [1.1-3.1] months; SQ, 2.3 (1.3-3.4) months), while median OS (IQR) in SACT-treated patients was 12.6 (6.1-24.5) months for NSQ and 10.3 (5.7-15.9) months for SQ. CONCLUSIONS: This real-world data analysis from a large Portuguese oncology hospital demonstrates a high disease burden for advanced NSCLC in the pre-immunotherapy era, with nearly one-quarter of patients not receiving SACT. Even in patients receiving SACT, median survival was only about 1 year.

Single-agent ibrutinib in RESONATE-2™ and RESONATE™ versus treatments in the real-world PHEDRA databases for patients with chronic lymphocytic leukemia.

After analyzing treatment patterns in chronic lymphocytic leukemia (CLL) (objective 1), we investigated the relative effectiveness of ibrutinib versus other commonly used treatments (objective 2) in patients with treatment-naïve and relapsed/refractory CLL, comparing patient-level data from two randomized registration trials with two real-world databases. Hazard ratios (HR) and 95% confidence intervals (CIs) were estimated using a multivariate Cox proportional hazards model, adjusted for differences in baseline characteristics. Rituximab-containing regimens were often prescribed in clinical practice. The most frequently prescribed regimens were fludarabine + cyclophosphamide + rituximab (FCR, 29.3%), bendamustine + rituximab (BR, 17.7%), and other rituximab-containing regimens (22.0%) in the treatment-naïve setting (n = 604), other non-FCR/BR rituximab-containing regimens (38.7%) and non-rituximab-containing regimens (28.5%) in the relapsed/refractory setting (n = 945). Adjusted HRs (95% CI) for progression-free survival (PFS) and overall survival (OS), respectively, with ibrutinib versus real-world regimens were 0.23 (0.14-0.37; p < 0.0001) and 0.40 (0.22-0.76; p = 0.0048) in the treatment-naïve setting, and 0.21 (0.16-0.27; p < 0.0001) and 0.29 (0.21-0.41; p < 0.0001) in the relapsed/refractory setting. When comparing real-world use of ibrutinib (n = 53) versus other real-world regimens in relapsed/refractory CLL (objective 3), adjusted HRs (95% CI) were 0.37 (0.22-0.63; p = 0.0003) for PFS and 0.53 (0.27-1.03; p < 0.0624) for OS. This adjusted analysis, based on nonrandomized patient data, suggests ibrutinib to be more effective than other commonly used regimens for CLL.

COPD Exacerbations Before and During COVID-19 in France, Germany, Italy, the UK and the US.

Background: Exacerbations of chronic obstructive pulmonary disease (COPD) were reported less frequently during the COVID-19 pandemic. We report real-world data on COPD exacerbation rates before and during this pandemic. Methods: Exacerbation patterns were analysed using electronic medical records or claims data of patients with COPD before (2017-2019) and during the COVID-19 pandemic (2020 through early 2022) in France, Germany, Italy, the United Kingdom and the United States. Data from each country were analysed separately. The proportions of patients with COPD receiving maintenance treatment were also estimated. Results: The proportion of patients with exacerbations fell 45-78% across five countries in 2020 versus 2019. Exacerbation rates in most countries were reduced by >50% in 2020 compared with 2019. The proportions of patients with an exacerbation increased in most countries in 2021. Across each country, seasonal exacerbation increases seen during autumn and winter in pre-pandemic years were absent during the first year of the pandemic. The percentage of patients filling COPD prescriptions across each country increased by 4.53-22.13% in 2019 to 9.94-34.17% in 2021. Conclusion: Early, steep declines in exacerbation rates occurred in 2020 versus 2019 across all five countries and were accompanied by a loss of the seasonal pattern of exacerbation.

Serum calcium and incident and fatal prostate cancer in the Swedish AMORIS study.

BACKGROUND: Observational studies have shown a positive association between intake of dairy products as well as serum levels of calcium and prostate cancer (PCa) risk. We studied the association between serum calcium and PCa while also accounting for levels of albumin, a protein to which calcium is bound. METHODS: A cohort based on 196,022 men with baseline information on calcium (mmol/L) and albumin (g/L) was selected from the Swedish Apolipoprotein MOrtality RISk study. Age-stratified multivariable Cox proportional hazard models were used to analyze associations between calcium and incident and fatal PCa risk. RESULTS: A total of 6,353 men were diagnosed with PCa and 731 died of PCa during mean follow-up of 12 years. A weak negative association was found between levels of calcium or albumin-corrected calcium and PCa risk (HR for quartiles of albumin-corrected calcium: 0.95 (0.89-1.02), 0.93 (0.86-1.00), and 0.91 (0.85-0.98) for the 2nd, 3rd, and 4th quartile compared to the 1st; p for trend: 0.012). BMI did not affect these findings. No association was found between calcium levels and fatal PCa. A positive association between Ca and death was observed when censoring for PCa [HR per SD: 1.14 (1.13-1.16)]. CONCLUSION: The weak negative association between Ca and PCa risk is likely explained by the relation between Ca and death. This illustrates the need to handle competing risks when defining whether Ca is involved in PCa etiology or whether it acts as a marker of other metabolic events in the causal pathway.

Burden of pulmonary arterial hypertension in England: retrospective HES database analysis.

BACKGROUND: The clinical and economic burden of pulmonary arterial hypertension (PAH) is poorly understood outside the United States. This retrospective database study describes the characteristics of patients with PAH in England, including their healthcare resource utilisation (HCRU) and associated costs. METHODS: Data from 1 April 2012 to 31 March 2018 were obtained from the National Health Service (NHS) Digital Hospital Episode Statistics database, which provides full coverage of patient events occurring in NHS England hospitals. An adult patient cohort was defined using an algorithm incorporating pulmonary hypertension (PH) diagnosis codes, PAH-associated procedures, PH specialist centre visits and PAH-specific medications. HCRU included inpatient admissions, outpatient visits and Accident and Emergency (A&E) attendances. Associated costs, calculated using national tariffs inflation-adjusted to 2017, did not include PAH-specific drugs on the High Cost Drugs list. RESULTS: The analysis cohort included 2527 patients (68.4% female; 63.6% aged ⩾50 years). Mean annual HCRU rates ranged from 2.9 to 3.2 for admissions (21-25% of patients had ⩾5 admissions), 9.4-10.3 for outpatient visits and 0.8-0.9 for A&E attendances. Costs from 2013 to 2017 totalled £43.2M (£33.9M admissions, £8.3M outpatient visits and £0.9M A&E attendances). From 2013 to 2017, mean cost per patient decreased 13% (from £4400 to £3833) for admissions and 13% (from £1031 to £896) for outpatient visits, but increased 52% (from £81 to £123) for A&E attendances. CONCLUSION: PAH incurs a heavy economic burden on a per-patient basis, highlighting the need for improved treatment strategies able to reduce disease progression and hospitalisations.The reviews of this paper are available via the supplemental material section.

Treatment and outcomes for early non-small-cell lung cancer: a retrospective analysis of a Portuguese hospital database.

AIM: This observational study evaluated treatment patterns and survival for patients with stage I-IIIA non-small-cell lung cancer (NSCLC). MATERIALS & METHODS: Adults newly diagnosed with NSCLC in 2012-2016 at IPO-Porto hospital were included. Treatment data were available for patients diagnosed in 2015-2016. RESULTS: 495 patients were included (median age: 67 years). The most common treatments were surgery alone or with another therapy (stage I: 66%) and systemic anticancer therapy plus radiotherapy (stage II: 54%; stage IIIA: 59%). One-year OS (95% CI) for patients with stage I, II and IIIA NSCLC (diagnosed 2012-2016) were 92% (88-96), 71% (62-82) and 69% (63-75), respectively; one-year OS (95% CI) for treated patients with stage I-II or stage IIIA NSCLC (diagnosed 2015-2016) were 89% (81-97) and 86% (75-98) for non-squamous cell and 76% (60-95) and 49% (34-70) for squamous cell NSCLC. CONCLUSION: Treatment advances are strongly needed for stage I-IIIA NSCLC, especially for patients with squamous cell histology.

Dose increase beyond labelled dose of biologics is associated with incremental pharmacy costs: results from a real-world study in the UK.

Background: There is limited evidence regarding biologics dosing patterns and its costs among psoriasis patients in the United Kingdom (UK). Objective: This retrospective study assessed biologics dose increase beyond labelled dose and associated UK pharmacy costs in moderate to severe psoriasis patients. Methods: Adult psoriasis patients on biologic prescription for ≥12 continuous months between January 2010 and March 2015 with their diagnosis recorded in the UK Hospital Treatment Insights Database within one month of such prescription were included. The proportion of patients receiving ≥30% higher the average daily maintenance dose as per the UK product label, and associated 12-month costs were reported. Results: The study included 362 patients, receiving adalimumab (48%), etanercept (17%), ustekinumab (12%), and infliximab (23%). Beyond labelled dose increase was noted in 14% adalimumab, 20% etanercept, 18% ustekinumab and 28% infliximab patients with an associated mean annual extra cost per patient of £7936, £5912, £2422 and £2275, respectively. Conclusion: Dose increase beyond labelled dose of biologics was commonly observed in moderate to severe psoriasis in the UK and resulted in substantial annual incremental pharmacy costs.

PHEDRA: using real-world data to analyze treatment patterns and ibrutinib effectiveness in hematological malignancies.

AIM: PHEDRA (Platform for Haematology in EMEA: Data for Real World Analysis) is a unique, noninterventional project based on secondary data collection from real-world (RW) patient-level (health record) databases to understand treatment patterns in hematological malignancies. It compares ibrutinib's effectiveness with alternative treatments using RW data (RWD) and randomized clinical trials data. MATERIALS & METHODS: RWD are cleaned, validated, harmonized into a Common Data Model, and analyzed statistically alongside randomized clinical trial data. Treatment outcomes include overall and progression-free survival. RESULTS: To date, RWD (four databases) are available for 2840 patients in three indications, collected between 1990 and 2017. CONCLUSION: PHEDRA is an innovative approach to generate evidence to inform optimal treatment decisions in RW settings.