Quality of care assessment for non-small cell lung cancer patients: transforming routine care data into a continuous improvement system.

PURPOSE: The complexity of cancer care requires planning and analysis to achieve the highest level of quality. We aim to measure the quality of care provided to patients with non-small cell lung cancer (NSCLC) using the data contained in the hospital's information systems, in order to establish a system of continuous quality improvement. METHODS/PATIENTS: Retrospective observational cohort study conducted in a university hospital in Spain, consecutively including all patients with NSCLC treated between 2016 and 2020. A total of 34 quality indicators were selected based on a literature review and clinical practice guideline recommendations, covering care processes, timeliness, and outcomes. Applying data science methods, an analysis algorithm, based on clinical guideline recommendations, was set up to integrate activity and administrative data extracted from the Electronic Patient Record along with clinical data from a lung cancer registry. RESULTS: Through data generated in routine practice, it has been feasible to reconstruct the therapeutic trajectory and automatically calculate quality indicators using an algorithm based on clinical practice guidelines. Process indicators revealed high adherence to guideline recommendations, and outcome indicators showed favorable survival rates compared to previous data. CONCLUSIONS: Our study proposes a methodology to take advantage of the data contained in hospital information sources, allowing feedback and repeated measurement over time, developing a tool to understand quality metrics in accordance with evidence-based recommendations, ultimately seeking a system of continuous improvement of the quality of health care.

Clinical effectiveness of dimethyl fumarate in multiple sclerosis patients from Argentina.

BACKGROUND: We assessed the effectiveness, safety and patient-reported outcomes (PROs) of dimethyl fumarate (DMF) in real-world clinical practice in patients with multiple sclerosis (PwMS) from Argentina. METHODS: We conducted a multicenter ambispective cohort study in Argentina between September 2020 and March 2023. Changes in annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) score, magnetic resonance imaging (MRI), no evidence of disease activity (NEDA), PROs (depression, anxiety, fatigue, burden of treatment and quality of life), and safety data were collected at clinical visits performed every 6 months for at least 24 months. RESULTS: We included 161 PwMS (64% female). DMF treatment was associated with a significant reduction in ARR from baseline after 24 months of treatment (from 0.87 to 0.23, p < 0.001). Disability progression was observed in 27.9% vs. 9.3% pre- and post-DMF, and disability improvement was found in 13% of patients from baseline to month 24. MRI activity was significantly reduced compared with baseline. Fatigue, depression, and quality of life scores were significantly improved from baseline to 24 months. Flushing was the most frequent adverse event reported in 19.2%. No significant reduction was observed in the hospitalization rate pre- and post-DMF (19.8% vs. 5.6%, p = 0.32). During follow-up, 135 (83%) patients were relapse-free, 110 (68.3%) were MRI free activity (Gad + lesion) and 108 (67%) reached NEDA. CONCLUSIONS: DMF significantly reduced disease activity in PwMS from Argentina with a good safety profile in real-world settings. A significant impact on the quality of life during follow-up was found.

Bridging Real-World Data Gaps: Connecting Dots Across 10 Asian Countries.

The economic trend and the health care landscape are rapidly evolving across Asia. Effective real-world data (RWD) for regulatory and clinical decision-making is a crucial milestone associated with this evolution. This necessitates a critical evaluation of RWD generation within distinct nations for the use of various RWD warehouses in the generation of real-world evidence (RWE). In this article, we outline the RWD generation trends for 2 contrasting nation archetypes: "Solo Scholars"-nations with relatively self-sufficient RWD research systems-and "Global Collaborators"-countries largely reliant on international infrastructures for RWD generation. The key trends and patterns in RWD generation, country-specific insights into the predominant databases used in each country to produce RWE, and insights into the broader landscape of RWD database use across these countries are discussed. Conclusively, the data point out the heterogeneous nature of RWD generation practices across 10 different Asian nations and advocate for strategic enhancements in data harmonization. The evidence highlights the imperative for improved database integration and the establishment of standardized protocols and infrastructure for leveraging electronic medical records (EMR) in streamlining RWD acquisition. The clinical data analysis and reporting system of Hong Kong is an excellent example of a successful EMR system that showcases the capacity of integrated robust EMR platforms to consolidate and produce diverse RWE. This, in turn, can potentially reduce the necessity for reliance on numerous condition-specific local and global registries or limited and largely unavailable medical insurance or claims databases in most Asian nations. Linking health technology assessment processes with open data initiatives such as the Observational Medical Outcomes Partnership Common Data Model and the Observational Health Data Sciences and Informatics could enable the leveraging of global data resources to inform local decision-making. Advancing such initiatives is crucial for reinforcing health care frameworks in resource-limited settings and advancing toward cohesive, evidence-driven health care policy and improved patient outcomes in the region.

Real-world experience with pertuzumab and trastuzumab combined with chemotherapy in neoadjuvant treatment for patients with early-stage HER2-positive breast cancer: the NEOPERSUR study.

PURPOSE: HER2-targeted therapies have dramatically improved outcomes of patients with HER2-positive breast cancer (BC), as demonstrated in neoadjuvant trials. This study aims to provide real-world evidence on the use and effectiveness of combined pertuzumab, trastuzumab and chemotherapy (CT) in early-stage HER2-positive BC. METHODS: A retrospective, multicentre study was conducted on patients diagnosed with HER2-positive early BC treated with neoadjuvant pertuzumab and trastuzumab plus CT at 13 Spanish sites. The primary endpoint was pathological complete response (pCR). RESULTS: A total of 310 patients were included. Pertuzumab and trastuzumab were combined with anthracyclines and taxanes, carboplatin and docetaxel, and taxane-based CT in 77.1%, 16.5%, and 6.5% of patients, respectively. Overall, the pCR rate was 62.2%. The pCR was higher amongst patients with hormone receptor-negative tumours and with tumours expressing higher levels of Ki-67 (> 20%). After postoperative adjuvant treatment, 13.9% of patients relapsed. Those patients who did not achieve pCR, with tumours at advanced stages (III), and with node-positive disease were more likely to experience distant relapse. Median overall survival (OS) and distant disease-free survival (D-DFS) were not reached at the study end. The estimated mean OS and D-DFS times were 7.5 (95% CI 7.3-7.7) and 7.3 (95% CI 7.1-7.5) years, respectively (both were significantly longer amongst patients who achieved pCR). Grade 3-4 anti-HER2 related toxicities were reported in six (1.9%) patients. CONCLUSION: Neoadjuvant pertuzumab and trastuzumab plus CT achieve high pCR rates in real-life patients with HER2-positive early BC, showing an acceptable safety profile. Innovative adjuvant strategies are essential in patients at high risk of distant disease recurrence.

Societal costs and survival of patients with biopsy-verified non-alcoholic steatohepatitis: Danish nationwide register-based study.

INTRODUCTION AND OBJECTIVES: Studies on the societal burden of patients with biopsy-confirmed non-alcoholic fatty liver disease (NAFLD) are sparse. This study examined this question, comparing NAFLD with matched reference groups. MATERIALS AND METHODS: Nationwide Danish healthcare registers were used to include all patients (≥18 years) diagnosed with biopsy-verified NAFLD (1997-2021). Patients were classified as having simple steatosis or non-alcoholic steatohepatitis (NASH) with or without cirrhosis, and all matched with liver-disease free reference groups. Healthcare costs and labour market outcomes were compared from 5 years before to 11 years after diagnosis. Patients were followed for 25 years to analyse risk of disability insurance and death. RESULTS: 3,712 patients with biopsy-verified NASH (n = 1,030), simple steatosis (n = 1,540) or cirrhosis (n = 1,142) were identified. The average total costs in the year leading up to diagnosis was 4.1-fold higher for NASH patients than the reference group (EUR 6,318), 6.2-fold higher for cirrhosis patients and 3.1-fold higher for simple steatosis patients. In NASH, outpatient hospital contacts were responsible for 49 % of the excess costs (EUR 3,121). NASH patients had statistically significantly lower income than their reference group as early as five years before diagnosis until nine years after diagnosis, and markedly higher risk of becoming disability insurance recipients (HR: 4.37; 95 % CI: 3.17-6.02) and of death (HR: 2.42; 95 % CI: 1.80-3.25). CONCLUSIONS: NASH, simple steatosis and cirrhosis are all associated with substantial costs for the individual and the society with excess healthcare costs and poorer labour market outcomes.

Outcomes with atezolizumab in metastatic urothelial cancer: real-world data from a single institution.

PURPOSE: Immune checkpoint inhibitors (ICIs) have been incorporated in the treatment of metastatic urothelial carcinoma (mUC) upon platinum-based chemotherapy according to the positive results of large clinical trials. Nevertheless, results from unselected populations reflecting real-world data (RWD) are highly informative to the clinician. We reviewed daily clinical practice outcomes in patients with mUC who received atezolizumab in our institution. METHODS: Here we evaluated the clinical activity and safety of atezolizumab in an unselected population of mUC patients who received atezolizumab between 2018 and 2022 reflecting RWD. Efficacy and safety information were retrospectively collected. RESULTS: A total of 63 patients were included. The mean age was 68 years and the objective response rate was 14.3%. The median progression-free survival was 3 months and the median overall survival 6 months. At 1 year, 42% of the patients were alive. ECOG (0 vs 1) and neutrophil-lymphocytes ratio < 2 at the start of ICI were positive prognostic factors that discriminated between long vs short survivors. Overall tolerance was good with no new safety signals. Five patients (17%) had treatment-related adverse events grade ≥ 2 that required corticosteroids. CONCLUSION: In this retrospective study, atezolizumab was an effective and tolerable treatment option for patients with mUC after progression to platinum-based chemotherapy. Yet, patient selection remains critical to improve outcomes.

Real-world evidence: experiences and challenges for decision making in Latin America.

OBJECTIVE: The Health Technology Assessment (HTA) process aims to optimize health system funding of technologies. In recent years there has been an increase in what is known as Real-World Evidence (RWE) as a complement to clinical trials. The objective of Health Technology Assessment International's Latin American Policy Forum 2022 was to explore the utility of incorporating RWE into HTA and decision-making processes in the region. METHODS: This article is based on a background document, survey, and the deliberative work of the country representatives who participated in the Forum. RESULTS: There is a growing interest in the use of Real-World Data / Real-World Evidence in HTA processes in Latin America, although currently there are no specific local guidelines for RWE use by HTA agencies. At present, its use is limited to certain areas such as adding context to HTA reports, the evaluation of adverse events, or cost estimation.Potential future uses of RWE were identified, including the creation of risk-sharing agreements, the assessment of technology performance in routine practice, providing information on outcomes that are not so easily evaluated in clinical trials (e.g., the identification of specific subpopulations or quality of life), and the estimation of input parameters for economic evaluations. CONCLUSIONS: The participants agreed that there are several areas presenting significant potential to expand the application of RWD/RWE and that the development of normative frameworks for its use could be helpful.

Real-world evidence of neoadjuvant chemotherapy for breast cancer treatment in a Brazilian multicenter cohort: Correlation of pathological complete response with overall survival.

OBJECTIVES: We aimed to evaluate the pCR rate in patients receiving NAC for the treatment of breast cancer (BC) in a multicenter cohort in Brazil. Additionally, we aimed to use RWD to assess the impact of pCR on OS and DFS. METHODS: This was a retrospective, multicenter cohort study that included female patients over 18 years of age who were diagnosed with nonmetastatic breast cancer and received NAC. OS and DFS at five years were estimated by the Kaplan‒Meier method. Additionally, we conducted a multivariate analysis to identify factors that were significantly associated with pCR and OS. RESULTS: From 2011 to 2020, 1891 patients were included in the study, and 421 (22,3%) achieved pCR (ypT0 ypN0). Considering the presence of residual DCIS, pCR was achieved in 467 patients (23,5%). The pCR rate varied between the subtypes: HER-2+ (p = 0,016) and clinical stage IIIA and IIIB (p < 0,001). Among HER-2+ patients, those who received trastuzumab had a significantly higher pCR rate than those who did not receive trastuzumab (p < 0.0001). Similarly, patients with TNBC who received treatment with platinum-based regimens also showed higher pCR rates (p < 0.0001). OS was grouped according to pCR status, and the OS rate was 88,3% in the pCR group and 58.1% in the non-pCR group (p < 0.0001). The five-year DFS was 92.2% in the pCR group and 64.3% in the non-pCR group (p < 0.0001). CONCLUSION: The pCR rate and its prognostic value varied across BC subtypes. In our study, pCR could be used as a surrogate of favorable clinical outcome, as it was associated with higher OS and DFS rates.

Challenges and opportunities in building a health economic framework for personalized medicine in oncology.

Personalized medicine has allowed for knowledge at an individual level for several diseases and this has led to improvements in prevention and treatment of various types of neoplasms. Despite the greater availability of tests, the costs of genomic testing and targeted therapies are still high for most patients, especially in low- and middle-income countries. Although value frameworks and health technology assessment are fundamental to allow decision-making by policymakers, there are several concerns in terms of personalized medicine pharmacoeconomics. A global effort may improve these tools in order to allow access to personalized medicine for an increasing number of patients with cancer.

Real-World Data and Paper-Based Disease Registries in the Small Island Developing State of Barbados During the COVID-19 Pandemic.

Significant data is being produced on the impact of COVID-19 on aspects of clinical care. However, less is known about the impact on real-world health data. The US Food and Drug Administration defines real-world data as "data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources," including disease registries.1 The methodology used by the Barbados National Registry (BNR)-active pursuit of first-hand clinical data using paper-based charts from multiple sources-makes it an ideal example of real-world data. Real-world data can overcome the barriers to clinical trials often present in small island developing states. This paper reviews the impact of the COVID-19 pandemic on the data of the BNR within the context of the real-world data cycle. Data collected retrospectively for 2016-2018, undergoing traceback during the pandemic, demonstrated a greater reliance on death certificate registration. A 38% reduction in the collection of new cases was noted in the postpandemic period compared to data collected in previous periods. The lack of access to source data delayed cancer registry reporting. We conclude that, given the challenges highlighted during the COVID-19 pandemic, more effort should be placed on providing timely access to real-world data for public health decision-making, particularly in small island developing states.

Minimally invasive surgery as a new clinical standard for bone anchored hearing implants-real-world data from 10 years of follow-up and 228 surgeries.

Purpose: To explore the clinical practice development of different surgical techniques when installing bone-anchored hearing implants and their associated trends in outcomes. Design: Retrospective study of 228 bone-anchored hearing implants in 200 patients, performed over a 10-year period between 2012 and 2022 in a referral hospital. Method: Real-world data of demography, etiology, surgical setup, complications, and audiological outcomes were collected. Eligibility criteria from clinical practice were applied. Results: The minimally invasive technique is associated with shorter surgery duration, 20 vs. 44 min as compared to a linear incision technique. The minimally invasive technique was also associated with a lower occurrence of complications when compared to linear incision techniques (intraoperative; 1.8% vs. 4.9%, postoperative; 49% vs. 66%). Most differences were seen in complications relating to skin and wound healing. Conclusion: Adoption of a minimally invasive surgical technique for the installations of bone-anchored hearing implants can reduce surgical complexity without compromising safety aspects or clinical benefits.

Neoadjuvant Trastuzumab and Pertuzumab in Combination with Standard Chemotherapy for HER2-Positive Early Breast Cancer: Real-World Practice in Cuba.

PURPOSE: Dual HER2 blockade chemotherapy is the standard of care for localized HER2+ breast cancer (BC). However, despite the efficacy of neoadjuvant therapy, relapses occurring in around 10% of patients highlight the need to improve its clinical approach. Therefore, this study aimed to evaluate the effectiveness/safety of neoadjuvant therapy with subcutaneous (SC) trastuzumab- pertuzumab chemotherapy (real world) to extend the evidence, which comes mainly from clinical trials (selected population; intravenous [IV] trastuzumab). MATERIALS AND METHODS: A prospective, longitudinal, observational study in a Cuban hospital. POPULATION: women aged ≥18 years with histologically confirmed HER2+ early-stage BC (2017-2021) eligible for neoadjuvant treatment (IV pertuzumab, SC trastuzumab, taxane-based chemotherapy). The aim was to determine the pathological complete response (pCR) rate to this scheme, its safety, and the impact of patient's characteristics on the outcomes. RESULTS: Eighty-seven women were included: n=29 (DPT [docetaxel-IV pertuzumab- SC trastuzumab 600 mg; 4 cycles]); n=58 (ddAC-DPT [dose-dense anthracycline-based scheme+DPT]; 8 cycles). The median age was 57 years (range 30-83), ECOG 0: 97%. Time from diagnosis to treatment (median) was 28 days. The overall pCR rate was 62.1% (55.2%, DPT; 66.5%, ddAC-DPT; p =0.351); HR+, 47.7% vs. HR-, 76.7% (p=0.006). There were no statistically significant differences based on nodal status, stage, or Ki-67 levels. Overall, 94.2% of patients experienced ≥1 adverse event related to treatment, all of them grade 1-3 and more common with ddAC-DPT. The main cause of treatment delays (n=19; ddAC-DPT, 16; DPT, 3) was treatment-related toxicities. CONCLUSION: Neoadjuvant trastuzumab (SC) and pertuzumab plus chemotherapy for HER2+ early-stage BC showed benefits in a real-life setting, with an acceptable safety profile.

Observaciones en el mundo real sobre la efectividad del sistema flash de monitorización de glucosa en la práctica clínica en Chile / Real-world observations of the effectiveness of the flash glucose monitoring in clinical practice in Chile

OBJETIVO: Evaluar el tiempo en rango de glucosa y su asociación con otras medidas del control glicémico establecidas por el consenso internacional del tiempo en rango en usuarios de vida real del sistema flash de monitorización de glucosa FreeStyle LibreTM en Chile. MÉTODOS: Se analizaron los datos provenientes de la base de datos Freestyle Libre™ entre diciembre de 2014 y enero de 2022. Las lecturas se dividieron en 10 grupos (deciles) del mismo tamaño (cada decil contenía aproximadamente 498 usuarios) en función del tiempo en rango. Para cada decil se calculó la media de determinaciones diarias, el promedio de glucosa, la HbA1c, la desviación estándar de glucosa, el coeficiente de variación de la glucosa, el tiempo en rango, el tiempo de glucosa (porcentaje) por encima de 250 mg/dL (TA250), el tiempo de glucosa (porcentaje) por encima de 180 mg/dL (TA180), el tiempo por debajo (porcentaje) de 70 mg/dL (TB70) y el tiempo por debajo (porcentaje) de 54 mg/dL (TB54). RESULTADOS: Desde diciembre de 2014 hasta enero de 2022 hubo 4984 lectores. El grupo con el mayor tiempo en rango mostró significativamente una menor glucosa promedio que el grupo con el tiempo en rango más bajo (decil 1: media 248,3 mg/dL, decil 10: media 113,2 mg/L, diferencia ­135,1 mg/dL, p<0.05). Asimismo, el mayor tiempo en rango se asoció con una menor desviación estándar (decil 1: media 93,7mg/dL, decil 10: media 26,7mg/L, diferencia: -67,0 mg/ dL, p<0,05), menor coeficiente de variación (decil 1: media 37,8%, decil 10: media 23,3%, diferencia: -14,5%, p<0,05), menor TA250 (decil 1: media 46,5%, decil 10: media 0,2%, diferencia: -46,3%, p<0.05), menor TA180 (decil 1: media 73,9%, decil 10: media 3,8%, diferencia: -70,1%, p<0.05), menor TB70 (decil 5: mediana 6,13%, decil 10: mediana 1,70%, diferencia: -4,43%, p<0.05) y menor TB54 (decil 5: mediana 1,79%, decil 10: mediana 0,12%, diferencia: -1,67%, p<0.05). El mayor tiempo en rango se asoció también significativamente con más determinaciones diarias (decil 1: media 11,4, decil 10: media 16,6, diferencia: 5,2, p<0,05). La frecuencia media de las determinaciones entre todos los lectores fue de 14,7 determinaciones diarias. CONCLUSIONES: En los pacientes con diabetes en Chile, el empleo del sistema flash de monitorización demuestra la asociación entre el mayor tiempo en rango, la reducción de la variabilidad de la glucosa y un menor riesgo de hiperglucemias e hipoglicemias y también con un mayor compromiso.

OBJECTIVE: To evaluate glucose time in range and its association with other metrics of glucose control established by the International Consensus on TIR amongst real-life patients using the Flash Glucose Monitoring system FreeStyle LibreTM in Chile. METHODS: Data from the Freestyle Libre™ database between December 2014 and January 2022 were analyzed. Readers were divided into 10 groups (deciles) of the same size (each decile had approximately 498 users) according to time in range. For each decile of time in range, the mean of daily scans, average glucose, estimated HbA1c, glucose standard deviation, glucose coefficient of variation, time in range, glucose time (percentage) above 250 mg/dL (TA250), and glucose time (percentage) above 180 mg/dL (TA180), and the median of glucose time (percentage) below 70 mg/dL (TB70) and glucose time (percentage) below 54 mg/dL (TB54), were calculated. RESULTS: From December 2014 to January 2022, there were 4984 readers. The group with the highest TIR showed significantly lower average glucose than the group with the lowest TIR (decile 1: mean 248.3 mg/dL, decile 10: mean 113.2 mg/L, difference: ­135.1 mg/dL, p<0.05). In addition, more time in range was associated with a lower glucose standard deviation (decile 1: mean 93.7 mg/dL, decile 10: mean 26.7 mg/L, difference: -67.0 mg/dL, p<0.05), lower glucose coefficient of variation (decile 1: mean 37.8%, decile 10: mean 23.3%, difference: -14.5%, p<0.05), lower TA250 (decile 1: mean 46.5%, decile 10: mean 0.2%, difference: -46.3%, p<0.05),lower TA180 (decile 1: mean 73.9%, decile 10: mean 3.8%, difference: -70.1%, p<0.05), lower TB70 (decile 5: median 6.13%, decile 10: median 1.70%, difference: -4.43%, p<0.05) and lower TB54 (decile 5: median 1.79%, decile 10: median 0.12%, difference: -1.67%, p<0.05). Greater TIR was also associated with significantly more daily scans (decile 1: mean 11.4, decile 10: mean 16.6, difference: 5.2, p<0.05). Mean scan frequency amongst all readers was 14.7 daily scans. CONCLUSIONS: In patients with diabetes from Chile, the use of the flash glucose monitoring system demonstrates the association between greater TIR, reduced glucose variability, and reduced risk of hyperglycemia and hypoglycemia, and also its association with greater engagement.

Better Clinical Results in Rheumatoid Arthritis Patients Treated Under a Multidisciplinary Care Model When Compared with a National Rheumatoid Arthritis Registry.

Purpose: To describe clinical characteristics and effectiveness of health care in patients with rheumatoid arthritis (RA) as part of a multidisciplinary care model (MCM) in a specialized rheumatology center, compared with the results of a national registry of RA (NARRA) as evidence of real-world management. Patients and Methods: We conducted a real-world study (July 1, 2018 to June 30, 2019) based on an analysis of electronic health records of a cohort of RA patients managed with the "Treat-to-Target" strategy in a specialized rheumatology center in Colombia with an MCM, compared with the NARRA that includes different models of usual care. Results: We have analyzed 7053 subjects with RA treated at a specialized rheumatology center and 81,492 patients from the NARRA. Cohorts were similar in their baseline characteristics, with women in predominance and diagnosis age close to 50 years. At the time of diagnosis, a higher proportion of clinical diagnostic test use and rheumatology consultation access was observed in the specialized rheumatology center than in the national registry (4-6 per year versus three or less). In addition, higher proportions of patients in remission and low disease activity were reported for the specialized rheumatology center, with a >40% amount of data lost in the national registry. Pharmacological management was similar regarding the analgesic use. In the specialized center, Certolizumab was more frequently used than in the NARRA registry; also, there were significant differences in methotrexate, leflunomide, and sulfasalazine use, being higher in the specialized rheumatology center. Conclusion: The MCM of a specialized center in RA can guarantee comprehensive care, with better access to all the services required to manage the disease. It ensures specialist management and evidence-based care that facilitates the achievement of therapeutic objectives. In addition, better patient records and follow-ups are available to evaluate health outcomes.

Early Real-World Data to Assess Benefits and Risks of COVID-19 Vaccines: A Systematic Review of Methods.

Since the authorization of the first COVID-19 vaccines in December 2020, multiple studies using real-world data (RWD) have been published to assess their effectiveness/safety profile. This systematic review aimed to characterize the methods and outcomes of studies using RWD for assessment of COVID-19 vaccines, four months after vaccine approval. MEDLINE and EMBASE were searched to identify published studies until 6 May 2021. Two independent researchers selected relevant publications and extracted data from included studies. The risk of bias was assessed using New-Castle Ottawa tools. After screening 1086 studies, 15 were included. Out of the 15 studies, 12 (80%) followed a cohort design, 8 (53%) were based on USA data, 7 (47%) assessed health care professionals, and 14 articles (93%) assessed the BNT162b2 vaccine. Data sources included institutional databases, electronic health records, and patient-generated data. The primary endpoint mainly described was SARS-CoV-2-infection. Hospitalization and mortality were assessed in 2 studies. For the comparability domain, six studies (40%) had a high risk of bias. A few months after the beginning of COVID-19 vaccination, Real-world Evidence (RWE) provided timely safety surveillance and comparative effectiveness with findings that showed similar findings to Randomized control trial (RCT). Most of the initiatives assessed BNT162b2 and were conducted in the USA and used healthcare workers' data.

Developing a real-world database for oncology: a descriptive analysis of breast cancer in Argentina.

Introduction: Registries based on Real-World Data (RWD) are those obtained outside of systematised and randomised clinical trials. They allow the collection of information from a large number of patients and enable the participation of a significant number of professionals. PrecisaXperta is a web platform developed for this purpose with more than 2 years of operation, parameterised for oncology. Its design allows the construction of an epidemiological database in real time and exportable for processing. Objective: To describe the characteristics and operation of this online data recording tool, explain how it was developed and analyse the quality of the information recorded, taking as an example the data obtained for breast cancer. Materials and methods: Physicians, computer scientists and data science analysts participated in the development. Patient data, history, educational level, diagnosis, staging, molecular markers, quality of life, types of treatments, progression and response, imaging, complications, adverse events are some of the fields included. Data treatment in terms of encryption, anonymisation, protection and validation is also explained. The selected breast cancer data for description were processed with medium-level statistical programmes, since the number required to apply Big Data engines is not yet available. Results: From a total of 6,892 solid tumours, 1,892 were breast cancer and 1,654 were selected that complied with a data set minimum elaborated ad hoc. Cases from 13 provinces showed a geolocation bias according to the place of practice of the professionals in the collaborative network. The predominant lack of data was detected in molecular markers (ki67) and correlativity in some lines of treatment. Inconsistencies in dates and therapeutic schemes were also detected. Data curation made it possible to exclude them. The age of the patients was 55.3 ± 11.88 years. At the time of diagnosis, the predominance was in stage I: 36.48% and II 30.06%, with positive hormone receptors in 1,424 (89.96%) cases. The predominant treatments were hormonal (61.54%) and target directed with 30.85% for HER2(+) and 39.14% for HER2(-) accompanied in most cases (85.9%) by some period of chemotherapy. Immunotherapy was much less represented (0.36%). Data were processed, homogenised, pooled and presented and made accessible in a form suitable for application to RWD analyses. Conclusions: PrecisaXperta fulfils this purpose of systematising the information to facilitate its loading with its simple and intuitive interface. From the analysis of the data obtained in breast cancer, it is clear that some fields should be mandatory in order to improve the quality of the information. The results describing the registered breast cancers give us a surface view of the affected population and prepare us to design future studies when we have local Big Data. This type of development, with continuous improvements and online results, will allow with its dissemination, that the participating professionals have information of what happens in the real world, having available in a democratic way, the epidemiology to be able to study, publish and investigate with these data.

Índices de internações, de mortalidade e de custos associados à doença pulmonar obstrutiva crônica (DPOC) no Distrito Federal após mudança do Protocolo Estadual de 2018 ­ Dados do mundo real / Hospitalization, mortality rates and costs associated with chronic obstructive pulmonary disease (COPD) in Distrito Federal, Brazil, before and after the change in 2018 state COPD protocol version ­ Real-world data

Objetivo: Avaliar os índices de hospitalização, mortalidade e custos associados à doença pulmonar obstrutiva crônica (DPOC) após mudança do Protocolo de Atenção à Saúde da Secretaria do Distrito Federal (SES-DF) em 2018 por um medicamento da mesma classe terapêutica, porém em dispositivo inalatório diferente. Métodos: Foi realizado um estudo observacional, transversal, em uma coorte de pacientes adultos internados em dois períodos específicos: gosto/2017 a julho/2018 (MAT 1 ­ vigência do Protocolo antigo) e agosto/2018 a julho/2019 (MAT 2 ­ vigência do novo Protocolo). Foram avaliados pacientes internados com diagnóstico principal de DPOC de acordo com a Classificação Estatística Internacional de Doenças e Problemas Relacionados com a Saúde Versão 10 (CID-10) estabelecida pelo protocolo de atenção à saúde e registrada no Datasus. Os desfechos avaliados foram número de internações, mortalidade e custos totais associados à internação por DPOC. Resultados: Após análise dos dados relacionados à DPOC, a comparação entre MAT 1 (agosto/2017 a julho/2018) e MAT 2 (agosto/2018 a julho/2019) resultou no aumento na ocorrência de importantes desfechos: +131,3% no número de internações com passagens em unidade de terapia intensiva (UTI) (n = 16 no MAT 1 vs. n = 37 no MAT 2, p < 0,01), +101,0% na frequência de internações com passagem em UTI sobre o total de internações (2,4% no MAT 1 vs. 4,8% no MAT 2, p = 0,01), +566,7% nos óbitos de pacientes internados em UTI (n = 3 no MAT 1 vs. n = 20 no MAT 2, p < 0,01) e +52,9% nos custos totais de internação (R$ 828.761 no MAT 1 vs. R$ 1.267.318 no MAT 2, p = 0,03). Conclusão: Durante os períodos analisados, houve aumento no uso de UTI, na mortalidade em UTI e nos custos totais relacionados à DPOC. A atualização do protocolo alterou tanto o dispositivo inalatório (névoa suave para pó seco) quanto a molécula (tiotrópio para glicopirrônio) e poderia ser um dos fatores responsáveis por esses desfechos, no entanto as limitações desta análise observacional não podem determinar um impacto causal da mudança do protocolo, pois outras variáveis também podem ter levado às diferenças descritas.

Objective: Evaluate the rates of hospitalization, mortality and costs associated with chronic obstructive pulmonary disease (COPD) before and after the change in COPD Treatment Protocol of State Health Secretariat of Distrito Federal (SES-DF). Methods: An observational, cross-sectional study was conducted in a cohort of hospitalized adult patients in two specific periods: August/2017 to July/2018 (MAT 1 ­ old version of Protocol in place) and August/2018 to July/2019 (MAT 2 - after the new Protocol version). Hospitalized patients with COPD as primary diagnosis according to International Statistical Classification of Diseases and Related Health Problems Version 10 (ICD-10) established by COPD Treatment Protocol and registered in DATASUS database were evaluated. The outcomes assessed were the number of hospitalizations, mortality and total costs ssociated with COPD hospitalizations. Results: After analyzing the data related to COPD, the comparison between MAT 1 (August/2017 to July/2018) and MAT 2 (August/2018 to July/2019) periods resulted in an increase in the occurrence of relevant outcomes: +131.3% in the number of hospitalizations with intensive care unit (ICU) admissions (n = 16 in MAT 1 vs. n = 37 in MAT 2, p < 0.01), +101.0% in the frequency of hospitalizations with ICU admissions over total number of hospitalizations (2.4% in MAT 1 vs. 4.8% in MAT 2, p = 0.01), +566.7% in deaths of patients admitted in ICU (n = 3 in MAT 1 vs. n = 20 in MAT 2, p < 0.01), +52.9% in total costs associated with COPD hospitalizations (R$ 828,761 in MAT 1 vs. R$ 1,267,318 in MAT 2, p = 0.03). Conclusion: During the analyzed periods, an increase in ICU usage, mortality in ICU and total costs related to COPD was observed. The update in the protocol switched the inhaler device (from soft mist to dry powder) and also the molecule (tiotropium to glycopyrronium) and could be potentially one of the factors responsible for these endpoints, however the limitations of this observational analysis cannot determine a causal impact of the protocol change as other variables could also have led to the differences described.

Lupus en Argentina. Pacientes no respondedores al tratamiento estándar y belimumab como posible opción. Datos del registro RELESSAR / Lupus in Argentina. Non-responders to standard treatment and belimumab as a possible option. RELESSAR record data

Introducción: el lupus es una enfermedad compleja y varias veces de difícil abordaje. Alcanzar la remisión es uno de los objetivos, incorporando opciones terapéuticas. Objetivos: describir las características generales de los pacientes según el estado de la enfermedad y el uso de belimumab. Materiales y métodos: estudio de corte transversal, registro RELESSAR. Se definió el estado de la enfermedad como: remisión: SLEDAI=0 y sin corticoides; baja actividad de la enfermedad: SLEDAI >0 y ≤4 y sin corticoides; control no óptimo: SLEDAI >4 y cualquier dosis de corticoides. Resultados: se incluyeron 1.277 pacientes, 23,4% en remisión, 12,6% en baja actividad y 63,8% con control no óptimo. En este último grupo eran más jóvenes y con menor duración de la enfermedad; presentaban mayores índices de actividad y cronicidad, y mayor empleo de inmunosupresores. Solo el 22,3% de los pacientes con criterio potencial de uso de belimumab (lupus eritematoso sistémico activo a pesar del tratamiento estándar) lo recibía en ese momento. Las variables asociadas a hospitalizaciones fueron: terapia con corticoides, ciclofosfamida y mayor SLICC. Conclusiones: se refleja la complejidad del manejo de estos pacientes y se visualizan aspectos estructurales como la desigualdad. El uso del belimumab resultaría beneficioso en los pacientes seleccionados.

Introduction: lupus is a complex disease and often difficult to approach. Achieving remission is one of the objectives, incorporating therapeutic options. Objectives: to describe the characteristics of the patients and the use of belimumab, according to the status of the disease. Materials and methods: cross-sectional study. Patients of the RELESSAR registry. Stratification: Remission: SLEDAI=0 and without corticosteroids. Low disease activity SLEDAI> 0 and ≤4 and without corticosteroids and non-optimal control: SLEDAI> 4 and any dose of corticosteroids. Results: a total of 1,277 patients were included, 23.4% in remission, 12.6% in low disease activity and 63.8% in non-optimal control. The last group was younger and had a shorter duration of the disease. They had higher activity and chronicity indices and greater use of immunosuppressants. Only 22.3% of the patients with potential criteria for the use of belimumab (activity disease despite standard treatment) were receiving it. The variables associated with hospitalizations were: corticosteroids, cyclophosphamide and higher SLICC. Those associated with severe infection: mycophenolate mofetil, azathioprine, corticosteroids, and higher SLICC. Conclusions: the complexity of the management of these patients is reflected, visualizing structural aspects such as inequality. The use of belimumab could be beneficial in selected patients.

Achievement of Developmental Milestones Recorded in Real Time: A Mobile App-Based Study.

OBJECTIVE: To clarify the characteristics of milestone achievement of Japanese infants using data assessed and recorded in real time via a Web-based app by caregivers. STUDY DESIGN: Between 2014 and 2019, the achievement of developmental milestones of 16 627 Japanese infants were recorded via the mobile app Papatto Ikuji along with pertinent information including sex, birth date, anthropometric measurements, and information on feeding. The milestones consisted of 20 items belonging to 4 domains: personal-social, fine motor-adaptative, language, and gross motor. The distribution of age at milestone achievement was compared with 4 conventional normative data. Subgroup analyses according to sex, season of birth, and feeding methods were performed with restricted mean survival time analysis. RESULTS: Age distributions of milestone achievement were generally similar in the app-based and conventional data. The exception was "Smile responsively," for which the age at achievement seemed higher in the app-based data. Subgroup analyses showed female-dominant sex differences in the achievement of person-social and language milestones. The results also showed seasonality in achievement of gross motor milestones, with earlier achievement in infants born in winter compared with those born in summer. CONCLUSIONS: We describe mobile app-based data on developmental milestone achievements that were recorded in real time. The app-based data were generally comparable with conventional normative data, suggesting that its potential to assess the development of children in real time. This could be used complementarily with the current well-child visits at scheduled time points.