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Therapeutic interventions for ANCA-associated vasculitides (AAV) dramatically changed over the last two decades. Especially the introduction of rituximab into the therapeutic portfolio significantly improved outcome data, whereby relapse rates in previous cohorts were mostly reported from only up to two-year periods. With the present study, we thought to retrospectively investigate the overall relapse rates in a large European AAV cohort. Data of patients with newly diagnosed GPA and MPA (retrospectively classified on ACR/EULAR 2022 criteria) from four tertiary referral centers in Germany and Switzerland were collected between 2000 and 2021. Events were captured during routine out-patient visits and hospital admissions. A relapse was considered at the time of any increased vasculitis activity, i.e. therapeutic necessity to increase glucocorticoids ≥10 mg prednisolone per day. First relapse occurrence was analyzed as time-to-event analysis and number of cases for each group. Differences were compared by ANOVA- and X2-test, respectively. Our data set comprised a total of 338 patients, 46.2% women (mean age of 59.2±14.7 years). Of these, 203 cases (60.1%) were classified as GPA and 135 (39.9%) as MPA. At initial presentation, 74.9% of patients exhibited renal, 50.9% pulmonary and 35.2% ENT involvement. Over a median follow-up period of 58.7 months (IQR 29.4 to 101.7) we observed an overall relapse rate of 35.8% in the entire cohort and median time to first relapse was 32.6 months (IQR 11.9 to 56.9). Following three years after initial diagnosis, we observed a significant difference, yielding 46 relapses (22.7%) in GPA and 19 (14.1%) in MPA patients at that time (p<0.05). Over the entire follow-up, 86 patients with GPA experienced at least one relapse (42.4%) whereas in MPA only 35 (25.9%) patients relapsed (p<0.01). In addition, log-rank test revealed a significant difference in relapse over time (p<0.05, Figure 1). The median time to the first relapse did not differ significantly between GPA and MPA patients (GPA: 27.9 months, IQR 13.6 to 55.9, vs. MPA: 35.1 months, IQR 8.6 to 55.9, p=0.458). In our European AAV cohort, we observed an overall relapse rate of 35.8% for both AAV entities with the first relapse occurring after a median of 32.6 months. GPA patients exhibited consistently higher relapse rates than those with MPA over the entire observational period. We propose that future analyzes of relapse rates should cover a minimum of three years to capture roughly half of all expected events. NIL. NIL. Stefan Krämer: None declared, Thomas Rauen Consultant of: Vifor, Kristian Pruin: None declared, Teresa Anslinger: None declared, Martin Busch: None declared, Tobias Schmitt: None declared, Raoul Bergner Speakers bureau: Abbvie, Bristol Myers Squibb, Chugai, Glaxo Smith Kline, Galapagos, MSD, Novartis, Consultant of: Galapagos, Vifor, Sebastian Mosberger: None declared, Thomas Neumann Speakers bureau: Roche Pharma (Suisse) AG, GlaxoSmithKline AG, Celgene Switzerland, UCB-Pharma AG, Janssen-Cilag AG, Vifor Pharma Switzerland, Novartis Switzerland, Consultant of: Roche Pharma (Suisse) AG, GlaxoSmithKline AG, Celgene Switzerland, AstraZeneca GmbH, Janssen-Cilag AG, Amgen Switzerland AG, Vifor Pharma Switzerland, Grant/research support from: Vifor Pharma Switzerland. [Display omitted]

Registry-based randomized controlled trials are defined as pragmatic trials that use registries as a platform for case records, data collection, randomization, and follow-up. Recently, the application of registry-based randomized controlled trials has attracted increasing attention in health research to address comparative effectiveness research questions in real-world settings, mainly due to their low cost, enhanced generalizability of findings, rapid consecutive enrollment, and the potential completeness of follow-up for the reference population, when compared with conventional randomized effectiveness trials. However several challenges of registry-based randomized controlled trials have to be taken into consideration, including registry data quality, ethical issues, and methodological challenges. In this article, we summarize the advantages, challenges, and areas for future research related to registry-based randomized controlled trials.

Background & objectives: Information on recent cancer statistics is important for planning, monitoring and evaluating cancer control activities. This article aims to provide an update on the cancer incidence estimates in India by sex, age groups and anatomical sites for the year 2022. Methods: The National Cancer Registry Programme Report 2020, reported the cancer incidence from 28 Population-Based Cancer Registries (PBCRs) for the years 2012-2016. This was used as the basis to calculate cancer estimates in India. Information pertaining to the population at risk was extracted from the Census of India (2001 and 2011) for the estimation of age-sex stratified population. PBCRs were categorised into the respective State and regions of the country to understand the epidemiology of cancer. The age-specific incidence rate for each specific anatomical site of cancer was applied to the estimated population to derive the number of cancer cases in India for 2022. Results: The estimated number of incident cases of cancer in India for the year 2022 was found to be 14,61,427 (crude rate:100.4 per 100,000). In India, one in nine people are likely to develop cancer in his/her lifetime. Lung and breast cancers were the leading sites of cancer in males and females, respectively. Among the childhood (0-14 yr) cancers, lymphoid leukaemia (boys: 29.2% and girls: 24.2%) was the leading site. The incidence of cancer cases is estimated to increase by 12.8 per cent in 2025 as compared to 2020. Interpretation & conclusions: The cancer incidence is continuing to increase in India. The new estimates will be helpful in planning cancer prevention and control activities through the intervention of early detection, risk reduction and management.

Hematopoietic stem cell transplantation (HSCT) is an established procedure for many acquired and congenital disorders of the hematopoietic system. A record number of 42 171 HSCT in 37 626 patients (16 030 allogeneic (43%), 21 596 autologous (57%)) were reported by 655 centers in 48 countries in 2015. Trends include continued growth in transplant activity over the last decade, with the highest percentage increase seen in middle-income countries but the highest absolute growth in the very-high-income countries in Europe. Main indications for HSCT were myeloid malignancies 9413 (25%; 96% allogeneic), lymphoid malignancies 24 304 (67%; 20% allogeneic), solid tumors 1516 (4%; 3% allogeneic) and non-malignant disorders 2208 (6%; 90% allogeneic). Remarkable is the decreasing use of allogeneic HSCT for CLL from 504 patients in 2011 to 255 in 2015, most likely to be due to new drugs. Use of haploidentical donors for allogeneic HSCT continues to grow: 2012 in 2015, a 291% increase since 2005. Growth is seen for all diseases. In AML, haploidentical HSCT increases similarly for patients with advanced disease and for those in CR1. Both marrow and peripheral blood are used as the stem cell source for haploidentical HSCT with higher numbers reported for the latter.

Researchers often analyze cancer registry data to assess for differences in survival among cancer treatments. However, the retrospective, nonrandomized design of these analyses raises questions about study validity. To examine the extent to which comparative effectiveness analyses using observational cancer registry data produce results concordant with those of randomized clinical trials. In this comparative effectiveness study, a total of 141 randomized clinical trials referenced in the National Comprehensive Cancer Network Clinical Practice Guidelines for 8 common solid tumor types were identified. Data on participants within the National Cancer Database (NCDB) diagnosed between 2004 and 2014, matching the eligibility criteria of the randomized clinical trial, were obtained. The present study was conducted from August 1, 2017, to September 10, 2019. The trials included 85 118 patients, and the corresponding NCDB analyses included 1 344 536 patients. Three Cox proportional hazards regression models were used to determine hazard ratios (HRs) for overall survival, including univariable, multivariable, and propensity score-adjusted models. Multivariable and propensity score analyses controlled for potential confounders, including demographic, comorbidity, clinical, treatment, and tumor-related variables. The main outcome was concordance between the results of randomized clinical trials and observational cancer registry data. Hazard ratios with an NCDB analysis were considered concordant if the NDCB HR fell within the 95% CI of the randomized clinical trial HR. An NCDB analysis was considered concordant if both the NCDB and clinical trial P values for survival were nonsignificant (P ≥ .05) or if they were both significant (P < .05) with survival favoring the same treatment arm in the NCDB and in the randomized clinical trial. Analyses using the NCDB-produced HRs for survival were concordant with those of 141 randomized clinical trials in 79 univariable analyses (56%), 98 multivariable analyses (70%), and 90 propensity score models (64%). The NCDB analyses produced P values concordant with randomized clinical trials in 58 univariable analyses (41%), 65 multivariable analyses (46%), and 63 propensity score models (45%). No clinical trial characteristics were associated with concordance between NCDB analyses and randomized clinical trials, including disease site, type of clinical intervention, or severity of cancer. The findings of this study suggest that comparative effectiveness research using cancer registry data often produces survival outcomes discordant with those of randomized clinical trial data. These findings may help provide context for clinicians and policy makers interpreting observational comparative effectiveness research in oncology.

Background According to the International Rare Diseases Research Consortium (IRDiRC), a known rare disease (RD) should be diagnosable within a year. This study sought: firstly, to ascertain how long it takes to obtain the diagnosis of a RD in Spain, along with its associated time trend; and secondly, to identify and measure diagnostic delay (defined by the IRDiRC as any period exceeding a year) by reference to the characteristics of RDs and the persons affected by them. Methods Using data sourced from the Spanish Rare Diseases Patient Registry, we performed a descriptive analysis of the time elapsed between symptom onset and diagnosis of each RD, by sex, age and date of symptom onset, and type of RD. We analysed the time trend across the period 1960–2021 and possible change points, using a Joinpoint regression model and assuming a Poisson distribution. The multivariate analysis was completed with backward stepwise logistic regression. Results Detailed information was obtained on 3304 persons with RDs: 56.4% had experienced delay in diagnosis of their RDs, with the mean time taken being 6.18 years (median = 2; IQR 0.2–7.5). Both the percentage of patients with diagnostic delay and the average time to diagnosis underwent a significant reduction across the study period (p < 0.001). There was a higher percentage of diagnostic delays: in women (OR 1.25; 95% CI 1.07–1.45); in cases with symptom onset at age 30–44 years (OR 1.48; 95% CI 1.19–1.84): and when analysed by type of RD, in mental and behavioural disorders (OR 4.21; 95% CI 2.26–7.85), followed by RDs of the nervous system (OR 1.39; 95% CI 1.02–1.88). Conclusions This is the first study to quantify time to diagnosis of RDs in Spain, based on data from a national registry open to any RD. Since over half of all persons affected by RDs experience delay in diagnosis, new studies are needed to ascertain the factors associated with this delay and the implications this has on the lives of patients and their families.

Background Standardization of procedures for data abstraction by cancer registries is fundamental for cancer surveillance, clinical and policy decision-making, hospital benchmarking, and research efforts. The objective of the current study was to evaluate adherence to the four components (completeness, comparability, timeliness, and validity) defined by Bray and Parkin that determine registries’ ability to carry out these activities to the hospital-based National Cancer Database (NCDB). Methods Tbis study used data from U.S. Cancer Statistics, the official federal cancer statistics and joint effort between the Centers for Disease Control and Prevention (CDC) and the National Cancer Institute (NCI), which includes data from National Program of Cancer Registries (NPCR) and Surveillance, Epidemiology, and End Results (SEER) to evaluate NCDB completeness between 2016 and 2020. The study evaluated comparability of case identification and coding procedures. It used Commission on Cancer (CoC) standards from 2022 to assess timeliness and validity. Results Completeness was demonstrated with a total of 6,828,507 cases identified within the NCDB, representing 73.7% of all cancer cases nationwide. Comparability was followed using standardized and international guidelines on coding and classification procedures. For timeliness, hospital compliance with timely data submission was 92.7%. Validity criteria for re-abstracting, recording, and reliability procedures across hospitals demonstrated 94.2% compliance. Additionally, data validity was shown by a 99.1% compliance with histologic verification standards, a 93.6% assessment of pathologic synoptic reporting, and a 99.1% internal consistency of staff credentials. Conclusion The NCDB is characterized by a high level of case completeness and comparability with uniform standards for data collection, and by hospitals with high compliance, timely data submission, and high rates of compliance with validity standards for registry and data quality evaluation.

AbstractObjectiveTo determine outcomes and safety of endovascular treatment for acute ischaemic stroke, due to proximal intracranial vessel occlusion in the anterior circulation, in routine clinical practice.DesignOngoing, prospective, observational cohort study.Setting16 centres that perform endovascular treatment in the Netherlands.Participants1488 patients included in the Multicentre Randomised Controlled Trial of Endovascular Treatment for Acute Ischaemic Stroke in the Netherlands (MR CLEAN) Registry who had received endovascular treatment, including stent retriever thrombectomy, aspiration, and all alternative methods for acute ischaemic stroke within 6.5 hours from onset of symptoms between March 2014 and June 2016.Main outcome measuresThe primary outcome was the modified Rankin Scale (mRS) score, ranging from 0 (no symptoms) to 6 (death) at 90 days after the onset of symptoms. Secondary outcomes were excellent functional outcome (mRS score 0-1), good functional outcome (mRS score 0-2), and favourable functional outcome (mRS score 0-3) at 90 days; score on the extended thrombolysis in cerebral infarction scale at the end of the intervention procedure; National Institutes of Health Stroke Scale score 24-48 hours after intervention; and complications that occurred during intervention, hospital admission, or three months’ follow up period. Outcomes and safety variables in the MR CLEAN Registry were compared with the MR CLEAN trial intervention and control arms.ResultsA statistically significant shift was observed towards better functional outcome in patients in the MR CLEAN Registry compared with the MR CLEAN trial intervention arm (adjusted common odds ratio 1.30, 95% confidence interval 1.02 to 1.67) and the MR CLEAN trial control arm (1.85, 1.46 to 2.34). The reperfusion rate, with successful reperfusion defined as a score of 2B-3 on the extended thrombolysis in cerebral infarction score, was 58.7%, the same as for patients in the MR CLEAN trial. Duration from onset of stroke to start of endovascular treatment and from onset of stroke to successful reperfusion or last contrast bolus was one hour shorter for patients in the MR CLEAN Registry. Symptomatic intracranial haemorrhage occurred in 5.8% of patients in the MR CLEAN Registry compared with 7.7% in the MR CLEAN trial intervention arm and 6.4% in the MR CLEAN trial control arm.ConclusionIn routine clinical practice, endovascular treatment for patients with acute ischaemic stroke is at least as effective and safe as in the setting of a randomised controlled trial.

We estimated autism spectrum disorder (ASD) prevalence in 7–9 year-old children in 2015 using data from three nationwide health registry systems (Denmark, Finland, Iceland) and two French population-based regional registries. Prevalence ranged from 0.48% in South-East France to 3.13% in Iceland (South-West France: 0.73%, Finland: 0.77%, Denmark: 1.26%). Male/female ratios ranged from 3.3 in Finland to 5.4 in South-West France. Between 12% (Denmark) and 39% (South-West France) of cases were diagnosed with intellectual disability. The variations in population-based ASD prevalence across four European countries with universal health care practices likely reflect variation in detection, referral and diagnosis practices and autism awareness across these areas. Using established population-based data systems is an efficient approach to monitor ASD prevalence trends over time.

Background China has made tremendous progresses in serving the needs of its people living with rare diseases in the past decade, especially over the last 5 years. The Chinese government’s systematic approach included a series of coordinated initiatives, amongst these are: forming the Rare Disease Expert Committee (2016), funding the “Rare Diseases Cohort Study” (2016–2020), and publishing its first “Rare Disease Catalog” (2018). Herein, we present the National Rare Diseases Registry System (NRDRS)—China’s first national rare diseases registry, and the analysis of cases registered in the first 5 years ending Dec 31, 2020. Results The total 62,590 cases covered 166 disease/disease types, forming 183 disease cohorts. The data from nearly 22% of them (13,947 cases) is also linked to valuable biological samples. The average age of definitive diagnosis was 30.88 years; 36.07% of cases were under 18 years of age. Regional distribution analysis showed 60% of cases were from the more developed, wealthier East and North China, suggesting the local availability of quality care and patients’ financial status were key access factors. Finally, 82.04% of cases were registered from the five clinical departments: Neurology, Endocrine, Hematology, Cardiovascular, and Nephrology, suggesting that either these are most affected by rare diseases, or that there were disease non-specific ascertainment factors. Conclusions The preliminary analysis of the first 5-year’s data provides unique and valuable insight on rare disease distribution in China, and higlights the directions for enhancing equity, scale and utility.