Case Report Form Research Articles

A clinical study to monitor prescription patterns, clinical outcomes, and adverse drug reactions among patients of various interstitial lung diseases attending respiratory medicine outpatient department at tertiary care hospital in Northern India.

The aim of this study was to monitor prescription patterns, clinical outcomes, and adverse drug reactions (ADR) among patients of various interstitial lung diseases (ILDs). This prospective study was conducted in the Department of Pharmacology and Therapeutics in collaboration with the Department of Respiratory Medicine, King George's Medical University, Lucknow, for a period of 12 months (October 2020-September 2021). A total of 77 patients were enrolled after satisfying the inclusion and exclusion criteria. The prescriptions were collected, and necessary details were noted on the case report form. After completion of the study, the data were analyzed for prescription patterns, clinical outcomes, and quality of life with the help of a validated questionnaire-King's Brief ILD (KBILD) questionnaire. At the same time, ADRs, if any, were assessed using Hartwig's Severity Assessment Scale and Naranjo Causality Assessment Scale. The most common ILD was acute/chronic hypersensitivity pneumonitis (HP). Average number of drugs per encounter was 4.45. Crepitations were the most common clinical signs. Clubbing and rhonchi were reported maximum in idiopathic pulmonary fibrosis. It was found that psychological, breathlessness and activities, chest symptoms, and total KBILD reduced significantly after 3 months as compared to baseline with a statistically significant difference as P < 0.01. ADRs were found in 23.38% (18) of the subjects. Maximum ADR reported was gastritis (9.09%), followed by hepatitis (3.90%). The high proportion of patients clinically diagnosed with HP in our study highlights the importance of a detailed environmental exposure history in the diagnostic evaluation of patients with ILD to avoid inaccurate diagnoses. ADR-related hospital admissions are a significant problem in the health-care system.

ACBC Study Protocol – Acute Colon Resection Versus Bridge to Colon Surgery with Stent Placement or Stoma Diversion: A Prospective Cohort Study

Abstract Background: Approximately 70% of all colon cancer emergencies result from colonic obstruction. Data from the Swedish Colorectal Cancer Registry indicate that patients undergoing emergency surgical resections for colon cancer face higher overall 30-day morbidity and increased mortality rates—5.8% at 30 days and 11.2% at 90 days—compared to those undergoing elective procedures, which have mortality rates of 1.1% and 2.0%, respectively. Other concerns about acute colon cancer resections are oncological outcomes. Long-term outcomes are significantly worse, with higher rates of recurrence and cancer-related death, regardless of the cancer stage or the use of adjuvant chemotherapy. Objectives: To evaluate the short- and long-term outcomes of primary resection for malignant colonic obstruction compared to only decompression using a self-expandable endoscopic metallic stent or diverting stomas as the first intervention (bridge to surgery) followed by later oncological resection. Design: This is a prospective observational study with data collected through the Swedish Colorectal Cancer Registry with an additional electronic case report form. Settings: Swedish multicenter study at both university and regional hospitals. Patients and Methods: Patients with suspected acute malignant large bowel obstruction due to colonic adenocarcinoma will be identified and followed. Analysis will be performed on an intention-to-treat basis. Descriptive statistics of the study cohort will be presented. Primary endpoints will be subjected to multivariate analyses adjusted for confounders. Main Outcome Measures: 30-day severe morbidity (Clavien–Dindo &gt; 3a), 30- and 90-day mortality rates, and overall survival after three and five years. Sample Size: We aim to include 1000 patients, with an interim analysis after the recruitment of 500 patients. Results: The study is ongoing. Conclusions: Acute colon obstruction is associated with substantial morbidity and mortality. We hypothesize that patients with malignant obstruction benefit from avoidance of emergency cancer resection and that bridge to surgery leads to decreased short-term morbidity and mortality and improved long-term oncological outcomes. Limitations: The main limitation is the risk of selection bias when the study is not a randomized controlled trial, which would be a standard process. A prospective observational study is, nevertheless, considered a pragmatic compromise between feasibility and ability to answer the research question. Conflicts of Interest: None.

Comparison of Medicare Claims-based Clostridioides difficile infection classification to chart review using a linked cohort

Background: Medicare claims are frequently used to study Clostridioides difficile infection (CDI) epidemiology. Categorizing CDI based on location of onset and potential exposure is critical in understanding transmission patterns and prevention strategies. While claims data are well-suited for identifying prior healthcare utilization exposures, they lack specimen collection and diagnosis dates to assign likely location of onset. Algorithms to classify CDI onset and healthcare association using claims data have been published, but the degree of misclassification is unknown. Methods: We linked patients with laboratory-confirmed CDI reported to four Emerging Infections Program (EIP) sites from 2016-2020 to Medicare beneficiaries using residence, birth date, sex, and hospitalization and/or healthcare exposure dates. Uniquely linked patients with fee-for-service Medicare A/B coverage and complete EIP case report forms were included. Patients with a claims CDI diagnosis code within ±28 days of a positive CDI test reported to EIP were categorized as hospital-onset (HO), long-term care facility onset (LTCFO), or community-onset (CO, either healthcare facility-associated [COHCFA] or community-associated [CA]) using a previously published algorithm based on claim type, ICD-10-CM code position, and duration of hospitalization (if applicable). EIP classifies CDI into these categories using positive specimen collection date and other information from chart review (e.g. admit/discharge dates). We assessed concordance of EIP and claims case classifications using Cohen’s kappa. Results: Of 10,002 eligible EIP-identified CDI cases, 7,064 were linked to a unique beneficiary; 3,451 met Medicare A/B fee-for-service coverage inclusion criteria. Of these, 650 (19%) did not have a claims diagnosis code ±28 days of the EIP specimen collection date (Table); 48% (313/650) of those without a claims diagnosis code were categorized by EIP as CA CDI. Among those with a CDI diagnosis code, concurrence of claims-based and EIP CDI classification was 68% (κ=0.56). Concurrence was highest for HO and lowest for COHCFA CDI. A substantial number of EIP-classified CO CDIs (30%, Figure) were misclassified as HO using the claims-based algorithm; half of these had a primary ICD-10 diagnosis code of sepsis (226/454; 50%). Conclusions: Evidence of CDI in claims data was found for 81% of EIP-reported CDI cases. Medicare classification algorithms concurred with the EIP classification in 68% of cases. Discordance was most common for community-onset CDI patients, many of whom were hospitalized with a primary diagnosis of sepsis. Misclassification of CO-CDI as HO may bias findings of claims-based CDI studies.

The association between unemployment and treatment among adults with hemophilia

BackgroundPeople with hemophilia often experience pain and suffer from comorbidities related to their bleeding disorder. Consequently, unemployment due to disability is prevalent among people with hemophilia. ObjectivesTo explore associations between unemployment due to disability and treatment while adjusting for known risk factors for unemployment. MethodsCollecting data from 20 hemophilia centers from 15 European countries, the Age-related DeVelopments ANd ComorbiditiEs (ADVANCE) in hemophilia study recruited 785 participants aged 40 years and over with hemophilia A or B. A comprehensive electronic case report form included items related to patient characteristics, demographic information, past and current treatment regimens, and medical history, including a lifelong history of comorbidities. Baseline data from the ADVANCE study was analyzed using descriptive statistics and logistic regression models. ResultsEmployment status was available for 756 of 785 participants aged 40 to 88 years (median, 53 years). We used regression analysis to compare people with hemophilia who were fully employed with those who were unemployed due to disability. This analysis included 424 participants. Using multivariable logistic regression, we found that age (odds ratio [OR], 1.07; P < .01), severe hemophilia (OR, 10.81; P < .01), current smoker (OR, 2.53; P < .01), and psychiatric disorder (OR, 4.18; P = .02) were associated with increased odds of unemployment due to disability. In contrast, prophylactic treatment (OR, 0.44; P = .01) was associated with decreased odds. ConclusionOur analysis suggests that by maintaining factor levels above a critical threshold (3%-5%), prophylactic treatment for people with hemophilia could help avoid unemployment due to disability. While prophylaxis is more costly and can be burdensome, the benefits to material well-being and quality of life could be substantial.

Simultaneous ST-elevation in lead augmented vector right (aVR) and III in non-ST-elevation acute coronary syndromes.

The value of ST-elevation in lead augmented vector right (aVR) remains controversial in clinical practice. This study aimed to investigate the association of simultaneous ST-elevation in lead aVR and III with angiographic findings and clinical outcomes in patients with non-ST-elevation acute coronary syndromes (NSTEACS). In this observational study, patients who had been diagnosed with NSTEACS and presented with ST-elevation in lead aVR and without ST-elevation in any other two contiguous leads were enrolled from January 2018 to June 2019. Demographic, baseline clinical, angiographic and interventional characteristics as well as clinical outcomes were collected and recorded on standardized case report forms. A total of 157 patients meeting the criteria were finally enrolled in this study and classified into two groups according to the presence of ST-elevation in lead III. Patients in the two groups were similar in average age and previous history of hypertension, diabetes mellitus, hyperlipidemia, chronic kidney disease, stroke, and peripheral vascular diseases (all P>0.05). Patients with ST-elevation in lead III tended to present with myocardial hypertrophy in the echocardiography (P=0.02). The cases with ST-elevation in lead III showed higher high sensitivity troponin T (hs-TnT; P=0.08) and creatinine kinase MB isoenzyme (CK-MB; P<0.01) whereas those without ST-elevation in lead III showed higher N-terminal pro brain natriuretic peptide (NT-proBNP; P=0.02). Of note, patients with ST-elevation in lead III presented with more ST-depression in multiple leads [especially in lead I, augmented vector left (aVL), V3-V6] as well as higher degree of ST-depression (all P<0.05) and were more likely to develop multi-vessel and left main trunk (LM) lesions (P=0.04), with 20% of the cases having a LM lesion and 60% having triple vessel lesions. Patients with ST-elevation in lead III were at increased risk of 3-year major adverse cardiovascular events (MACEs), despite no significant statistical difference between the two groups (hazard ratio =1.29; P=0.26). The NSTEACS cases with simultaneous ST-elevation in lead III and aVR tended to present with more multiple leads with ST-depression, higher degree of ST-depression, and more LM or multi-vessel lesions, suggesting a broader range of severe myocardial ischemia. The concurrent presentation of ST-elevation in lead III and aVR may play a vital role in the diagnosis, risk-stratification, and prediction of poor prognosis during the management of NSTEACS patients.

Real-World Safety and Effectiveness of Letermovir in Patients Undergoing Allogenic Hematopoietic Stem Cell Transplantation: Final Results of Post-Marketing Surveillance in Japan.

Cytomegalovirus (CMV) is a common opportunistic infection after allogenic hematopoietic stem cell transplantation (allo-HSCT). Letermovir, an inhibitor of CMV DNA terminase, is approved for CMV prophylaxis in allo-HSCT patients. We report the final results of post-marketing surveillance of letermovir in Japan. The case report forms were drafted in part by the Japanese Data Center for Hematopoietic Cell Transplantation using data elements in the Transplant Registry Unified Management Program and sent to individual HSCT centers to decrease the burden of reporting. Hematopoietic stem cell transplantation patients who received letermovir between May 2018 and May 2022 were registered. Data collected included physician-assessed adverse events/adverse drug reactions and clinical effectiveness (development of CMV disease, CMV antigen status, and use of preemptive therapy). A total of 821 HSCT patients were included in the safety analyses. Adverse drug reactions occurred in 11.33% of patients, with serious adverse drug reactions in 3.05%. The five most common adverse drug reactions were nausea (1.58%), renal impairment (1.46%), and acute graft versus host disease, CMV test positive, and hepatic function abnormal (0.61% each). A total of 670 patients were eligible for effectiveness analyses. Among these patients, 16.57% and 28.66% required preemptive therapy through week 14 and week 48, respectively. In addition, relatively few patients developed CMV disease throughout the follow-up period (1.34% at week 14 and 3.85% at week 48). This final analysis of post-marketing surveillance with up to 48 weeks follow-up period in Japan provides further evidence supporting the safety profile and effectiveness of letermovir for CMV prophylaxis in patients undergoing allo-HSCT in real-world settings.

The National Institutes of Health COVID-19 Neuro Databank/Biobank: Creation and Evolution.

Diverse neurological conditions are reported associated with the SARS-CoV-2 virus; neurological symptoms are the most common conditions to persist after the resolution of acute infection, affecting 20% of patients 6 months after acute illness. The COVID-19 Neuro Databank (NeuroCOVID) was created to overcome the limitations of siloed small local cohorts to collect detailed, curated, and harmonized de-identified data from a large diverse cohort of adults with new or worsened neurological conditions associated with COVID-19 illness, as a scientific resource. A Steering Committee including US and international experts meets quarterly to provide guidance. Initial study sites were recruited to include a wide US geographic distribution; academic and non-academic sites; urban and non-urban locations; and patients of different ages, disease severity, and comorbidities seen by a variety of clinical specialists. The NeuroCOVID REDCap database was developed, incorporating input from professional guidelines, existing common data elements, and subject matter experts. A cohort of eligible adults is identified at each site; inclusion criteria are: a new or worsened neurological condition associated with a COVID-19 infection confirmed by testing. De-identified data are abstracted from patients' medical records, using standardized common data elements and five case report forms. The database was carefully enhanced in response to feedback from site investigators and evolving scientific interest in post-acute conditions and their timing. Additional US and international sites were added, focusing on diversity and populations not already described in published literature. By early 2024, NeuroCOVID included over 2,700 patient records, including data from 16 US and 5 international sites. Data are being shared with the scientific community in compliance with NIH requirements. The program has been invited to share case report forms with the National Library of Medicine as an ongoing resource for the scientific community. The NeuroCOVID database is a unique and valuable source of comprehensive de-identified data on a wide variety of neurological conditions associated with COVID-19 illness, including a diverse patient population. Initiated early in the pandemic, data collection has been responsive to evolving scientific interests. NeuroCOVID will continue to contribute to scientific efforts to characterize and treat this challenging illness and its consequences.

Research protocol - Evaluating data quality in the Netherlands Perinatal Registry (Perined): A data comparison study using hospital records from the IUGR Risk Selection (IRIS) study

Background The quality of registry based studies depends largely on the data accuracy of the registries. The Dutch Perinatal Registry (Perined) is a nationwide database comprising perinatal data digitally provided by different healthcare providers. Perined data are used for comparing outcomes across regions and healthcare institutions as well as for quality analyses and research purposes. However, little is known about the data quality of the Perined database. Therefore, the aim of this study is to assess the quality of Perined data compared to hospital records and case report forms (CRFs) that were part of the IUGR Risk Selection (IRIS) study. Methods In this comparison study data from Perined and the IRIS Study will be used. The IRIS study was a large cluster-randomized trial investigating the effectiveness of routine third trimester ultrasonography in reducing severe adverse perinatal outcomes among Dutch low-risk pregnant women. A subsample of the IRIS study of neonates being at risk of severe adverse perinatal outcomes and their mothers will be used. Baseline demographic data were collected by midwives from participating women at inclusion (around 22 weeks’ gestation) using CRFs, and in-depth neonatal and maternal clinical data were retrieved from hospital records by trained research assistants. These latter IRIS study data were linked and compared to Perined data. Completeness of Perined data will be calculated for every variable. The reliability will be assessed as the percent of agreement between Perined and hospital record data or the CRF-based data. Additionally, intra-class correlation coefficients will be calculated for continuous variables, and Kappa and ‘Prevalence-and-Bias-Adjusted Kappa’ will be calculated for categorical variables. Discussion The results of the proposed comparison study will provide users of Perined data insight in its data quality. This will serve as an example of the accuracy of registry based data used in maternal and neonatal care research.

Clozapine Withdrawal Catatonia: A Case Series and Review of Literature

Background: Catatonia has been reported with withdrawal of medications. Among the various psychotropics, clozapine has been implicated to cause catatonia when abruptly withdrawn. The data regarding clozapine withdrawal catatonia are scarce and are mostly available in the form of case reports and series. Aim: In this case series, we present three cases of clozapine withdrawal catatonia and review the available literature on clozapine withdrawal clozapine. Results: All the three patients developed catatonia within 48 h to 14 days of stoppage of clozapine in the doses of 100–350 mg/day. Two of the patients experiencing clozapine withdrawal improved with reinstitution of clozapine in the previous doses along with benzodiazepines. However, one patient additionally required electroconvulsive therapy (ECT) for the management of withdrawal catatonia. A review of literature showed that clozapine withdrawal catatonia has male preponderance and has been reported with withdrawal of clozapine doses of 50–550 mg/day. The catatonia usually starts in 48 h to 2 weeks of stopping clozapine. Available literature suggests that most of the patients have been managed with restarting of clozapine, along with lorazepam and occasional patients require ECT. Conclusions: To conclude our case series and the available review of literature suggests that sudden discontinuation of clozapine can lead to withdrawal catatonia. Our cases also highlight the importance of reviewing treatment history, especially of abrupt discontinuation of clozapine when a patient on clozapine present with catatonia. This can be very helpful in deciding about further management.

Tips and tricks for successfully conducting a multicenter study

Multicenter studies play a crucial role in medical research and advancement, facilitating the application of new knowledge to clinical practice. These studies are associated with multiple benefits but are more complex than those involving a single center. With the philosophy that most of the qualities required to lead a multicenter study depend on attitude and can be learned, developed, and improved, in this manuscript, we share with the reader a series of recommendations that we consider important for successfully conducting such studies. The tips and tricks that will be discussed in detail are as follows: effectively leading the project; clearly defining viable and relevant objectives; designing a clear and detailed protocol; carefully selecting centers and collaborating investigators; meticulously designing the case report form; centrally managing the project efficiently; maintaining fluent communication with investigators; and, finally, designing a clear authorship policy and ensuring the appropriate publication of the study results. We hope that these suggestions encourage potential researchers to conduct multicenter studies, thereby collectively enhancing the quality of research and its application to clinical practice.

Surgical aspects and outcomes after nephrectomy for Wilms tumour in sub-Saharan Africa: A report from Wilms Africa Phase II-CANCaRe Africa.

Wilms tumour (WT) is one of the common and curable cancer types targeted by the Global Initiative for Childhood Cancer. Tumour excision is essential for cure. This analysis focuses on surgical outcomes of patients with WT in sub-Saharan Africa. We implemented a risk-stratified WT treatment guideline as a multicentre, prospective study across eight hospitals and six countries. Eligibility criteria were age 6months to 16years, unilateral WT, surgery performed after preoperative chemotherapy and diagnosed between 1 January 2021 and 31 December 2022. Data collection included a specific surgical case report form (CRF). The study registered 230 patients, among whom 164 (71.3%) had a nephrectomy. Ninety-eight percent of patients had a completed surgical CRF. Out 164 patients, 50 (30.5%) had distant metastases. Median tumour diameter at surgery was 11.0cm. Lymph node sampling was done in 122 (74.3%) patients, 34 (20.7%) had intraoperative tumour rupture, and for 18 (10.9%), tumour resection involved en bloc resection of another organ. Tumour size at surgery was significantly correlated with tumour rupture (p<.01). With a median follow-up of 17months (range: 2-33), 23 (14.0%) patients have relapsed. Twenty-two (13.4%) patients abandoned treatment post nephrectomy. Two-year event-free survival was 60.4%±4.7% with treatment abandonment as an event. Survival post nephrectomy is challenged by treatment abandonment, treatment-related mortality and relapse. Large tumours after preoperative chemotherapy were associated with a higher risk of tumour rupture. Earlier diagnosis and access to radiotherapy are expected to improve survival.

QOL-09. EQUI AND HIPPO: A STUDY ASSESSING THE FEASIBILITY AND ACCEPTABILITY OF HORSE THERAPY FOR CHILDREN, ADOLESCENTS AND YOUNG ADULTS IN REMISSION FROM A MEDULLOBLASTOMA

Abstract INTRODUCTION Psycho-social and motor disorders are common long-term sequalae in patients treated for a medulloblastoma during childhood. They often require long-term, multidisciplinary rehabilitation and post-therapeutic care that can be difficult to maintain over time. To address this common problem, we hypothesized that a horse therapy can re-engage children in their rehabilitation. The expected benefits of the horse therapy involve both psycho-social skills thought animal mediation (equitherapy) and motor skills using the horse’s three-dimensional movement (hippotherapy). MATERIALS AND METHODS We conducted a single-center prospective interventional trial including patients treated for medulloblastoma during childhood, aged 10 to 26, in complete remission. The intervention consisted on weekly 2-hours sessions of equitherapy and hippotherapy with a specialized physiotherapist and psychologist over a 4-month period (16 sessions per patient) in groups of 2 participants. The primary objective was to determine the feasibility of a rehabilitation program combining equitherapy and hippotherapy. The secondary objective was to assess changes in motor skills, balance, emotional feelings, self-esteem and quality of life at the end of the sessions, at 4 months and at 6 months using questionnaires and standardized and validated measurements. RESULTS The study began in April 2023 and is still ongoing. Six participants were included and four ended the intervention period. On this preliminary cohort, the recruitment rate was excellent with 100% of patients offered the program agree to take part. Three participants completed the program with a 100% adherence rate. All participants were very satisfied with their participation in the program. Analysis of second objective will be presented in the form of case reports. CONCLUSION The expected outcome of this study is to define a framework for the application of this practice, enabling a model of this rehabilitative management, and then to lead to dissemination and recognition of this activity, enabling larger-scale studies.

Exploring the Clinical and Genetic Landscape of Angelman Syndrome: Patient-Reported Insights from an Italian Registry.

Background: The Angelman Syndrome Registry (RISA) was developed as a retrospective study with the following objectives: to evaluate the clinical history of individuals with Angelman Syndrome (AS) in Italy and compare it with the existing literature; to investigate the feasibility of gathering data by directly involving participants in the data collection process; and to explore the relationship between different symptoms and genotypes. Methods: Established in 2018, RISA enrolled a total of 82 participants, with 62 (75.6%) providing complete data. Demographic, clinical, and genetic information was collected using electronic case report forms. Descriptive statistics characterized the sample, while associations between genotype and clinical characteristics were examined. Results: Descriptive analysis revealed a median participant age of 8.0 years, with males comprising 48.8% of the sample. Deletion (58.1%) was the most common genotype. The majority (82.2%) experienced epilepsy, with seizures typically onset before 3 years of age. Most patients (86.2%) required multiple anti-epileptic drugs for control, with generalized tonic-clonic seizures and atypical absence seizures being most prevalent. The deletion group exhibited more severe developmental delays and a trend towards higher seizure severity. Sleep problems affected 69.4% of participants, characterized by difficulties in sleep onset and maintenance. Conclusions: This study offers valuable insights into the clinical history and genetic characteristics of AS in Italy, consistent with the prior literature. Additionally, it underscores the efficacy of patient registries in capturing comprehensive data on rare diseases such as AS, highlighting their potential to advance research and enhance patient care.

Status and influencing factors of frailty in hospitalized patients with chronic heart failure: A cross-sectional study.

To investigate the frailty status of inpatients with chronic heart failure (CHF) and analyse its influencing factors, so as to provide evidence for the early identification of high-risk groups and frailty management. Early identification of frailty can guide the development and implementation of holistic and individualized treatment plans. However, at present, the frailty of patients with CHF has not attracted enough attention. A cross-sectional study. From June 2022 to June 2023, a convenience sample of 256 participants were recruited at a hospital in China. Multivariate logistic regression analysis was used to explore the influencing factors of frailty in patients with CHF, and an ROC curve was drawn to determine the cut-off values for each influencing factor. STROBE checklist guides the reporting of the manuscript. A total of 270 questionnaires were sent out during the survey, and 256 valid questionnaires were ultimately recovered, resulting in an effective recovery rate of 94.8%. The incidence of frailty in hospitalized patients with CHF was 68.75%. Multivariable logistic regression analysis showed that age, self-care ability, nutritional risk, Kinesiophobia and NT-proBNP were risk factors for frailty, while albumin and LVEF were protective factors. Multidimensional frailty was prevalent in hospitalized patients with CHF. Medical staff should take measures as early as possible from the aspects of exercise, nutrition, psychology and disease to delay the occurrence and development of frailty and reduce the occurrence of clinical adverse events caused by frailty. This study emphasizes the importance of the early identification of multidimensional frailty and measures can be taken to delay the occurrence and development of frailty through exercise, nutrition, psychology and disease treatment. Patients contributed through sharing their information required for the case report form and filling out questionnaires.

Quality assessment of radiotherapy in the prospective randomized SENOMAC trial

Background and purposeRecommendations for regional radiotherapy (RT) of sentinel lymph node (SLN)-positive breast cancer are debated. We here report a RT quality assessment of the SENOMAC trial. Materials and MethodsThe SENOMAC trial randomized clinically node-negative breast cancer patients with 1–2 SLN macrometastases to completion axillary lymph node dissection (cALND) or SLN biopsy only between 2015–2021. Adjuvant RT followed national guidelines. RT plans for patients included in Sweden and Denmark until June 2019 were collected (N = 1176) and compared to case report forms (CRF). Dose to level I (N = 270) and the humeral head (N = 321) was analyzed in detail. ResultsCRF-data and RT plans agreed in 99.3 % (breast/chest wall) and in 96.6 % of patients (regional RT). Congruence for whether level I was an intended RT target was lower (78 %). In accordance with Danish national guidelines, level I was more often an intended target in the SLN biopsy only arm (N = 334/611, 55 %,) than in the cALND arm (N = 174/565, 31 %,). When an intended target, level I received prescribed dose to 100 % (IQR 98–100 %) of the volume. However, even when not an intended target, full dose was delivered to > 80 % of level I (IQR 75–90 %). The intentional inclusion of level I in the target volume more than doubled the dose received by ≥ 50 % of the humeral head. ConclusionCongruence between CRF data and RT plans was excellent. Level I received a high dose coverage even when not intentionally included in the target. Including level I in target significantly increased dose to the humeral head.

British Axial Spondyloarthritis Inception Cohort (BAxSIC): a protocol for a multicentre real-world observational cohort study of early axial spondyloarthritis.

Timely diagnosis remains a challenge in axial SpA (axSpA). In addition, data are scarce on the impact of diagnostic delay and disease progression in affected individuals. The British Axial Spondyloarthritis Inception Cohort (BAxSIC) study aims to investigate the impact of newly diagnosed axSpA, the natural history of the disease and the effect of diagnostic delay on disease outcomes. BAxSIC is a prospective, multicentre, observational study. Eligible participants are adults (≥16 years of age), with a physician-confirmed diagnosis of axSpA in the 6 months prior to study entry, recruited from secondary and tertiary rheumatology centres in the UK. Participants will be followed up for 3 years, with in-person visits at baseline and 24 months. In addition, patient self-reported assessments will be recorded remotely via the online electronic case report form (eCRF) at 6, 12, 18, 30 and 36 months. The first patient was enrolled in BAxSIC in June 2023. Recruitment is currently ongoing and is planned to end in June 2026. Initial results will be available in 2027. Since opening, the trial has undergone two protocol amendments. The BAxSIC study is the first inception cohort designed to investigate the impact of diagnostic delay on clinical presentation and long-term functional outcomes in patients with axSpA in the UK. With an innovative, patient-led virtual longitudinal data collection model, data generated from this study will help inform and improve the care of people newly diagnosed with axSpA. ClinicalTrials.gov (http://clinicaltrials.gov), NCT05676775.

Real-World Study of Adjuvant Biosimilar Trastuzumab-dkst for HER2-Positive Breast Cancer Treatment in a Brazilian Population.

Biological monoclonal antibodies play a pivotal role in cancer treatment, with biosimilars significantly enhancing their accessibility. In Brazil's ethnically diverse setting, real-world evidence is crucial for assessing the effectiveness and applicability of these therapies in routine clinical practice. We performed a multicentric, observational, prospective real-world study on biosimilar trastuzumab-dkst for adjuvant treatment of early HER2-positive breast cancer in Brazilian patients. Data were collected using a case-report form. Of the 176 recruited, we present data from the first 59 patients (mean age 51.7 ± 12.9years) who had completed treatment with trastuzumab-dkst. The mean time from diagnosis to the first adjuvant treatment with trastuzumab-dkst was 5.5 ± 2.7months. Of the patients, 59% of patients achieved at least a 30-month follow-up. The 31.7-month invasive disease-free survival rate (IDFS) was 94.5% (95% CI 83.9-98.2%) and median IDFS was not achieved, since only three patients had invasive disease recurrence. The overall survival rate was 100% until the last assessment. The observed adverse events were similar to those presented by other studies using biosimilar or reference trastuzumab. Four serious adverse events (8.5%) were observed. A reduction in left ventricular ejection fraction of at least 10% was observed in 16.9% of participants. There was no treatment interruption, and three participants (5.1%) had their trastuzumab-dkst dose reduced. Our study reinforces the existing pivotal data, underscoring the real-world efficacy and safety of biosimilar trastuzumab-dkst in the adjuvant treatment for early HER2-positive breast cancer. The preliminary long-term effectiveness and safety data we present further validate trastuzumab-dkst's role as a cost-saving alternative in oncological care. These findings have important implications for improving patient access to crucial treatments and for the more efficient use of healthcare resources. NCT03892655.

The First Reported Foodborne Botulism Outbreak in Riyadh, Saudi Arabia: Lessons Learned

BackgroundBotulism has not been previously reported in the Kingdom of Saudi Arabia. This rare and sometimes fatal foodborne illness is caused by neurotoxins and primarily results from consuming home-canned fruits, vegetables, dairy, and seafood products & it can lead to paralysis.ObjectiveThe purpose of this study was to evaluate the clinical features of patients who developed botulism in Riyadh in 2024 after consuming mayonnaise from a well-known local chain of restaurants in Riyadh, Saudi Arabia.MethodsWe conducted a retrospective analysis of medical records and interviewed patients or their attendants for all hospitalized cases of foodborne botulism at Riyadh First Health Cluster. For each patient, a standard case report form was completed, containing information on demographics, clinical aspects, botulinum test results, and type of exposure. Descriptive statistics were applied to assess the data. During the outbreak, nineteen patients with foodborne diseases were admitted to Riyadh First Health Cluster Hospitals. Following thorough physical examinations, botulism was suspected in each case.ResultsEight of the 19 suspected foodborne illness patients fully satisfied the botulism case definition requirements set forth by the Saudi Arabian Public Health Authority (Weqaya). Among these eight patients, 2 (25%) were male and 6 (75%) were female, with a mean age of 23.25 ± 9.29 years (range: 12–38 years). The incubation period for our patients was 36.25 ± 26.26 h. Notable symptoms included dysphagia in all eight patients (100%), dysarthria, generalized weakness, nausea and vomiting in seven patients (88%), diplopia in four patients (50%), and stomach discomfort in three patients (38%). Of the eight cases, six required intubation, one mimicked brain death, and two were stable. The presence of Clostridium botulinum spores as the cause of the outbreak was confirmed by detecting botulinum spores in contaminated food.ConclusionDiplopia and dysarthria were the most common early sign of botulism. Early manifestations may include respiratory symptoms without any musculoskeletal symptoms. or nausea, vomiting and disorientation.

Utility of automated data transfer for cancer clinical trials.

e23011 Background: The burden of data collection on site staff for cancer clinical trials is steadily increasing, and inefficiencies in data entry into electronic data capture (EDC) systems lead to poor data quality and delays in reporting. Software that facilitates automated transfer of mapped structured data from the electronic health record (EHR) to the study EDC system can help to address these challenges. We examined the impact of multi-site usage of an embedded point and click EHR-to-EDC tool on study data capture across multiple cancer clinical trials. Methods: We conducted a retrospective analysis of usage of an EHR-to-EDC tool among research teams at 3 Academic Medical Centers (AMCs) for 4 industry-sponsored Phase 1 cancer clinical trials from the start of use of the tool at each AMC to a cutoff date of 12/31/23. We assessed the types and volume of structured data accessible via FHIR APIs and amenable for transfer (“mapped”) across protocols, the volume of data transferred per protocol, and the time for data transfer, defined as time from case report form (CRF) launch to submission in EDC and time spent in the software application overall. Descriptive statistics were generated for all study metrics. Results: During the 10 month observation period, the EHR-to-EDC tool was used by 8 users to transfer data for 25 patients enrolled across the 4 protocols. Table 1 shows the percentage of total CRF forms mapped and breakdown of proportion of mapped fields per protocol. Overall, 8,597 individual data points (87% labs, 10% vitals and 3% concomitant medications) were pushed for 718 unique CRF submissions. The average number of individual data points and unique CRF submissions per patient were as follows: 400 and 36 (Study A), 420 and 36 (Study B), 251 and 16 (Study C), 270 and 21 (Study D). The average total time spent in the tool per CRF submission was 2.16 minutes (range 1.17-7.00 minutes, inclusive of potential inactive time, application launch and data search). The average time to complete a CRF was 46 seconds (range 15-65 seconds). Conclusions: Use of EHR-to-EDC software facilitated efficient automated transfer of a high volume of study data due to EDC-required repeat use of mapped CRF forms. The impact appeared consistent across protocols, with dozens of unique CRF submissions and hundreds of individual data points pushed per patient for each study. Time for CRF completion was low and uniform across users. This study supports the growing adoption of EHR-to-EDC technology among sites and sponsors, and highlights the evolving potential to further improve and facilitate clinical trial data entry, as tool capabilities are developed beyond structured fields to enable unstructured data capture and transfer. [Table: see text]

Safety and Efficacy of Lemborexant in Insomnia Patients: Results of a Postmarketing Observational Study of Dayvigo® Tablets.

A prospective, postmarketing observational study was conducted to evaluate the safety and efficacy of lemborexant (LEM) tablets in daily clinical practice in Japan. No other studies of a similar size have been conducted since the marketing approval of LEM, making this the first report of its kind. Insomnia patients (n=550) administered LEM (5-10 mg daily) for the first time were enrolled. Adverse events were collected for target events (somnolence, parasomnia, narcolepsy and associated conditions, suicidal ideation and suicidal behavior). Overall improvement of insomnia symptoms was assessed by the investigator based on the patient's complaint. Subjective sleep onset latency (sSOL) and subjective total sleep time (sTST) were investigated as sleep parameters. A case report form was obtained from 539 patients. The incidence of adverse drug reactions (ADRs) was 7.65% for somnolence, 1.76% for nightmares, 0.59% for abnormal dreams, and 0.20% for sleep paralysis. No serious ADRs or ADRs related to suicidal ideation or suicidal behavior were observed. The efficacy rate at the final evaluation was 80.83%. Decreased sSOL and increased sTST were observed as assessed starting from Week 8 of treatment. Based on the results of this study, the safety result was consistent with the safety profile described in the current package insert. Efficacy results also indicated that LEM is clinically useful.