Electronic Case Report Form Research Articles

Trial in Progress: A Real-World Cohort Study Reporting Patient Profiles, Clinical Outcomes, and Unmet Medical Need of Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Eculizumab in the Essen Center in Germany

Trial in Progress: A Real-World Cohort Study Reporting Patient Profiles, Clinical Outcomes, and Unmet Medical Need of Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Eculizumab in the Essen Center in Germany

A Preliminary Analysis of FLAM (Italian Non-Interventional Multi-center Study of FLT3 mutated AML patients): FLT3 Receptor Gene Mutational Analysis and FLT3 Inhibitors Administration in the Real-Life Clinical Practice

A Preliminary Analysis of FLAM (Italian Non-Interventional Multi-center Study of FLT3 mutated AML patients): FLT3 Receptor Gene Mutational Analysis and FLT3 Inhibitors Administration in the Real-Life Clinical Practice

The therapeutic management of South African dyslipidaemic patients at very high cardiovascular risk (CARDIO TRACK): a cross-sectional study.

Dyslipidaemia is a major modifiable risk factor for atherosclerotic cardiovascular disease. At the time the study was conducted, guidelines recommended a low-density lipoprotein cholesterol (LDL-C) target of less than 1.8 mmol/l and a reduction of at least 50% if the baseline LDL-C was between 1.8 and 3.5 mmol/l in patients with either very high cardiovascular risk or established atherosclerosis. In South Africa, there is a paucity of data on attainment of LDL-C goal in patients with very high cardiovascular risk who are on maximum tolerated statin with or without ezetimibe. The aim was to assess the percentage of very high cardiovascular risk South African patients with dyslipidaemia not reaching an LDL-C goal of less than 1.8 mmol/l, despite maximum tolerated statin with or without ezetimibe. This was a multi-centre, observational, cross-sectional study conducted at 15 private healthcare sector sites and one public sector site. Adults (> 18 years) with very high cardiovascular risk of familial hypercholesterolaemia receiving stable, maximum-tolerated statin therapy for at least four weeks prior to their latest lipid profile were enrolled into the study, and electronic case report forms were completed after written informed consent was provided. LDL-C goal attainment was modelled, first assuming an increase in the statin dose to the registered maximum, followed by the addition of ezetimibe or a PCSK9-inhibitor. In total, 507 patients were screened, of whom 492 were eligible for study participation. One patient was excluded from the analysis because of a missing LDL-C value. Most participants were male (male 329, 67%; female 162, 33%). Most patients were either obese (223, 46.0%) or overweight (176, 36.3%). Hypertension and diabetes mellitus were frequent co-morbidities and were found in 381 (77.6%) and 316 (64.4%) patients, respectively. Eighty (16.3%) patients reported current smoking. Only 68 (13.8%) patients were taking ezetimibe in addition to a statin. Reasons for not using ezetimibe included no requirement for ezetimibe in the opinion of the treating physician (229, 48.7%), cost (149, 31.7%), Physician's choice (39, 8.3%), or other (53, 11.3%). Only 161 (32.8%) of the patients attained their goal LDL-C level. In our modelling analysis, increasing the statin dose to the registered maximum and adding ezetimibe brought an additional 34.5% of patients to goal, while adding a PCSK9-inhibitor, irrespective of any other changes to lipid-lowering therapy brought over 90% of not-at-goal patients to goal. Most study participants were not at LDL-C goal despite maximum-tolerated statin, highlighting the need for treatment intensification in this high-risk population. Although intensifying treatment by adding a PCSK9-inhibitor brought more patients to goal, the initial addition of ezetimibe would be more reasonable, given the cost of PCSK9-inhibitors.

A New Case Manager for Diabetic Patients: A Pilot Observational Study of the Role of Community Pharmacists and Pharmacy Services in the Case Management of Diabetic Patients.

The adherence of type 2 diabetes mellitus (DM2) patients with an individual care plan (ICP) is often not satisfactory, nor does it allow for a significant improvement in outcome, because of poor accessibility to services, poor integration of pathway articulations, poor reconciliation with the patient’s life, or the lack of a constant reference person. The purpose of this study was to evaluate the contribution of community pharmacists and pharmacy services in improving adherence with periodic controls in DM2. The study was conducted at a rural pharmacy. A sample of 40 patients was calculated with respect to a historical cohort and subsequently enrolled. Clinical and personal data were collected in an electronic case report form. Pharmacists acting as a case manager followed patients carrying out their ICP developed by an attending physician. Some of the activities foreseen by the ICP, such as electrocardiogram, fundus examination, and self-analysis of blood and urine, were carried out directly in the pharmacy by the pharmacist through the use of telemedicine services and point of care units. Activities that could not be performed in the pharmacy were booked by the pharmacist at the accredited units. Examination results were electronically reported by the pharmacist to the attending physician. The primary endpoint was the variation in patient adherence with the ICP compared to a historical cohort. Secondary endpoints were variation in waiting time for the examinations, mean percentage change in glycated hemoglobin (HbA1c) and low-density lipoprotein (LDL) cholesterol levels and blood pressure, impact on healthcare-related costs, and perceived quality of care. Adherence to the ICP significantly increased. Waiting times were reduced and clinical outcomes improved with conceivable effects on costs. Patients appreciated the easier access to services. Community pharmacists and pharmacy services represent ideal actors and context that, integrated in the care network, can really favor ICP adherence and obtain daily morbidity reduction and cost savings through proper disease control and an early diagnosis of complications.

Health utility of type 2 diabetes patients using basal insulin in China: results from the BEYOND II study.

To derive the health utility scores of type 2 diabetes (T2D) patients using basal insulin (BI) with diverse characteristics in China. The study used the data of insulin-using T2D patients on BI treatment enrolled in the BEYOND II study, which is a multi-center, observational study from 78 hospitals nationwide. The 3-level EQ-5D (EQ-5D-3L) questionnaire was administered to each patient to derive their health utility scores using the EQ-5D-3L value set for China. Patients' clinical and sociodemographic information were retrieved from their electronic case report form (eCRF). Ordinary least-square models with different specifications were explored to identify the best-fitting model to predict the utility scores. The sample (n = 12,583) achieved a mean (standard deviation) EQ-5D-3L utility score of 0.936 (0.120). According to the model, a Chinese male who was younger than 59years, not underweight, diagnosed with T2D shorter than 10years, with controlled plasma glucose and free of diabetes complications/comorbidities, would have a mean utility of 0.993. Being female, older age, underweight, and higher plasma glucose, longer diabetes duration was negatively related to EQ-5D-3L scores. Comorbidities and seven of eleven complications were associated with utility decrement. Interactions between some complications were also discovered. The derived health utility scores for diabetes complications could facilitate the assessment of the cost-effectiveness of health interventions for Chinese insulin-using T2D patients.

EFFICACY AND SAFETY OF ACLIDINIUM BROMIDE IN PATIENTS WITH COPD, CARDIOVASCULAR RISK FACTORS, AND CHRONIC BRONCHITIS IN THE ASCENT STUDY

EFFICACY AND SAFETY OF ACLIDINIUM BROMIDE IN PATIENTS WITH COPD, CARDIOVASCULAR RISK FACTORS, AND CHRONIC BRONCHITIS IN THE ASCENT STUDY

Composite type-2 biomarker strategy versus a symptom–risk-based algorithm to adjust corticosteroid dose in patients with severe asthma: a multicentre, single-blind, parallel group, randomised controlled trial

SummaryBackgroundAsthma treatment guidelines recommend increasing corticosteroid dose to control symptoms and reduce exacerbations. This approach is potentially flawed because symptomatic asthma can occur without corticosteroid responsive type-2 (T2)-driven eosinophilic inflammation, and inappropriately high-dose corticosteroid treatment might have little therapeutic benefit with increased risk of side-effects. We compared a biomarker strategy to adjust corticosteroid dose using a composite score of T2 biomarkers (fractional exhaled nitric oxide [FENO], blood eosinophils, and serum periostin) with a standardised symptom–risk-based algorithm (control).MethodsWe did a single-blind, parallel group, randomised controlled trial in adults (18–80 years of age) with severe asthma (at treatment steps 4 and 5 of the Global Initiative for Asthma) and FENO of less than 45 parts per billion at 12 specialist severe asthma centres across England, Scotland, and Northern Ireland. Patients were randomly assigned (4:1) to either the biomarker strategy group or the control group by an online electronic case-report form, in blocks of ten, stratified by asthma control and use of rescue systemic steroids in the previous year. Patients were masked to study group allocation throughout the entirety of the study. Patients attended clinic every 8 weeks, with treatment adjustment following automated treatment-group-specific algorithms: those in the biomarker strategy group received a default advisory to maintain treatment and those in the control group had their treatment adjusted according to the steps indicated by the trial algorithm. The primary outcome was the proportion of patients with corticosteroid dose reduction at week 48, in the intention-to-treat (ITT) population. Secondary outcomes were inhaled corticosteroid (ICS) dose at the end of the study; cumulative dose of ICS during the study; proportion of patients on maintenance oral corticosteroids (OCS) at study end; rate of protocol-defined severe exacerbations per patient year; time to first severe exacerbation; number of hospital admissions for asthma; changes in lung function, Asthma Control Questionnaire-7 score, Asthma Quality of Life Questionnaire score, and T2 biomarkers from baseline to week 48; and whether patients declined to progress to OCS. A secondary aim of our study was to establish the proportion of patients with severe asthma in whom T2 biomarkers remained low when corticosteroid therapy was decreased to a minimum ICS dose. This study is registered with ClinicalTrials.gov, NCT02717689 and has been completed.FindingsPatients were recruited from Jan 8, 2016, to July 12, 2018. Of 549 patients assessed, 301 patients were included in the ITT population and were randomly assigned to the biomarker strategy group (n=240) or to the control group (n=61). 28·4% of patients in the biomarker strategy group were on a lower corticosteroid dose at week 48 compared with 18·5% of patients in the control group (adjusted odds ratio [aOR] 1·71 [95% CI 0·80–3·63]; p=0·17). In the per-protocol (PP) population (n=121), a significantly greater proportion of patients were on a lower corticosteroid dose at week 48 in the biomarker strategy group (30·7% of patients) compared with the control group (5·0% of patients; aOR 11·48 [95% CI 1·35–97·83]; p=0·026). Patient choice to not follow treatment advice was the principle reason for loss to PP analysis. There was no difference in secondary outcomes between study groups and no loss of asthma control among patients in the biomarker strategy group who reduced their corticosteroid dose.InterpretationBiomarker-based corticosteroid adjustment did not result in a greater proportion of patients reducing corticosteroid dose versus control. Understanding the reasons for patients not following treatment advice in both treatment strategies is an important area for future research. The prevalence of T2 biomarker-low severe asthma was low.FundingThis study was funded, in part, by the Medical Research Council UK.

Various Combination of Antiviral Treatment of COVID-19 Pneumonia; A Preliminary Observational Study

Background: Coronavirus disease 2019 (COVID-19) has emerged serious medical crisis worldwide. No regimen has been approved as gold standard of treatment. There were no report of other combinations of antiviral cocktail therapy. Methods: We compared time to recovery of COVID-19 pneumonia patients who were treated with various combinations of lopinavir/ritonavir, darunavir /ritonavir, favipiravir, hydroxychloroquine, or no antiviral treatment with lopinavir/ritonavir plus high-dose oseltamivir. Results: Lopinavir/ritonavir plus high-dose oseltamivir had the best efficacy in terms of time to recovery compared to no antiviral treatment (P=0.003) and darunavir/ritonavir plus hydroxychloroquine and favipiravir (P =0.02). Lopinavir/ritonavir plus favipiravir had the borderline efficacy in terms of time to recovery compared to no antiviral treatment (P=0.05).There was no superior efficacy in terms of time to recovery of the combinations of darunavir/ritonavir plus hydroxychloroquine with or without favipiravir compared with no antiviral treatment (P>0.05). Darunavir/ritonavir plus hydroxychloroquine with or without favipiravir had similar efficacy to Lopinavir/ritonavir plus favipiravir (P>0.05). The patients who were treated with hydroxychloroquine 400-1200 mg/day had a significantly increased risk of death (P=0.04). Conclusions: The combination of lopinavir/ritonavir plus high-dose oseltamivir exhibited a trend of superiority compared to the combination of darunavir/ritonavir plus hydroxychloroquine and favipiravir and no antiviral treatment. However, the efficacy of the combinations of antiviral cocktail treatment of COVID-19 pneumonia should be further investigated in a randomized control study registerd as NCT04303299.Trial Registration: NCT04303299.Funding Statement: No support from any organization for the submitted work.Declaration of Interests: No conflicts of interest.Ethics Approval Statement: The electronic case report form and results of this study was acknowledge and approved by the independent ethics committee of the Ministry of Public Health for the NCT04303299 study, which we plan to start in the next phase as an open-label randomized controlled trial.

Cost of Human Immunodeficiency Virus (HIV) and Determinants of Healthcare Costs in HIV-Infected Treatment-Naive Patients Initiated on Antiretroviral Therapy in Germany: Experiences of the PROPHET Study

The purpose of the prospective clinical and pharmacoeconomic outcomes study of different first-line antiretroviral treatment strategies (PROPHET) was to examine the healthcare costs of human immunodeficiency virus (HIV)-infected persons in Germany treated with different antiretroviral therapy (ART) strategies and to identify variables associated with high costs. The setting was a 24-month prospective multicenter observational cohort study in a German HIV-specialized care setting from 2014 to 2017. A microcosting approach was used for the estimation of healthcare costs. Data were obtained via electronic case report forms. The costs were calculated from both the societal and the statutory health insurance perspective. Regression models were performed that took into consideration the impact of several independent variables. Four hundred thirty-four patients from 24 centers throughout Germany were included. Average annual healthcare costs were €20 118 (standard deviation [SD] €6451) per patient from the societal perspective (n= 336) and €17 306 (SD €4106) from the statutory health insurance perspective (n= 292). Expenditures for the ART medication had the highest impact. Total costs declined in the second year of therapy. There was a significant association between the amount of total cost and clinical or therapeutic variables from both perspectives; a diagnosis of acquired immune deficiency syndrome (AIDS) led to higher costs as well as the chosen ART strategy. Age also increased cost from the statutory health insurance perspective. The main cost driver of the healthcare costs for HIV-positive patients was antiretroviral drug expenses. Further variables that influenced the costs were identified. The results provide a detailed overview of the resource use of patients in the PROPHET cohort.

Coronavirus Disease 2019 in Solid Organ Transplant: A Multicenter Cohort Study.

BackgroundThe COVID-19 pandemic has led to significant reductions in transplantation, motivated in part by concerns of disproportionately more severe disease among solid organ transplant (SOT) recipients. However, clinical features, outcomes, and predictors of mortality in SOT recipients are not well-described.MethodsWe performed a multi-center cohort study of SOT recipients with laboratory-confirmed COVID-19. Data were collected using standardized intake and 28-day follow-up electronic case report forms. Multivariable logistic regression was used to identify risk factors for the primary endpoint, 28-day mortality, among hospitalized patients.ResultsFour hundred eighty-two SOT recipients from >50 transplant centers were included: 318 (66%) kidney or kidney/pancreas, 73 (15.1%) liver, 57 (11.8%) heart, and 30 (6.2%) lung. Median age was 58 (IQR 46-57), median time post-transplant was 5 years (IQR 2-10), 61% were male, and 92% had ≥1 underlying comorbidity. Among those hospitalized (376 [78%]), 117 (31%) required mechanical ventilation, and 77 (20.5%) died by 28 days after diagnosis. Specific underlying comorbidities (age >65 [aOR 3.0, 95%CI 1.7-5.5, p<0.001], congestive heart failure [aOR 3.2, 95%CI 1.4-7.0, p=0.004], chronic lung disease [aOR 2.5, 95%CI 1.2-5.2, p=0.018], obesity [aOR 1.9, 95% CI 1.0-3.4, p=0.039]) and presenting findings (lymphopenia [aOR 1.9, 95%CI 1.1-3.5, p=0.033], abnormal chest imaging [aOR 2.9, 95%CI 1.1-7.5, p=0.027]) were independently associated with mortality. Multiple measures of immunosuppression intensity were not associated with mortality.ConclusionsMortality among SOT recipients hospitalized for COVID-19 was 20.5%. Age and underlying comorbidities rather than immunosuppression intensity-related measures were major drivers of mortality.

PrescrAIP: A Pan-European Study on Current Treatment Regimens of Auto-Immune Pancreatitis.

Introduction: Treatment of autoimmune pancreatitis (AIP) is based solely on consensus and has yet to become standardized. Consequently, therapeutic regimens vary greatly between countries and centers, and largely depend on the experience of the physician. At this moment, the optimal regimen for inducing disease remission and preventing relapse is unknown.Objectives: The primary objective of this study is to describe current treatment regimens used in Europe, and to compare their effectiveness in inducing remission and preventing and treating relapse. The secondary objectives are: to identify risk factors for relapse; to assess the diagnostic accuracy of the Unified-AIP criteria; to assess the performance of the M-ANNHEIM score for predicting relapse; and to assess long-term outcomes including pancreatic exocrine insufficiency and pancreatic cancer.Methods: This is an international, retrospective, observational cohort study, performed in over 40 centers from 16 European countries. Eligible are all patients diagnosed with AIP from 2005 onwards, regardless of the used diagnostic criteria. Data on study subjects will be retrieved from the hospital's electronic medical records and registered with a standardized, web-based, electronic case report form (eCRF). To compare the effectiveness of treatment regimens in inducing remission, preventing relapse, and treating relapse, subjects will be stratified in groups based on: type of therapy; initial therapy dose; cumulative therapy dose; therapy tapering speed and duration; and having received maintenance therapy or not.Ethics and Dissemination: Ethical and/or institutional review board approvals are obtained by all participating centers according to local regulations. The study complies with the General Data Protection Regulation (GDPR). All manuscripts resulting from the study will be submitted to peer-reviewed journals.Conclusion: This is the first pan-European retrospective registry for AIP. It will produce the first large-scale data on treatment of European patients with AIP, providing answers on the use and effectiveness of treatment regimens. In the future, this collaboration may provide a network for continuation into a prospective European registry.

Successful linkage of French large-scale national registry populations to national reimbursement data: Improved data completeness and minimized loss to follow-up

Registries, a cornerstone of contemporary medicine, frequently suffer from incomplete documentation and losses to follow-up. By linking data to a single-payer national claims database, national registries may be enriched and the quality enhanced. To explore the value of data from the French Système National des Données de Santé (SNDS) as a resource to enhance the quality of registries when combined with data from electronic case report forms, and to assess the power to minimize data gaps and losses to follow-up. A probabilistic algorithm was developed to link and match records in the SNDS with patient data from the electronic case report forms of two registries on transcatheter aortic valve implantation: FRANCE-2 and FRANCE-TAVI. The algorithm created patient profiles from transcatheter aortic valve implantation procedures in the SNDS, matching them as closely as possible to the profiles in the registry databases. The objective was to achieve 90% linkage of the populations. The linked database was analysed for completeness and loss to follow-up. For validation, mortality curves for the linked registry cohorts were compared with those for the original populations. A total of 34,397 unique registries entries were identified, and 89.9% of patients in the SNDS could be linked. Rates of losses to follow-up over 2 years were 1.0% in the linked FRANCE-TAVI population compared with 40.3% based on electronic case report form documentation. For FRANCE-2, 3-year rates of losses to follow-up were 1.7% and 6.1%, respectively. Mortality curves for populations based on SNDS and electronic case report form data were practically superimposable. Linking data from a single-payer national claims database to national registries using a probabilistic approach is feasible and can close data gaps and practically abolish losses to follow-up in the registry population.

Dynamics of anti-malarial antibodies in non-immune patients during and after a first and unique Plasmodium falciparum malaria episode

BackgroundMalaria is a major travel medicine issue. Retrospective confirmation of a malaria episode diagnosed in an endemic area can have relevant implications in transfusional medicine in Europe, where blood donors are excluded from donation on the basis of positive malaria serology. However, there is scarce evidence on the dynamics of anti-malarial antibodies after a first malaria episode in non-immune individuals. The first aim of this study was to describe the dynamics of anti-malarial antibodies in a first malaria episode in non-immune travellers. Secondary objectives were to assess the sensitivity of serology for a retrospective diagnosis in non-immune travellers diagnosed while abroad and to discuss the implications in transfusional medicine.MethodsRetrospective analysis of the results of an indirect fluorescence antibody test (IFAT) for malaria available for patients with a first malaria episode by Plasmodium falciparum and admitted at the IRCCS Sacro Cuore Don Calabria hospital in a 14-year period. The antibody titres were collected at baseline and during further follow up visits. Epidemiological, demographic and laboratory test results (including full blood count and malaria parasite density) were anonymously recorded in a study specific electronic Case Report Form created with OpenClinica software. Statistical analysis was performed with SAS software version 9.4.ResultsThirty-six patients were included. Among them, all but two were Europeans (one African and one American). Median length of fever before diagnosis was 2 days (IQR 1–3). Thirty-five patients had seroconversion between day 1 and day 4 from admission, and the titre showed a sharply rising titre, often to a very high level in a few days. Only a single patient remained negative in the first 5 days from admission, after which he was no more tested. Six patients were followed up for at least 2 months, and they all showed a decline in IFAT titre, tending to seroreversion (confirmed in one patient with the longest follow up, almost 4 years).ConclusionsSerology demonstrated reliable for retrospective diagnosis in non-immune travellers. The decline in the anti-malarial titre might be included in the screening algorithms of blood donors, but further studies are needed.

A Cohort of Patients with COVID-19 in a Major Teaching Hospital in Europe.

Background: Since the confirmation of the first patient infected with SARS-CoV-2 in Spain in January 2020, the epidemic has grown rapidly, with the greatest impact on the region of Madrid. This article describes the first 2226 adult patients with COVID-19, consecutively admitted to La Paz University Hospital in Madrid. Methods: Our cohort included all patients consecutively hospitalized who had a final outcome (death or discharge) in a 1286-bed hospital of Madrid (Spain) from 25 February (first case admitted) to 19 April 2020. The data were manually entered into an electronic case report form, which was monitored prior to the analysis. Results: We consecutively included 2226 adult patients admitted to the hospital who either died (460) or were discharged (1766). The patients’ median age was 61 years, and 51.8% were women. The most common comorbidity was arterial hypertension (41.3%), and the most common symptom on admission was fever (71.2%). The median time from disease onset to hospital admission was 6 days. The overall mortality was 20.7% and was higher in men (26.6% vs. 15.1%). Seventy-five patients with a final outcome were transferred to the intensive care unit (ICU) (3.4%). Most patients admitted to the ICU were men, and the median age was 64 years. Baseline laboratory values on admission were consistent with an impaired immune-inflammatory profile. Conclusions: We provide a description of the first large cohort of hospitalized patients with COVID-19 in Europe. Advanced age, male sex, the presence of comorbidities and abnormal laboratory values were more common among the patients with fatal outcomes.

Proceedings of the 2019 GRAPPA Collaborative Research Network Meeting.

At the 2019 Group for Research and Assessment of Psoriasis and Psoriatic Arthritis-Collaborative Research Network annual meeting, the group presented its progress in selecting a database platform; items to include in an electronic case report form (eCRF); and standardized operating procedures (SOP) for the collection, processing, storage, and transport of biomaterial. A pilot investigator-initiated study was also proposed that, in addition to addressing an area of unmet need, would allow for the testing of both the eCRF and SOP.

Real-world outcomes of pembrolizumab plus carboplatin plus paclitaxel or nab-paclitaxel in non-small cell lung cancer (NSCLC).

e21717 Background: Pem+carbo+pac or nab-pac are approved first-line (1L) treatments for metastatic squamous NSCLC since 10/30/2018. Difference in real-world outcomes of these triplet combinations are unknown. Methods: Providers from community oncology practices in the USA reviewed the charts of consecutive NSCLC patients initiating 1L treatment with either pem+carbo+pac (“pac”) or pem+carbor+nab-pac (“nab-pac”) from 06/01/18-12/31/18; data were collected 11/22/2019-12/23/2019. Patient characteristics, treatment patterns, grade 3/4 toxicities, disease response, date of progression/death were retrospectively abstracted into an electronic case report form (eCRF). Univariate statistics were used to compare demographics, clinical characteristics and treatment patterns between cohorts. Overall survival (OS) from 1L was calculated using the Kaplan-Meier method. A Cox proportional hazards model was used to compare the risk of death between the two cohorts adjusted for multiple variables. Results: eCRFs were completed for 254 patients: nab-pac = 115, pac = 139. Median follow-up was 13.1 mos in both cohorts. No differences in demographics/clinical characteristics were noted (table). Median duration of therapy was 3.9 mos (nab-pac) and 3.8 mos (pac) (p = 0.58). Objective response rate: nab-pac = 69.6%, pac = 69.8% (p = 0.96). Receipt of maintenance therapy: nab-pac = 44.4%, pac = 48.9% (p = 0.47) . Median time to progression was 6.4 mos for nab-pac (n = 32) and 3.5 mos for pac (n = 29) (p = 0.10). 13.9% of nab-pac and 7.9% of pac patients had grade 3/4 toxicities (p = 0.16). At data cut-off, 18 nab-pac (15.7%) and 30 pac (21.6%) patients were deceased. Median OS from initiation of 1L was not reached. 12-month OS: nab-pac = 87.8% (95% CI: 81.8-93.8), pac = 79.3% (95% CI: 72.5-86.2). Adjusting for sex, age, race, PD-1 expression level, charlson comorbidity index (CCI) and ECOG-PS patients treated with nab-pac had a 40% reduction in mortality risk (HR = 0.55, 95% CI: 0.30-1.01, p = 0.05). Conclusions: No significant differences in demographics and clinical characteristics between patients treated with nab-pac or pac were observed. Median duration of treatment was equivalent with a marginally longer 12-month survival rate, and a lower adjusted mortality risk, among patients treated with nab-pac. [Table: see text]

Real-world disease burden for patients (pts) with myelofibrosis (MF) treated with ruxolitinib (RUX).

e19539 Background: MF is an aggressive myeloid neoplasm and until recently RUX was the only treatment available. Real-world, long-term outcomes in pts with MF have not been described. Methods: Providers from multiple community oncology clinics in the USA reviewed the charts of consecutive pts with MF (primary or post-polycythemia vera/essential thombocythemia MF) who received RUX at any time from Jan 1, 2015 to Dec 31, 2017; data were collected Nov 15–26, 2019. Pt characteristics, treatment patterns, and outcomes (symptoms, blood counts, disease transformation) were retrospectively abstracted into an electronic case report form (eCRF) at each visit during the first 6 months, at any RUX dose modification; and at 12, 24, and 36 months post-RUX initiation. Pt characteristics, treatment patterns, rationale for dose modification, and treatment duration are described for pts during the first 6 months of therapy. Results: eCRFs were completed for 104 pts with MF treated with RUX. 42 pts received a RUX dose modification and 62 did not. Median follow-up from RUX initiation was 23.5 months. In the first 6 months of therapy, 40.4% of pts had a RUX dose modification (76.2% dose reduced; 23.8% dose increased). The most common reason (70%) for dose increase was titration to therapeutic dose. The most common reason (75%) for dose reduction was hemoglobin 8–10 g/dL or platelet count 50–75 × 109. The starting dose of RUX was higher for no RUX dose modification pts (mean total daily dose 34.9 mg vs 27.0 mg, P &lt; 0.01). RUX dose-modified pts were older vs pts with no RUX dose modification ( P = 0.01); International Prognostic Scoring System (IPSS) risk score ( P = 0.89) and the proportion of pts with primary MF ( P = 0.06) were similar (Table). Overall, 64.4% of RUX treated pts discontinued therapy (RUX dose-modified 78.6%; no RUX dose modification 54.8%, P = 0.01). Median duration of RUX treatment was 11.0 months (RUX dose modified 10.6 months; no RUX dose modification 12.2 months; log rank P = 0.12). Conclusions: In this real-world study, 64.4% of pts discontinued RUX during follow-up. Higher rates of discontinuation and shorter time to discontinuation were noted for pts with a dose modification during the first 6 months of therapy. Given the short duration of therapy observed in this study as compared with published clinical trials, additional MF treatments are needed that offer pt benefit. [Table: see text]

Pattern of coronary arterial lesions amongst Saudi Arabians: a cross-sectional coronary fluoroscopic angiography study.

IntroductionCoronary artery disease (CAD) is a major cardiovascular disease (CVD) that affects a large population globally. This study aimed at determining coronary arterial lesions (CAL), particularly in terms of age, gender, coronary artery/arteries involved, number of lesions, and dominant coronary artery in the Kingdom of Saudi Arabia (KSA).MethodsA cross-sectional study was conducted at the King Khalid Hospital and Prince Sultan Centre for Health Care in Al-Kharj between January 2017 and March 2018. The patients with CAD lesion/s, fulfilling the inclusion criteria, were recruited from the cardiovascular medicine unit. Demographic information and the location and extent of their CAD lesions were extracted and documented in electronic case report form (eCRF). SPSS 22.0 was used for statistical analysis, and p value ≤ 0.05 was considered as significant.ResultsOf the 262 patients, male and female preponderance was 74.8% and 25.2%, respectively. The majority of the patients were adults above the age of 50 (72%). About half of all patients were active smokers (53%). Diabetes, hypertension, and hyperlipidaemia were recorded in 63%, 53.7% and 25% respectively. The incidence of cardiovascular lesions was documented after coronary angiography; left circumflex artery lesions had the highest incidence (85.3%), followed by left anterior descending artery lesions (82.4%) and right circumflex artery lesions (74.3%). Left main coronary artery lesions had the lowest incidence (10.3%). Most patients (59.6%) had three concomitant lesions, whereas a minority of patients had two (22.8%) and one lesion (17.7%).ConclusionThe pattern of CALs is different among the Saudi population as compared to other countries.

The iCRF Generator: Generating interoperable electronic case report forms using online codebooks

Semantic interoperability of clinical data is essential to preserve its meaning and intent when the data is exchanged, re-used or integrated with other data. Achieving semantic operability requires the use of a communication standard, such as HL7, as well as (functional) information standards. Manually mapping clinical data to a medical thesaurus, such as SNOMED CT, is complicated and requires expert knowledge of both the dataset, including its context, and the thesaurus. As an alternative, the (re-)use of codebooks, data definitions which may already have been mapped to a thesaurus, can be a viable approach. We've developed the iCRF Generator, a Java program that can generate the core of an interoperable electronic case report form (iCRF) for several of the major electronic data capture systems (EDCs). To build their CRFs, users can select one or more items from established codebooks, available from an online system called ART-DECOR. By providing an easy to use method to create CRFs for multiple EDCs based on the same codebooks, interoperability can be more easily attained.

The iCRF Generator: Generating interoperable electronic case report forms using online codebooks

Semantic interoperability of clinical data is essential to preserve its meaning and intent when the data is exchanged, re-used or integrated with other data. Achieving semantic operability requires the use of a communication standard, such as HL7, as well as (functional) information standards. Manually mapping clinical data to a medical thesaurus, such as SNOMED CT, is complicated and requires expert knowledge of both the dataset, including its context, and the thesaurus. As an alternative, the (re-)use of codebooks, data definitions which may already have been mapped to a thesaurus, can be a viable approach. We’ve developed the iCRF Generator, a Java program that can generate the core of an interoperable electronic case report form (iCRF) for several of the major electronic data capture systems (EDCs). To build their CRFs, users can select one or more items from established codebooks, available from an online system called ART-DECOR. By providing an easy to use method to create CRFs for multiple EDCs based on the same codebooks, interoperability can be more easily attained.