Long-term Follow-up Of Patients Research Articles

Age-based comparison of patients with heart failure treated with sacubitril-valsartan in France: a real-world evaluation

Abstract Background Sacubitril-Valsartan (S/V) was approved in Europe in 2015, for treatment of adults with symptomatic chronic heart failure with reduced left ventricular ejection fraction (HFrEF). In France, S/V had benefited from an early access program (2015-2018) and became available in community pharmacy since October 2018. Purpose Our study aimed to describe characteristics of patients who received S/V in France and to assess real-life mortality in this population regarding age. Methods This non-interventional retrospective study utilized existing claims data from the French National Healthcare Data System (SNDS) allowing long-term follow-up of patients who benefited from at least one reimbursement of S/V. Patient data between April 2012 and December 2020 were extracted from the database. Prespecified subgroup analyses were conducted for subjects aged <75 and ≥75 y to describe characteristics of patients at first reimbursement of S/V. Cardiovascular mortality and all-cause mortality were estimated using Kaplan-Meier method. Results Between 2015 and 2020, 104,910 patients initiated S/V and were followed for an average of 20 months; 42.6% (44,743 patients) were ≥75 y. Mean (SD) age difference between the 2 subgroups was 20.3 years [62.2 (9.6) vs 82.5 (5.2) in patients <75 and ≥75 y respectively]. Patients ≥75 y had a higher prevalence of atrial fibrillation (50.6% vs 31.2%) and chronic renal failure (20.9% vs 10.1%) and were less co-treated with mineralocorticoid-receptor antagonists (21.8% vs 40.5%) and ß-Blockers (64.0% vs 73.8%) compared to those <75 y. Three months after first dispensation, 48.3%, 35.3%, and 16.4% of patients aged ≥75 y received S/V dosages of 24/26mg, 49/51mg, and 97/103mg respectively, compared to 31.4%, 38.2%, and 30.4% for patients aged <75 y. The mean (SD) exposure period was 14.7 (13.6) months in patients ≥75 y vs 19.0 (15.5) months in <75 y patients. As expected, given the age difference, patients ≥75 y had a higher probability of all-cause mortality at 12-month 16.5% vs 6.2% in patients <75 y. Furthermore, 12-month cardiovascular mortality – evaluated during the early access program period (2015-2017) – in patients ≥75 y was 11.0% vs 4.8% in patients <75 y. Figures 1 and 2 show survival curves for cardiovascular mortality and all-cause mortality of these 2 subgroups. Of interest, cardiovascular mortality rate at 12 months was 7% in this study, compared to 6% in PARADIGM-HF, and all-cause mortality at 12-month was higher (10,6% vs 7%), likely due to the profile of the patients, who were older and have more comorbidities in real life. Finally, very few patients were hospitalized for reasons related to angioedema, 16/44,743 in ≥75 y patients and 26/60,167 in those <75 y. Conclusion This is the first French real-world study providing exhaustive nationwide data on S/V mortality over a period of almost 6 years in a very large population of elderly with a mean age of 70.9 y vs 63.8 y in PARADIGM-HF.Figure 1Figure 2

Long-term follow-up after infective endocarditis: comparison of patients who have undergone cardiac surgery versus a conservative therapy with and without an indication for cardiac surgery

Abstract Background Infective endocarditis (IE) is still a serious disease, with a high mortality despite cardiac surgery and modern conservative therapy. However, data on long-term follow-up are sparse. Methods Retrospective single centre registry on the hospital course and long-term follow-up of patients with IE who have undergone cardiac surgery or a conservative therapy with and without an indication for cardiac surgery. Results Between 1/2013 and 12/2016, 171 IE patients were treated at our hospital. A follow-up with either patient contact, review of hospital charts or physician contact was performed in 2022. Due to lost to follow-up (n=4) or refusal to participate after discharge (n=1), the final evaluation was performed with 166 patients (97.1%). They were followed-up for a median 2385.0 (between 2156 and 2773) days. 139 (81.3%) of the patients were operated (surgery) and 32 (18.7%) were treated conservatively. Amongst the conservatively treated group, 34.4% (11/32) of the patients had an indication for surgery, but it was decided not to perform it (cons-with) and 65.6% (21/32) had no indication for surgery (cons-without). Operated patients were younger compared to cons treated patients (65.1 ± 13.0 vs 72.8 ± 15.7, p = 0.00016). Whereas hospital mortality was not different between both groups (21.6 % vs 21.9 %, p = 0.97112), calculated 5y mortality was higher in der cons treated group (71.0 % vs 49.6 %, p=0.03168). Conclusions These data on long-term follow-up after IE show a high total mortality amongst all patients regardless of type of therapy, but higher mortality rate after 5 years amongst conservative treated patients (71%) compared toa surgical treated (49.6% p= 0.03168). The highest mortality rate after 5 years (100%) was detected in the cons-with compared to 55% in the cons-without group. More than 2/3 of deaths were due to IE. However, operated patients were younger and had less co-morbidities. Furthermore, rehospitalisation rate for recurrent IE was also high.

Impact of the underlying rhythm during cavotricuspid isthmus ablation on the incidence of future new-onset atrial fibrillation: insights from an international multi-centre registry

Abstract Background Radiofrequency catheter ablation of the cavotricuspid isthmus (RF-CTI) is the estabslished treatment of typical atrial flutter (AFL). After RF-CTI for AFL patients without previously documented atrial fibrillation (AF) may also develop AF. It is unknown whether the presence or absence of AFL at the time of ablation can predict the future occurence of AF. Purpose To compare baseline characteristics, procedural data, and long-term follow-up (FU) of patients with AFL. Methods Consecutive patients with documented AFL undergoing RF-CTI were enrolled in a multi-center registry in five centers in two countries between February 2017 and July 2023. For patients with AFL, entrainment mapping was performed for confirming CTI dependent AFL. Patients were categorized into two groups, those with AFL and those with sinus rhythm (SR) during RF-CTI. FU data were collected, including clinical assessments, 12-lead electrocardiogram (ECG) and 24-hour Holter-ECGs during each visit. Results Of the 949 subjects, 36% were excluded from the analysis due to a history of AF. A total of 574 patients were included (mean age 68 +/- 11 yrs, 14% females), 62% presented in AFL group and 38 % SR group during CTI. Median FU was 364 days. Patients in AFL group were older (P < 0.029), more likely to have diabetes (P = 0.026), and were less frequently treated with antiarrhythmic drugs (P < 0.001). In the first 12 months of FU, 40% of the patients underwent three FU examinations, while 77% of the patients underwent at least one 24-hour Holter-ECGs during FU. In the Kaplan-Meier analysis patients in AFL group significantly less often developed AF during FU (log-rank, P < 0.016, central ). In the multivariate Cox regression analysis, sinus rhythm during ablation (hazard ratio 1.474; 95% CI 1.043-2.084; P = 0.028), male gender (hazard ratio 1.722; 95% CI 1.116-2.658; P = 0.014) and coronary artery disease (hazard ratio 1.711; 95% CI 1.145-2.555; P = 0.009) were identified as predictors for the occurence of AF during FU. Conclusion Over a one year follow-up, new onset AF occurs significantly less frequently in patients with ongoing atrial flutter during RF-CTI ablation.

Chronotropic incompetence as a critical predictor for cardiovascular outcomes in heart failure with preserved ejection function

Abstract Background Chronotropic incompetence (CI), linked with exercise intolerance, is a prevalent feature of heart failure with preserved ejection fraction (HFpEF). Our study aimed to explore the impact of CI on cardiovascular outcomes in HFpEF patients. Methods From November 2019 to December 2022, we enrolled 359 subjects who underwent invasive cardiopulmonary exercise testing (iCPET) due to heart failure symptoms. After excluding individuals without HFpEF (those with resting pulmonary capillary wedge pressure [PCWP] < 15mmHg or exercise PCWP < 25mmHg), we identified 113 patients for long-term follow-up. Our assessment of cardiovascular outcomes encompassed hospitalization for heart failure (HHF) and cardiovascular death. We employed Cox regression analysis to evaluate the associations between exercise hemodynamic parameters and outcomes. Results Among the cohort, 85 (75.2%) subjects with CI were older (71 vs. 66 years) and more frequently women (61 vs. 28%) compared to those without CI. At peak exercise, patients with CI exhibited significantly reduced left atrial booster strain, impaired right ventricle-arterial coupling, decreased systemic vascular resistance, cardiac output (CO), stroke volume, VO2, respiratory exchange ratio, and minute ventilation/carbon dioxide production (VE/VCO2) slope. A significant increase in PCWP (36 ± 10 vs. 26 ± 11mmHg, p=0.012) was noted in patients with CI. Over a median follow-up of 22.6 months, 20 (17.7%) subjects experienced cardiovascular events. Multivariate Cox proportional analyses revealed that CI (Hazard ratio [HR] 1.725, 95% confidence intervals [CI] 1.212-2.413) and an elevated PCWP/CO slope (HR 1.829, 95% CI 1.331-2.382) were predictive of outcomes. Conclusions Patients with CI demonstrated heightened filling pressure, a steeper VE-VCO2 slope, and reduced VO2 compared to those without CI. These factors likely contribute to adverse cardiovascular outcomes in HFpEF patients with CI.

Development and validation of a training course on proton pump inhibitor deprescription for general practitioners in a rural continuing medical education program: a pilot study

BackgroundDespite being cornerstone medications for managing gastrointestinal disorders, proton pump inhibitors (PPI) have raised concerns due to inappropriate prescribing and overutilization, their potential side effects, and interactions with other medications. General practitioners (GPs) provide long-term patient follow-up and are targets to promote PPI deprescribing to reach the widest possible population. GPs practicing in rural settings encounter unique challenges as their numbers dwindle and their workload increases. Hence, targeted educational interventions are crucial to promote appropriate prescribing practices in such underserved areas.MethodsWe developed a continuing medical education (CME) program focused on PPI deprescribing for GPs in rural settings. The program comprised of an interactive training session featuring clinical cases, an open discussion, and distribution of educational materials. We assessed the program’s effectiveness using a two-level Kirkpatrick model, evaluating participant satisfaction and knowledge levels through pre- and post-course questionnaires.ResultsThirty-three GPs participated, with 61.9% working in semi-rural and 38.1% in rural areas (21 responded to the 1st questionnaire, 14 to the 2nd ). Median medical experience was 6 years, with 61.9% serving as internship supervisors. Despite 95.2% acknowledging PPI overprescription, none had previously participated in dedicated PPI CME programs. The open discussion session provided valuable insights into various topics related to PPI use and gastrointestinal health. Participants expressed high satisfaction with the program (average rating of 9.1/10) and 92.9% reported changes in practice, including increased awareness of inappropriate PPI prescriptions. Indeed, 92.9% of GPs identified inappropriate PPI use following the course. 57.1% of participants utilized the provided educational materials. The main practice changes observed included an increased reassessment rate of PPI indications (71.4% at each renewal after vs. 19% before, 28.6% non-systematically after vs. 66.8% before, 0% rarely after vs. 14.3% before; p = 0.006), the necessity for more than one consultation to deprescribe (64.3% after vs. 23.8% before; p = 0.021), systematic utilization of gradual cessation of PPI (100% vs. 61.9%; p = 0.039) and more frequent use of additional medication (92.9% vs. 57.1%; p = 0.022), primarily antiacids (92.3%).ConclusionsOur study underscores the effectiveness of targeted CME programs in promoting appropriate prescribing practices and enhancing knowledge among GPs in rural settings. Despite the challenges encountered in deprescribing PPI, the program facilitated proactive approaches in managing treatment discontinuation failures. Tailored educational interventions are essential for mitigating medication prescribing challenges and improving patient outcomes in rural primary care settings.Trial registrationsNot applicable.

Evaluation of retinal nerve fiber layer and choroidal structure in obese children and adolescents

Aim: Obesity-related vascular damage and endothelial dysfunction have deleterious effects on the ocular vasculature. It was aimed to examine optical coherence tomography (OCT) parameters in obese and overweight children and to define their relationship with metabolic markers in this study. Methods: The patient group consisted of 26 obese, 24 overweight patients aged between 8 and 18 years. The control group consisted of 25 healthy children with normal body mass index (BMI). This was a cross-sectional observational study. Serum glucose, lipid parameters, and homeostasis model assessment of insulin resistance (HOMA-IR) were investigated. Measurement of choroidal thickness was performed with Cirrus HD-OCT (Carl Zeiss Meditec Inc., Dublin, CA, USA). Retinal nerve fiber layer (RNFL) thickness was determined by an automatic computer algorithm without the need for user measurement. Results: There were no differences in subfoveal, nasal, temporal choroidal thickness, and RNFL between obese, overweight, and control groups (p>0.05). A positive (linear) moderate relationship was found between RNFL and the HOMA-IR of 26 patients in the obese group (r=0.389) (p=0.049). A positively weak correlation was found between height and RNFL in obese patient group (r=0.264, p=0.028). Conclusion: In the study, RNFL thickness increased as HOMA-IR level increased in obese children and adolescents. RNFL decreased as the height increased in obese children and adolescents. We believe that more comprehensive data about the effect of obesity on RNFL and choroidal thickness will be obtained with prospective studies in which the obese patient group with insulin resistance is taken separately and disease durations are defined, and long-term patient follow-up is performed.

Occlusive retinal vasculitis and scleritis following brolucizumab treatment: A case report.

Brolucizumab is an anti-vascular endothelial growth factor agent. Clinical trials have demonstrated excellent efficacy of brolucizumab for neovascular age-related macular degeneration in terms of both visual and anatomic outcomes. However, compared with conventional anti-vascular endothelial growth factor therapy, this new treatment has a higher incidence of complications, particularly the development of occlusive retinal vasculitis. In this case report, we describe a patient who developed occlusive retinal vasculitis following brolucizumab treatment for age-related macular degeneration, followed by scleritis 141 days later. A 67-year-old Japanese man with a diagnosis of polypoidal choroidal vasculopathy in his right eye has received 18 intravitreal injections of aflibercept in the past 4 years. Because of a decline in treatment efficacy, intravitreal brolucizumab injection (IVBr) was initiated. However, 17 days after the second IVBr, the patient developed extensive occlusive retinal vasculitis with intraocular inflammation. Occlusive retinal vasculitis in the right eye was diagnosed as a complication of brolucizumab therapy. Corticosteroid treatment was initiated. The occlusive retinal vasculitis resolved 121 days after the second IVBr, and corticosteroid treatment was discontinued on day 138. However, on day 158 after the second IVBr, scleritis with intraocular inflammation developed. By day 184 after the second IVBr, both the scleritis and intraocular inflammation had resolved with the resumption of topical corticosteroid treatment. This case underscores the potential for brolucizumab-induced scleritis and emphasizes the importance of recognizing and promptly managing this complication. Furthermore, it highlights the need for long-term careful follow-up in patients who develop occlusive retinal vasculitis after brolucizumab treatment.

The Role of Atrial Premature Complexes in Exercise Test in Predicting Atrial Fibrillation in Patients Without Obstructive Coronary Artery Disease.

Atrial fibrillation (AF) is usually triggered by frequent atrial premature complexes (APC) and atrial tachycardias originated in the pulmonary veins. The aim of the current study is to clarify the relationship between AF and APCs observed during treadmill exercise testing through long-term patient follow-up. Our study only examined the data of patients who did not have any obstructive coronary artery disease and had an exercise test. In total, 1559 patients were included in this research. The study data were divided into two groups according to the development of AF during follow-up. The patients who developed any type of AF during the follow-up period were classified as AF (+). Mean follow-up time for AF (+) and (-) groups were 48 and 47 months, respectively. In the univariable analysis, age, LAAP, and the presence of APCs (HR: 3.906, 95% CI: 2.848-5.365, p < 0.001) during the treadmill exercise test were significantly associated with the development of AF. In the multivariable analysis, age (adjusted HR: 1.063, 95% CI: 1.043-1.083, p < 0.001) and the presence of APCs during the treadmill exercise test (adjusted HR: 2.504, 95% CI: 1.759-3.565, p < 0.001) emerged as independent risk factors for the development of AF. The AF-free survival was significantly lower in the APCs (+) patients compared with the APCs (-) patients (log rank p < 0.001). Our study revealed that individuals without obstructive CAD who exhibited frequent APCs during treadmill exercise tests were more likely to develop AF.

Epidemiological Evidence for Upper Respiratory Infections as a Potential Risk Factor for Meniere's Disease: A Korean National Health Sample Cohort Study.

Meniere's disease (MD) is a chronic inner ear disorder characterized by tinnitus, ear fullness, episodic vertigo, and fluctuating hearing loss, which significantly impacts quality of life and poses management challenges. Recent evidence suggests that upper respiratory infections (URIs) may contribute to MD's onset. This study examines the potential link between URIs and MD using data from the Korean National Health Insurance Service-National Sample Cohort (2002-2019). We analyzed 19,721 individuals with MD and 78,884 matched controls, adjusting for demographic factors and comorbidities using propensity score matching. Our results showed that individuals with a URI within one year prior to the index date exhibited a 2.01-fold greater likelihood of developing MD (95% confidence interval [CI] = 1.91-2.11, p < 0.001), while those with URIs within two years demonstrated a 1.54-fold higher probability (95% CI = 1.50-1.59, p < 0.001). Furthermore, we found that even remote URIs occurring up to two years before the index date significantly increased the risk of developing MD, underscoring the need for long-term patient follow-up. Overall, our study suggests that individuals with a history of URI may have an elevated risk of developing MD over multiple time frames, regardless of demographic or health profiles.

Baroreflex activation therapy in advanced heart failure: A long-term follow-up.

Baroreceptor activation therapy (BAT) is a promising new treatment strategy for patients with heart failure with reduced ejection fraction (HFrEF). It provides symptomatic relief, improvement in left ventricular function and reduction of cardiac biomarkers. Data regarding the long-term effect of BAT on HFrEF are scarce. This retrospective, monocentric study aimed to assess long-term outcome in patients who underwent BAT. Patients with HFrEF who received BAT at Hannover Medical School between 2014 and 2023 were followed until the latest available follow-up. Symptom burden, echocardiography and laboratory testing were assessed before BAT implantation and in subsequent follow-ups. Twenty-three patients (mean age 66±10years, 83% male) with HFrEF were included in the study. Aetiology of heart failure was ischaemic in 70%. The majority of patients (96%) suffered from New York Heart Association (NYHA) III with a mean left ventricular ejection fraction (LVEF) of 23±8% and N-terminal pro-B-type natriuretic peptide (NT-proBNP) of 2463±2922pg/mL. A complication occurred in one patient during BAT implantation (4%). The mean follow-up was 3±2 (max. 7.5) years. BAT reduced NYHA classification in 12 patients (52%) after 1year, of which one patient remained in ameliorated NYHA for 7.5years. Echocardiographic evaluation revealed significant improvement in LVEF by 9±9% after 1year (P<0.001) and by 11±9% (P=0.005) after 2years. In addition, BAT mildly reduced NT-proBNP in the first 2years [non-significantly after 1year by 396±1006pg/mL and significantly after 2years by 566±651pg/mL (P=0.039)]. Seven patients reaching the recommended replacement time underwent device exchange. Four patients died during observation time. BAT resulted in a substantial reduction in NYHA classification and improvement in LVEF that lasted over long-term follow-up in many patients. NT-proBNP level decreased interim in long-term follow-up. These findings highlight the long-term efficacy and potential benefits of BAT as a therapeutic intervention for patients with HFrEF.

Effect of Different Injury Morphology of the Endplate on Intervertebral Disc Degeneration: Retrospective Cohort Study.

To describe a simplified classification scheme for endplate injury morphology based on 3D CT and to examine possible associations between endplate injury morphology and vertebral space and other variables such as type of fracture and disc degeneration in a group of patients with thoracolumbar fractures. This study was a retrospective cohort study. We collected patients with thoracolumbar fractures admitted from January 2015 to August 2020 and divided them into three groups based on the morphology of endplate injury (45 cases of mild endplate injury, 54 cases of moderate endplate injury, and 42 cases of severe endplate injury, SEI). Data of vertebral body and intervertebral space height and angle, the Pfirrmann grade, endplate healing morphology were collected during preoperative, postoperative, and long-term follow-up of patients in each group. One-way analysis of variance (ANOVA), chi-squared test, and repeated measurement ANOVA were used to compare and analyze the influence of endplate injury morphology on patient prognosis. Most moderate injuries to the endplate (fissure-type injury) and severe injuries (irregular depression-type injury, Schmorl's node-type injury) resulted in significant disc degeneration in the long-term transition. This study also showed significant differences in the height of the anterior margin of the injured spine and the intervertebral space height index during this process. The current study suggests that although the region of injury in endplate fissure-type injury is small preoperatively, it may be a major factor in leading to severe disc degeneration, loss of intervertebral height, and Cobb angle in the long term. The results of our study therefore may allow surgeons to predict the prognosis of patients with thoracolumbar fractures and guide their treatment.

The left atrial appendage exclusion for prophylactic stroke reduction (leaaps) trial: rationale and design

IntroductionLeft atrial appendage exclusion (LAAE) has been shown in randomized trials to reduce ischemic stroke risk in patients undergoing cardiac surgery with known atrial fibrillation (AF). Many patients undergoing cardiac surgery without pre-existing AF are at risk of stroke and may benefit from LAAE. MethodsLeft Atrial Appendage Exclusion for Prophylactic Stroke Reduction (LeAAPS) is an international, prospective, randomized, multicenter, blinded trial evaluating the effectiveness of LAAE in preventing ischemic stroke or systemic embolism in patients undergoing cardiac surgery at increased risk of AF and ischemic stroke. The trial will enroll 6500 patients at increased risk of stroke in whom a cardiac surgery is planned at 250 sites worldwide. Eligible patients are ≥18 years old, have no pre-existing AF but are at increased risk for AF and stroke (based on age, CHA2DS2-VASc score, left atrium size or brain natriuretic peptide). Patients are randomized 1:1 to receive either LAAE with AtriClip or no LAAE during cardiac surgery. Healthcare providers outside of the operating room and the patient will be blinded to allocation. The primary effectiveness endpoint is the first occurrence of ischemic stroke, systemic arterial embolism, or surgical or endovascular LAA closure. The powered secondary effectiveness endpoint is ischemic stroke or systemic arterial embolism. The primary safety endpoint is the occurrence of one of the following events (through 30 days): pericardial effusion requiring percutaneous or surgical treatment, peri-operative major bleeding, deep sternal wound infection, or myocardial infarction. Other endpoints include mortality, rehospitalizations, clinically diagnosed AF, transient ischemic attack, and cognitive and quality of life assessments. Follow-up is every 6 months for a minimum of 5 years; primary analysis occurs when 469 patients have had an ischemic stroke or systemic embolism. ConclusionThe results of the LeAAPS trial will demonstrate whether LAAE with AtriClip at the time of other routine cardiac surgery reduces stroke or systemic arterial embolism during long-term follow-up in patients at high risk of stroke without pre-existing AF. Trial RegistrationClinicalTrials.gov, Identifier: NCT05478304, https://clinicaltrials.gov/study/NCT05478304?term=%20NCT05478304&rank=1

Antral mucosa healing at long-term follow-up in patients with corpus atrophic gastritis and concomitant antral gastritis may mimic autoimmune gastritis

Background and aimCorpus atrophic gastritis (CAG) is defined as autoimmune when the antrum is spared, representing this element a crucial diagnostic criterium of autoimmune gastritis. In contrast, CAG with concomitant antral gastritis (AG), atrophic or non-atrophic, is generally attributed to H. pylori infection. During the natural history of CAG, possible antrum healing has been supposed. The current study aimed to assess the antral mucosa histopathological changes at long-term follow-up (FU) with respect to baseline in patients with CAG and concomitant atrophic or non-atrophic gastritis AG. MethodsRetrospective study on 130 patients with histologically diagnosed CAG with atrophic or non-atrophic AG. Mean FU gastroscopy was at 40.6 (range 4–192) months. Patients with confirmed CAG (n = 117; median age 66, range 20–87 years; 67.5 % F) were finally included. At baseline, 47 (40.2 %) had non-atrophic and 70 (59.8 %) atrophic AG. Helicobacter pylori (Hp) infection was present at histology in 27.3 % of patients, all treated. ResultsAt FU, 30/117(25.6 %) patients showed a complete antral healing; 11/29(37.9 %) were Hp positive at baseline, cured in all but one. Atrophic AG regressed in 16/70(22.8 %) patients. Both, antral healing and regression of antral AG, were found to be similar in Hp-cured and not-cured/ naïve-negatives patients (p > 0.05). ConclusionIn a subset of CAG patients, AG may regress at long-term FU irrespective of Hp cure, thus mimicking autoimmune atrophic gastritis and raising concerns about its current histopathological diagnostic criteria.

The thinking of several issues in the diagnosis and treatment of pancreatic cystic tumor

The incidence of pancreatic cystic neoplasms increased dramatically. The clinically optimal management guideline for pancreatic cysts has continued to improve in previous decades. Some issues in diagnosis and treatment require careful consideration and decision-making to achieve the best therapeutic effect and avoid overtreatment. This article analyzes the new progress in the differential diagnosis of pancreatic cystic tumors, surgical decision-making and scope of surgery, and long-term follow-up of patients who have not undergone surgery. In general, the current diagnosis and treatment decisions for pancreatic cystic tumors are more comprehensive and scientific as evidence becomes increasingly abundant, emphasizing the risks and benefits of precise diagnosis and treatment and balanced treatment.

Seroprevalence of SARS-CoV-2 in pediatric hematology-oncology patients.

The COVID-19 pandemic disproportionately affected persons with underlying medical conditions. SARS-CoV-2 infection susceptibility and vaccine effectiveness in pediatric hematology-oncology patients were unknown. From February to July 2022, anti-spike and anti-nucleocapsid Ig were assayed in 354 pediatric hematology-oncology subjects, including 53 oncology patients receiving chemotherapy (cancer), 150 patients with sickle cell disease (SCD), and 151 benign consult and long-term follow-up patients (controls). Participants completed a questionnaire. Frequencies of COVID-19 infection, defined by positive PCR/antigen test or anti-nucleocapsid Ig, were 62% in cancer, 71% in SCD, 52% in controls, with SCD statistically different than controls (p=.001). Infection was associated with COVID-19 exposure, Hispanic/Latino or Black/African American ethnicity, multi-family dwelling, sports participation; COVID-19 booster decreased association with infection. In COVID-19-positive cancer patients, 58% had positive anti-nucleocapsid and 76% had positive anti-spike (≥10U/mL), compared to essentially 100% seroconversion in SCD and controls (p<.0001, p=.01, respectively). Infection led to high anti-spike (≥2500U/mL) in 12% cancer, 14% SCD, and 15% controls (p=.93). Vaccination resulted in anti-spike positivity in 90% cancer, 100% SCD, and 100% controls (p=.06), and in high anti-spike in 20% cancer, 47% SCD, and 41% controls (p=.36). Of boosted subjects, one of two cancer, 6/6 SCD, and 19/19 controls exhibited high anti-spike. Cancer patients demonstrated similar SARS-CoV-2 infection frequency as controls, but diminished antibody response to infection and vaccination. SCD patients exhibited seroconversion indistinguishable from controls. Vaccination was associated with higher frequency of high anti-spike than infection; vaccination plus booster was most effective in eliciting high anti-spike antibody detectable beyond 90days.

529P Paucisymptomatic late onset Pompe disease: lessons from the clinical, radiological and histopathological long-term follow-up of an untreated patient

529P Paucisymptomatic late onset Pompe disease: lessons from the clinical, radiological and histopathological long-term follow-up of an untreated patient

OA14.06 Divarasib Single-Agent Long-Term Follow-up and Atezolizumab Combination Treatment in Patients with KRAS G12C-Positive NSCLC

OA14.06 Divarasib Single-Agent Long-Term Follow-up and Atezolizumab Combination Treatment in Patients with KRAS G12C-Positive NSCLC

Long-term follow-up of Chinese patients with methylmalonic acidemia of the cblC and mut subtypes

Long-term follow-up of Chinese patients with methylmalonic acidemia of the cblC and mut subtypes

Endoscopic Submucosal Dissection for Early Gastric Cancer Exceeding Expanded Criteria-Long-Term Outcomes from the German ESD Registry.

Background and aims: Endoscopic submucosal dissection (ESD) has become a standard treatment for early gastric cancer (EGC), often fulfilling guideline criteria (GC) or expanded criteria (EC). When lesions exceed the EC, surgical resection is recommended. However, a subgroup of these patients are not treated surgically. The aim of this study was to investigate the long-term follow-up of patients after ESD for EGC outside the EC (out of indication; OI). Methods: Patients who were included in the prospective German ESD registry were analyzed when ESD was performed for EGC. Patients were stratified in three groups according to histopathological features (GC, EC and OI). The results were evaluated in terms of patient characteristics, procedure characteristics and follow-up data. Results: Over a 48-month period, 195 patients from 14 German centers were included. In total, 71 lesions (36.4%) met the guideline criteria, 70 lesions (35.9%) corresponded to the expanded criteria and 54 lesions (27.7%) turned out to be OI. The R0 resection rate was significantly higher for the GC and EC groups than for the OI group (94.4% vs. 84.3% vs. 55.6%, p < 0.001). Additional surgery was not performed in 72% (39/54) of patients in the OI group. During a mean follow-up of 37 months, overall survival showed no significant difference between the EC and OI groups when endoscopic follow-up was performed without additional surgery (p = 0.064). Conclusions: The results show that a good long-term survival can be achieved after ESD for patients with OI lesions without additional surgery. The treatment decision has to be made on an individual basis, taking the patient's comorbidities and the risk of surgical resection into account.

Comprehensive genetic analysis for identification of monogenic disorders and selection of appropriate treatments in pediatric patients with persistent thrombocytopenia

Persistent thrombocytopenia is caused by various diseases, including immune thrombocytopenia, inherited thrombocytopenia, and inherited bone marrow failure syndromes. Considering the large number of genes responsible for inherited disorders, comprehensive genetic analysis is required to diagnose monogenic disorders. In this study, we enrolled 53 pediatric patients with persistent thrombocytopenia exhibiting visually small or normal-sized platelets. We performed whole-exome sequencing, including 56 genes responsible for inherited thrombocytopenia, and evaluated clinical parameters according to disease type. Among 53 patients, 12 patients (22.6%) were diagnosed with monogenic disorders. Nine patients had a family history of thrombocytopenia. Pathogenic or novel variants of genes responsible for inherited thrombocytopenia were identified in three and six patients, respectively. The variants in genes for inherited thrombocytopenia with large or giant platelets were unexpectedly identified in six patients. Pathogenic variants in genes for inherited bone marrow failure syndromes with systemic features were identified in three patients with atypical symptoms. Since the definitive diagnostic methods for immune thrombocytopenia are limited, and a substantial number of patients with inherited thrombocytopenia are at a high risk of developing malignancies, comprehensive genetic analysis is indispensable for selecting appropriate therapies, avoidance of unnecessary treatments for immune thrombocytopenia, and long-term follow-up of patients with inherited thrombocytopenia.