Long-term Follow-up Data Research Articles

Comparison of continuity index for drag versus point-by-point ablation with the use of proactive esophageal cooling

Abstract Background Proactive esophageal cooling was recently FDA cleared to reduce the likelihood of esophageal injury during radiofrequency (RF) ablation for treatment of atrial fibrillation (AF). Long-term follow-up data have also shown improved freedom from arrhythmia with proactive esophageal cooling compared to luminal esophageal temperature (LET) monitoring during pulmonary vein isolation (PVI). One hypothesized mechanism is the greater lesion continuity attainable with the use of cooling, which can be quantified by the Continuity Index (which increases as continuity decreases). Increasing the Continuity Index increases gap formation, reduces isolation, and leads to greater AF recurrence; however, the difference in Continuity Index between ablating in a point-by-point fashion versus a dragging fashion has not previously been analyzed. Purpose To prospectively measure the Continuity Index of PVI cases and compare the dragging technique to point-by-point technique when using proactive esophageal cooling or LET monitoring. Methods We determined the Continuity Index for 101 patients: 77 prospectively utilizing proactive esophageal cooling, 24 retrospectively recorded using LET monitoring. Cases were completed by four operators, three utilizing point-by-point technique, and one using the drag technique. The Continuity Index was calculated by incrementing one unit for each lesion that did not border the previous lesion during the PVI (simplifying the original description of the Continuity Index). Results For cases using esophageal cooling and performing RF ablation using the drag technique, the average Continuity Index was 8.5 (range: 1 to 14). For point-by-point operators using esophageal cooling, the average Continuity Index was 1.2 (range: 0 to 9), representing a difference of 7.3 between the dragging technique and point-by-point ablation (p < .001). Patients receiving LET monitoring and RF ablation using the drag technique had Continuity Indices of 23.8 (range 15 to 30). Patients receiving LET monitoring and RF ablation using point-by-point ablation had Continuity Indices of 28.4 (range 16 to 50). Figure 1 compares these findings. Only 4 patients (5%) in the esophageal cooling group had a Continuity Index above 6 on either side (all in the dragging group), versus 100% (24) of patients in the LET monitoring group. Conclusion Proactive esophageal cooling during PVI is associated with a high degree of lesion continuity, as represented by the low Continuity Index. The drag technique resulted in a higher index than the point-by-point technique, but both provided significantly lower Continuity Indices (greater lesion continuity) than cases using LET monitoring. This may offer a mechanism for the greater freedom from arrhythmia seen to date with proactive cooling in long-term follow-up.Figure 1.Comparison of Continuity Index

Long-term follow-up of single-chamber atrial pacing - system upgrade and wenckebach block point behavior: potential implications for leadless AAI pacing?

Abstract Introduction Until recently leadless pacemakers (PM) did not support atrial pacing. A novel dual chamber leadless PM system now provides treatment option for a wide range of indications. Single-chamber atrial stimulation (AAI) for the treatment of symptomatic sick sinus syndrome (SSS) is scarcely used due to concerns about the potential development of atrioventricular block (AVB) and development of paroxysmal atrial fibrillation (AF). Long-term follow-up data of AAI-PM is however scarce. Little is known about the role of serial Wenckebach block point (WBP) measurements in AAI-PM patients for risk stratification. Purpose To assess the incidence of dual chamber upgrade of patients treated with AAI-PM for SSS during long-term follow-up and to investigate the role of serial WBP measurements for risk stratification. Methods Prospectively enrolled patients undergoing implantation of an AAI-PM between October 2002 and December 2023. WBP was measured at implantation and serially during follow up. Results We included a total of 178 patients, of which 58% were female and the median age at implantation was 78 [IQR 71 – 83] years. The median follow-up duration was 6.5 [IQR 2.0 – 9.7] years. 24 patients (13.5%) received a system upgrade to DDD including 3 patients (1.7%) with a mode change to ventricular pacing only (VVI). Adjusted for years of follow-up the upgrade rate was 2.1% per year. Median time to system upgrade was 5.2 [IQR 1.6 – 8.7] years. Reasons for system upgrade were high-degree AVB (38%), low WBP (17%), atrial arrhythmias (33%), unclear syncope (8%) and infection (4%). Median WBP at implantation was 130 [120 – 140] bpm, showing a significant decline over time in the upgrade-group with 105 [95 – 130] bpm compared to the rest of the cohort with 130 [120-130] bpm (p = 0.009). A decline in WBP >20 bpm was found in 17.9% patients and a decline of 10-20 bpm in 33.7% of patients. New-onset atrial arrythmias were found in 36 patients (20%), 29% in the upgrade group and 19% in the rest of the cohort (p = 0.3). Neither age at implantation, sex, WBP at implantation, nor atrial arrhythmia prior to implantation were predictive for the need of a future system upgrade. Conclusion In this cohort of patients undergoing AAI-PM implantation for SSS, a system upgrade was required in 2% per year. The WBP remained stable in 48% of patients. Therefore, AAI-PM due to SSS emerges as a judicious and secure therapeutic modality, warranting increased consideration in the light of new treatment options for leadless pacing.

Long-term prognostic impact of fasting plasma glucose and myocardial flow reserve beyond other risk factors and heart disease phenotypes

Abstract Background Cardiometabolic risk factors, including high fasting plasma glucose (hFPG), are emerging determinants of residual mortality risk in patients with coronary artery disease (CAD) or heart failure (HF). Coronary microvascular dysfunction, encompassing a broad spectrum of traditional and emerging risk factors, might be a comprehensive risk predictor in these patients. Purpose To assess whether hFPG and global myocardial blood flow (MBF) reserve measured by positron emission tomography (PET), expressing global coronary function, may predict long-term prognosis beyond other risk factors and the presence of obstructive CAD or left ventricular (LV) dysfunction associated with HF. Methods Among 281 consecutive patients undergoing cardiac PET/CT because of stable chest pain or dyspnea we retrospectively collected long-term follow-up data in 103 patients (mean age 61±10 years, 74 males) in whom MBF measurements, coronary angiography and clinical data were available. Disease phenotypes included obstructive CAD (35%), LV dysfunction without obstructive CAD (43%) or none (22%). Results The mean age was 61±10 years (74 males, 72%). Among CVD risk factors, high LDL-cholesterol (hypercholesterolemia under treatment or LDL-C> 130 mg/dL) was present in 72%, high systemic blood pressure (hypertension under treatment or SBP>130/85 mmHg) in 51%, smoking habits in 49%, obesity (BMI>30 Kg/m2) in 22%, type 2 diabetes under treatment in 17%, low HDL-cholesterol (HDL-C< 40 mg/dL in males and <50 mg/dL in females) in 51%, high triglycerides (TG>150 mg/dL) in 27% and high fasting plasma glucose (FPG>100 mg/dL) in 33%. Global MBF reserve was <2 in 68% of patients. In a median follow-up of 10.9 years (7.8-13.9 IQR), 39 patients (37.8%) died (13.6% cardiac death). At multivariable Cox analyses including all risk factors and MBF reserve, age (HR 1.06, 95% CI 1.02-1.11), high FPG (HR 2.28, 95% CI 1.09-4.79) and depressed MBF reserve (HR 4.79, 95% CI 2.14-10.69) were the only independent predictors of death (Global χ2 36.74, P=0.0001). When disease phenotypes were added to the model, age (HR 1.07, 95% CI 1.02-1.12), high FPG (HR 2.18, 95% CI 1.02-4.63) and depressed MBF reserve (HR 4.47, 95% CI 1.96-10.18) remained independent predictors of death (Global χ2 37.41, P=0.0004) (Figure 1). Figure 2 plots the observed all cause death rate in patients with different disease phenotypes stratified for absence/presence of high FPG or MBF reserve <2. Death rate progressively increases according to disease phenotypes and is significantly associated with high FPG (P=0.049) or impaired MBF reserve (P=0.012) in each patient group. Conclusions These results suggest a strong long-term prognostic role of high FPG and depressed MBF reserve in a selected high-risk population of patients with high prevalence of obstructive CAD or HF.Figure 1 Figure 2

Transcatheter Edge-to-Edge Repair of the Mitral Valve in Four Dogs: Preliminary Results Regarding Efficacy and Safety

Mitral valve disease is a common heart disease in dogs. The aim of this study was to describe the cases of dogs that underwent mitral valve repair via a V-clamp device and to provide long-term follow-up data on cardiac function after mitral valve repair. Four dogs with mitral valve regurgitation who experienced coughing and dyspnea underwent surgical mitral valve repair between December 2023 and March 2024. The patients were evaluated via transthoracic and transesophageal echocardiography. Echocardiography revealed mitral valve leaflet regurgitation. Mitral valve repair was performed under general anesthesia using a V-clamp device introduced through an introducer wire guide. Echocardiography was conducted at baseline and during the six-month follow-up. Blood analysis results after surgical repair were normal. Follow-up echocardiography revealed no complications related to the procedure, with all dogs demonstrating improved respiratory signs and quality of life after repair. No adverse reactions were reported after surgery. A V-clamp device was used to repair mitral valve regurgitation in four dogs. Mitral valve repair via a V-clamp device is another treatment option for mitral valve disease in dogs.

Short- and Long-Term Outcomes of Prenatally Identified Congenital Aqueductal Stenosis by Fetal MRI.

Providing accurate prenatal prognostication for expectant parents is challenging due to limited literature on factors impacting outcomes in children with congenital aqueductal stenosis (CAS). This study stratified CAS patients into isolated or complex categories (presence of additional intra- or extra-cranial anomalies or genetic syndromes) and evaluated both short- and long-term outcomes. Additionally, the role of ventricular rupture was assessed. This was a single center retrospective-cohort study of CAS patients who underwent fetal MRI over a 10-year period. Of 140 patients with CAS, 107 (76%) were complex and 33 (24%) were isolated. There were no differences in the size of ventricular enlargement or incidence of ventricular rupture between the two groups. 14 pregnancies were terminated, 9 experienced fetal demise/stillbirth, and there were 21 post-natal deaths. Outcomes at the time of hospital discharge were available for 86 patients and long-term follow-up data for 64. CSF diversion (via ventriculoperitoneal shunt) was performed in 95% of complex and 71% of isolated CAS patients. Acutely, no differences were noted in seizures (complex: 10%; isolated: 18%) or respiratory support but there was an increased risk for feeding support. Risks for non-ambulatory status (complex: 32% vs. isolated: 0%), epilepsy (complex: 56% vs. isolated: 19%) and long-term gastrostomy tube assisted feeding (complex: 25.5% vs. isolated: 0%) were significantly greater with complex CAS. The presence of rupture did not impact clinical outcome. Poor clinical outcome was associated with complex CAS. Ventricular rupture alone did not portend a poor outcome. Prenatal counseling can tailor prognostication by CAS type.

Long-term follow-up of methotrexate and cytarabine in adult patients with Langerhans cell histiocytosis.

The optimal treatment strategy for adult Langerhans cell histiocytosis (LCH) remains unclear. Our previous study demonstrated the remarkable efficacy of combined methotrexate and cytarabine (Ara-C) [MA] therapy in patients newly diagnosed with LCH, with a median follow-up of 2 years. The present article reports long-term follow-up data spanning a median of 78 months (6.5 years) from a single-arm, single-centre, prospective phase 2 clinical trial (NCT02389400) conducted between January 2014 and December 2020. Ninety-five adults with newly diagnosed LCH exhibiting multisystem disease or multifocal single-system involvement underwent MA therapy every 35 days for six cycles. Methotrexate (1 g/m2) was administered by 24 h infusion on day 1 and AraC (0.1 g/m2) by 24 h infusion for 5 days. The primary end-point was event-free survival (EFS). The median patient age was 32 years (range 18-65 years). The overall response rate was 89.5%. Seven patients in this cohort died, and 38 experienced disease reactivation. No degenerative central nervous system diseases were observed. The estimated 6-year overall survival (OS) and EFS rates were 93.2% and 55.2% respectively. Multivariate analysis revealed that risk organ (RO) involvement at baseline (hazard ratio [HR] 6.135 [95% confidence interval (CI) 1.185-32.259]; p = 0.031) and age >40 years at diagnosis (HR 7.299 [95% CI 1.056-21.277]; p = 0.042) were associated with inferior OS. RO (HR 2.604 [95% CI 1.418-4.762]; p = 0.002) and skin (HR 2.232 [95% CI 1.171-4.255]; p = 0.015) involvement at baseline were poor prognostic factors for EFS. Regarding adverse events, four patients developed a second primary malignancy. In conclusion, the MA regimen was a valid and safe therapeutic approach for adult patients newly diagnosed with LCH.

Upper Airway Stimulation for Children and Adolescents with Down Syndrome: Long-Term Follow-Up.

Hypoglossal nerve stimulation (HGNS) is safe and effective for patients with Down syndrome (DS) and severe persistent obstructive sleep apnea (OSA). Long-term outcomes for this patient population have not been evaluated. A prospective single-group multicenter cohort study with 1-year follow-up was conducted between 2015 and 2021 among 42 adolescent patients with DS and severe persistent OSA. Here, we evaluate long-term outcomes in this patient cohort. Patients were evaluated with polysomnogram (PSG) at three timepoints: pre-implantation (timepoint 1), 1-year post-implantation (timepoint 2), and long-term follow-up (timepoint 3). Long-term follow-up data were available for 33 of 42 patients. Mean (SD) of timepoint 3 was 4.0 (1.9) years after implantation. Using a therapy response definition of a 50% decrease in Apnea Hypopnea INdez (AHI) from timepoint 1, the response rate was 69.7% (23/33) at timepoint 2 and 87.9% (29/33) at timepoint 3. From timepoint 1, there was a mean (SD) decrease in AHI of 12.7 (13.4) events/h at timepoint 2 and 15.7 (13.1) events/h at timepoint 3. The mean percentage change in AHI between timepoints 1 and 2 was -51.1% (95% CI: -32.8% to -69.3%) and between timepoints 1 and 3 was -59.6% (95% CI: -42.0% to -77.3%). Patients with DS and severe persistent OSA who undergo HGNS implantation may continue to experience improvement in PSG parameters at long-term follow-up. Future studies are needed to assess additional long-term outcomes in this patient population, including neurocognition and quality of life. 3 Laryngoscope, 2024.

Prenatal finding of isolated ventricular septal defect: genetic association, outcomes and counseling.

The innovation in ultrasound has greatly promoted the prenatal diagnosis of ventricular septal defect. As a minor lesion of congenital heart disease, the prenatal genetic counseling of isolated ventricular septal defect faces some challenges, including the true genetic correlationship, selection of appropriated testing methods to identify deleterious mutations, and avoidance of overdiagnosis and overintervention. Researchers have explored the prenatal diagnosis efficiency of commonly used cytogenetic and molecular genetic technologies. Small insertions/deletions and monogenic variants with phenotypic heterogeneity play important role and contribute to the comprehend of pathogenesis. Isolated ventricular septal defect fetuses without genetic finding and extracardiac structural abnormality generally have good pregnancy outcome. Long-term follow-up data is needed to describe the comprehensive map, such as the potential missed diagnosis especially late-onset syndromes, the impact on the quality of life and life expectancy. When conducting prenatal genetic counseling, strict adherence to ethical principles is needed to ensure that the rights of all parties involved are fully protected. Clinicians should carefully evaluate the risks and benefits and provide parents with sufficient information and advice to enable them to make informed decisions.

Changes in Use of Migraine Medications, Healthcare Resource Utilization, and Associated Direct Costs Over 12Months Following Initiation of Erenumab: A US Retrospective Real-World Analysis.

Erenumab-aooe is approved for the preventive treatment of migraine in adults. Recent publications have evaluated migraine medication use during the 6 months after starting erenumab, but longer-term follow-up data are limited. The objective of this study was to describe 12-month medication use and changes in healthcare resource utilization (HRU) and associated direct costs among patients initiating erenumab. We identified adult patients with an erenumab claim in the Merative MarketScan Commercial and Medicare Databases from May 2018 through September 2019. Eligible patients had ≥ 12months of continuous medical and pharmacy coverage before (pre-index period) and after (post-index period) the index date (first erenumab claim) in addition to pre-index evidence of migraine. Patients were stratified by post-index-period adherence to erenumab, defined as ≥ 80% of days covered (adherent) or < 80% of days covered (non-adherent). Outcomes were measured pre- and post-index, and differences between these periods were described. Among 7528 eligible patients, the mean (standard deviation) age was 45.1 (11.4) years and 85.4% were female; 38.5% of patients were adherent to erenumab. Most patients used acute or traditional migraine-preventive medications pre-index, with reductions in use observed post-index (acute medication was used by 95.6% of patients pre-index, compared to 92.3% post-index; traditional preventive medication was used by 89.6% of patients pre-index, compared to 81.9% post-index). Reductions were observed for HRU of emergency room visits (-3.8%) and brain- and other head-imaging studies (-7.5%). Overall costs associated with acute and traditional preventive medications were reduced (-$764), but costs for HRU increased slightly ($76). When stratifying by adherence and combining costs for acute and traditional preventive medications and HRU, adherent patients had cost decreases (-$1947), while non-adherent patients had cost increases ($101). Most patients initiating erenumab had prior use of acute and traditional migraine-preventive therapies. The reduction in acute and traditional migraine-preventive medication use and HRU over the 12-month follow-up supports the long-term clinical benefits of erenumab in the real-world setting.

Meta-analysis of clinical outcomes of robot-assisted partial nephrectomy and classical open partial nephrectomy.

Robotic-assisted partial nephrectomy (RAPN) has emerged as a promising alternative to classical partial nephrectomy (CPN). This study aimed to compare the outcomes of RAPN and CPN for treating localized renal tumors through a meta-analysis of available literature. Chinese databases, such as CNKI, Chinese Science and Technology Periodicals Database (VIP), and Wanfang Full-text Database, were searched using Chinese search terms, and all published articles on PubMed and Web of Science were searched using English search terms. Articles on Localized Renal Tumors were included. RevMan5.3 software was used for meta-analysis. The funnel plots were drawn using Stata software to assess publication bias. This study aimed to identify the differences between robotic-assisted partial nephrectomy and classic partial nephrectomy in patients with localized renal tumors. In total, 67 articles with 17677 patients were included. The results demonstrate the advantages of RAPN over CPN for localized renal tumors. Compared to CPN and RAPN had significant differences in intraoperative blood loss, hospital stay duration, incidence of perioperative complications, and proportion of patients requiring blood transfusion. Regarding surgical outcomes, RAPN showed more favorable results regarding the incidence of positive resection margins, postoperative decline in glomerular filtration rate (GFR), postoperative local recurrence rate, and proportion of Trifecta achieved. However, there was no significant difference between RAPN and CPN regarding 5-year tumor-specific survival rates. The study suggests that robotic-assisted partial nephrectomy is a viable alternative to classic surgery for renal tumors. The strengths of this study are the use of a comprehensive search strategy and the inclusion of studies published in both English and Chinese. The limitations of this study are the small sample size and the need for long-term follow-up data. RAPN and CPN have similar overall survival outcomes for treating localized renal tumors. However, RAPN may offer advantages in terms of perioperative outcomes and preservation of renal function. Further studies are needed to confirm these findings and to identify the optimal surgical approach for individual patients.

What is known about suicide prevention gatekeeper training and directions for future research.

Suicide prevention training that teaches skills to support a person experiencing thoughts of suicide and create community support networks, often termed, "gatekeeper" training (GKT), has been a longstanding pillar of international, national, and local suicide prevention efforts. GKT aims to improve knowledge, attitudes, and self-efficacy in identifying individuals at risk for suicide, hopefully enhancing one's willingness and ability to intervene with a person experiencing a crisis. However, little is known about GKT's effectiveness in creating the essential behavior change (e.g., increase in intervening behaviors) it sets out to accomplish. This paper explores the history and theoretical background of GKT, reviews the current state of research on GKT, and provides framing and recommendations for next steps to advance research and practice around GKT. Through positioning GKT appropriately within the field of suicide prevention, we argue that the field of suicide prevention needs more rigorous research around GKT that includes long-term follow-up data on usage of skills learned during training, data on outcomes of those who have received an intervention from a trained gatekeeper, and the integration of implementation science to further our understanding of which trainings are appropriate for which helpers.

The negative outlook: Long-term follow up of ThyroSeq negative and low-risk nodules.

Molecular testing of thyroid nodules is an essential tool to help risk stratify nodules with indeterminate cytology. Although ThyroSeq testing has been around for over a decade, there is a paucity of long-term follow-up data on cytologically indeterminate nodules that are determined to be molecularly negative or low-risk. The objective of this study is to assess the outcomes of nodules with indeterminate cytology (Bethesda III or IV) and negative or low-risk ThyroSeq results. This is a single academic institution retrospective cohort study. Patients with at least one thyroid nodule sampled with fine-needle aspiration who underwent ThyroSeq testing from 2012 to 2018 and had negative or low-risk ThyroSeq results on a cytologically indeterminate sample (n=159 patients, 167 nodules) were included in the study. Outcomes include the false-negative rate and negative predictive value of each test version, as well as follow-up length for each nodule. There were 159 patients with a mean age of 58 years (7-84 years) included in this study; the majority were female (81.8%). The mean follow-up was 4.0 years. Of 167 nodules, three were found to be malignant on resection (1.8%). The negative predictive value for the entire cohort was 98.2% and it was 89.3% for the surgical series. ThyroSeq testing has good negative predictive value and can help risk stratify cytologically indeterminate nodules. Routine follow-up allows for safe monitoring of nodules for features suggestive of malignancy.

Cost-effectiveness of TOMAC therapy for medication-refractory restless legs syndrome: an updated analysis based on Extension study data

Aims Patients with medication-refractory restless legs syndrome (RLS) have limited therapeutic options available for symptom relief. Tonic motor activation (TOMAC) presents a novel, non-invasive therapy for this patient population. The aim of this study was to conduct an updated cost-effectiveness analysis of TOMAC therapy based on recently available longer-term follow-up data. Materials and Methods A previously published decision-analytic Markov model was utilized to project strategy-specific costs and outcomes over three-years and lifetime for TOMAC compared to status quo treatment (control). Cohort characteristics were based on 24-week clinical data from the extension cohort of the RESTFUL study, which included longer-term follow-up of RESTFUL completers who were assigned to continue or discontinue TOMAC use (TOMAC and control, respectively). The primary analysis outcome was the incremental cost-effectiveness ratio (ICER) in $ per quality-adjusted life year (QALY) gained, calculated using change in International RLS Study Group Rating Scale (IRLS) score as the effect measure. Extensive sensitivity analyses were performed. Results TOMAC treatment improved IRLS by 5.9 versus control, resulting in estimated utility improvement of +0.05. Over three-years and lifetime, TOMAC added 0.14 and 0.73 QALYs, at incremental costs of $6,751 and $27,440, resulting in projected ICERs of $47,609 and $37,823 per QALY gained, respectively. TOMAC was found to be cost-effective across all tested sensitivity analyses. Limitations and Conclusion The inclusion of longer follow-up data in the current analysis confirms earlier exploratory cost-effectiveness findings and suggest TOMAC therapy may provide a high-value treatment option for patients with medication-refractory RLS.

Complete Response to Locoregional Therapy Plus Immunotherapy for Hepatocellular Carcinoma

Previous studies showed that 42% to 50% of patients with locally advanced hepatocellular carcinoma (HCC) achieved complete remission (CR) after combined locoregional therapy (LRT) plus immunotherapy (IO). However, data on predictors of CR and long-term clinical outcomes without surgery and after discontinuation of IO are lacking. To assess the long-term clinical outcomes among patients with unresectable HCC who achieved CR after LRT-IO and were placed on a watch-and-wait protocol. This cohort study included patients with unresectable HCC who achieved CR after LRT-IO in 2 prospective studies between January 2018 and December 2022. The time of data cutoff was June 2023. Radiologic CR was defined per modified Response Evaluation Criteria in Solid Tumors. All patients underwent close surveillance after CR without surgical interventions, and IO was discontinued. All patients had received stereotactic body radiotherapy followed by anti-programmed cell death protein 1 or anti-programmed death ligand 1 therapy. Forty-nine patients had received a dose of transarterial chemoembolization before stereotactic body radiotherapy. The primary outcome was the 3-year overall survival (OS) rate. Secondary outcomes included the 3-year time-to-progression rate, 3-year local control rate, and relapse pattern. Factors associated with CR were analyzed using multivariate analyses. A total of 63 patients were enrolled (58 male [92.1%]; median age, 69 years [range, 18-90 years]); 38 patients (60.3%) had macrovascular invasion, and the median tumor diameter was 10 cm (range, 3.8-31.1 cm). The median follow-up time was 34.7 months (95% CI, 6.5-64.6 months). Twenty-nine patients (46.0%) achieved CR. The patients achieving CR had a significantly better 3-year OS rate than patients not achieving CR (75.5% [95% CI, 58.2%-98.3%] vs 28.1% [95% CI, 7.4%-29.4%]; P < .001). Among the 29 patients with CR, the 3-year time-to-progression rate was 58.7% (95% CI, 38.7%-79.1%) and the 3-year local control rate was 90.5% (95% CI, 78.2%-100%). Ten patients (34.5%) developed recurrence; among them, 6 (60.0%) with solitary intrahepatic disease relapse underwent curative surgical treatment. The absence of tumor vascular invasion (odds ratio, 0.30; 95% CI, 0.10-0.89) and the sum of the largest lesion diameters of 8 cm or less (odds ratio, 0.26; 95% CI, 0.07-0.98) were associated with CR. This cohort study of LRT-IO with long-term follow-up data found a durable response in patients with locally advanced unresectable HCC. Long-term survival was attainable in patients with radiologic CR. Further randomized clinical trials are warranted.

Comment on, "Decoding pediatric spinal tumors: a single-center retrospective case series on etiology, presentation, therapeutic strategies, and outcomes".

The article "Decoding pediatric spinal tumors: a single-center retrospective case series on etiology, presentation, therapeutic strategies, and outcomes" by Lenga et al. (2024) provides essential insights into pediatric spinal tumors, a rare and challenging area of medical research. The authors present a thorough analysis of clinical presentations, treatment strategies, and patient outcomes, offering valuable data to refine therapeutic approaches and improve outcomes. However, the study's retrospective design, confined to a single center, introduces potential biases and limits the generalizability of findings. The lack of long-term follow-up data and a control group further restricts the study's scope. Future research should prioritize multi-center collaborations, incorporate control groups, and extend follow-up durations to better understand long-term outcomes. The establishment of standardized treatment protocols is also recommended to enhance consistency in managing pediatric spinal tumors across diverse clinical settings.

Prenatal Surgery for Open Fetal Spina Bifida in Patients with Obesity: A Review of Current Evidence and Future Directions.

Background: Obesity rates have significantly increased globally, affecting up to 40% of women of childbearing age in the United States. While prenatal repair of open fetal spina bifida has shown improved outcomes, most fetal surgery centers exclude patients with a body mass index (BMI) ≥ 35 kg/m2 based on criteria from the Management of Myelomeningocele Study (MOMS) trial. This exclusion raises concerns about healthcare equity and highlights a significant knowledge gap regarding the safety and efficacy of fetal spina bifida repair in patients with obesity. Objective: To review the current state of knowledge regarding open fetal surgery for fetal spina bifida in patients with obesity, focusing on safety, efficacy, and clinical considerations. Methods: A comprehensive literature search was conducted using the PubMed and EMBASE databases, covering articles from the inception of the databases to April 2024. Studies discussing fetal surgery for neural tube defects and documenting BMI measurements and their impact on surgical outcomes, published in peer-reviewed journals, and available in English were included. Quantitative data were extracted into an Excel sheet, and data synthesis was conducted using the R programming language (version 4.3.3). Results: Three retrospective studies examining outcomes of prenatal open spina bifida repair in a total of 43 patients with a BMI ≥ 35 kg/m2 were identified. These studies did not report significant adverse maternal or fetal outcomes compared to patients with lower BMIs. Our pooled analysis revealed a perinatal mortality rate of 6.1% (95% CI: 1.76-18.92%), with 28.0% (95% CI: 14.0-48.2%) experiencing the premature rupture of membranes and 82.0% (95% CI: 29.2-98.0%) delivering preterm (<37 weeks). Membrane separation was reported in 10.3% of cases (95% CI: 3.3-27.7%), the mean gestational age at birth was 34.3 weeks (95% CI: 32.3-36.3), and the average birth weight was 2651.5 g (95% CI: 2473.7-2829.4). Additionally, 40.1% (95% CI: 23.1-60.0%) required a ventriculoperitoneal shunt. Conclusion: While current evidence suggests that fetal spina bifida repair may be feasible in patients with obesity, significant limitations in the existing body of research were identified. These include small sample sizes, retrospective designs, and a lack of long-term follow-up data. There is an urgent need for large-scale, prospective, multicenter studies to definitively establish the safety and efficacy of fetal spina bifida repair in patients with obesity. Such research is crucial for developing evidence-based guidelines, improving clinical outcomes, and addressing healthcare disparities in this growing patient population with obesity.

Long-term major events after hospital discharge for out-of-hospital cardiac arrest

BackgroundCardiac arrest remains a global health issue with limited data on long-term outcomes, particularly regarding recurrent cardiovascular events in patients surviving out-of-hospital cardiac arrest. (OHCA). We aimed to describe the long-term occurrence of major cardiac event defined by hospital admission for cardiovascular events or death in OHCA hospital survivors, whichever came first. Our secondary objective were to assess separately occurrence of hospital admission and death, and to identify the factors associated with major event occurrence. We hypothesized that patients surviving an OHCA has a protracted increased risk of cardiovascular events, due to both presence of the baseline conditions that lead to OHCA, and to the cardiovascular consequences of OHCA induced acute ischemia-reperfusion.MethodsConsecutive OHCA patients from three hospitals of Sudden Death Expertise Center (SDEC) Registry, discharged alive from 2011 to 2015 were included. Long-term follow-up data were obtained using national inter-regime health insurance information system (SNIIRAM) database and the national French death registry. The primary endpoint was occurrence of a major event defined by hospital admission for cardiovascular events and death, whichever came first during the follow-up. The starting point of the time-to-event analysis was the date of hospital discharge. The follow-up was censored on the date of the first event. For patients without event, follow-up was censored on the date of December, 29th, 2016.ResultsA total of 306 patients (mean age 57; 77% male) were analyzed and followed over a median follow-up of 3 years for hospital admission for cardiovascular event and 6 years for survival. During this period, 38% patients presented a major event. Hospital admission for cardiovascular events mostly occurred during the first year after the OHCA whereas death occurred more linearly during the all period. A previous history of chronic heart failure and coronary artery disease were independently associated with the occurrence of major event (HR 1.75, 95%CI[1.06-2.88] and HR 1.70, 95%CI[1.11-2.61], respectively), whereas post-resuscitation myocardial dysfunction, cardiogenic shock and cardiologic cause of cardiac arrest did not.ConclusionSurvivors from OHCA must to be considered at high risk of cardiovascular event occurrence whatever the etiology, mainly during the first year following the cardiac arrest and should require closed monitoring.

Using Quality Improvement to Expand and Align State Public Health Long-Term Follow-up Data Collection Across Newborn Screening Conditions

Context: Despite the undeniable success of newborn screening (NBS), numerous barriers exist regarding long-term follow-up (LTFU) of children with conditions included in NBS. Furthermore, there is a focus on condition-specific follow-up with no national guidelines for standard quality measures collected by state public health LTFU programs. Program: Minnesota Department of Health (MDH) Longitudinal Follow-up for NBS. Implementation: A state public health LTFU for NBS quality improvement (QI) project was carried out with collaboration between MDH project team members and a QI Steering Team who provided clinical, public health, education, caregiver, and community perspectives and expertise to MDH. Relevant measures were selected from existing data elements, and additional measures were developed based on exchange between MDH project team and the QI Steering Team. Potential data sources were explored and prioritized. Processes for querying existing data sources such as death records were refined, new sources such as electronic health records and paper/PDF health records were established, and data collection was piloted. The Minnesota Electronic Disease Surveillance System was modified, and an electronic data form was created to promote consistent data abstraction from sources. Throughout the project, progress was evaluated and shared. At project conclusion, MDH project team and the QI Steering Team reviewed project outcomes and approaches to evolve the project into ongoing surveillance. Evaluation: Five common public health LTFU measures were determined. Overall, 77% of attempted measures were successfully collected. Primary and secondary data sources were adopted. In addition, collected data resulted in 7% of cases that were closed to further public health LTFU, often related to a move out of state. Discussion: This project established the feasibility of state public health LTFU surveillance of outcomes and health care use by collecting a common data set applicable across NBS conditions.

Congenital Brain Tumors: Surgical Outcomes and Long-Term Prognostic Factors

ObjectiveTo evaluate long-term outcomes of surgical resection for congenital brain tumors (CBTs) in infants under one year of age and to identify factors related to survival. MethodsOur retrospective study analyzed infants who underwent gross total (GTR) or subtotal resection (STR) for CBTs between 2001 and 2019. Data were obtained from medical records, including demographics, clinical presentation, diagnosis, tumor characteristics, and presence of hydrocephalus. Additional factors such as pre- and/or postoperative ventriculoperitoneal shunt (VPS) placement and adjuvant chemotherapy or radiotherapy were also reviewed. Cox regression analysis was used to identify factors associated with survival. ResultsThe study included 70 patients, with median age at surgery of 198.5 days, and 28 (40%) were girls. Seizures (31.4%) and vomiting (24.3%) were the most common presenting symptoms. High-grade tumors were present in 29 (41.4%) patients. GTR was achieved in 64.3% of cases, with surgical mortality rate of 7.1%. Overall survival rates at 5 and 10 years were 78% and 63%, respectively. Long-term follow-up data were available for 61 patients (87%), with median follow-up of 74.2 months. Among 45 long-term survivors, 55.5% had neurological sequelae. Factors associated with reduced survival included high-grade, preoperative hydrocephalus, larger tumor size, and VPS placement. The extent of resection improved survival only in low-grade tumor cases. Multivariable Cox regression analysis identified tumor grade and size as independent predictors of poor prognosis. ConclusionsSurgical resection remains crucial for treating CBTs in infants under one year, yet the aggressive nature of malignant tumors results in suboptimal outcomes regarding prognosis.

Health outcomes 3 months and 6 months after molnupiravir treatment for COVID-19 for people at higher risk in the community (PANORAMIC): a randomised controlled trial.

No randomised controlled trials have yet reported on the effectiveness of molnupiravir on longer term outcomes for COVID-19. The PANORAMIC trial found molnupiravir reduced time to recovery in acute COVID-19 over 28 days. We aimed to report the effect of molnupiravir treatment for COVID-19 on wellbeing, severe and persistent symptoms, new infections, health care and social service use, medication use, and time off work at 3 months and 6 months post-randomisation. This study is a follow-up to the main analysis, which was based on the first 28 days of follow-up and has been previously reported. For this multicentre, primary care, open-label, multi-arm, prospective randomised controlled trial conducted in the UK, participants were eligible if aged at least 50 years, or at least 18 years with a comorbidity, and unwell 5 days or less with confirmed COVID-19 in the community. Participants were randomly assigned to the usual care group or molnupiravir group plus usual care (800 mg twice a day for 5 days), which was stratified by age (<50 years or ≥50 years) and vaccination status (at least one dose: yes or no). The primary outcome was hospitalisation or death (or both) at 28 days; all longer term outcomes were considered to be secondary outcomes and included self-reported ratings of wellness (on a scale of 0-10), experiencing any symptom (fever, cough, shortness of breath, fatigue, muscle ache, nausea and vomiting, diarrhoea, loss of smell or taste, headache, dizziness, abdominal pain, and generally feeling unwell) rated as severe (moderately bad or major problem) or persistent, any health and social care use, health-related quality of life (measured by the EQ-5D-5L), time off work or school, new infections, and hospitalisation. Between Dec 8, 2021, and April 27, 2022, 25 783 participants were randomly assigned to the molnupiravir plus usual care group (n=12 821) or usual care group (n=12 962). Long-term follow-up data were available for 23 008 (89·2%) of 25 784 participants with 11 778 (91·9%) of 12 821 participants in the molnupiravir plus usual care group and 11 230 (86·6%) of 12 963 in the usual care group. 22 806 (99·1%) of 23 008 had at least one previous dose of a SARS-CoV-2 vaccine. Any severe (3 months: adjusted risk difference -1·6% [-2·6% to -0·6%]; probability superiority [p(sup)]>0·99; number needed to treat [NNT] 62·5; 6 months: -1·9% [-2·9% to -0·9%]; p(sup)>0·99, NNT 52·6) or persistent symptoms (3 months: adjusted risk difference -2·1% [-2·9% to -1·5%]; p(sup)>0·99; NNT 47·6; 6 months: -2·5% [-3·3% to -1·6%]; p(sup)>0·99; NNT 40) were reduced in severity, and health-related quality of life (measured by the EQ-5D-5L) improved in the molnupiravir plus usual care group at 3 months and 6 months (3 months: adjusted mean difference 1·08 [0·65 to 1·53]; p(sup)>0·99; 6 months: 1·09 [0·63 to 1·55]; p(sup)>0·99). Ratings of wellness (3 months: adjusted mean difference 0·15 (0·11 to 0·19); p(sup)>0·99; 6 months: 0·12 (0·07 to 0·16); p(sup)>0·99), experiencing any more severe symptom (3 months; adjusted risk difference -1·6% [-2·6% to -0·6%]; p(sup)=0·99; 6 months: -1·9% [-2·9% to -0·9%]; p(sup)>0·99), and health-care use (3 months: adjusted risk difference -1·4% [-2·3% to -0·4%]; p(sup)>0·99; NNT 71·4; 6 months: -0·5% [-1·5% to 0·4%]; p(sup)>0·99; NNT 200) had high probabilities of superiority with molnupiravir treatment. There were significant differences in persistence of any symptom (910 [8·9%] of 10 190 vs 1027 [11%] of 9332, NNT 67) at 6 months, and reported time off work at 3 months (2017 [17·9%] of 11 274 vs 2385 [22·4%] of 10 628) and 6 months (460 [4·4%] of 10 562 vs 527 [5·4%] of 9846; NNT 100). There were no differences in hospitalisations at long-term follow-up. In a vaccinated population, people treated with molnupiravir for acute COVID-19 felt better, experienced fewer and less severe COVID-19 associated symptoms, accessed health care less often, and took less time off work at 6 months. However, the absolute differences in this open-label design are small with high numbers needed to treat. UK Research and Innovation and National Institute for Health and Care Research.