Patient Population Research Articles

Treatment Responses of Pakistani Nasopharyngeal Carcinoma Patients Treated with Induction Chemotherapy Followed by Concurrent Chemoradiotherapy

Objective: To investigate the response of nasopharyngeal cancer patients to induction chemotherapy, followed by concurrent chemoradiation, in our patient population. Study Design: Retrospective cohort study

De novo Postoperative Postictal Psychosis Improved with Vagal Nerve Stimulation: A Case Report

Post-ictal psychosis can manifest in patients with intractable temporal lobe epilepsy following temporal lobectomy, presenting with psychiatric symptoms such as hallucinations, depression, and aggressive behavior. This case study details a 43-year-old male with refractory focal epilepsy who developed de novo post-ictal psychosis after undergoing left anterior temporal lobectomy and hippocampal ablation. Initially, the patient achieved seizure control, but later experienced recurrent focal seizures and subsequent post-ictal psychosis, characterized by aggression, paranoia, and hallucinations. Despite multiple hospitalizations and trials of antipsychotic medications, no improvement was observed. Following the placement of a vagal nerve stimulator, the patient’s psychotic episodes ceased, allowing for the discontinuation of antipsychotic medications. This case highlights the critical need for psychiatric monitoring in epilepsy patients post temporal lobectomy and underscores the potential of vagal nerve stimulator in managing peri-ictal psychotic episodes. Further research is essential to elucidate the mechanisms by which vagal nerve stimulator exerts its antipsychotic effects and to establish guidelines for its use in this patient population.

Abstract B027: Adapting communication resources for clinic staff in a multisite FQHC to improve HPV vaccination encounters with Spanish-speaking parents of adolescents

Abstract Introduction: Adopting evidenced-based strategies (EBS) targeting clinic staff in safety-net settings can improve adolescent HPV vaccination rates. Still, adaptations of EBS are needed to reach racial/ethnic minority patients and families effectively. As part of a larger implementation study on HPV vaccination, we examined and addressed adaptation needs for HPV vaccine communication resources to support clinic staff serving Hispanic adolescent FQHC patients. Methods: In October 2022-January 2023, we partnered with a large multi-site FQHC in Los Angeles, CA to deliver two clinic staff trainings designed to i) improve HPV knowledge and communication strategies; and ii) discuss HPV vaccine workflow. Informed by the Practice Change Model, we completed baseline qualitative interviews with clinic team members (providers, clinic staff, and leadership) and provider and clinic staff surveys to inform the development, implementation, and adaptation of clinic staff training on HPV vaccine knowledge and communication strategies. We then conducted site visits and key-informant interviews with clinic staff immediately post-training. Notes from site visits and key-informant interviews were transcribed to identify potential barriers to adoption and opportunities for adaptation. Results: Baseline data indicated 95% of clinic staff identified as Hispanic and 94% reported speaking a language other than English. Notes from site visits and key-informant interviews with clinic staff indicated clinic staff identified the need for Spanish language messaging to communicate HPV and cancer prevention messages to monolingual Spanish families. As a result, we adapted Spanish-language messages from the 2023 Health Resources and Services Administration toolkit and American Cancer Society to develop a handout with scripts that address top concerns about HPV vaccination with monolingual Spanish-speaking parents of adolescents to that were identified from baseline data collection. Bilingual-bicultural staff then validated messages and confirmed cultural relevance. Developed materials were then presented to clinic leadership to review and suggest further refinement before delivering them to clinic staff. Discussion: Key-informant interviews and post-training site visits played an important role in identifying opportunities to adapt EBS designed to improve HPV vaccination among culturally diverse patients. To better serve diverse communities disproportionately impacted by HPV-associated cancers, clinic staff need resources with HPV vaccine communication messages available in languages that reflect the needs of their patient population. Citation Format: Samantha Garcia, Adrienne Martinez-Hollingsworth, Sheila Salinas Navarro, Sharon Hudson, Zehui He, Emily Dang, Lawrence Palinkas, Lourdes Baezconde-Garbanati, Jennifer Tsui. Adapting communication resources for clinic staff in a multisite FQHC to improve HPV vaccination encounters with Spanish-speaking parents of adolescents [abstract]. In: Proceedings of the 17th AACR Conference on the Science of Cancer Health Disparities in Racial/Ethnic Minorities and the Medically Underserved; 2024 Sep 21-24; Los Angeles, CA. Philadelphia (PA): AACR; Cancer Epidemiol Biomarkers Prev 2024;33(9 Suppl):Abstract nr B027.

Abstract A161: Racial and ethnic differences in receipt of guideline-recommended hepatocellular carcinoma surveillance in a high Risk, diverse primary care patient population: A study in two large integrated healthcare systems in California and Hawai‘i

Abstract Guideline-recommended ultrasounds for hepatocellular carcinoma (HCC) surveillance target patients with cirrhosis and certain liver conditions, yet uptake remains low, particularly among racial/ethnic minority groups. Underuse may be attributed to factors at the patient, provider, healthcare system, and neighborhood levels. However, in-depth analyses identifying contributory factors are lacking. We investigated the presence of racial/ethnic disparities in receipt of recommended surveillance among high-risk patients as a retrospective analysis of electronic health record (EHR) data from Kaiser Permanente Hawai‘i (KPHI) and Sutter Health in California, both of which serve numerous Asian American, Hispanic, Native Hawaiian (NH), other Pacific Islander (PI), and multiracial/ethnic patients. We included patients with ≥1 in-person encounter (2000-2017), no history of liver cancer, and a clinical indication of surveillance eligibility (cirrhosis, chronic hepatitis B or C, or a high FIB-4 score ≥2.67) with a minimum EHR follow-up of 18 months post-eligibility. Recommended surveillance is biannual abdominal imaging (ultrasound, CT, MRI) or alpha-1 fetoprotein tests. We categorized eligible patients as 'ever surveilled' if conducted at least once during follow-up and by the proportion of follow-up time they were up-to-date with surveillance (PTUDS). The cohort includes 54,597 patients (9,488 from KPHI; 45,109 from Sutter); current results are for the KPHI subset only. Among N=9,488 patients (median age: 52; 46% female; 26% NHW, 14% NH, 11% Filipino, 10% multiracial/ethnic) median follow-up was 91 months and 82% were surveilled at least once, with a mean PTUDS of 14% (SD=19%) overall and 19% (SD=21%) among the surveilled. We used sex- and age-adjusted log-risk regression for risk ratio (RR) of ‘ever surveilled’ vs. not and linear regression for PTUDS; NHW is the reference group in all models. We found that NH (RR=0.96, 95%CI 0.93-1.00), PI (RR=0.91, 95%CI 0.97-0.95) and multiracial/ethnic (RR=0.95, 95%CI 0.91-0.99) patients were significantly less likely to be ever surveilled, and that PI (beta=-0.02, 95% CI -0.04-0.01) race/ethnicity was significantly associated with lower PTUDS, while Chinese (beta=0.04, 95% CI 0.02-0.06), Korean (beta=0.04, 95%CI 0.01-0.07), Vietnamese (beta=0.05, 95%CI 0.02-0.09), and other Asian (beta=0.03, 95%CI 0.00-0.07) ethnicities were significantly associated with higher PTUDS. Although not statistically significant, surveillance rates were lower for American Indian/Alaska Native and Black groups, and higher for Japanese and Non-Hispanic Other race/ethnicity groups. Continued analyses will incorporate Sutter data, employ fully adjusted models with clustering by health system, and examine patient, provider, and neighborhood drivers of these disparities. Our findings indicate racial/ethnic disparities in HCC surveillance receipt, particularly noting the heterogeneity within Asian populations, and underscore the importance of analyzing disaggregated data for Asian American groups to fully understand and address these disparities. Citation Format: Enyao Y. Zhang, Mindy DeRouen, Alison J. Canchola, Aly M. Cortella, Pushkar Inamdar, Janet N. Chu, Sixiang Nie, Mai Vu, Ma Somsouk, Michele M. Tana, Anna D. Rubinsky, Iona Cheng, Mi-Ok Kim, Mark Segal, Chanda Ho, Yihe G. Daida, Su-Ying Liang, Hashem B. El-Serag, Scarlett L. Gomez, Salma Shariff-Marco, Caroline A. Thompson. Racial and ethnic differences in receipt of guideline-recommended hepatocellular carcinoma surveillance in a high Risk, diverse primary care patient population: A study in two large integrated healthcare systems in California and Hawai‘i [abstract]. In: Proceedings of the 17th AACR Conference on the Science of Cancer Health Disparities in Racial/Ethnic Minorities and the Medically Underserved; 2024 Sep 21-24; Los Angeles, CA. Philadelphia (PA): AACR; Cancer Epidemiol Biomarkers Prev 2024;33(9 Suppl):Abstract nr A161.

Abstract B160: Assessing patient experience in cancer clinical trial participants: Results from a rapid umbrella review with an equity lens

Abstract Introduction: Clinical trials are an important part of cancer care, yet research assessing patient experience in cancer clinical trials is sparse. Ensuring that the patient voice is heard and valued in the context of clinical trials is critical to the delivery of high-quality, patient-centered, and equitable cancer care. Furthermore, understanding the aspects of cancer clinical trials that are important to and reported by patients may inform strategies to address barriers to clinical trial participation, improve diversity in clinical trials, and advance health equity, particularly among populations underrepresented in clinical research. This rapid umbrella review sought to understand how patient experience is assessed in cancer clinical trials and to identify opportunities to integrate an equity lens. Methods: PubMed, Embase, Cochrane, and CINAHL databases were searched for systematic reviews that identify and describe development, use, or testing of tools/instruments assessing patient experience of cancer clinical trial patients. The search strategy was collaboratively developed with an NIH Librarian, using a combination of key terms to describe: 1) patient experience, 2) clinical trials, 3) healthcare delivery, and ) systematic reviews. The search was limited to publications in the English language and a date range of 2018-2024. One co-author completed the title and abstract review based on the inclusion criteria. Two reviewers will then independently completed the full text review, quality appraisal, and data abstraction for all included studies. All authors will synthesize data into matrix tables and narratively summarize into key themes. Discrepancies were resolved through discussion. Results: Out of 1,751 yielded search results, 151 articles were preliminarily identified. Upon further reading of the full texts, the final list of included articles meeting inclusion criteria will be reported using Covidence. Summary findings from this rapid umbrella review will include the number of studies identified, clinical trials population, a description of the patient experience measures, domains, findings, as well as opportunities for health equity integration using the PROGRESS-Plus Framework. Conclusion: Equitable representation in clinical trials will require intentional efforts to understand and enhance the experience of patients on clinical trials across diverse patient populations, as well as address barriers to participation. However, there are several research gaps that limit our understanding in this area, including a lack of consistent definitions, patient experience domains; availability and use of standardized, validated measures for clinical trial populations, and knowledge about what factors differ and influence patients’ experiences, particularly among populations underrepresented in clinical research. Attention to these gaps will facilitate a greater understanding of patient needs, experiences, and barriers, and may promote actionable strategies to increase access to, inclusion of, and diversity in cancer clinical trials. Citation Format: Brenda Adjei, Katrina Makres, Eric Juarez, Sae-Jin E Lee, Lynne Padgett. Assessing patient experience in cancer clinical trial participants: Results from a rapid umbrella review with an equity lens [abstract]. In: Proceedings of the 17th AACR Conference on the Science of Cancer Health Disparities in Racial/Ethnic Minorities and the Medically Underserved; 2024 Sep 21-24; Los Angeles, CA. Philadelphia (PA): AACR; Cancer Epidemiol Biomarkers Prev 2024;33(9 Suppl):Abstract nr B160.

Abstract B135: Association of residential economic and racial segregation with metastatic disease at diagnosis in pediatric patients with solid tumors

Abstract Background: Disparities in pediatric cancer outcomes by race and ethnicity may be influenced by neighborhood characteristics, particularly residential economic and racial segregation. Prior to a new cancer diagnosis, pediatric patients are vulnerable to structural barriers embedded in their neighborhoods. In particular, residential economic and racial segregation may impede access to initial care for early cancer detection, increasing the likelihood of presenting with distant metastatic disease at diagnosis that can translate to excess morbidity and mortality. This study assesses the association of residential economic and racial segregation as measured by the Index of Concentration at the Extremes (ICE) with the likelihood of presenting with distant metastatic disease (as compared to local or locoregional disease) among children newly diagnosed with cancer. Methods: We studied a cohort of pediatric patients diagnosed with solid tumors at Children’s Healthcare of Atlanta between 2010 and 2016. Patients’ ZIP codes were linked to the ICE, which was constructed at the ZIP code level using American Community Survey 2012-2016 five-year estimates. The ICE was categorized into tertile 1 (most deprived) through tertile 3 (most privileged). Multivariable logistic regression models were used to assess the association between residential economic and racial segregation, defined by tertiles of the ICE, and the likelihood of presenting with distant metastatic disease, as determined by initial cancer staging. All models adjusted for patient- and disease-level covariates, including age, sex at birth, health insurance type, and cancer type. Results: Among 829 patients identified in our analytic sample, 27.6% had distant metastatic disease at diagnosis. Living in the most deprived (ICE tertile 1) versus the most privileged (ICE tertiles 2/3) neighborhoods was significantly associated with a higher likelihood of presenting with distant metastatic disease at diagnosis (adjusted odds ratio [OR] = 1.46; 95% confidence interval [CI] = 1.03 to 2.07; p=0.03). Additionally, non-Hispanic Black patients living in the most deprived neighborhoods were more likely to have distant metastatic disease at diagnosis compared to non-Hispanic White patients living in the most privileged neighborhoods (adjusted OR = 1.83; 95% CI = 1.15 to 2.92; p=0.002). Conclusion: This study provides new insights into how residential economic and racial segregation is associated with metastatic disease at diagnosis among children presenting with a new solid tumor. Our findings highlight the need for geographically targeted interventions to improve community care, resource allocation and investment, and public policies aimed at supporting this vulnerable patient population. Citation Format: Alexandra Cathcart, Sharon M. Castellino, Lu Zhang, Karen Wasilewski-Masker, Andrew L. Hong, Heeju Sohn, Xu Ji. Association of residential economic and racial segregation with metastatic disease at diagnosis in pediatric patients with solid tumors [abstract]. In: Proceedings of the 17th AACR Conference on the Science of Cancer Health Disparities in Racial/Ethnic Minorities and the Medically Underserved; 2024 Sep 21-24; Los Angeles, CA. Philadelphia (PA): AACR; Cancer Epidemiol Biomarkers Prev 2024;33(9 Suppl):Abstract nr B135.

Abstract C102: Polyethnic-1000: Advancing cancer genomics by studying ethnically diverse, underserved patient populations in New York

Abstract C102: Polyethnic-1000: Advancing cancer genomics by studying ethnically diverse, underserved patient populations in New York

Low Versus Standard Intensity Heparin Protocols in Adults Maintained on Extracorporeal Membrane Oxygenation: A Retrospective Cohort Study

Background: Patients maintained on extracorporeal membrane oxygenation (ECMO) often require systemic anticoagulation to prevent circuit clotting and systemic thromboembolic complications. The optimal intensity of anticoagulation to balance the risk of bleeding and prevention of thrombotic complications in this patient population is not well described. Objective: To compare bleeding events in patients on ECMO anticoagulated with standard vs low intensity heparin protocols. Methods: This single-center, retrospective cohort study included adult patients on VA- or VV-ECMO and anticoagulated with low or standard intensity heparin protocols. The primary outcome was the incidence of major bleeding; secondary outcomes included the incidence of minor bleeding, thrombotic complications, heparin-induced thrombocytopenia, in-hospital mortality, time in therapeutic range, anti-Xa correlation with aPTT, intensive care unit and hospital lengths of stay, oxygenator exchanges, and rate of protocol switching. Results: A total of 27 patients (14 low intensity, 13 standard intensity) were included. There were six major bleeding events in the low intensity group and four in the standard intensity group ( P = 0.69); there were four minor bleeding events in the low intensity group and five in the standard intensity group ( P = 0.69). Seven patients in the standard intensity group switched protocols; zero patients in the low intensity group switched protocols ( P = 0.002). There were no differences in any other outcomes. Conclusions: There was no difference in the incidence of any bleeding or thrombotic events when using a low vs standard intensity heparin protocol in patients on ECMO. A low intensity heparin strategy for patients on ECMO may be feasible and safe.

Facilitators and challenges of implementing a digital patient education programme for rheumatoid arthritis into clinical practice

BackgroundThe integration of telehealth interventions into clinical practice is frequently delayed, hindering the full adoption. Previously, we developed a digital patient education (PE) programme for self-management in rheumatoid arthritis (RA). While the programme design considered crucial factors to ensure the likelihood of success in clinical practice, there is a need for a systematic evaluation of implementation perspectives. The purpose of this study was to explore perspectives crucial to implementation of a digital PE programme in clinical practice.MethodsThe non-adoption, abandonment, scale-up, spread and sustainability (NASSS) framework was used to evaluate the successes and challenges of implementing the digital PE programme. We included a data set consisting of qualitative focus group discussions involving study nurses, rheumatologists, and leaders from rheumatology departments. Data analysis was guided by a deductive content analysis approach. Further we included data from earlier studies pertaining to the programme’s implementation, comprising the programme development process, a randomized controlled trial evaluating the programme’s effectiveness, and a qualitative study exploring patients’ perspectives of the programme.ResultsFacilitators and challenges of importance to implementation of digital PE were identified. While a wide range of patients could benefit from using digital PE, future implementation should aim for an even broader group than those studied. Both patients and healthcare providers embraced the technology, and the fact that it did not require specific technical skills enhances its potential for success. However, offering digital PE should be based on individual assessments, and expanding its use will require organizational adjustments. An adaptable structure is needed to accommodate unforeseen care needs that may arise following the use of digital PE at home. There was indication of some reluctance among healthcare providers toward the programme shown by concerns about changing roles, which could impact the adoption of the program.ConclusionsThe design and ease of use of the technology, the program’s effectiveness, its availability, and the potential to release healthcare resources may encourage the implementation of digital patient education. Challenges associated with implementing this mode of care pertains to the condition and the patient population, user adoption of the technology, and the organization of patient education.Trial registrationThe study is registered by the Central Denmark Region Scientific Committee (no. 1-16-02-52-19).

Correlation of gastrointestinal symptom rating scale and frequency scale for the symptoms of gastroesophageal reflux disease with endoscopic findings

Background Gastroesophageal Reflux Disease (GERD) is caused by the reflux of gastric contents into the esophagus and has a 13% global prevalence that is increasing. GERD symptoms negatively impact physical, social, and emotional quality of life. The Frequency Scale for the Symptoms of GERD (FSSG) and the Gastrointestinal Symptom Rating Scale (GSRS) determine the efficacy of treatment but may not correlate with endoscopically estimated esophageal mucosal injury severity. We aimed to probe the correlation between FSSG, GSRS, and esophageal injury severity to evaluate whether these scores can predict GERD severity. Methods A total of 2962 patients who underwent physical examinations, including upper gastrointestinal endoscopy, at the Kyoto Kuramaguchi Medical Center, Japan, were enrolled in this study. Upper gastrointestinal endoscopy was used to diagnose fundic mucosal atrophy, reflux esophagitis based on the Los Angeles (LA) classification, gastroesophageal flap value function (GEFV) based on Hill’s classification, and Barrett’s esophagus. Endoscopic diagnoses were examined for correlations with FSSG and GSRS scores. Results In reflux esophagitis, FSSG and GSRS scores correlated with LA-B and LA-C endoscopic diagnosis but not with LA-M and LA-A endoscopic findings. Multiple regression analysis results were similar. FSSG scores reflected advanced fundic gland mucosal atrophy, while GSRS scores associated with high grade of GEFV. Conclusions This is the first report to examine the correlation between FSSG and GSRS scores and endoscopic findings in a relatively large patient population. Our findings suggest that these scores can diagnose the severity of reflux esophagitis.

Irinotecan hydrochloride liposome HR070803 in combination with 5-fluorouracil and leucovorin in locally advanced or metastatic pancreatic ductal adenocarcinoma following prior gemcitabine-based therapy (PAN-HEROIC-1): a phase 3 trial

Liposomal irinotecan has shown promising antitumor activity in patients with advanced or metastatic pancreatic ductal adenocarcinoma (PDAC) who have undergone prior gemcitabine-based therapies. This randomized, double-blind, parallel-controlled, multicenter phase 3 study (NCT05074589) assessed the efficacy and safety of liposomal irinotecan HR070803 combined with 5-fluorouracil (5-FU) and leucovorin (LV) in this patient population. Patients with unresectable, locally advanced, or metastatic PDAC who had previously received gemcitabine-based therapies were randomized 1:1 to receive either HR070803 (60 mg/m2 anhydrous irinotecan hydrochloride, equal to 56.5 mg/m2 free base) or placebo, both in combination with 5-FU (2000 mg/m2) and LV (200 mg/m2), all given intravenously every two weeks. The primary endpoint of the study was overall survival (OS). A total of 298 patients were enrolled and received HR070803 plus 5-FU/LV (HR070803 group, n = 149) or placebo plus 5-FU/LV (placebo group, n = 149). Median OS was significantly improved in the HR070803 group compared to the placebo group (7.4 months [95% CI 6.1–8.4] versus 5.0 months [95% CI 4.3–6.0]; HR 0.63 [95% CI 0.48–0.84]; two-sided p = 0.0019). The most common grade ≥ 3 adverse events in the HR070803 group were increased gamma-glutamyltransferase (19.0% versus 11.6% in placebo group) and decreased neutrophil count (12.9% versus 0 in placebo group). No treatment-related deaths occurred in the HR070803 group, while the placebo group reported one treatment-related death (abdominal infection). HR070803 in combination with 5-FU/LV has shown promising efficacy and manageable safety in advanced or metastatic PDAC in the second-line setting, representing a potential option in this patient population.

Optimizing tacrolimus dosing in Hispanic renal transplant patients: insights from real-world data

AimTacrolimus, an immunosuppressant used to prevent organ rejection in renal transplant patients, exhibits high inter-patient variability, necessitating therapeutic drug monitoring. Early post-transplant tacrolimus exposure in Hispanics is understudied. Although genotypic information is linked to pharmacokinetic differences, its clinical application remains limited. This study aimed to use a real-world data-driven, pharmacokinetic model-based approach for tacrolimus in Hispanics to determine a suitable initial dose and design an optimal dose titration strategy by simulations to achieve plasma trough concentration target levels of 10–12 ng/mL at the earliest.MethodsSparse concentration-time data of tacrolimus were obtained from electronic medical records for self-identified Hispanic subjects following renal transplant. Rich pharmacokinetic literature data was leveraged to estimate structural pharmacokinetic model parameters, which were then fixed in the current analysis. Only apparent clearance was estimated with the sparse tacrolimus data and potential covariates were identified. Simulations of various starting doses and different dose titration strategies were then evaluated.ResultsThe analysis included 121 renal transplant patients with 2,215 trough tacrolimus concentrations. A two-compartment transit absorption model with allometrically scaled body weight and time-varying hematocrit on apparent clearance adequately described the data. The estimated apparent clearance was 13.7 L/h for a typical patient weighing 70 kg and at 30% hematocrit, demonstrating a 40% decrease in clearance compared to other patient populations. Model based simulations indicated the best initial dose for the Hispanic population is 0.1 mg/kg/day. The proposed titration strategy, with three dose adjustments based on trough levels of tacrolimus, increased the proportion of patients within the target range (10–12 ng/mL) more than 2.5-fold and decreased the proportion of patients outside the therapeutic window by 50% after the first week of treatment.ConclusionHispanic renal transplant population showed an estimated 40% decrease of apparent clearance in the typical patient compared to other populations with similar characteristics. The proposed dose adjustment attained the target range rapidly and safely. This study advocates for tailored tacrolimus dosing regimens based on population pharmacokinetics to optimize therapy in Hispanic renal transplant recipients.

Risk Stratification and Management of Acute Respiratory Failure in Patients With Neuromuscular Disease

Objectives: Guillain-Barré syndrome (GBS) and myasthenia gravis (MG) are the most common causes of acute neuromuscular respiratory failure resulting in ICU admission. This synthetic narrative review summarizes the evidence for the prediction and management of acute neuromuscular respiratory failure due to GBS and MG. Data Sources: We searched PubMed for relevant literature and reviewed bibliographies of included articles for additional relevant studies. Study Selection: English-language publications were reviewed. Data Extraction: Data regarding study methodology, patient population, evaluation metrics, respiratory interventions, and clinical outcomes were qualitatively assessed. Data Synthesis: No single tool has sufficient sensitivity and specificity for the prediction of acute neuromuscular respiratory failure requiring mechanical ventilation. Multimodal assessment, integrating history, examination maneuvers (single breath count, neck flexion strength, bulbar weakness, and paradoxical breathing) and pulmonary function testing are ideal for risk stratification. The Erasmus GBS Respiratory Insufficiency Score is a validated tool useful for GBS. Noninvasive ventilation can be effective in MG but may not be safe in early GBS. Airway management considerations are similar across both conditions, but dysautonomia in GBS requires specific attention. Extubation failure is common in MG, and early tracheostomy may be beneficial for MG. Prolonged ventilatory support is common, and good functional outcomes may occur even when prolonged ventilation is required. Conclusions: Multimodal assessments integrating several bedside indicators of bulbar and respiratory muscle function can aid in evidence-based risk stratification for respiratory failure among those with neuromuscular disease. Serial evaluations may help establish a patient’s trajectory and to determine timing of respiratory intervention.

Barriers to Offering Organ Preservation for Rectal Cancer in a Predominantly Hispanic Safety Net Hospital.

Total neoadjuvant therapy (TNT) for locally advanced rectal cancer (LARC) has shown promise in achieving pathologic complete response (pCR) and enabling organ preservation through watch-and-wait (WW) strategies. However, implementation of WW protocols in diverse patient populations and safety-net hospitals faces unique challenges. The objective of this study is to evaluate TNT outcomes and identify barriers to WW implementation in a predominantly Hispanic safety-net hospital in South Texas. A retrospective review was conducted of 40 LARC patients treated with TNT at an academic tertiary referral center in South Texas between 2018 and 2023. Patient demographics, disease characteristics, and pCR rates were analyzed. A survey of multidisciplinary providers assessed perceived institutional and patient-related barriers to WW implementation. The cohort was 70% Hispanic, with a median age of 54 years. Most patients had advanced disease at diagnosis (57.5% T4, 65% N2). The pCR rate was 18.5% (5/27) among patients undergoing surgery. Re-review of MRIs for pCR patients revealed that 2/5 had minimal residual disease. The provider survey identified MRI quality variability, lack of dedicated treatment coordinators, and concerns about patient compliance and financial barriers as key obstacles to WW implementation. Despite advanced disease presentation in a predominantly Hispanic population, TNT achieved pCR rates comparable to international trials. Institutional and patient-level barriers to WW were identified, informing the development of a tailored WW protocol for this unique patient population.

Industry Perspective on First-in-Human and Clinical Pharmacology Strategies to Support Clinical Development of T-Cell Engaging Bispecific Antibodies for Cancer Therapy.

T-cell-engaging bispecific antibodies (TCEs) that target tumor antigens and T cells have shown great promise in treating cancer, particularly in hematological indications. The clinical development of TCEs often involves a lengthy first-in-human (FIH) trial with many dose-escalation cohorts leading up to an early proof of concept (POC), enabling either a no-go decision or dose selection for further clinical development. Multiple factors related to the target, product, disease, and patient population influence the efficacy and safety of TCEs. The intricate mechanism of action limits the translatability of preclinical models to the clinic, thereby posing challenges to streamline clinical development. In addition, unlike traditional chemotherapy, the top dose and recommended phase II doses (RP2Ds) for TCEs in the clinic are often not guided by the maximum tolerated dose (MTD), but rather based on the integrated dose-response assessment of the benefit/risk profile. These uncertainties pose complex challenges for translational and clinical pharmacologists (PK/PD scientists), as well as clinicians, to design an efficient clinical study that guides development. To that end, experts in the field, under the umbrella of the American Association of Pharmaceutical Scientists, have reviewed learnings from published literature and currently marketed products to share perspectives on the FIH and clinical pharmacology strategies to support early clinical development of TCEs.

External ear malformations and cardiac and renal anomalies: A systematic review and meta-analysis

Context External Ear Malformations (EEM) continue to be a common malformation seen in the pediatric patient population. This study aims to further elucidate the correlation between EEM and cardiac and renal anomalies. Objective A systematic review and meta-analysis to study the incidence of cardiac and renal anomalies associated with syndromic and isolated (EEM). Data sources The literature search spanned multiple databases, including Google Scholar, PubMed, Scopus, Web of Science, and MEDLINE. Study selection Studies must be focused on EEM and cardiac and/or renal anomalies. Only articles written in English were included. Data extraction General study characteristics, number of EEM patients, number of cardiac and renal anomalies and whether cases were syndromic were extracted from the studies. Results Of 1,058 initial studies, 33 were included for meta-analyses. Mean JBI score for all included studies was 92.06%, indicating acceptable study quality. Interrater reliability was high, with a Cohen kappa score for all studies of 0.94. The resulting pooled prevalence of cardiac abnormalities was 20% [95% CI:13–28%], while renal abnormalities were 13% [95% CI: 7–20%]. The most common anomalies were VSD (3.725%) and renal agenesis (2.04%). The presence of syndrome data across studies was not a significant modifier of prevalence rates. Limitations Primary limitation is due to heterogeneity in individual study methodology and reporting standards. Conclusions These results highlight a higher prevalence of cardiac-related conditions than renal anomalies in patients with both syndromic and non-syndromic EEM in the included studies, underscoring the need for thorough clinical evaluations.

Survey of Medical Access for Inpatients in Center for Cleft Lip and Palate Treatment: Social Media Usage Preferences and Influencing Factors in China

This study investigated the role of social media in the process of cleft lip and palate patients selecting surgeons. Patients utilized social media to obtain information about their health care providers and treatment teams and relied on this information to guide their decisions on where to seek medical care. A retrospective survey was conducted among patients who underwent surgery in our department from December 2020 to May 2023. Through an anonymous questionnaire titled “Factors Influencing the Decision to Choose a Particular Cleft Surgeon,” we explored how patients discovered their primary surgeon via social media and the factors they deemed most important in selecting their surgeon. The questionnaire was completed by patients or their guardians. The cleft lip and palate patient population exhibited a distinct geographical distribution. A total of 548 individuals participated in the survey on media usage. Search engines (Baidu) were the most frequently visited (30.5%), followed by short video platforms (TikTok and Kwai), accounting for 21.7%, the hospital’s official website at 20.8%, and WeChat at 15%. The primary factors influencing the selection of surgeons were the surgeon’s reputation (66.2%), the hospital/medical team’s reputation (49.2%), the quality of communication during consultations (15.4%), and the surgical outcomes depicted in postoperative photos (6.9%). The Internet remains the primary source of health information. Therefore, it is crucial to not only provide accurate medical information but also effectively communicate complex medical knowledge through easily accessible media channels in a manner that is comprehensible to patients.

Barriers to implementation of rapid identification and antimicrobial susceptibility testing technologies in the clinical microbiology laboratory: an American perspective

Abstract Clinical microbiology laboratories are responsible for confirming the aetiology of infectious diseases and providing antimicrobial susceptibility testing results. Traditional culture-based testing can be augmented by more rapid testing modalities to provide clinically actionable information as quickly as possible. Despite improvements in patient outcomes, many clinical microbiology laboratories are facing challenges to in-sourcing these technologies. Depending on a multitude of factors, including size, location and patient population served, these barriers may affect some laboratories and hospital systems to greater or lesser extents than others. It will be up to each individual facility to ascertain its ability to overcome barriers. To aid in this self-assessment, we present for thoughtful consideration a discussion of the barriers to implementation of rapid identification and antimicrobial susceptibility testing technologies, with specific attention to matters of financial cost, staff expertise, operational issues and stakeholder buy-in.

Risk Stratification in Nonischemic Dilated Cardiomyopathy Using CMR Imaging

ImportanceAccurate risk stratification of nonischemic dilated cardiomyopathy (NIDCM) remains challenging.ObjectiveTo evaluate the association of cardiac magnetic resonance (CMR) imaging–derived measurements with clinical outcomes in NIDCM.Data SourcesMEDLINE, Embase, Cochrane Library, and Web of Science Core Collection databases were systematically searched for articles from January 2005 to April 2023.Study SelectionProspective and retrospective nonrandomized diagnostic studies reporting on the association between CMR imaging–derived measurements and adverse clinical outcomes in NIDCM were deemed eligible.Data Extraction and SynthesisPrespecified items related to patient population, CMR imaging measurements, and clinical outcomes were extracted at the study level by 2 independent reviewers. Random-effects models were fitted using restricted maximum likelihood estimation and the method of Hartung, Knapp, Sidik, and Jonkman.Main Outcomes and MeasuresAll-cause mortality, cardiovascular mortality, arrhythmic events, heart failure events, and major adverse cardiac events (MACE).ResultsA total of 103 studies including 29 687 patients with NIDCM were analyzed. Late gadolinium enhancement (LGE) presence and extent (per 1%) were associated with higher all-cause mortality (hazard ratio [HR], 1.81 [95% CI, 1.60-2.04]; P < .001 and HR, 1.07 [95% CI, 1.02-1.12]; P = .02, respectively), cardiovascular mortality (HR, 2.43 [95% CI, 2.13-2.78]; P < .001 and HR, 1.15 [95% CI, 1.07-1.24]; P = .01), arrhythmic events (HR, 2.69 [95% CI, 2.20-3.30]; P < .001 and HR, 1.07 [95% CI, 1.03-1.12]; P = .004) and heart failure events (HR, 1.98 [95% CI, 1.73-2.27]; P < .001 and HR, 1.06 [95% CI, 1.01-1.10]; P = .02). Left ventricular ejection fraction (LVEF) (per 1%) was not associated with all-cause mortality (HR, 0.99 [95% CI, 0.97-1.02]; P = .47), cardiovascular mortality (HR, 0.97 [95% CI, 0.94-1.00]; P = .05), or arrhythmic outcomes (HR, 0.99 [95% CI, 0.97-1.01]; P = .34). Lower risks for heart failure events (HR, 0.97 [95% CI, 0.95-0.98]; P = .002) and MACE (HR, 0.98 [95% CI, 0.96-0.99]; P < .001) were observed with higher LVEF. Higher native T1 relaxation times (per 10 ms) were associated with arrhythmic events (HR, 1.07 [95% CI, 1.01-1.14]; P = .04) and MACE (HR, 1.06 [95% CI, 1.01-1.11]; P = .03). Global longitudinal strain (GLS) (per 1%) was not associated with heart failure events (HR, 1.06 [95% CI, 0.95-1.18]; P = .15) or MACE (HR, 1.03 [95% CI, 0.94-1.14]; P = .43). Limited data precluded definitive analysis for native T1 relaxation times, GLS, and extracellular volume fraction (ECV) with respect to mortality outcomes.ConclusionThe presence and extent of LGE were associated with various adverse clinical outcomes, whereas LVEF was not significantly associated with mortality and arrhythmic end points in NIDCM. Risk stratification using native T1 relaxation times, extracellular volume fraction, and global longitudinal strain requires further evaluation.

A Cost-Utility Analysis of Ferric Derisomaltose Versus Ferric Carboxymaltose in Patients with Iron Deficiency Anemia in China.

Intravenous (IV) iron is the recommended treatment for patients with iron deficiency anemia (IDA) unresponsive to oral iron treatment, in whom oral iron is contraindicated, or where rapid iron replenishment is required. Ferric derisomaltose (FDI) and ferric carboxymaltose (FCM) are high-dose, rapid-infusion, IV iron formulations that have recently been compared in three head-to-head randomized controlled trials (RCTs), which showed significantly higher incidence of hypophosphatemia after administration of FCM than FDI. The present study objective was to evaluate the cost-utility of FDI versus FCM in a population of patients with IDAin China. A previously-published patient-level simulation model was used to model the cost-utility of FDI versus FCM in China. The number of infusions of FDI and FCM was modeled based on the approved posology of the respective formulations using simplified tables of iron need in a population of patients with body weight and hemoglobin levels informed by a Chinese RCT of FCM. Data on the incidence of hypophosphatemia was obtained from the PHOSPHARE-IDA RCT, while data on disease-related quality of life were obtained from SF-36v2 data from the PHOSPHARE-IBD RCT. Over the 5-year time horizon, patients received 3.98 courses of iron treatment on average, requiring 0.90 fewer infusions of FDI than FCM (7.69 vs. 6.79). This resulted in iron procurement and administration cost savings of renminbi (RMB) 206 with FDI (RMB 3,519 vs. RMB 3,312). Reduced incidence of hypophosphatemia-related fatigue resulted in an increase of 0.07 quality-adjusted life years and further cost savings of RMB 782 over 5years, driven by reduced need for phosphate testing and replenishment. FDI was therefore the dominant intervention. The results showed that FDI would improve patient quality of life and reduce direct healthcare expenditure versus FCM in patients with IDA in China.