Optimal Clinical Practice Research Articles

Asia-Pacific consensus recommendations on the management of generalized pustular psoriasis.

Generalized pustular psoriasis (GPP) is a rare, chronic, heterogeneous, and potentially life-threatening disease characterized by primary, sterile, and macroscopically visible pustules with or without systemic symptoms. There are ethnic differences in the genetic mutations associated with GPP that might affect the clinical manifestations and treatment responses. Currently, there is limited evidence from the patient population in the Asia-Pacific (APAC) region, resulting in a general paucity of information on the effective management of patients with GPP in this region. This modified Delphi panel study aimed to identify current evidence and gain advanced insights to facilitate the development of a regionally tailored APAC consensus on the management of GPP. A systematic literature review (SLR) was conducted to identify published literature and develop consensus statements on (i) definition and clinical course, (ii) diagnosis of GPP, (iii) treatment outcomes, goals, and monitoring measures, and (iv) optimal management strategies and clinical practices. Statements were rated by a panel of dermatologists in two rounds, with the threshold for consensus at ≥80% agreement. Twenty experts from the APAC region reached consensus on 106 statements that were developed based on the SLR and experts' collective expertise. The experts agreed that GPP is a rare, severe, and potentially life-threatening condition that is distinct from plaque psoriasis. This consensus emphasized the importance of a tailored treatment strategy taking into account the GPP flare severity and each patient's unique clinical circumstances. The experts reached consensus on the severity classification of GPP flares and recommended first-line and maintenance treatment options for adult GPP, childhood GPP, and GPP in pregnancy. These consensus outcomes have been synthesized into treatment algorithms to guide dermatologists in the APAC region in their clinical decision-making processes.

Evaluation of control arm quality in recent radiation oncology randomized clinical trials.

295 Background: Randomized controlled trials (RCTs) are designed to provide the highest levels of evidence for clinical practice; consequently, RCTs are the mainstay for creating guidelines and approving new treatments. It is ethically essential for patients assigned to the control arm in RCTs to receive standard-of-care treatment. This serves to protect patients, optimize adequate treatment, and ensures that RCT findings are compared to current standard-of-care therapy – an ethical imperative of RCT design and implementation. Oncologic medical trials investigating new systemic agents for cancer have a high proportion of RCTs with inadequate control arms (PMID 31046071). It is unknown whether this finding is prevalent in oncologic trials investigating radiation therapy (RT) for cancer. Methods: We identified registered clinical trials investigating RT in patients with cancer over the past decade. ClinicalTrials.gov was queried for RT as the intervention and cancer and other related terms as the condition between 1/1/13 and 1/1/23. Exclusion criteria included trials with incomplete status or non-oncologic indications. Trials were categorized by indication/primary disease site, first posted date, investigational arm, control arm, and sponsor/collaborator. Each control arm was analyzed, and the standard of care was determined according to National Comprehensive Cancer Center Network (NCCN) guidelines at the time of first posting on ClinicalTrials.gov. Results: A total of 508 interventional studies with results registered on ClinicalTrials.gov were included. Of these, 360 single-arm studies were excluded. 116 studies investigating a treatment other than RT were excluded. 20 studies that were not completed were excluded. The remaining 12 studies were included in the final analysis. Of the included trials, 2 each investigated RT usage in central nervous system, prostate, head and neck, and breast disease sites, and 1 each for lung, hepatobiliary, rectal, and bone disease sites. A majority of the trials were industry-funded (83%), and over 2/3 of studies took place in the United States (75%). 100% of the trials were found to have an adequate control arm per the corresponding NCCN guidelines. Conclusions: 100% of oncologic RCTs investigating radiation therapy for cancer were found to have an adequate control arm. This finding contrasts with medical oncology trials, which have been shown to have a high proportion of RCTs with inadequate control arms. This disparity highlights the importance of designing oncologic RCTs with adequate control arms, which is crucial for providing the highest level of evidence to guide optimal clinical practice and ensure the safety of patients. These findings indicate that the field of Radiation Oncology is ensuring ethical trial design with adequate control arm treatments and emphasizes the need for a continued focus on improving the quality of ethical oncologic research trials.

Safety and quality of parenteral nutrition: Areas for improvement and future perspectives.

This article is based on presentations and discussions held at the International Safety and Quality of Parenteral Nutrition (PN) Summit (held November 8-10, 2021, at Charleston, SC, and Bad Homburg, Germany) and aims to raise awareness concerning unresolved issues associated with the PN process and potential future directions, including a greater emphasis on patients' perspectives and the role of patient support. Ensuring that every patient in need receives adequate PN support remains challenging. It is important to have a standardized approach to identify nutritional risk and requirements using validated nutritional screening and assessment tools. Gaps between optimal and actual clinical practices need to be identified and closed, and responsibilities in the nutrition support team clarified. Use of modern technology opens up opportunities to decrease workloads or liberate resources, allowing a more personalized care approach. Patient-centered care has gained in importance and is an emerging topic within clinical nutrition, in part because patients often have different priorities and concerns than healthcare professionals. Regular assessment of health-related quality of life, functional outcomes, and/or overall patient well-being should all be performed for PN patients. This will generate patient-centric data, which should be integrated into care plans. Finally, communication and patient education are prerequisites for patients' commitment to health and for fostering adherence to PN regimes. Moving closer to optimal nutritional care requires input from healthcare professionals and patients. Patient-centered care and greater emphasis on patient perspectives and priorities within clinical nutrition are essential to help further improve clinical nutrition.

Identifying ideal pharmacist‐to‐patient ratios for the successful provision of clinical pharmacy services

AbstractClinical pharmacists add significant value to the health care team and are associated with reduced hospital mortality, drug costs, length of stay, and adverse drug reactions. However, there remains a paucity of evaluative data regarding optimal clinical pharmacy practice models. Identifying optimal patient (or bed)‐to–clinical pharmacist ratios is one mechanism to assess the allocation of resources. The ACCP Task Force on Clinical Pharmacy Services was charged with developing a white paper to examine optimal ratios in different care settings, considering confounders and other factors that affect the interpretation of ratio data. The committee performed an extensive search of the pharmacy, medical, and nursing literature to identify existing data on provider‐to‐patient ratios. Recommendations on ratios are mainly from survey data or statements from various health care organizations. Many factors affect the capacity of clinical pharmacists to achieve patient care and non‐patient care responsibilities. Examples include general differences in baseline expectations; nonstandardized program performance metrics; variable hospital/practice setting types; variable patient acuity and complexity; competing priorities and other resources; industry‐wide rates of and variability in addressing burnout; inconsistent means for justifying numbers and historical corporate structures; and external endorsements and mandates. Although some areas of practice such as critical care pharmacy have more robust evidence for specific ratios, others such as pediatrics are far less clear. A clinical pharmacist ratio must be established as a starting point. This will allow for better allocation today and serve as a springboard for future determinations. Furthermore, clinical pharmacist ratios should be preserved to offer reliable patient care 365 days per year. As clinical pharmacist services shift from beneficial to essential, continuous daily patient care services must be the practice model moving forward.

Awareness on antibiotic prescription for endodontic cases among Dentists and Endodontists in Saudi Arabia

The knowledge of Dentists and Endodontists in Saudi Arabia regarding antibiotic prescriptions during and after endodontic treatment is of interest. A self-designed questionnaire survey was utilized to assess the knowledge of dentists across various cities in Saudi Arabia concerning antibiotic usage guidelines for endodontic purposes. A total of 391 participants were included in the study, and the questionnaire was distributed through social platforms like WhatsApp, Instagram, Facebook Messenger, and email. Results revealed that over 80% of participants acknowledged the need for antibiotics in cases of acute apical abscess with cellulitis, with amoxicillin being the most recommended antibiotic by dentists. Interestingly, there was no statistically significant difference in knowledge based on experience or graduation group. In conclusion, while participants demonstrated adequate knowledge about antibiotic prescriptions in endodontic cases, continued awareness of updated guidelines, including the WHO's Essential Medicines List (EML), guidelines by the European Society of Endodontology (ESE) and American Association Endodontists (AAE) is essential for optimal clinical practice.

Accessibility, availability and common practices regarding genetic testing for epilepsy across Europe: A survey of the European Reference Network EpiCARE.

The increasingly rapid pace of advancement in genetic testing may lead to inequalities in technical and human resources with a negative impact on optimal epilepsy clinical practice. In this view, the European Reference Network (ERN) for Rare and Complex Epilepsies EpiCARE conducted a survey addressing several aspects of accessibility, availability, costs, and standard practices on genetic testing across ERN EpiCARE centers. An online Google form was sent to 70 representatives of ERN EpiCARE centers. Descriptive statistics and qualitative analysis were used for data presentation. We received 45 responses (1/center) representing 23 European countries with a better representation of Western Europe. Forty-five percent of the centers did not have access to all available types of genetic testing, mainly reflecting the limited availability of whole-genome sequencing (WGS). Thirty-five percent of centers report cost coverage only for some of the available tests, while costs per test varied significantly (interquartile range IQR ranging from 150 to 1173 euros per test across centers). Urgent genetic testing is available in 71.7% of countries (time-to-urgent result: 2 day to 2 months). The average time-to-result of specific tests in case of non-urgent prescription has a significant variance across centers, with the biggest range observed for whole-exome sequencing (6-128 weeks, IQR: 27 weeks). The percentage of agreement among the experts regarding the choice of genetic test at first intention in specific clinical situations was in all cases less than 50 percent (34.9% to 47% according to the proposed scenarios). Costs, time to deliver the results to the clinician, and type of first-line genetic testing vary widely across Europe, even in countries where ERN EpiCARE centers are present. Increased availability of genetic tests and guidance for optimal test choices in epilepsy remains essential to avoid diagnostic delays and excess health costs. The survey of the ERN EpiCARE highlights disparities in genetic testing for epilepsy across 45 ERN EpiCARE centers in 23 European countries. The findings reveal variable access to certain genetic tests, with lowest access to WGS. Costs and time-to-results vary widely. Urgent genetic testing is available in 71.7% of countries. Agreement among experts on first-line genetic tests for specific patient scenarios is below 50%. The study emphasizes the need for improved test availability and guidance to avoid diagnostic delays and unnecessary costs. EpiCARE has the mission to contribute in homogenizing best practices across Europe.

Evaluating the therapeutic and reconstructive efficacy of flap transplantation techniques in managing nasal tissue deficiency resulting from post-rhinoplasty surgical infections.

Rhinoplasty is a frequently performed aesthetic surgery with a high procedural volume. Despite the operational ease and safety profile of biocompatible materials such as silicone and inflatable prosthetics, postoperative complications like surgical site infections can lead to significant clinical challenges. These complications necessitate effective therapeutic and reconstructive interventions. This study aims to evaluate the efficacy of different flap transplantation techniques for early treatment of post-rhinoplasty nasal tissue deficiencies. A retrospective study was conducted from May 2018 to May 2023, involving 38 patients divided into a control group receiving standard anti-infective treatment and an observation group undergoing early flap grafting procedures. Efficacy was evaluated using objective metrics-namely, nasal appearance, function and flap condition-each assessed through a 10-point scoring system, and subjective metrics such as patient satisfaction gauged through self-administered questionnaires. Statistical analyses were performed using SPSS version 27.0. Both groups were statistically comparable in terms of demographics such as age, marital status and educational background. The observation group displayed significantly improved outcomes in terms of nasal aesthetics (mean score 7.92 ± 1.00), nasal function (mean score 8.47 ± 0.51) and flap condition (mean score 7.89 ± 1.12) compared to the control group. Flap transplantation techniques demonstrated superior therapeutic and reconstructive efficacy in the management of nasal tissue deficiencies arising from post-rhinoplasty surgical infections. These findings contribute to evidence-based recommendations for optimal clinical practice.

The Promotoer, a brain-computer interface-assisted intervention to promote upper limb functional motor recovery after stroke: a statistical analysis plan for a randomized controlled trial

BackgroundElectroencephalography (EEG)-based brain-computer interfaces (BCIs) allow to modulate the sensorimotor rhythms and are emerging technologies for promoting post-stroke motor function recovery. The Promotoer study aims to assess the short and long-term efficacy of the Promotoer system, an EEG-based BCI assisting motor imagery (MI) practice, in enhancing post-stroke functional hand motor recovery. This paper details the statistical analysis plan of the Promotoer study.MethodsThe Promotoer study is a randomized, controlled, assessor-blinded, single-centre, superiority trial, with two parallel groups and a 1:1 allocation ratio. Subacute stroke patients are randomized to EEG-based BCI-assisted MI training or to MI training alone (i.e. no BCI). An internal pilot study for sample size re-assessment is planned. The primary outcome is the effectiveness of the Upper Extremity Fugl-Meyer Assessment (UE-FMA) score. Secondary outcomes include clinical, functional, and user experience scores assessed at the end of intervention and at follow-up. Neurophysiological assessments are also planned. Effectiveness formulas have been specified, and intention-to-treat and per-protocol populations have been defined. Statistical methods for comparisons of groups and for development of a predictive score of significant improvement are described. Explorative subgroup analyses and methodology to handle missing data are considered.DiscussionThe Promotoer study will provide robust evidence for the short/long-term efficacy of the Promotoer system in subacute stroke patients undergoing a rehabilitation program. Moreover, the development of a predictive score of response will allow transferring of the Promotoer system to optimal clinical practice. By carefully describing the statistical principles and procedures, the statistical analysis plan provides transparency in the analysis of data.Trial registrationClinicalTrials.gov NCT04353297. Registered on April 15, 2020.

ChatGPT's Epoch in Rheumatological Diagnostics: A Critical Assessment in the Context of Sjögren's Syndrome.

The rise of artificial intelligence in medical practice is reshaping clinical care. Large language models (LLMs) like ChatGPT have the potential to assist in rheumatology by personalizing scientific information retrieval, particularly in the context of Sjögren's Syndrome. This study aimed to evaluate the efficacy of ChatGPT in providing insights into Sjögren's Syndrome, differentiating it from other rheumatological conditions. A database of peer-reviewed articles and clinical guidelines focused on Sjögren's Syndrome was compiled. Clinically relevant questions were presented to ChatGPT, with responses assessed for accuracy, relevance, and comprehensiveness. Techniques such as blinding, random control queries, and temporal analysis ensured unbiased evaluation. ChatGPT's responses were also assessed using the 15-questionnaire DISCERN tool. ChatGPT effectively highlighted key immunopathological and histopathological characteristics of Sjögren's Syndrome, though some crucial data and citation inconsistencies were noted. For a given clinical vignette, ChatGPT correctly identified potential etiological considerations with Sjögren's Syndrome being prominent. LLMs like ChatGPT offer rapid access to vast amounts of data, beneficial for both patients and providers. While it democratizes information, limitations like potential oversimplification and reference inaccuracies were observed. The balance between LLM insights and clinical judgment, as well as continuous model refinement, is crucial. LLMs like ChatGPT offer significant potential in rheumatology, providing swift and broad medical insights. However, a cautious approach is vital, ensuring rigorous training and ethical application for optimal patient care and clinical practice.

What makes a good clinical practice experience in radiography and sonography? An exploration of qualified clinical staff and student perceptions

IntroductionClinical practice can be a challenging experience for some healthcare students. The quality of the clinical experience can have a profound effect on the overall student learning experience in clinical practice and attrition. The aim of the study was to explore the perceptions of an optimal clinical practice experience by radiography and sonography students and qualified clinical staff. MethodsA qualitative research design was adopted utilising focus groups (n = 5). The study population comprised of qualified radiography/sonography staff (n = 10) from across a number of placement sites used by City, University of London and radiography and sonography students (n = 15) from the same institution. Full verbatim transcriptions were analysed thematically. ResultsFour key themes emerged: 1) favourable/unfavourable traits, 2) creating an optimal learning environment 3) challenges and 4) considerations for clinical education. Key factors for a positive learning experience included clinical supervisors being approachable, whilst encouraging and empowering students. Qualified radiography/sonography clinical staff highlighted student motivation as an important aspect for successful placement learning. ConclusionThe study has provided an insight into a number of positive attributes that enhance student learning experiences whilst on clinical placement. Unhelpful attributes and challenges, such as student motivation and time limitations, were also revealed. It is important that supervisors within the clinical departments actively work towards the provision of a positive learning experience. Implications for practiceThis study can better inform clinical staff regarding the importance of enhancing the student learning experience and facilitating high quality learning within the clinical department. Moreover, to encourage clinical staff to ensure robust “support” is established for students on placement.

Clinical applications of next-generation sequencing in the diagnosis of genetic disorders in Korea: a narrative review

Background: Next-generation sequencing (NGS) technologies have revolutionized genetic testing and enabled efficient screening of various genetic conditions in clinical settings. However, the clinical application of genetic test results presents numerous significant challenges. This review aims to provide a comprehensive overview of key concepts for the clinical application of NGS, including (1) technical aspects and limitations, (2) variant classification, (3) clinical interpretation, (4) familial testing and genetic counseling, and (5) ethical considerations.Current Concepts: In short-read-based NGS, several limitations exist in detecting genomic variations, including repetitive sequences or complex structural variations. The variant classification process can be influenced by suspected genomic conditions and the accessibility of genomic databases. Therefore, the final genetic diagnosis depends on the physician’s discretion, which relies on the genotype-phenotype correlations and reverse phenotyping through additional evaluations. Familial testing can help trace the origin of variants and allele segregation and aid in variant interpretation, risk assessment, disease prevention, and family planning. In addition to addressing the clinical impact of genetic results, genetic counselors should also consider potential consequences related to ethical, legal, and social issues, including family dynamics.Discussion and Conclusion: NGS-based genetic testing is a promising diagnostic tool for genetic disorders, but proper variant interpretation and clinical evaluation are crucial for optimal clinical practice. Notably, ethical considerations and regulatory measures are required to prepare for the next era of genomic medicine.

Establishing a prognostic model based on immune-related genes and identification of BIRC5 as a potential biomarker for lung adenocarcinoma patients

BackgroundLung adenocarcinoma (LUAD) is an extraordinarily malignant tumor, with rapidly increasing morbidity and poor prognosis. Immunotherapy has emerged as a hopeful therapeutic modality for lung adenocarcinoma. Furthermore, a prognostic model (based on immune genes) can fulfill the purpose of early diagnosis and accurate prognostic prediction.MethodsImmune-related mRNAs (IRmRNAs) were utilized to construct a prognostic model that sorted patients into high- and low-risk groups. Then, the prediction efficacy of our model was evaluated using a nomogram. The differences in overall survival (OS), the tumor mutation landscape, and the tumor microenvironment were further explored between different risk groups. In addition, the immune genes comprising the prognostic model were subjected to single-cell RNA sequencing to investigate the expression of these immune genes in different cells. Finally, the functions of BIRC5 were validated through in vitro experiments.ResultsPatients in different risk groups exhibited sharply significant variations in OS, pathway activity, immune cell infiltration, mutation patterns, and immune response. Single-cell RNA sequencing revealed that the expression level of BIRC5 was significantly high in T cells. Cell experiments further revealed that BIRC5 knockdown markedly reduced LUAD cell proliferation.ConclusionThis model can function as an instrumental variable in the prognostic, molecular, and therapeutic prediction of LUAD, shedding new light on the optimal clinical practice guidelines for LUAD patients.

Interferon-beta exposure in-utero and the risk of infections in early childhood.

Knowledge within the field of multiple sclerosis treatment during pregnancy is vital to ensure the most optimal clinical practice. Immunomodulatory treatment in pregnancy could in theory affect the normal development and maturation of the immune system of the fetus with a potential increased risk of infections, consequently. We therefore set out to investigate whether exposure to interferon-beta in utero affected the risk of acquiring infections in early childhood. This retrospective matched cohort study utilized data from the Danish Multiple Sclerosis Registry linked with national Danish registries to identify all children born of mothers with MS in Denmark from 1998 to 2018. The study included 510 children exposed to interferon-beta in utero. The children were matched 1:1 on various of demographic characteristics with children born to mothers with untreated MS and 1:3 with children born to mothers without MS. Each child was followed for up to five years. Using individual-level data, we investigated all-cause mortality, rate of hospital admissions due to infections, and redeemed prescriptions of antibiotics. The primary statistical model applied was a negative binomial regression analysis. We found no differences in childhood mortality, for hospital admissions the rate ratio compared to healthy controls was 0.79 (0.62-1.00). Regarding antibiotic prescriptions, the results were similar (RR 1.00 (0.90-1.11). Furthermore, we found no certain dose-response relationship between interferon-beta exposure duration and hospital admission rate (P=0.47) or redeemed antibiotic prescription (P=0.71). Exposure to interferon-beta during gestation has little to no impact on the risk of acquiring significant infections during the first five years of childhood.

Palliative short-course radiotherapy (RAPASH study) in patients with rectal cancer.

Palliative radiation therapy (RT) is used to treat symptomatic rectal cancer although clinical benefits and toxicities are poorly documented. There is no consensus about the optimal RT regimen and clinical practice undergoes significant changes. Our aim was to evaluate the efficacy and toxicity of short-course (SC) RT in this setting of patients. Charts from patients with locally advanced disease not candidates for standard treatment or with symptomatic metastatic rectal cancer treated with SCRT (25 Gy/5 fractions in 5 consecutive days) were retrospectively reviewed. Clinical outcome measures were symptomatic response rate and toxicity. From January 2007 to December 2017, 59 patients (median age 80 years) received SCRT; 53 were evaluable. The median follow-up was 8 months (range, 1-70). Clinical response to RT for bleeding, pain and tenesmus was 100%, 95% and 89%, respectively. The compliance with the treatment was 100% and no patient experienced acute severe (≥ grade 3) toxicities. Median time to symptoms recurrence was 11 months (range 3-69). Globally, the median overall survival was 12 months. SCRT is a safe and effective regimen in symptomatic rectal cancer and may be considered the regimen of choice for standard treatment in unfit patients.

#3074 CHANGES IN THE CLINICAL CHARACTERISTICS AND MANAGEMENT OF PATIENTS WITH AHUS OVER 10 YEARS: TRENDS FROM THE GLOBAL AHUS REGISTRY

Abstract Background and Aims Atypical haemolytic uraemic syndrome (aHUS) is a rare disease predominantly caused by alternative complement pathway dysregulation. Prior to 2011, before the targeted complement inhibitor eculizumab became available, aHUS frequently led to end-stage kidney disease (ESKD) and early death. Treatment with eculizumab led to notable improvements in outcomes; whether patient characteristics and management has continued to change over time is unknown. Using data from the Global aHUS Registry, we assessed clinical characteristics and management of patients with aHUS over 10 years to identify any potential trends. Method All patients enrolled in the Global aHUS Registry from 2012–2022 were included. Patients were categorised according to age at aHUS onset (adult [≥18 years] vs paediatric [<18 years]); onset of aHUS was defined as the earliest of initial symptom presentation, aHUS diagnosis, or first recorded thrombotic microangiopathy (TMA). Patient characteristics were summarised using descriptive analysis. Results Of the 1994 patients enrolled in the registry between 2012 and 2022, 33 (1.7%) were missing data on age at aHUS onset. Of the remaining 1961 patients, 813 (41.5%) were paediatric and 1148 (58.5%) were adult. Changes in patient characteristics and management are presented in Fig. 1. Plasma exchange/plasma infusion (PE/PI) prior to and including the year of enrolment was less common in paediatric than adult patients (58.5% vs. 71.3%) between 2012–2013 and declined substantially in paediatric (19.6%) relative to adult (55.1%) patients by 2020–2022. Similar proportions of paediatric patients required dialysis at the time of enrolment between 2012–2013 (5.1%) and 2020–2022 (5.9%), while numerically lower proportions of adults required dialysis over time (2012–2013, 15.2%; 2020–2022, 6.5%). The rate of paediatric patients requiring a kidney transplant prior and up to enrolment dropped from 2012–2013 (28.4%) to 2020–2022 (5.9%); however, the rate among adult patients remained comparable (2012–2013, 29.2%; 2020–2022, 27.1%). A decrease in the proportion of patients with a reported identified pathogenic variant and/or anti-CFH antibodies was observed in paediatric and adult patients between 2012–2013 (paediatric, 59.1%; adult, 45.5%) and 2020–2022 (paediatric, 49.0%; adult, 30.8%). The proportion of patients with a reported triggering event increased from 2012–2013 (paediatric, 6.3%; adult, 12.4%) to 2020–2022 (paediatric, 9.8%; adult, 28.0%). In this cohort, a total of 1208 patients were treated with eculizumab/ravulizumab between 2012 and 2022 (paediatrics, 476; adults, 732). Between 2012–2013, the median time from aHUS onset to treatment initiation was 94.9 days for paediatric patients (blue) and 51.1 days for adult patients (orange); this fell to 13.1 days in paediatrics and 24.1 days in adults between 2020–2022 (Fig. 2). Conclusion Utilisation of PE/PI to treat aHUS has decreased more in paediatric than adult patients over time. A decreased requirement for transplant in paediatric patients was evident, suggesting more paediatrics are now being diagnosed and treated earlier, leading to better outcomes with fewer patients progressing to renal failure and requiring kidney transplant. The proportion of both paediatric and adult patients with reported pathogenic variants and/or anti-CFH antibodies decreased over time and may reflect the rate at which a clinical constellation consistent with aHUS is identified. Moreover, aHUS may be recognised more frequently in the presence of a triggering condition, or a lower barrier for clinicians to suspect aHUS in recent years. Patients were more rapidly treated with a complement inhibitor, likely due to better awareness in the clinical community around complement inhibitors and the importance of early treatment initiation. Although patients with aHUS are being treated more promptly in recent years than 10 years ago, more work is needed, especially in adults, to move towards optimal clinical practice.

Update on Familial Adenomatous Polyposis-Associated Desmoid Tumors.

Desmoid tumors (DT) represent the second high risk of tumor in familial adenomatous polyposis (FAP) patients. Although FAP-associated DTs (FAP-DT) are caused by germline mutations in the adenomatous polyposis coli (APC) gene, extracolonic manifestations, sex, family history, genotype, and the ileal pouch anal anastomosis procedure are all linked to the development of DTs in FAP patients. Multidisciplinary management has replaced aggressive surgery as the preferred treatment of DTs. There is growing evidence to support the use of active surveillance strategy as first-line treatment for FAP-DT patients. Radiotherapy for intra-abdominal desmoids is now rarely used because of severe late toxicity. Pharmacotherapy, however, represents a promising future with the improvement of traditional cytotoxic drugs and the investigation of targeted drugs. Although nonsurgery treatment has been used widely nowadays, surgery remains the mainstay when symptomatic or life-threatening DTs are present. Further research will be needed for more optimal clinical practice.

High-risk HPV testing improves accuracy in detection of CIN2+ lesions in ASC-H postmenopausal women? An academic hospital experiences.

Reflex human papilloma virus (HPV) testing with "atypical squamous cells, cannot exclude high-grade squamous lesion (ASC-H)" cytologic diagnosis is not recommended by American Society for Colposcopy and Cervical Pathology guidelines. Studies have shown human papillomavirus (HPV)-negative ASC-H patients of increased age are low risk for cervical intraepithelial neoplasia 2 or worse (CIN2+) lesions on colposcopic follow-up. We retrospectively assessed the efficacy of reflex HPV testing in postmenopausal women with ASC-H in the Los Angeles County hospitals and clinics in a 5-year period. Of a total 85 clinically postmenopausal women with ASC-H, 31 (36.5%) women were found to have CIN2+ lesions on follow-up biopsy and five of them were HPV-negative. Of the women with CIN2+ lesions and positive HPV, 13 (41.9%) were high-risk HPV (hrHPV) 16/18/45 positive and 13 (41.9%) were hrHPV-other subtype positive. Women with positive HPV had an over 3-fold increased risk of developing CIN2+ lesions (P= 0.008). Relative risk of hrHPV16/18/45 was 1.79-fold higher than that of hrHPV-other subtype. The positive predictive value and negative predictive value of hrHPV were 49.1% and 84.4%, respectively. CIN2+ detection rate in Hispanic women with positive hrHPV was higher than in non-Hispanic women (53.8% versus 35.7%). Overall, postmenopausal women with ASC-H cytology result and negative hrHPV were less likely to develop CIN2+ lesions, whereas about half of ASC-H postmenopausal women develop CIN2+ lesions if hrHPV positive, especially if hrHPV 16/18/45 positive. Therefore, triaging ASC-H postmenopausal women with cotesting or, ideally, hrHPV genotyping should be considered as optimal clinical practice to avoid overtreatment.

The story of retinal polarization scanning—from inspiration to commercialization

As pediatric ophthalmologists, we recognize pain points and limitations to optimal clinical practice with virtually every clinical encounter. These limitations inspire many of us to imagine innovative devices, programs, or protocols that might address one or more clinical challenges. But the path to commercialization of any novel idea travels through a haunted house of unexpected barriers, through a junkyard of failures, and through a seemingly endless string of heartbreak hills.

Valuing the Partnership Between the Veterans Health Administration and Academic Medicine.

Valuing the Partnership Between the Veterans Health Administration and Academic Medicine.

‘I had nothing. It’s just life experience that helped me through that situation’: Australian audiologists’ perspectives on audiological clinical practice for traumatic brain injury and rehabilitation

ABSTRACT Objective Australian Audiologists’ perspectives on standard non-specialized clinical practice in the context of Traumatic Brain Injury (TBI) were examined, including the perceived barriers to optimal service provision. Design A qualitative research design utilizing semi-structured interviews was conducted using purposive sampling. Nine participants were interviewed about their understanding of the impact of TBI on hearing and balance; identification, diagnosis and management of auditory and vestibular dysfunction following TBI; barriers to service delivery; training relating to complex clients (i.e., TBI); and awareness of referral pathways. Results Three major themes, each with subthemes, were evident in the data. The major themes reflected general considerations of audiological professional culture and specific issues related to knowledge of TBI and clinical practice with patients. Analysis revealed that professional culture seemed to act as a contextual barrier and interacted with the perceived lack of TBI related knowledge to hinder optimal clinical practice in this patient population. Conclusion Application of the biopsychosocial model, including interdisciplinary care in the management of patients with TBI, is needed. An improvement in theoretical and practical knowledge encompassing the wide-ranging effects of TBI is critical for the optimal audiological service delivery.