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Leigh Ramos-Platt,1 Lauren Elman,2 Perry B Shieh3 1Department of Pediatrics, Keck School of Medicine, University of Southern California and Children’s Hospital of Los Angeles, Los Angeles, CA, USA; 2Department of Neurology, University of Pennsylvania, Philadelphia, PA, USA; 3Department of Neurology and Pediatrics, University of California Los Angeles, Los Angeles, CA, USACorrespondence: Perry B Shieh, Department of Neurology and Pediatrics, University of California Los Angeles, Los Angeles, CA, USA, Email pshieh@mednet.ucla.eduAbstract: Spinal muscular atrophy (SMA) is a rare, progressive neuromuscular disorder that, until recently, was the most common inherited cause of infant mortality. Since 2016, three disease-modifying therapies have emerged, nusinersen, onasemnogene abeparvovec-xioi, and risdiplam, leading to a transformation in the SMA treatment landscape, changes in disease trajectories, and a profound impact on clinical care. This environment poses a challenge to making informed treatment decisions, including initial treatment choice, treatment changes, and potential use of combination therapies as new data emerge. To better understand factors that influence physician-patient decision-making, a roundtable discussion was convened by Biogen (sponsor) with a panel of four US SMA experts. This report shares the panel’s opinions and clinical experiences, with the goals of helping clinicians and people with SMA and their families to better understand the factors influencing real-world treatment decisions and stimulating a broader discussion in the SMA community. The panelists highlighted that patients are often heavily involved in treatment decisions, and physicians must be aware of current data to guide patients in making the best decisions. Thus, in the absence of data from head-to-head treatment comparisons, physicians’ roles include reviewing treatment options and describing what is known of the benefits, challenges, and potential side effects of each therapy with patients and families. For infants and young children, the panelists expressed a sense of urgency for early intervention to minimize motor function loss, whereas the goal for adults is long-term disease stabilization. In the panelists’ experience, factors that influence patients’ decisions to change to an alternative therapy include convenience, administration route, novelty of therapy, and hope for improved function, while reasons for returning to a previous therapy include a perception of decreased efficacy and side effects. Ongoing clinical trials and analyses of real-world experiences should further inform treatment decisions and optimize patient outcomes.Graphical Abstract: Keywords: nusinersen, onasemnogene abeparvovec-xioi, risdiplam, spinal muscular atrophy

This research aimed to use a computational approach to aid infection prevention strategies during the predesign phase of healthcare environment planning. The objective of this study was to decompose and analyze health system catchment area 'outside design basis factors' such as demographic, public health, and environmental characteristics that may contribute to infection causing pathogen spread in healthcare environments using state of Florida data as a context for analysis. Supervised Machine Learning methodologies were used to determine relationships between Socioecological System (SES) factors and observed incidence rates of specific Hospital Acquired Infections (HAI). Results of the analysis suggest that relevant regional population SES factors, when included as independent variables, can forecast the potential co-occurrence of specific types of HAI in regional health system service areas. Outcomes of the analysis suggest that demographic and regional characteristics are significantly related to the predictive incidence of Antimicrobial-resistant infections in healthcare settings in Florida Agency for Healthcare Administration (ACHA) regions. The outcomes of this research suggest the benefit of using SES computational modeling for decision-making in health system Architectural Programming and predesign. This process allows for manifesting meaningful patterns by applying quantitative methods to analyze patient population characteristics that may influence the prevalence of pathogen spread within the interior environments of healthcare inpatient settings. This information contributes to a deeper understanding of context-specific priorities for operationalizing human-centered Infection Prevention through Design (PtD) infrastructure and planning. [ABSTRACT FROM AUTHOR]

Although we are unaware of the existence of a recognized fetal MRI qualification, individuals who perform fetal MRI should have undergone specialized training in collaboration with a teaching center, enabling them to perform a state-of-the-art fetal MRI examination after exposure to a sufficient number of cases. Introduction Fetal magnetic resonance imaging (MRI) is an important diagnostic imaging adjunct to ultrasonography[1], especially for evaluation of the fetal brain, lungs and bowel[2] and the placenta[3]. Factors influencing the decision to perform fetal MRI include, but are not limited to: experience/equipment of the ultrasound and MRI facilities, accessibility to MRI, maternal conditions such as obesity, abdominal scarring and oligohydramnios, gestational age, safety concerns, legal considerations regarding termination of pregnancy (TOP) and parental wishes after appropriate counseling[8]. [Extracted from the article]

Objective: Tracheostomy in the neurocritical care population is associated with poorer outcomes. This study hypothesised that a multidisciplinary approach to tracheostomy care can improve outcomes. Methods: This study was a prospective longitudinal study of all tracheostomised patients in the neurocritical care units of a quaternary centre over 17 years. All patients were managed by a tracheostomy team with a constant core membership of an intensive care consultant, speech and language therapist, and physiotherapist with consultant ENT input. Results: A total of 51 per cent of patients were decannulated in hospital at an average of 48 (neuromedical) and 57.6 (neurosurgical) days. Of the 42 per cent of patients transferred to another facility with a tracheostomy tube in situ, 37.5 per cent were at an advanced stage of tracheostomy weaning. Complication rates were low at 4.8 per cent with no tracheostomy associated mortalities. Conclusion: A multidisciplinary approach can enable good outcomes in the neurocritical care population. Consistency of care spanning the step-down from critical to ward-level care is crucial to improving outcomes. [ABSTRACT FROM AUTHOR]

Objective: The Nuchal Translucency Quality Review (NTQR) program has provided standardized education, credentialing and epidemiological monitoring of nuchal translucency (NT) measurements since 2005. Our aim was to review the effect on NT measurement of provider characteristics since the program's inception.Methods: We evaluated the distribution of NT measurements performed between January 2005 and December 2019, for each of the three primary performance indicators of NT measurement (NT median multiples of the median (MoM), SD of log10 NT MoM and slope of NT with respect to crown-rump length (CRL)) for all providers within the NTQR program with more than 30 paired NT/CRL results. Provider characteristics explored as potential sources of variability included: number of NT ultrasound examinations performed annually (annual scan volume of the provider), duration of participation in the NTQR program, initial credentialing by an alternative pathway, provider type (physician vs sonographer) and number of NT-credentialed providers within the practice (size of practice). Each of these provider characteristics was evaluated for its effect on NT median MoM and geometric mean of the NT median MoM weighted for the number of ultrasound scans, and multiple regression was performed across all variables to control for potential confounders.Results: Of 5 216 663 NT measurements from 9340 providers at 3319 sites, the majority (75%) of providers had an NT median MoM within the acceptable range of 0.9-1.1 and 85.5% had NT median MoM not statistically significantly outside this range. Provider characteristics associated with measurement within the expected range of performance included higher volume of NT scans performed annually, practice at a site with larger numbers of other NT-credentialed providers, longer duration of participation in the NTQR program and alternative initial credentialing pathway.Conclusions: Annual scan volume, duration of participation in the NTQR program, alternative initial credentialing pathway and number of other NT-credentialed providers within the practice are all associated with outcome metrics indicating quality of performance. It is critical that providers participate in ongoing quality assessment of NT measurement to maintain consistency and precision. Ongoing assessment programs with continuous feedback and education are necessary to maintain quality care. © 2021 International Society of Ultrasound in Obstetrics and Gynecology. [ABSTRACT FROM AUTHOR]