Risk Information Sources for Snow Disaster Risk Preparedness in Scotland

Heavy snow disruptions are common and costly occurrences in the UK, including Scotland. Yet, heavy snow remains an underresearched aspect of disaster risks in Scotland. This study critically examined the 2018 heavy snow event in Scotland referred to as the “Beast from the East” (BfE) in order to explore the different sources of information used by the public in preparation for and response to heavy snow emergencies. Our study also examined the effectiveness of BfE risk communication between authorities and the public and sought to determine if there is a relationship between risk information received and the intention to mitigate risk. Data were collected through a semistructured survey from (n = 180) residents of the Annandale and Eskdale region of Dumfries and Galloway, Scotland. Our analysis shows that public authority information sources were the most sought-after information sources, followed by online and web sources. We found statistically significant differences between groups (such as age, gender, and mobility/disability) in terms of using risk information sources. Further analysis shows that the relationship between information received and the intention to mitigate risks is not linear but influenced by intervening variables such as work pressures, financial commitment, and stakeholders’ expectations. We argue that where full adherence to official risk advice is required, policymakers should carefully consider issues around these three factors.

Practice patterns for chronic hypoparathyroidism: data from patients and physicians in France

Context. Recent guidelines have provided recommendations for the care of patients with chronic hypoparathyroidism. Very little is known about actual physicians’ practices or their adherence to such guidelines. Objective. To describe the practice patterns and their compliance with international guidelines. Design. Cohort studies: Épi-Hypo (118 Physicians and 107 patients, from 09/2016 to 12/2019) and ePatients (110 patients, November 2019). Methods. Internet-based cohorts involving all settings at a nationwide level (France). Participants were i) physicians treating patients with chronic hypoparathyroidism and patients with chronic hypoparathyroidism either participating in the ii) Épi-Hypo study (Épi-Hypo 2019 patients) or iii) Hypoparathyroidism France, the national representative association (ePatients). Results. The physicians’ specialties were mainly endocrinology (61%), nephrology (28%), family medicine (2.5%), pediatrics (2.5%), rheumatology (2%) or miscellaneous (4%). Forty-five percent were practicing in public universities. The median number of pharmaceutical drug classes prescribed was 3 per patient. The combination of active vitamin D and calcium salt was given to 59% and 58% of ePatients and Épi-Hypo 2019 patients, respectively. Eighty-five percent of ePatients and 87% of physicians reported monitoring plasma calcium concentrations at a steady state at least twice a year. In 32% and 26% of cases, respectively, ePatients and physicians reported being fully in accordance with international guidelines that recommend targeting symptoms, plasma calcium and phosphate values, and urine calcium excretion. Conclusions. The care of patients with chronic hypoparathyroidism involves physicians with very different practices, so guidelines should include and target not only endocrinologists. Full adherence to the guidelines is low in France.

Real-World Application of a Procalcitonin Monitoring Protocol in a Pediatric Intensive Care Unit

OBJECTIVE Procalcitonin (PCT) is a biomarker used as an indicator for inflammation and bacterial infections. In October 2018, our PICU implemented a PCT monitoring protocol incorporating cutoffs established in previous studies to help guide antibiotic decision-making in patients undergoing sepsis evaluation. The study objective was to evaluate adherence to the protocol with regard to PCT monitoring and antibiotic use. METHODS This retrospective review included PICU patients with systemic inflammatory response syndrome ages > 1 month to 18 years with at least 1 PCT level and blood culture obtained during the 9 months following protocol implementation. Patients were excluded if they received < 48 hours of antibiotic therapy, were neutropenic, or had antibiotics initiated at another hospital. Patients were evaluated for protocol adherence, defined as antibiotic continuation or discontinuation per protocol guidance without excess PCT monitoring. Descriptive statistics were employed. RESULTS Out of 100 patients evaluated, 50 patients were included. Full adherence was observed in 17 patients (34%). Reasons for non-adherence were excess PCT monitoring (54.5%), antibiotic continuation (30.3%), or both (15.2%). Of patients who were non-adherent due to antibiotic continuation, 61.5% had a positive respiratory viral panel (RVP). A total of 49 excess PCT levels were drawn, resulting in an additional $2,000 in health care costs and $15,000 in patient charges. CONCLUSIONS Overall, the impact of our PCT monitoring protocol was difficult to evaluate due to non-adherence, but it highlights potential areas of focus for improving PCT monitoring and antimicrobial stewardship, such as inclusion of RVP results.

INFLUENCE OF DENTAL PHOTOPOLYMERIZATION LAMPS ON THE DEVELOPMENT OF POST-FILLING SENSITIVITY

The article discusses the main types of photopolymerization lamps used in therapeutic dentistry for light polymerization of photocurable composite materials. Their characteristics, advantages and disadvantages, which can lead to the development of complications in the treatment of caries in the form of post-restoration sensitivity, are analyzed. Recommendations for optimal operation with these devices are formulated.Relevance. Often, post-filling pain occurs in experienced doctors with full adherence to the treatment protocol, when, it would seem, nothing should cause complications, overlooking the fact that the reason may lie in the use of a polymerizing device. An important problem today is the lack of awareness of doctors about the peculiarities of working with light-curing devices, depending on their characteristics. According to a survey by N.V. Petrazhitskaya, more than 60% do not know what kind of photopolymerizing lamp they use in their work, about 90% cannot indicate the luminous flux power of their device for polymerisation of the composite, 81% do not regularly check it. Therefore, the problem of the optimal use of light-curing lamps requires additional study.

DURATION OF CLINICAL EFFECT OF LOW-DOSE SUBLINGUAL-PERORAL ALLERGEN-SPECIFIC IMMUNOTHERAPY OF BRONCHIAL ASTHMA

Background. Allergen-specific immunotherapy (ASIT) affects the immune response and the clinical course of bronchial asthma (BA). Objective. To evaluate the clinical efficacy of sublingual-oral low-dose ASIT in asthma based on the results of 5-year follow-up. Material and Methods. ASIT was carried out with a mixture of household allergens in 51 patients aged 18-60 years old. The patient's opinion, the number and severity of BA exacerbations, the duration of post-immunotherapeutic remission and the factors influencing it, adherence to treatment, and the safety of treatment were taken into account. Results. The effectiveness of ASIT according to the patient’s opinion: 45.1% - excellent, 49% - good result. In allergic asthma, remission after ASIT was significantly higher than in mixed asthma. Full adherence to treatment (3 courses of ASIT) - in 76.5% (39/51) of patients. There were no adverse reactions during the entire observation period. The mixed BA phenotype, contact with the allergen in everyday life, and smoking had a negative effect. Positive - high compliance to therapy. Conclusions. Sublingual-oral ASIT is an effective and safe method of treating allergic and mixed asthma. The duration of post-immunotherapeutic remission of BA for 3 years was observed after 3 courses of ASIT.

Continuous use of glycomacropeptide in the nutritional management of patients with phenylketonuria: a clinical perspective

Background In phenylketonuria (PKU), modified casein glycomacropeptide supplements (CGMP-AA) are used as an alternative to the traditional phenylalanine (Phe)-free L-amino acid supplements (L-AA). However, studies focusing on the long-term nutritional status of CGMP-AA are lacking. This retrospective study evaluated the long-term impact of CGMP-AA over a mean of 29 months in 11 patients with a mean age at CGMP-AA onset of 28 years (range 15–43) [8 females; 2 hyperphenylalaninaemia (HPA), 3 mild PKU, 3 classical PKU and 3 late-diagnosed]. Outcome measures included metabolic control, anthropometry, body composition and biochemical parameters. Results CGMP-AA, providing 66% of protein equivalent intake from protein substitute, was associated with no significant change in blood Phe with CGMP-AA compared with baseline (562 ± 289 µmol/L vs 628 ± 317 µmol/L; p = 0.065). In contrast, blood tyrosine significantly increased on CGMP-AA (52.0 ± 19.2 μmol/L vs 61.4 ± 23.8 μmol/L; p = 0.027). Conclusions Biochemical nutritional markers remained unchanged which is an encouraging finding in adults with PKU, many of whom are unable to maintain full adherence with nutritionally fortified protein substitutes. Longitudinal, prospective studies with larger sample sizes are necessary to fully understand the metabolic impact of using CGMP-AA in PKU.

Continuous use of glycomacropeptide in the nutritional management of patients with phenylketonuria: a clinical perspective

Background: In phenylketonuria (PKU), modified casein glycomacropeptide supplements (CGMP-AA) are used as an alternative to the traditional phenylalanine (Phe)-free L-amino acid supplements (L-AA). However, studies focusing on the long-term nutritional status of CGMP-AA are lacking. This retrospective study evaluated the long-term impact of CGMP-AA over a mean of 29 months in 11 patients with a mean age at CGMP-AA onset of 28 years (range 15 to 43) [8 females; 2 hyperphenylalaninaemia (HPA), 3 mild PKU, 3 classical PKU and 3 late-diagnosed]. Outcome measures included metabolic control, anthropometry, body composition and biochemical parameters. Results: CGMP-AA, providing 66% of protein substitute intake, was associated with no significant change in blood Phe with CGMP-AA compared with baseline (562 ± 289 µmol/L vs 628 ± 317 µmol/L; p=0.065). In contrast, blood tyrosine significantly increased on CGMP-AA (52.0 ± 19.2 μmol/L vs 61.4 ± 23.8 μmol/L; p=0.027). Conclusions: Biochemical nutritional markers remained unchanged which is an encouraging finding in adults with PKU, many of whom are unable to maintain full adherence with nutritionally fortified protein substitutes. Longitudinal, prospective studies with larger sample sizes are necessary to fully understand the metabolic impact of using CGMP-AA in PKU.

Continuous use of glycomacropeptide in the nutritional management of patients with phenylketonuria: a clinical perspective

Background: In phenylketonuria (PKU), modified casein glycomacropeptide supplements (CGMP-AA) are used as an alternative to the traditional phenylalanine (Phe)-free L-amino acid supplements (L-AA). However, studies focusing on the long-term nutritional status of CGMP-AA are lacking. This retrospective study evaluated the long-term impact of CGMP-AA over a mean of 29 months in 11 patients with a mean age at CGMP-AA onset of 28 years (range 15 to 43) [8 females; 2 hyperphenylalaninaemia (HPA), 3 mild PKU, 3 classical PKU and 3 late-diagnosed]. Outcome measures included metabolic control, anthropometry, body composition and biochemical parameters. Results: CGMP-AA, providing 66% of protein substitute intake, was associated with no significant change in blood Phe with CGMP-AA compared with baseline (562 ± 289 µmol/L vs 628 ± 317 µmol/L; p=0.065). In contrast, blood tyrosine significantly increased on CGMP-AA (52.0 ± 19.2 μmol/L vs 61.4 ± 23.8 μmol/L; p=0.027). Conclusions: Biochemical nutritional markers remained unchanged which is an encouraging finding in adults with PKU, many of whom are unable to maintain full adherence with nutritionally fortified protein substitutes. Longitudinal, prospective studies with larger sample sizes are necessary to fully understand the metabolic impact of using CGMP-AA in PKU.

Adherence and a Potential Trade-Off Currently Faced in Optimizing Hemophilia Treatment

INTRODUCTION Severe hemophilia, i.e., <1% normal FVIII level (A) or FIX level (B), are congenital bleeding disorders characterized by uncontrolled bleeding. The clinical benefits of prophylactic FVIII/IX replacement therapy are well understood, but require adherence to a schedule of routine infusions. Optimal adherence is associated with better joint outcomes and lower rates of chronic pain. Nonetheless a lack of patient-reported data has to date limited our understanding of the patient burden associated with adherence to treatment, and the relationship between adherence and the ability to work, among people living with hemophilia in the US. Data from the Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study reported a high proportion of adults with hemophilia B receiving routine infusions (at least one infusion per month), showing a negative impact on their ability to work, and people receiving routine infusions were more likely than people treated on-demand to report an inability to work in most situations. The ability of people living with hemophilia to participate in the labor force, without barriers to job choice or working hours, is a key outcome in the drive to achieve health equity. The objective of the analysis is to examine the relationship between adherence and the labor force participation of people with severe hemophilia in the US. METHODS This analysis draws data from a patient-reported study, the 'Cost of Severe Hemophilia Across the US: A Socioeconomic Survey' (CHESS US+). Conducted in 2019, the CHESS US+ study is a cross-sectional patient-centered study of adults with severe hemophilia in the US. A patient-completed questionnaire collected data on clinical, economic, and humanistic outcomes, for a 12-month retrospective period. This analysis examines labor force participation and employment status (full-time, part-time, unemployed, retired) and chronic pain categorized by 'none', low-level ('1-5'), and high-level ('6-10'). The analysis was stratified by adherence to treatment, self-reported on a 1-10 scale, from "not at all" to "fully", categorized into low (1-6), moderate (7-9) and full (10) adherence. Results are presented as mean (standard deviation) or N (%). RESULTS The analysis comprised 356 people with severe hemophilia A (73%) and B (27%) who participated in CHESS US+ study. In Table 1, the baseline characteristics of the study population are stratified by full adherence (N = 119), moderate adherence (N=134) and low adherence (N=103). Having no chronic pain was most prevalent in the full adherence group (37.7%), compared to moderate (8.3%) or low (13.9%) adherence cohorts. Chronic pain, both low- and high-levels were least prevalent among people with full adherence. Moreover, people with low adherence were disproportionately more likely to have high-levels of chronic pain relative to moderate adherence or full adherence (Table 1). Unemployment, however, was highest in full adherence (21.1%), and people with full adherence were also least likely to be in full-time employment (42%). The full-time employment rate decreased as adherence declined from full to moderate (Table 1), and was comparable in people with low adherence (57.3%) or moderate adherence (54.5%). CONCLUSIONS This analysis of CHESS US+ examined the complex relationship between labor market outcomes and adherence to treatment, among adults with severe hemophilia in the US. Adherence was associated with lower rates of chronic pain, representing the importance of achieving an optimal treatment strategy. Nonetheless, patients achieving optimal adherence were less likely to be in full-time employment, and more likely to be part-time or unemployed, comparatively. Together, these data characterize a trade-off in clinical outcomes versus workforce participation, and suggest that the goal of achieving health equity may currently still be unmet. Disclosures Burke: HCD Economics: Current Employment; University of Chester: Current Employment; F. Hoffmann-La Roche Ltd: Consultancy. Asghar:HCD Economics: Current Employment. Misciattelli:Freeline: Current Employment, Current equity holder in publicly-traded company. Kar:Freeline: Current Employment, Current equity holder in publicly-traded company. Morgan:uniQure: Consultancy; HCD Economics: Current Employment. Dhillon:HCD Economics: Current Employment; F. Hoffmann-La Roche Ltd: Other: All authors received editorial support for this abstract, furnished by Scott Battle, funded by F. Hoffmann-La Roche Ltd, Basel, Switzerland. . O'Hara:HCD Economics: Current Employment, Current equity holder in private company; F. Hoffmann-La Roche Ltd: Consultancy.

The Impact of a Slow-Release Large Neutral Amino Acids Supplement on Treatment Adherence in Adult Patients with Phenylketonuria

The gold standard treatment for phenylketonuria (PKU) is a lifelong low-phenylalanine (Phe) diet supplemented with Phe-free protein substitutes. Adherence to therapy becomes difficult after childhood. Supplementing with large neutral amino acids (LNAAs) has been proposed as an alternative medication to Phe-free protein substitutes (i.e., amino acid mixtures). The aim of this study was to evaluate adherence to therapy and quality of life (QoL) in a cohort of sub-optimally controlled adult PKU patients treated with a new LNAA formulation. Twelve patients were enrolled in a 12-month-trial of slow-release LNAAs (1g/kg/day) plus a Phe-restricted diet. Medication adherence was measured with the Morisky Green Levine Medication Adherence Scale; the QoL was measured using the phenylketonuria-quality of life (PKU-QoL) questionnaire. Phe, tyrosine (Tyr) levels, and Phe/Tyr ratios were measured fortnightly. Before treatment, 3/12 patients self-reported a ‘medium’ adherence to medication and 9/12 reported a low adherence; 60% of patients reported a full adherence over the past four weeks. After 12 months of LNAA treatment, all patients self-reported a high adherence to medication, with 96% reporting a full adherence. Phe levels remained unchanged, while Tyr levels increased in most patients. The Phy/Tyr ratio decreased. All patients had a significant improvement in the QoL. LNAAs may give patients a further opportunity to improve medication adherence and, consequently, their QoL.