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1.
Pediatrics ; 2024 May 03.
Article in English | MEDLINE | ID: mdl-38699802

ABSTRACT

OBJECTIVES: To identify the most important attributes related to the process of achieving, and outcomes associated with, successful care for differences of sex development (DSD). METHODS: We developed a best-worst scaling survey administered to 520 DSD stakeholders, including individuals or family members of those with DSD, health care specialists, and patient support and advocacy representatives. Fourteen process-related attributes and 16 outcome-related attributes were identified through qualitative research. We estimated relative importance scores and coefficients from regression analysis to understand the relative importance of attributes and conducted latent class analysis to explore heterogeneity in preferences. RESULTS: The 3 most important process attributes were (1) good communication between care team and patient/family, (2) care team educated patient/family about condition, and (3) care team incorporates the values of patient/family. The 3 most important outcome attributes were (1) patient satisfaction, (2) patient mental health, and (3) treatment maintains physical health. Latent class analyses showed that respondents had heterogeneous preferences. For process-related attributes, we identified 3 respondent groups: "Patient autonomy and support" (46% of respondents), "Education and care transitions" (18%), and "Shared decision-making" (36%). For outcome-related attributes, we identified 2 respondent groups: "Preserving function and appearance" (59% of respondents) and "Patient health and satisfaction" (41%). CONCLUSIONS: Outcomes such as patient satisfaction and health were the most important outcome attributes, and good communication and education from the care team were the most important process attributes. Respondents expressed heterogeneous preferences for selected DSD care attributes that providers should consider to improve satisfaction with and quality of DSD care.

2.
Acad Pediatr ; 2024 Mar 26.
Article in English | MEDLINE | ID: mdl-38548263

ABSTRACT

OBJECTIVE: To improve oral health disparities and outcomes among US children impacted by dental caries, there is a need to understand the cost-effectiveness of a targeted, risk-based versus universal-based approach for caries prevention. METHODS: Health and economic outcomes were simulated in a cohort of 50,000 US children aged 1-18 years, comparing current practice (CP) to risk-based-prevention (RBP) and prevention-for-all (PFA) strategies using health care sector and limited societal perspectives. Prevention included biannual oral health exams and fluoride varnish application, and one-time dental sealant placement. The primary outcome is the cost-effectiveness ratio (ICER), defined as the additional cost per quality-adjusted life year (QALY) gained when comparing each strategy to the next least costly one. RESULTS: For RBP compared to CP, the ICER was US$83,000/QALY from the health care sector perspective; for PFA compared to RBP the ICER was US$154,000/QALY. Using a limited societal perspective that includes caregiver time spent attending dental or medical setting visits, RBP compared to CP yielded a ratio of $119,000/QALY and PFA compared to RBP was $235,000/QALY. Results were most sensitive to changes in the probability of pain from an episode of dental caries, costs for prevention and restoration, and the loss in health-related quality of life due to dental caries pain. Scenario analyses evaluating a reduced intensity of prevention services yielded lower ICERs. CONCLUSION: Using a risk-based approach that identifies and targets children at increased risk for dental caries to guide the delivery of prevention services represents an economic value similar to other pediatric prevention programs.

3.
Influenza Other Respir Viruses ; 18(2): e13255, 2024 Feb.
Article in English | MEDLINE | ID: mdl-38403302

ABSTRACT

We conducted a multicentre hospital-based test-negative case-control study to measure vaccine effectiveness (VE) against PCR-confirmed influenza in adult patients with severe acute respiratory infection (SARI) during the 2022/2023 influenza season in Europe. Among 5547 SARI patients ≥18 years, 2963 (53%) were vaccinated against influenza. Overall VE against influenza A(H1N1)pdm09 was 11% (95% CI: -23-36); 20% (95% CI: -4-39) against A(H3N2) and 56% (95% CI: 22-75) against B. During the 2022/2023 season, while VE against hospitalisation with influenza B was >55%, it was ≤20% for influenza A subtypes. While influenza vaccination should be a priority for future seasons, improved vaccines against influenza are needed.


Subject(s)
Influenza A Virus, H1N1 Subtype , Influenza Vaccines , Influenza, Human , Pneumonia , Adult , Humans , Influenza, Human/epidemiology , Influenza, Human/prevention & control , Seasons , Influenza A Virus, H1N1 Subtype/genetics , Influenza A Virus, H3N2 Subtype/genetics , Case-Control Studies , Vaccine Efficacy , Europe/epidemiology , Hospitalization , Hospitals , Vaccination
4.
Euro Surveill ; 29(8)2024 Feb.
Article in English | MEDLINE | ID: mdl-38390651

ABSTRACT

Influenza A viruses circulated in Europe from September 2023 to January 2024, with influenza A(H1N1)pdm09 predominance. We provide interim 2023/24 influenza vaccine effectiveness (IVE) estimates from two European studies, covering 10 countries across primary care (EU-PC) and hospital (EU-H) settings. Interim IVE was higher against A(H1N1)pdm09 than A(H3N2): EU-PC influenza A(H1N1)pdm09 IVE was 53% (95% CI: 41 to 63) and 30% (95% CI: -3 to 54) against influenza A(H3N2). For EU-H, these were 44% (95% CI: 30 to 55) and 14% (95% CI: -32 to 43), respectively.


Subject(s)
Influenza A Virus, H1N1 Subtype , Influenza Vaccines , Influenza, Human , Humans , Influenza, Human/epidemiology , Influenza, Human/prevention & control , Influenza B virus , Influenza A Virus, H3N2 Subtype , Vaccination , Case-Control Studies , Seasons , Hospitals , Primary Health Care
5.
Euro Surveill ; 29(3)2024 Jan.
Article in English | MEDLINE | ID: mdl-38240061

ABSTRACT

We conducted a multicentre hospital-based test-negative case-control study to measure the effectiveness of adapted bivalent COVID-19 mRNA vaccines against PCR-confirmed SARS-CoV-2 infection during the Omicron XBB lineage-predominant period in patients aged ≥ 60 years with severe acute respiratory infection from five countries in Europe. Bivalent vaccines provided short-term additional protection compared with those vaccinated > 6 months before the campaign: from 80% (95% CI: 50 to 94) for 14-89 days post-vaccination, 15% (95% CI: -12 to 35) at 90-179 days, and lower to no effect thereafter.


Subject(s)
COVID-19 Vaccines , COVID-19 , Humans , Case-Control Studies , COVID-19/prevention & control , SARS-CoV-2/genetics , Hospitalization , Europe/epidemiology , RNA, Messenger
6.
Eur J Public Health ; 34(1): 181-189, 2024 Feb 05.
Article in English | MEDLINE | ID: mdl-37889597

ABSTRACT

BACKGROUND: A pre-existing, well-established European influenza surveillance network known as I-MOVE enabled the rapid implementation of a European multi-country COVID-19 hospital surveillance network for surveillance of hospitalized COVID-19 cases in early 2020. This network included 257 hospitals in 11 surveillance sites across nine countries. We aimed to identify whether the surveillance objectives were relevant to public health actions, whether the surveillance system met its objectives, where and how shortcomings could be improved, and whether the system was sustainable. METHODS: We identified six key attributes (meeting objectives, usefulness, timeliness, data quality, simplicity and sustainability) to assess, using Centers for Disease Control and Prevention's evaluation framework. We analyzed pooled datasets, held interviews and group discussions with 10 participating and coordinating sites and gathered feedback through web surveys. RESULTS: There was overall agreement that the surveillance objectives had been met and being involved in a network of European partners had additional important benefits for stakeholders. While the publication of the outputs was not always sufficiently timely, data submission processes were considered straightforward and the key surveillance variables (age, sex, hospital admission and mortality data) were complete. The main challenges were identified as the collection of the large number of variables, limited available human resources and information governance and data protection laws. CONCLUSIONS: I-MOVE-COVID-19 delivered relevant and accurate data supporting the development and implementation of COVID-19 surveillance. Recommendations presented here identify learning opportunities to support preparedness and surveillance response for future pandemics. The applied evaluation framework in this study can be adapted for other European surveillance system evaluations.


Subject(s)
COVID-19 , Influenza, Human , Humans , COVID-19/epidemiology , Europe/epidemiology , Public Health , Influenza, Human/epidemiology , Pandemics
7.
PLoS One ; 18(12): e0295264, 2023.
Article in English | MEDLINE | ID: mdl-38096237

ABSTRACT

Anxiety about performing numerical calculations is becoming an increasingly important issue. Termed mathematics anxiety, this condition negatively impacts performance in numerical tasks which can affect education outcomes and future employment. The disruption account proposes poor performance is due to anxiety disrupting limited attentional and inhibitory resources leaving fewer cognitive resources for the current task. This study provides the first neural network model of math anxiety. The model simulates performance in two commonly-used tasks related to math anxiety: the numerical Stroop and symbolic number comparison. Different model modifications were used to simulate high and low math-anxious conditions by modifying attentional processes and learning; these model modifications address different theories of math anxiety. The model simulations suggest that math anxiety is associated with reduced attention to numerical stimuli. These results are consistent with the disruption account and the attentional control theory where anxiety decreases goal-directed attention and increases stimulus-driven attention.


Subject(s)
Anxiety Disorders , Anxiety , Humans , Anxiety/psychology , Mathematics , Learning , Neural Networks, Computer
8.
Euro Surveill ; 28(47)2023 11.
Article in English | MEDLINE | ID: mdl-37997666

ABSTRACT

IntroductionTwo large multicentre European hospital networks have estimated vaccine effectiveness (VE) against COVID-19 since 2021.AimWe aimed to measure VE against PCR-confirmed SARS-CoV-2 in hospitalised severe acute respiratory illness (SARI) patients ≥ 20 years, combining data from these networks during Alpha (March-June)- and Delta (June-December)-dominant periods, 2021.MethodsForty-six participating hospitals across 14 countries follow a similar generic protocol using the test-negative case-control design. We defined complete primary series vaccination (PSV) as two doses of a two-dose or one of a single-dose vaccine ≥ 14 days before onset.ResultsWe included 1,087 cases (538 controls) and 1,669 cases (1,442 controls) in the Alpha- and Delta-dominant periods, respectively. During the Alpha period, VE against hospitalisation with SARS-CoV2 for complete Comirnaty PSV was 85% (95% CI: 69-92) overall and 75% (95% CI: 42-90) in those aged ≥ 80 years. During the Delta period, among SARI patients ≥ 20 years with symptom onset ≥ 150 days from last PSV dose, VE for complete Comirnaty PSV was 54% (95% CI: 18-74). Among those receiving Comirnaty PSV and mRNA booster (any product) ≥ 150 days after last PSV dose, VE was 91% (95% CI: 57-98). In time-since-vaccination analysis, complete all-product PSV VE was > 90% in those with their last dose < 90 days before onset; ≥ 70% in those 90-179 days before onset.ConclusionsOur results from this EU multi-country hospital setting showed that VE for complete PSV alone was higher in the Alpha- than the Delta-dominant period, and addition of a first booster dose during the latter period increased VE to over 90%.


Subject(s)
COVID-19 , Humans , Adult , COVID-19/epidemiology , COVID-19/prevention & control , BNT162 Vaccine , RNA, Viral , SARS-CoV-2 , Vaccine Efficacy , Hospitalization , Europe/epidemiology
9.
Euro Surveill ; 28(47)2023 11.
Article in English | MEDLINE | ID: mdl-37997665

ABSTRACT

IntroductionThe I-MOVE-COVID-19 and VEBIS hospital networks have been measuring COVID-19 vaccine effectiveness (VE) in participating European countries since early 2021.AimWe aimed to measure VE against PCR-confirmed SARS-CoV-2 in patients ≥ 20 years hospitalised with severe acute respiratory infection (SARI) from December 2021 to July 2022 (Omicron-dominant period).MethodsIn both networks, 46 hospitals (13 countries) follow a similar test-negative case-control protocol. We defined complete primary series vaccination (PSV) and first booster dose vaccination as last dose of either vaccine received ≥ 14 days before symptom onset (stratifying first booster into received < 150 and ≥ 150 days after last PSV dose). We measured VE overall, by vaccine category/product, age group and time since first mRNA booster dose, adjusting by site as a fixed effect, and by swab date, age, sex, and presence/absence of at least one commonly collected chronic condition.ResultsWe included 2,779 cases and 2,362 controls. The VE of all vaccine products combined against hospitalisation for laboratory-confirmed SARS-CoV-2 was 43% (95% CI: 29-54) for complete PSV (with last dose received ≥ 150 days before onset), while it was 59% (95% CI: 51-66) after addition of one booster dose. The VE was 85% (95% CI: 78-89), 70% (95% CI: 61-77) and 36% (95% CI: 17-51) for those with onset 14-59 days, 60-119 days and 120-179 days after booster vaccination, respectively.ConclusionsOur results suggest that, during the Omicron period, observed VE against SARI hospitalisation improved with first mRNA booster dose, particularly for those having symptom onset < 120 days after first booster dose.


Subject(s)
COVID-19 , Pneumonia , Humans , Adult , COVID-19/prevention & control , COVID-19 Vaccines , Vaccine Efficacy , SARS-CoV-2 , Hospitalization , Europe/epidemiology , RNA, Messenger
10.
Pediatrics ; 152(2)2023 08 01.
Article in English | MEDLINE | ID: mdl-37465909

ABSTRACT

Guanidinoacetate methyltransferase (GAMT) deficiency is an autosomal recessive disorder of creatine biosynthesis due to pathogenic variants in the GAMT gene that lead to cerebral creatine deficiency and neurotoxic levels of guanidinoacetate. Untreated, GAMT deficiency is associated with hypotonia, significant intellectual disability, limited speech development, recurrent seizures, behavior problems, and involuntary movements. The birth prevalence of GAMT deficiency is likely between 0.5 and 2 per million live births. On the basis of small case series and sibling data, presymptomatic treatment with oral supplements of creatine, ornithine, and sodium benzoate, and a protein-restricted diet to reduce arginine intake, appear to substantially improve health and developmental outcomes. Without newborn screening, diagnosis typically happens after the development of significant impairment, when treatment has limited utility. GAMT deficiency newborn screening can be incorporated into the tandem-mass spectrometry screening that is already routinely used for newborn screening, with about 1 per 100 000 newborns screening positive. After a positive screen, diagnosis is established by finding an elevated guanidinoacetate concentration and low creatine concentration in the blood. Although GAMT deficiency is significantly more rare than other conditions included in newborn screening, the feasibility of screening, the low number of positive results, the relative ease of diagnosis, and the expected benefit of presymptomatic dietary therapy led to a recommendation from the Advisory Committee on Heritable Disorders in Newborns and Children to the Secretary of Health and Human Services that GAMT deficiency be added to the Recommended Uniform Screening Panel. This recommendation was accepted in January 2023.


Subject(s)
Language Development Disorders , Movement Disorders , Child , Humans , Infant, Newborn , Guanidinoacetate N-Methyltransferase/genetics , Creatine , Neonatal Screening/methods , Language Development Disorders/diagnosis , Movement Disorders/diagnosis , Movement Disorders/genetics , Movement Disorders/therapy
11.
Vaccine ; 41(29): 4239-4248, 2023 06 29.
Article in English | MEDLINE | ID: mdl-37291022

ABSTRACT

BACKGROUND: The epidemiology of circulating seasonal influenza strains changed following the 2009 pandemic influenza A(H1N1). A universal influenza vaccination recommendation has been implemented and new vaccine types have become available post-2009. The objective of this study was to evaluate the cost-effectiveness of routine annual influenza vaccination in the context of this new evidence. METHODS: A state transition simulation model was constructed to estimate the health and economic outcomes of influenza vaccination compared to no vaccination for hypothetical US cohorts stratified by age and risk status. Model input parameters were derived from multiple sources, including post-2009 vaccine effectiveness data from the US Flu Vaccine Effectiveness Network. The analysis used societal and healthcare sector perspectives and a one-year time horizon, except permanent outcomes were also included. The primary outcome was the incremental cost-effectiveness ratio (ICER) in dollars per quality-adjusted life years (QALYs) gained. RESULTS: Compared to no vaccination, vaccination yielded ICERs lower than $95,000/QALY for all age and risk groups, except for non-high-risk adults 18-49 years ($194,000/QALY). Vaccination was cost-saving for adults ≥50 years at higher risk for influenza-related complications. Results were most sensitive to changes in the probability of influenza illness. Performing the analysis from the healthcare sector perspective, excluding vaccination time costs, delivering vaccinations in lower-cost settings, and including productivity losses improved the cost-effectiveness of vaccination. Sensitivity analysis revealed that vaccination remains below $100,000/QALY for older persons ≥65 years at vaccine effectiveness estimates as low as 4 %. CONCLUSIONS: Cost-effectiveness of influenza vaccination varied by age and risk status and was less than $95,000/QALY for all subgroups, except for non-high-risk working-age adults. Results were sensitive to the probability of influenza illness and vaccination was more favorable under certain scenarios. Vaccination for higher risk subgroups resulted in ICERs below $100,000/QALY even at low levels of vaccine effectiveness or circulating virus.


Subject(s)
Influenza A Virus, H1N1 Subtype , Influenza Vaccines , Influenza, Human , Adult , Humans , Aged , Aged, 80 and over , Influenza, Human/epidemiology , Cost-Benefit Analysis , Vaccination/methods , Quality-Adjusted Life Years
12.
Euro Surveill ; 28(26)2023 06.
Article in English | MEDLINE | ID: mdl-37382887

ABSTRACT

BackgroundIn early 2020, the I-MOVE-COVID-19 hospital surveillance system was adapted from an existing influenza surveillance system to include hospitalised COVID-19 cases.AimTo describe trends in the demographic and clinical characteristics of hospitalised COVID-19 cases across Europe during the first 2 years of the pandemic, and to identify associations between sex, age and chronic conditions with admission to intensive care or high dependency units (ICU/HDU) and in-hospital mortality.MethodsWe pooled pseudonymised data from all hospitalised COVID-19 cases in 11 surveillance sites in nine European countries, collected between 1 February 2020 and 31 December 2021. Associations between sex, age and chronic conditions, with ICU/HDU admission and in-hospital mortality were examined using Pearson's chi-squared test, and crude odds ratio (OR) estimates with respective 95% confidence intervals (CI).ResultsOf 25,971 hospitalised COVID-19 cases, 55% were male, 35% were 75 years or older and 90% had a chronic underlying condition. Patients with two or more chronic underlying conditions were significantly more likely to die in-hospital from COVID-19 (OR: 10.84; 95% CI: 8.30-14.16) than those without a chronic condition.ConclusionThe surveillance demonstrated that males, those 75 years or older and those with chronic conditions were at greater risk of in-hospital death. Over the surveillance period, outcomes tended to improve, likely because of vaccinations. This surveillance has laid the groundwork for further research studies investigating the risk factors of hospitalised COVID-19 cases and vaccine effectiveness.


Subject(s)
COVID-19 , Humans , Male , Female , COVID-19/epidemiology , Hospital Mortality , Europe/epidemiology , Critical Care , Hospitalization
13.
Euro Surveill ; 28(21)2023 05.
Article in English | MEDLINE | ID: mdl-37227299

ABSTRACT

BackgroundBetween October 2022 and January 2023, influenza A(H1N1)pdm09, A(H3N2) and B/Victoria viruses circulated in Europe with different influenza (sub)types dominating in different areas.AimTo provide interim 2022/23 influenza vaccine effectiveness (VE) estimates from six European studies, covering 16 countries in primary care, emergency care and hospital inpatient settings.MethodsAll studies used the test-negative design, but with differences in other study characteristics, such as data sources, patient selection, case definitions and included age groups. Overall and influenza (sub)type-specific VE was estimated for each study using logistic regression adjusted for potential confounders.ResultsThere were 20,477 influenza cases recruited across the six studies, of which 16,589 (81%) were influenza A. Among all ages and settings, VE against influenza A ranged from 27 to 44%. Against A(H1N1)pdm09 (all ages and settings), VE point estimates ranged from 28% to 46%, higher among children (< 18 years) at 49-77%. Against A(H3N2), overall VE ranged from 2% to 44%, also higher among children (62-70%). Against influenza B/Victoria, overall and age-specific VE were ≥ 50% (87-95% among children < 18 years).ConclusionsInterim results from six European studies during the 2022/23 influenza season indicate a ≥ 27% and ≥ 50% reduction in disease occurrence among all-age influenza vaccine recipients for influenza A and B, respectively, with higher reductions among children. Genetic virus characterisation results and end-of-season VE estimates will contribute to greater understanding of differences in influenza (sub)type-specific results across studies.


Subject(s)
Influenza A Virus, H1N1 Subtype , Influenza Vaccines , Influenza, Human , Vaccine Efficacy , Adolescent , Child , Humans , Case-Control Studies , Influenza A Virus, H1N1 Subtype/genetics , Influenza A Virus, H3N2 Subtype/genetics , Influenza B virus/genetics , Influenza Vaccines/administration & dosage , Influenza, Human/epidemiology , Influenza, Human/prevention & control , Seasons , Vaccination , Denmark/epidemiology , Male , Adult , Middle Aged
14.
JAMA Netw Open ; 6(1): e2253582, 2023 01 03.
Article in English | MEDLINE | ID: mdl-36716030

ABSTRACT

Importance: COVID-19 vaccination rates in the US remain below optimal levels. Patient preferences for different attributes of vaccine products and the vaccination experience can be important in determining vaccine uptake decisions. Objective: To assess preferences for attributes of adult and pediatric COVID-19 vaccination among US adults. Design, Setting, and Participants: An online survey of a national panel of 1040 US adults was conducted in May and June 2021. A discrete choice analysis was used to measure the relative value of each attribute in the decision to choose a COVID-19 vaccination option for adults or children. Six attributes were used to described hypothetical vaccination options: vaccine effectiveness, mild side effects, rare adverse events, number of doses, time required for vaccination, and regulatory approval. Respondents chose between hypothetical vaccination profiles or no vaccination. Additional survey questions asked about vaccination beliefs, COVID-19 illness experience, COVID-19 risk factors, vaccination status, and opinions about the risk of COVID-19. Exposures: Respondents chose which vaccine profile they would prefer to receive for themselves (or no vaccination). Respondents then considered an identical set of profiles for a hypothetical child aged 0 to 17 years. Main Outcomes and Measures: Relative value of vaccination-related attributes were estimated using Bayesian logit regression. Preference profiles for subgroups were estimated using latent class analyses. Results: A total of 1040 adults (610 [59%] female; 379 participants [36%] with an age of 55 years and older years) responded to the survey. When asked about vaccination choices for themselves, participants indicated that vaccine effectiveness (95% vs 60%) was a significant attribute (ß, 9.59 [95% CrI, 9.20-10.00] vs ß, 0.41 [95% CrI, 0-0.80]). Respondents also preferred fewer rare adverse events (ß, 6.35 [95% CrI, 5.74-6.86), fewer mild side effects (ß, 5.49; 95% CrI, 5.12-5.87), 1 dose (ß, 5.41; 95% CrI, 5.04-5.78), FDA approval (ß, 6.01; 95% CrI, 5.64-6.41), and shorter waiting times (ß, 5.67; 95% CrI, 4.87-6.48). Results were very similar when framing the question as adult or child vaccination, with slightly stronger preference for fewer rare adverse events for children. Latent class analysis revealed 4 groups of respondents: (1) individuals sensitive to safety and regulatory status, (2) individuals sensitive to convenience, (3) individuals who carefully considered all attributes in making their choices, and (4) individuals who rejected the vaccine. Conclusions and Relevance: In this survey study of US adults, the identification of 4 distinct preference groups provides new information to guide communications to support vaccine decision making. In particular, the group that prioritize convenience (less time required for vaccination and fewer doses) may present an opportunity to create actionable strategies to increase vaccination uptake for both adult and pediatric populations.


Subject(s)
COVID-19 , Vaccines , Adult , Humans , Child , Female , Male , COVID-19 Vaccines/therapeutic use , Bayes Theorem , COVID-19/epidemiology , COVID-19/prevention & control , Vaccination
15.
Genet Med ; 25(2): 100330, 2023 02.
Article in English | MEDLINE | ID: mdl-36445366

ABSTRACT

Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is an X-linked condition caused by pathogenic variants in the iduronate-2-sulfatase gene. The resulting reduced activity of the enzyme iduronate-2-sulfatase leads to accumulation of glycosaminoglycans that can progressively affect multiple organ systems and impair neurologic development. In 2006, the US Food and Drug Administration approved idursulfase for intravenous enzyme replacement therapy for MPS II. After the data suggesting that early treatment is beneficial became available, 2 states, Illinois and Missouri, implemented MPS II newborn screening. Following a recommendation of the Advisory Committee on Heritable Disorders in Newborns and Children in February 2022, in August 2022, the US Secretary of Health and Human Services added MPS II to the Recommended Uniform Screening Panel, a list of conditions recommended for newborn screening. MPS II was added to the Recommended Uniform Screening Panel after a systematic evidence review reported the accuracy of screening, the benefit of presymptomatic treatment compared with usual case detection, and the feasibility of implementing MPS II newborn screening. This manuscript summarizes the findings of the evidence review that informed the Advisory Committee's decision.


Subject(s)
Iduronate Sulfatase , Mucopolysaccharidosis II , Child , Humans , Infant, Newborn , United States , Mucopolysaccharidosis II/diagnosis , Mucopolysaccharidosis II/genetics , Neonatal Screening , Iduronic Acid , Iduronate Sulfatase/therapeutic use , Glycosaminoglycans , Enzyme Replacement Therapy/methods
16.
Front Cardiovasc Med ; 9: 1011192, 2022.
Article in English | MEDLINE | ID: mdl-36330008

ABSTRACT

Women with advanced heart failure receive advanced surgical therapies such as durable left ventricular assist device (LVAD) implantation or heart transplantation at a rate much lower compared to males. Reasons for this discrepancy remain largely unknown. Much of what is understood reflects outcomes of those patients who ultimately receive device implant or heart transplantation. Females have been shown to have a higher mortality following LVAD implantation and experience higher rates of bleeding and clotting phenomena and right ventricular failure. Beyond outcomes, the literature is limited in the identification of pre-operative factors that drive lower than expected LVAD implant rates in this population. More focused research is needed to define the disparities in advance heart failure therapy delivery in women and other underserved populations.

17.
Euro Surveill ; 27(21)2022 05.
Article in English | MEDLINE | ID: mdl-35620997

ABSTRACT

IntroductionIn July and August 2021, the SARS-CoV-2 Delta variant dominated in Europe.AimUsing a multicentre test-negative study, we measured COVID-19 vaccine effectiveness (VE) against symptomatic infection.MethodsIndividuals with COVID-19 or acute respiratory symptoms at primary care/community level in 10 European countries were tested for SARS-CoV-2. We measured complete primary course overall VE by vaccine brand and by time since vaccination.ResultsOverall VE was 74% (95% CI: 69-79), 76% (95% CI: 71-80), 63% (95% CI: 48-75) and 63% (95% CI: 16-83) among those aged 30-44, 45-59, 60-74 and ≥ 75 years, respectively. VE among those aged 30-59 years was 78% (95% CI: 75-81), 66% (95% CI: 58-73), 91% (95% CI: 87-94) and 52% (95% CI: 40-61), for Comirnaty, Vaxzevria, Spikevax and COVID-19 Vaccine Janssen, respectively. VE among people 60 years and older was 67% (95% CI: 52-77), 65% (95% CI: 48-76) and 83% (95% CI: 64-92) for Comirnaty, Vaxzevria and Spikevax, respectively. Comirnaty VE among those aged 30-59 years was 87% (95% CI: 83-89) at 14-29 days and 65% (95% CI: 56-71%) at ≥ 90 days between vaccination and onset of symptoms.ConclusionsVE against symptomatic infection with the SARS-CoV-2 Delta variant varied among brands, ranging from 52% to 91%. While some waning of the vaccine effect may be present (sample size limited this analysis to only Comirnaty), protection was 65% at 90 days or more between vaccination and onset.


Subject(s)
COVID-19 , Influenza Vaccines , Influenza, Human , COVID-19/epidemiology , COVID-19/prevention & control , COVID-19 Vaccines , Europe/epidemiology , Humans , Influenza, Human/prevention & control , Primary Health Care , SARS-CoV-2 , Vaccination
19.
J Pediatr Urol ; 18(1): 36.e1-36.e17, 2022 02.
Article in English | MEDLINE | ID: mdl-34876378

ABSTRACT

INTRODUCTION: Utilizing a qualitative phenomenological design, the Defining Successful Outcomes and Trade-offs study examined stakeholder perspectives regarding optimal healthcare delivery and outcomes for individuals with a difference/disorder of sex development (DSD). OBJECTIVE: We describe study methods and provide an overview of themes and subthemes. STUDY DESIGN: Interviews were conducted with individuals with a DSD (n = 24), parents of those with a DSD (n = 19), healthcare providers (n = 37), and others (n = 30). Primary questions regarding clinical management of patients with DSD were: "What is a successful outcome?" and "How do you achieve it?" RESULTS: Themes included: understanding of DSD diagnosis and self-efficacy in management is necessary but complex; patient and family psychological well-being; support from others versus being stigmatized; affected person experiences physical health and accepts the implications of their condition; complexities in DSD decision making, roles and expectations; and knowledgeable providers and multidisciplinary teams are essential, notwithstanding persisting barriers. Participants recognized competing values potentially forcing trade-offs in decision making. DISCUSSION: Recognition of diverse and sometimes conflicting perspectives regarding optimal pathways of care and outcomes - both within and among those with DSD and their providers -promises to enhance shared decision making. CONCLUSION: Diverse perspectives and perceptions of trade-offs associated with DSD healthcare emphasize the need to tailor care for patients and families.


Subject(s)
Disorders of Sex Development , Disorders of Sex Development/diagnosis , Disorders of Sex Development/psychology , Disorders of Sex Development/therapy , Humans , Parents/psychology , Qualitative Research , Sexual Development
20.
Article in English | WPRIM (Western Pacific) | ID: wpr-972071

ABSTRACT

Background@#The aim of this study was to determine the effectiveness of community-based programs in preventing dengue thru systematic review and meta-analysis of randomized control trials.@*Methods@#All randomized control trials on the evaluation of the effectiveness of community-based programs in dengue prevention were searched on reliable databases (MEDLINE/PubMed, Embase, ClinicalTrials.gov, Google Scholar, etc.). From a total of 32 studies that were identified as of April 2021, there were 11 studies that were included after screening of the titles and abstracts by the review authors. A computer software Revman v.5.4 was used for the statistical analyses needed for the study. All the included studies were encoded in the said software and the effect of the interventions were estimated using odds ratio for the different measurable dengue indices along with an encoded 95% confidence interval@*Results@#The community-based programs used in the included studies were generally effective in preventing dengue using House index (OR=0.83 (95% CI=0.70-0.98), p=0.03), Container index (OR=0.61 (95% CI=0.50-0.74), p<0.01) and Breteau index (OR=0.92 (95% CI=0.81-1.04), p=0.18) as the outcome of measure. The subgroup analysis showed that environmental manipulation [(HI: OR=0.59 (95% CI=0.37-0.92), p= 0.02); (CI: OR=0.54 (95% CI=0.20-1.44), p=0.22); (BI: OR=0.58 (95% CI=0.33-0.88), p=0.010)] had a greater effect towards dengue prevention as compared to human behavior alone or in combination with it.@*Conclusion@#We conclude that environmental management or programs involving community participation is an effective strategy in dengue prevention. Community empowerment and capacity building are indeed important elements to achieve dengue control.


Subject(s)
Dengue , Meta-Analysis
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