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BACKGROUND: Debates on the allocation of medical resources during the coronavirus disease 2019 (COVID-19) pandemic revealed the need for a better understanding of immunological risk. Studies highlighted variable clinical outcomes of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infections in individuals with defects in both adaptive and innate immunity, suggesting additional contributions from other factors. Notably, none of these studies controlled for variables linked with social determinants of health. OBJECTIVE: To determine the contributions of determinants of health to risk of hospitalization for SARS-CoV-2 infection among individuals with inborn errors of immunodeficiencies. METHODS: This is a retrospective, single-center cohort study of 166 individuals with inborn errors of immunity, aged 2 months through 69 years, who developed SARS-CoV-2 infections from March 1, 2020, through March 31, 2022. Risks of hospitalization were assessed using a multivariable logistic regression analysis. RESULTS: The risk of SARS-CoV-2-related hospitalization was associated with underrepresented racial and ethnic populations (odds ratio [OR] 4.50; 95% confidence interval [95% CI] 1.57-13.4), a diagnosis of any genetically defined immunodeficiency (OR 3.32; 95% CI 1.24-9.43), obesity (OR 4.24; 95% CI 1.38-13.3), and neurological disease (OR 4.47; 95% CI 1.44-14.3). The COVID-19 vaccination was associated with reduced hospitalization risk (OR 0.52; 95% CI 0.31-0.81). Defects in T cell and innate immune function, immune-mediated organ dysfunction, and social vulnerability were not associated with increased risk of hospitalization after controlling for covariates. CONCLUSIONS: The associations between race, ethnicity, and obesity with increased risk of hospitalization for SARS-CoV-2 infection indicate the importance of variables linked with social determinants of health as immunological risk factors for individuals with inborn errors of immunity.
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COVID-19 , Enfermedades de Inmunodeficiencia Primaria , Humanos , COVID-19/epidemiología , SARS-CoV-2 , Estudios Retrospectivos , Estudios de Cohortes , Vacunas contra la COVID-19 , Obesidad , Hospitalización , Enfermedades de Inmunodeficiencia Primaria/epidemiologíaRESUMEN
Background: Debates on the allocation of medical resources during the COVID-19 pandemic revealed the need for a better understanding of immunologic risk. Studies highlighted variable clinical outcomes of SARS-CoV-2 infections in individuals with defects in both adaptive and innate immunity, suggesting additional contributions from other factors. Notably, none of these studies controlled for variables linked with social determinants of health. Objective: To determine the contributions of determinants of health to risk of hospitalization for SARS-CoV-2 infection among individuals with inborn errors of immunodeficiencies. Methods: This is a retrospective, single-center cohort study of 166 individuals with inborn errors of immunity, aged two months through 69 years, who developed SARS-CoV-2 infections from March 1, 2020 through March 31, 2022. Risks of hospitalization was assessed using a multivariable logistic regression analysis. Results: The risk of SARS-CoV-2-related hospitalization was associated with underrepresented racial and ethnic populations (odds ratio [OR] 5.29; confidence interval [CI], 1.76-17.0), a diagnosis of any genetically-defined immunodeficiency (OR 4.62; CI, 1.60-14.8), use of B cell depleting therapy within one year of infection (OR 6.1; CI, 1.05-38.5), obesity (OR 3.74; CI, 1.17-12.5), and neurologic disease (OR 5.38; CI, 1.61-17.8). COVID-19 vaccination was associated with reduced hospitalization risk (OR 0.52; CI, 0.31-0.81). Defective T cell function, immune-mediated organ dysfunction, and social vulnerability were not associated with increased risk of hospitalization after controlling for covariates. Conclusions: The associations between race, ethnicity, and obesity with increased risk of hospitalization for SARS-CoV-2 infection indicate the importance of variables linked with social determinants of health as immunologic risk factors for individuals with inborn errors of immunity. Highlights: What is already known about this topic? Outcomes of SARS-CoV-2 infections in individuals with inborn errors of immunity (IEI) are highly variable. Prior studies of patients with IEI have not controlled for race or social vulnerability. What does this article add to our knowledge ? For individuals with IEI, hospitalizations for SARS-CoV-2 were associated with race, ethnicity, obesity, and neurologic disease. Specific types of immunodeficiency, organ dysfunction, and social vulnerability were not associated with increased risk of hospitalization. How does this study impact current management guidelines? Current guidelines for the management of IEIs focus on risk conferred by genetic and cellular mechanisms. This study highlights the importance of considering variables linked with social determinants of health and common comorbidities as immunologic risk factors.
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Hereditary Angioedema (HAE) is a rare and disabling disease for which early diagnosis and effective therapy are critical. This revision and update of the global WAO/EAACI guideline on the diagnosis and management of HAE provides up-to-date guidance for the management of HAE. For this update and revision of the guideline, an international panel of experts reviewed the existing evidence, developed 28 recommendations, and established consensus by an online DELPHI process. The goal of these recommendations and guideline is to help physicians and their patients in making rational decisions in the management of HAE with deficient C1-inhibitor (type 1) and HAE with dysfunctional C1-inhibitor (type 2), by providing guidance on common and important clinical issues, such as: 1) How should HAE be diagnosed? 2) When should HAE patients receive prophylactic on top of on-demand treatment and what treatments should be used? 3) What are the goals of treatment? 4) Should HAE management be different for special HAE patient groups such as children or pregnant/breast feeding women? 5) How should HAE patients monitor their disease activity, impact, and control? It is also the intention of this guideline to help establish global standards for the management of HAE and to encourage and facilitate the use of recommended diagnostics and therapies for all patients.
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Hereditary angioedema (HAE) is a rare and disabling disease for which early diagnosis and effective therapy are critical. This revision and update of the global WAO/EAACI guideline on the diagnosis and management of HAE provides up-to-date guidance for the management of HAE. For this update and revision of the guideline, an international panel of experts reviewed the existing evidence, developed 28 recommendations, and established consensus by an online DELPHI process. The goal of these recommendations and guideline is to help physicians and their patients in making rational decisions in the management of HAE with deficient C1 inhibitor (type 1) and HAE with dysfunctional C1 inhibitor (type 2), by providing guidance on common and important clinical issues, such as: (1) How should HAE be diagnosed? (2) When should HAE patients receive prophylactic on top of on-demand treatment and what treatments should be used? (3) What are the goals of treatment? (4) Should HAE management be different for special HAE patient groups such as children or pregnant/breast-feeding women? and (5) How should HAE patients monitor their disease activity, impact, and control? It is also the intention of this guideline to help establish global standards for the management of HAE and to encourage and facilitate the use of recommended diagnostics and therapies for all patients.
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Angioedemas Hereditarios , Angioedemas Hereditarios/prevención & control , Angioedemas Hereditarios/terapia , Niño , Proteína Inhibidora del Complemento C1/genética , Proteína Inhibidora del Complemento C1/uso terapéutico , Consenso , Femenino , Humanos , EmbarazoRESUMEN
BACKGROUND: Treatment options for peanut allergy are limited. In previous clinical trials, epicutaneous immunotherapy with a patch containing 250-µg peanut protein (Viaskin Peanut 250 µg [VP250]) was well tolerated and statistically superior to placebo in desensitizing peanut-allergic children. OBJECTIVE: To examine the safety of VP250 in children, using a study design approximating potential real-world use. METHODS: REAL LIfe Use and Safety of EPIT (REALISE) is a phase 3 multicenter study consisting of a 6-month, randomized, double-blind, placebo-controlled period followed by open-label active treatment. Children aged 4 to 11 years with physician diagnosis of peanut allergy received daily treatment with placebo (6 months) or VP250 (up to 36 months). Data from the 6-month, randomized, controlled phase of REALISE are reported. RESULTS: Three hundred ninety-three children were randomized 3:1 to receive VP250 (n = 294) or placebo (n = 99) for 6 months; 284 (72.3%) children had a history of peanut anaphylaxis. According to parent diary, all participants receiving VP250 and 83.8% receiving placebo reported at least 1 episode of local skin reaction, with frequency decreasing over time. Only 4 participants (1.4%) receiving VP250 discontinued because of adverse events (AEs). Epinephrine was administered for allergic reactions attributed to VP250 in 7 children (2.4%), of whom 5 remained in the study; none involved severe anaphylaxis. Overall, AE rates were similar among participants with and without a history of peanut anaphylaxis. CONCLUSIONS: In a study designed to mirror real-world use, VP250 was observed to be well tolerated in peanut-allergic children, consistent with previous phase 2b and 3 studies.
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Anafilaxia , Hipersensibilidad al Cacahuete , Administración Oral , Alérgenos/uso terapéutico , Anafilaxia/etiología , Arachis , Niño , Desensibilización Inmunológica/métodos , Humanos , Factores Inmunológicos/uso terapéutico , Hipersensibilidad al Cacahuete/tratamiento farmacológicoRESUMEN
OBJECTIVE: Pediatric asthma is a costly and complex disease with proven interventions to prevent exacerbations. Finding the patients at highest risk of exacerbations is paramount given limited resources. Insurance claims identify all outpatient, inpatient, emergency, pharmacy, and diagnostic services. The objective was to develop a risk score indicating the likelihood of asthma exacerbation within the next year based on prior utilization. METHODS: A retrospective analysis of insurance claims for patients 2 to 18 years in a network in Massachusetts with 3 years of continuous enrollment in a commercial plan. Thirty-six potential predictors of exacerbation in the third year were assessed with a stepwise regression. Retained predictors were weighted relative to their contribution to asthma exacerbation risk and summed to create the Asthma Exacerbation Risk (AER) score. RESULTS: In a cohort of 28,196 patients, there were 10 predictors associated with the outcome of having an asthma exacerbation in the next year that depend on age, meeting the Healthcare Effectiveness Data and Information Set persistent asthma criteria, fill patterns of asthma medications and oral steroids, counts of nonexacerbation outpatient visits, an exacerbation in the last 6 months, and whether spirometry was performed. The AER score is calculated monthly from a claims database to identify potential patients for an asthma home-visiting program. CONCLUSIONS: The AER score assigns a risk of exacerbation within the next 12 months using claims data to identify patients in need of preventive services.
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Asma , Revisión de Utilización de Seguros , Asma/epidemiología , Niño , Estudios de Cohortes , Humanos , Lactante , Estudios Retrospectivos , Factores de RiesgoRESUMEN
Peanut allergy can result in life-threatening reactions and is a major public health concern. Oral immunotherapy (OIT) induces desensitization to food allergens through administration of increasing amounts of allergen. To dissect peanut-specific immunoglobulin E (IgE) and IgG responses in subjects undergoing OIT, we have developed AllerScan, a method that leverages phage-display and next-generation sequencing to identify the epitope targets of peanut-specific antibodies. We observe a striking diversification and boosting of the peanut-specific IgG repertoire after OIT and a reduction in pre-existing IgE levels against individual epitopes. High-resolution epitope mapping reveals shared recognition of public epitopes in Ara h 1, 2, 3, and 7. In individual subjects, OIT-induced IgG specificities overlap extensively with IgE and exhibit strikingly similar antibody footprints, suggesting related clonal lineages or convergent evolution of peanut-specific IgE and IgG B cells. Individual differences in epitope recognition identified via AllerScan could inform safer and more effective personalized immunotherapy.
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Desensibilización Inmunológica/métodos , Mapeo Epitopo/métodos , Epítopos/química , Inmunoglobulina E/sangre , Inmunoglobulina G/sangre , Omalizumab/uso terapéutico , Hipersensibilidad al Cacahuete/terapia , Albuminas 2S de Plantas/administración & dosificación , Albuminas 2S de Plantas/química , Antígenos de Plantas/administración & dosificación , Antígenos de Plantas/química , Arachis/química , Arachis/inmunología , Linfocitos B/inmunología , Linfocitos B/patología , Estudios de Casos y Controles , Epítopos/inmunología , Secuenciación de Nucleótidos de Alto Rendimiento , Ensayos Analíticos de Alto Rendimiento , Humanos , Proteínas de la Membrana/administración & dosificación , Proteínas de la Membrana/química , Hipersensibilidad al Cacahuete/genética , Hipersensibilidad al Cacahuete/inmunología , Hipersensibilidad al Cacahuete/patología , Biblioteca de Péptidos , Proteínas de Plantas/administración & dosificación , Proteínas de Plantas/química , Medicina de Precisión , Proteínas de Almacenamiento de SemillasRESUMEN
PURPOSE: We seek to determine how youth with chronic medical conditions experience alcohol screening and counseling. METHODS: Adolescents with type I diabetes, juvenile idiopathic arthritis, moderate persistent asthma, cystic fibrosis, attention deficit hyperactivity disorder, or inflammatory bowel disease were surveyed. Descriptive statistics and regression analysis quantified rates of asking and counseling about alcohol. RESULTS: Of 390 participants (75.1% white/non-Hispanic, 51.8% female, average age 16.4 years), 70% reported being asked about their alcohol use by a healthcare provider, and 76% reported receiving at least one message regarding alcohol and health. Of past year drinkers, 54% disclosed use to their provider. Only 2.0% of youth reported receiving the message "I should not drink." CONCLUSIONS: Most youth with chronic medical conditions were asked and counseled about alcohol use although few heard unambiguous recommendations to avoid alcohol consumption.
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Enfermedad Crónica/terapia , Consejo , Tamizaje Masivo , Consumo de Alcohol en Menores/prevención & control , Adolescente , Instituciones de Atención Ambulatoria , Femenino , Humanos , Masculino , Encuestas y CuestionariosAsunto(s)
Alérgenos/administración & dosificación , Hipersensibilidad a los Alimentos/diagnóstico , Accesibilidad a los Servicios de Salud/normas , Aceptación de la Atención de Salud/estadística & datos numéricos , Mejoramiento de la Calidad/organización & administración , Accesibilidad a los Servicios de Salud/estadística & datos numéricos , Humanos , Mejoramiento de la Calidad/estadística & datos numéricosRESUMEN
Food challenges are the criterion standard for establishing the presence or absence of food allergy. However, they remain underused because of their resource-intensive nature, inadequate reimbursement, and concern for the risk of anaphylaxis. Here, we review indications for performing food challenges, including scenarios of uncertain diagnosis, quality-of-life effects following food challenges, and the impact on office practice including coding and reimbursement issues. Demand for food challenges is likely to increase and allergists should be capable of providing this service to their patients when indicated.
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Hipersensibilidad a los Alimentos/diagnóstico , Pruebas Inmunológicas/métodos , Actitud del Personal de Salud , Humanos , Médicos , Calidad de VidaRESUMEN
BACKGROUND: Peanut oral immunotherapy, using a variety of approaches, has been previously shown to induce desensitization in peanut-allergic subjects, but no products have been approved for clinical use by regulatory agencies. OBJECTIVE: We performed the first phase 2 multicentered study to assess the safety and efficacy of AR101, a novel oral biologic drug product. METHODS: A randomized, double-blind, placebo-controlled trial was conducted at 8 US centers. Eligible subjects were 4 to 26 years old, sensitized to peanut, and had dose-limiting symptoms to ≤143 mg of peanut protein in a screening double-blind, placebo-controlled food challenge (DBPCFC). Subjects were randomized 1:1 to daily AR101 or placebo and gradually up-dosed from 0.5 to 300 mg/day. The primary endpoint was the proportion of subjects in each arm able to tolerate ≥443 mg (cumulative peanut protein) at exit DBPCFC with no or mild symptoms. RESULTS: Fifty-five subjects (29 AR101, 26 placebo) were enrolled. In the intention-to-treat analysis, 23 of 29 (79%) and 18 of 29 (62%) AR101 subjects tolerated ≥443 mg and 1043 mg at exit DBPCFC, respectively, versus 5 of 26 (19%) and 0 of 26 (0%) placebo subjects (both P < .0001). Compared with placebo, AR101 significantly reduced symptom severity during exit DBPCFCs and modulated peanut-specific cellular and humoral immune responses. Gastrointestinal (GI) symptoms were the most common treatment-related adverse events (AEs) in both groups, with 6 AR101 subjects (21%) withdrawing, 4 of those due primarily to recurrent GI AEs. CONCLUSIONS: In this study, AR101 demonstrated an acceptable safety profile and demonstrated clinical activity as a potential immunomodulatory treatment option in peanut-allergic children over the age of 4, adolescents, and young adults.
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Alérgenos/administración & dosificación , Arachis , Desensibilización Inmunológica , Hipersensibilidad al Cacahuete/terapia , Proteínas de Plantas/administración & dosificación , Administración Oral , Adolescente , Adulto , Niño , Preescolar , Método Doble Ciego , Femenino , Humanos , Masculino , Resultado del Tratamiento , Adulto JovenRESUMEN
BACKGROUND: Although previous single-center studies report the rate of anaphylaxis for oral food challenges (OFCs) as 9% to 11%, little is known regarding the epidemiology of clinical OFCs across multiple centers in the United States. OBJECTIVE: To examine the epidemiology, symptoms, and treatment of clinical low-risk OFCs in the nonresearch setting. METHODS: Data were obtained from 2008 to 2013 through a physician survey in 5 food allergy centers geographically distributed across the United States. Allergic reaction rates and the association of reaction rates with year, hospital, and demographics were determined using a linear mixed model. Meta-analysis was used to pool the proportion of reactions and anaphylaxis with inverse-variance weights using a random-effects model with exact confidence intervals (CIs). RESULTS: A total of 6,377 OFCs were performed, and the pooled estimate of anaphylaxis was 2% (95% CI, 1%-3%). The rate of allergic reactions was 14% (95% CI, 13%-16%) and was consistent during the study period (P = .40). Reaction rates ranged from 13% to 33%. Males reacted 16% more frequently than females (95% CI, 4%-37.5%; P = .04). Foods challenged in 2013 varied geographically, with peanut as the most challenged food in the Northeast, Midwest, and West and egg as the most challenged in the South. CONCLUSION: As the largest national survey of allergic reactions of clinical open OFCs in a nonresearch setting in the United States, this study found that performing clinical nonresearch open low-risk OFCs results in few allergic reactions, with 86% of challenges resulting in no reactions and 98% without anaphylaxis.
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Alérgenos/inmunología , Anafilaxia/epidemiología , Hipersensibilidad a los Alimentos/epidemiología , Adolescente , Anafilaxia/diagnóstico , Anafilaxia/fisiopatología , Arachis/química , Arachis/inmunología , Niño , Preescolar , Hipersensibilidad a los Alimentos/diagnóstico , Hipersensibilidad a los Alimentos/fisiopatología , Humanos , Incidencia , Lactante , Modelos Lineales , Prevalencia , Riesgo , Factores Sexuales , Pruebas Cutáneas , Estados Unidos/epidemiologíaRESUMEN
BACKGROUND: Oral food challenges (OFCs) are routinely used to confirm ongoing food allergy. Serum-specific IgE (sIgE) and skin prick testing (SPT) are imperfect predictors of which patients will pass OFCs. OBJECTIVE: The objective of this study was to describe the design and implementation of a Standardized Clinical Assessment and Management Plan (SCAMP) to study and iteratively improve sIgE and SPT thresholds to determine when and where to conduct OFCs for patients. METHODS: Allergists consulted recommended sIgE and SPT thresholds when ordering challenges although diversions were permitted. Criteria were iteratively improved after periodic analyses of challenge outcome and diversions. RESULTS: Over 3 years, allergists ordered 2368 food challenges for 1580 patients with histories of IgE-mediated reactions to food: 1386 in an outpatient clinic and 945 in a higher resource infusion center. Reactions to challenge were observed in 13% of clinic and 23% of infusion center challenges. Six patients challenged in clinic required treatment with epinephrine compared with 22 in the infusion center. The need for epinephrine was more common in patients with asthma-5% of asthmatic patients required epinephrine compared with 1% of nonasthmatic patients (P < .01). Recommended sIgE and SPT thresholds were incrementally changed and, using the control chart methodology, a significant decrease was noted in the proportion of challenges ordered in the higher resource location. CONCLUSIONS: By setting and continually refining sIgE and SPT recommendations using the SCAMP method, allergists can better determine the risk of severe reaction and triage patients to the appropriate setting for an OFC.
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Alérgenos/inmunología , Asma/diagnóstico , Asma/inmunología , Hipersensibilidad a los Alimentos/diagnóstico , Hipersensibilidad a los Alimentos/inmunología , Evaluación de Síntomas , Administración Oral , Adolescente , Niño , Preescolar , Alimentos , Humanos , Inmunización , Inmunoglobulina E/sangre , Lactante , Guías de Práctica Clínica como Asunto , Mejoramiento de la Calidad , Estándares de Referencia , Riesgo , Pruebas Cutáneas , Estados Unidos , Adulto JovenRESUMEN
BACKGROUND: Peanut oral immunotherapy is a promising approach to peanut allergy, but reactions are frequent, and some patients cannot be desensitized. The anti-IgE medication omalizumab (Xolair; Genentech, South San Francisco, Calif) might allow more rapid peanut updosing and decrease reactions. OBJECTIVE: We sought to evaluate whether omalizumab facilitated rapid peanut desensitization in highly allergic patients. METHODS: Thirty-seven subjects were randomized to omalizumab (n = 29) or placebo (n = 8). After 12 weeks of treatment, subjects underwent a rapid 1-day desensitization of up to 250 mg of peanut protein, followed by weekly increases up to 2000 mg. Omalizumab was then discontinued, and subjects continued on 2000 mg of peanut protein. Subjects underwent an open challenge to 4000 mg of peanut protein 12 weeks after stopping study drug. If tolerated, subjects continued on 4000 mg of peanut protein daily. RESULTS: The median peanut dose tolerated on the initial desensitization day was 250 mg for omalizumab-treated subjects versus 22.5 mg for placebo-treated subject. Subsequently, 23 (79%) of 29 subjects randomized to omalizumab tolerated 2000 mg of peanut protein 6 weeks after stopping omalizumab versus 1 (12%) of 8 receiving placebo (P < .01). Twenty-three subjects receiving omalizumab versus 1 subject receiving placebo passed the 4000-mg food challenge. Overall reaction rates were not significantly lower in omalizumab-treated versus placebo-treated subjects (odds ratio, 0.57; P = .15), although omalizumab-treated subjects were exposed to much higher peanut doses. CONCLUSION: Omalizumab allows subjects with peanut allergy to be rapidly desensitized over as little as 8 weeks of peanut oral immunotherapy. In the majority of subjects, this desensitization is sustained after omalizumab is discontinued. Additional studies will help clarify which patients would benefit most from this approach.
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Antialérgicos/uso terapéutico , Desensibilización Inmunológica , Omalizumab/uso terapéutico , Hipersensibilidad al Cacahuete/tratamiento farmacológico , Hipersensibilidad al Cacahuete/terapia , Adolescente , Adulto , Alérgenos/inmunología , Arachis/inmunología , Niño , Método Doble Ciego , Femenino , Humanos , Inmunoglobulina E/sangre , Masculino , Pruebas Cutáneas , Adulto JovenRESUMEN
BACKGROUND: In an effort to reduce barriers to screening for alcohol use in pediatric primary care, the National Institute on Alcoholism and Alcohol Abuse (NIAAA) developed a two-question Youth Alcohol Screening Tool derived from population-based survey data. It is unknown whether this screening tool, designed for use with general populations, accurately identifies risk among youth with chronic medical conditions (YCMC). This growing population, which comprises nearly one in four youth in the US, faces a unique constellation of drinking-related risks. METHOD: To validate the NIAAA Youth Alcohol Screening Tool in a population of YCMC, we performed a cross-sectional validation study with a sample of 388 youth ages 9-18 years presenting for routine subspecialty care at a large children's hospital for type 1 diabetes, persistent asthma, cystic fibrosis, inflammatory bowel disease, or juvenile idiopathic arthritis. Participants self-administered the NIAAA Youth Alcohol Screening Tool and the Diagnostic Interview Schedule for Children as a criterion standard measure of alcohol use disorders (AUD). Receiver operating curve analysis was used to determine cut points for identifying youth at moderate and highest risk for an AUD. RESULTS: Nearly one third of participants (n = 118; 30.4%) reported alcohol use in the past year; 86.4% (106) of past year drinkers did not endorse any AUD criteria, 6.8% (n = 8) of drinkers endorsed a single criterion, and 6.8% of drinkers met criteria for an AUD. Using the NIAAA tool, optimal cut points found to identify youth at moderate and highest risk for an AUD were ≥ 6 and ≥12 drinking days in the past year, respectively. CONCLUSIONS: The NIAAA Youth Alcohol Screening Tool is highly efficient for detecting alcohol use and discriminating disordered use among YCMC. This brief screen appears feasible for use in specialty care to ascertain alcohol-related risk that may impact adversely on health status and disease management.
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Consumo de Bebidas Alcohólicas/efectos adversos , Trastornos Relacionados con Alcohol/diagnóstico , Tamizaje Masivo , Poblaciones Vulnerables/estadística & datos numéricos , Adolescente , Trastornos Relacionados con Alcohol/epidemiología , Trastornos Relacionados con Alcohol/etiología , Niño , Estudios Transversales , Femenino , Humanos , Masculino , Curva ROC , Medición de Riesgo , Estados Unidos/epidemiologíaRESUMEN
Presently, medications approved for children with Hereditary Angioedema (HAE) are extremely limited. This is especially the case for children under 12 years of age. For this reason we reviewed and summarized the data on treatment of children with HAE. Available data indicate that plasma derived C1-inhibitor is a safe, effective treatment option for HAE in pediatric patients, including those below 12 years of age. Other therapies are also appear safe for the under 12 year of age, but less data are available. Importantly, home-based treatment of HAE in this age group appears to be safe and effective and can improve quality of life. These findings support current HAE consensus guidelines which strongly recommend the use of plasma derived C1-inhibitor as a first-line treatment in children and encourage home and self-treatment.