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1.
Allergy ; 77(7): 1961-1990, 2022 07.
Artículo en Inglés | MEDLINE | ID: mdl-35006617

RESUMEN

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.


Asunto(s)
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 , Embarazo
2.
J Allergy Clin Immunol ; 139(3): 873-881.e8, 2017 Mar.
Artículo en Inglés | MEDLINE | ID: mdl-27609658

RESUMEN

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.


Asunto(s)
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 Joven
3.
Ann Allergy Asthma Immunol ; 119(4): 339-348.e1, 2017 10.
Artículo en Inglés | MEDLINE | ID: mdl-28890356

RESUMEN

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.


Asunto(s)
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/epidemiología
4.
Pediatr Allergy Immunol ; 26(6): 537-44, 2015 Sep.
Artículo en Inglés | MEDLINE | ID: mdl-26111105

RESUMEN

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.


Asunto(s)
Angioedemas Hereditarios/tratamiento farmacológico , Proteína Inhibidora del Complemento C1/uso terapéutico , Factores Inmunológicos/uso terapéutico , Plasma/química , Adolescente , Factores de Edad , Angioedemas Hereditarios/diagnóstico , Angioedemas Hereditarios/inmunología , Niño , Preescolar , Proteína Inhibidora del Complemento C1/efectos adversos , Proteína Inhibidora del Complemento C1/aislamiento & purificación , Servicios de Atención de Salud a Domicilio , Humanos , Factores Inmunológicos/efectos adversos , Factores Inmunológicos/aislamiento & purificación , Guías de Práctica Clínica como Asunto , Autocuidado , Resultado del Tratamiento
5.
Pediatr Allergy Immunol ; 25(5): 420-7, 2014 Aug.
Artículo en Inglés | MEDLINE | ID: mdl-24313851

RESUMEN

Hereditary angioedema (HAE) is a lifelong illness characterized by recurrent swelling of the skin, intestinal tract, and, ominously, the upper airway. It is caused by inadequate activity of the protein C1-inhibitor, with dysfunction in the kallikrein/bradykinin pathway underlying the clinical symptoms. In addition to the physical symptoms, patients experience significant decrements in vocational and school achievement as well as in overall quality of life. Symptoms often begin in childhood and occur by age 20 in most patients, but life-threatening attacks are uncommon in the pediatric population. The availability of new therapies has transformed the management of HAE.


Asunto(s)
Angioedemas Hereditarios , Niño , Humanos
9.
Ann Allergy Asthma Immunol ; 110(3): 184-188.e2, 2013 Mar.
Artículo en Inglés | MEDLINE | ID: mdl-23548529

RESUMEN

BACKGROUND: Hereditary angioedema (HAE) is a rare disorder associated with episodic attacks of well-demarcated angioedema. Attacks that affect the larynx can result in life-threatening airway obstruction. OBJECTIVES: To examine efficacy and safety of ecallantide treatment for laryngeal HAE attacks. METHODS: Data were combined from 4 clinical studies (EDEMA2, EDEMA3, EDEMA4, and DX-88/19) evaluating 30 mg of subcutaneous ecallantide for treatment of acute HAE attacks. Efficacy was assessed using 2 validated, HAE-specific, patient-reported outcome measures. The change in Mean Symptom Complex Severity (MSCS) score indicates change in symptom severity; a negative score indicates improvement. The calculated minimally important difference (MID) for change in severity is -0.30. The Treatment Outcome Score (TOS) measures treatment response. A positive score indicates improvement; the calculated MID is 30. RESULTS: Overall, 98 patients received ecallantide for 220 laryngeal attacks. The mean ± SD change in MSCS score was -1.1 ± 0.73 and -1.6 ± 0.68 at 4 and 24 hours, respectively. The mean ± SD TOS was 73.5 ± 35.8 and 85.5 ± 27.8 at 4 and 24 hours, respectively. Median time to significant improvement was 185 minutes (95% confidence interval, 167-226). One attack required intubation. Four treatment-emergent serious adverse events were reported, including 2 HAE attacks that resulted in hospitalization and 2 anaphylactic reactions. One of these reactions required treatment with epinephrine, but both patients recovered fully. There were no deaths. CONCLUSION: In this large attack series, ecallantide was effective for treatment of laryngeal HAE attacks. There is a risk of hypersensitivity, including anaphylaxis, consistent with product labeling. As such, ecallantide should be administered under the supervision of a health care professional. TRIAL REGISTRATION: clinicaltrials.gov Identifiers: not applicable for EDEMA2 (trial was conducted before implementation of registration requirements); NCT00262080 for EDEMA3, NCT00457015 for EDEMA4, and NCT00456508 for DX-88/19.


Asunto(s)
Angioedemas Hereditarios/tratamiento farmacológico , Antiinflamatorios no Esteroideos/administración & dosificación , Péptidos/administración & dosificación , Adolescente , Adulto , Anciano , Anafilaxia/tratamiento farmacológico , Anafilaxia/etiología , Angioedemas Hereditarios/fisiopatología , Antiinflamatorios no Esteroideos/efectos adversos , Progresión de la Enfermedad , Epinefrina/uso terapéutico , Femenino , Humanos , Inyecciones Subcutáneas , Laringe/efectos de los fármacos , Laringe/fisiopatología , Masculino , Persona de Mediana Edad , Péptidos/efectos adversos , Riesgo , Resultado del Tratamiento , Adulto Joven
10.
Ann Allergy Asthma Immunol ; 110(3): 168-72, 2013 Mar.
Artículo en Inglés | MEDLINE | ID: mdl-23548526

RESUMEN

BACKGROUND: Effective treatment of acute attacks is critical in managing hereditary angioedema (HAE). Ecallantide, a plasma kallikrein inhibitor, is approved for the treatment of HAE attacks. Occasionally, a second dose is needed when treating attacks of HAE. OBJECTIVE: To evaluate the characteristics of HAE attacks requiring a second dose (dose B) of ecallantide. METHODS: Data from all ecallantide clinical trials (EDEMA2, EDEMA4, and DX-88/19) that allowed an open-label dose B were included in this analysis. Patient and attack characteristics potentially predictive of dose B after ecallantide were analyzed by logistic regression. A multivariate model was built using a backward selection process, incorporating variables from the univariate model with P < .20 and removing factors with the highest P value until only significant (P < .05) factors remained. RESULTS: The analysis included 732 ecallantide-treated HAE attacks in 179 patients. Dose B was required in 88 attacks (12.0%), most (80.5%) for incomplete response. By attack location, 31 of 325 abdominal attacks (9.5%), 17 of 158 laryngeal attacks (10.8%), and 40 of 242 peripheral attacks (16.5%) required dose B. On the basis of the univariate analysis, baseline severity (odds ratio = 1.33, P = .15) and peripheral attack (odds ratio = 1.80, P = .01) were identified as potential predictive factors; abdominal attacks had an inverse correlation (odds ratio = 0.64, P = .055). However, the multivariate analysis identified only peripheral attacks as statistically significantly correlated (P < .05) with dose B requirement. CONCLUSION: A single, 30-mg dose of ecallantide was effective for most HAE attacks (88.0%). Patients with peripheral attacks of HAE were more likely to require a second dose of ecallantide after 4 hours. TRIAL REGISTRATION: clinicaltrials.gov Identifiers: not applicable for EDEMA2 (trial was conducted before registration requirements were implemented), NCT00457015 for EDEMA4, and NCT00456508 for DX-88/19.


Asunto(s)
Angioedemas Hereditarios/diagnóstico , Angioedemas Hereditarios/tratamiento farmacológico , Antiinflamatorios no Esteroideos/administración & dosificación , Péptidos/administración & dosificación , Adulto , Angioedemas Hereditarios/fisiopatología , Antiinflamatorios no Esteroideos/efectos adversos , Progresión de la Enfermedad , Cálculo de Dosificación de Drogas , Femenino , Humanos , Masculino , Persona de Mediana Edad , Péptidos/efectos adversos , Valor Predictivo de las Pruebas , Pronóstico , Resultado del Tratamiento , Adulto Joven
11.
Allergy Asthma Proc ; 34(2): 155-61, 2013.
Artículo en Inglés | MEDLINE | ID: mdl-23484891

RESUMEN

Hereditary angioedema (HAE) is a rare disorder characterized by recurrent attacks of potentially life-threatening edema. The plasma kallikrein inhibitor ecallantide is approved for treatment of acute HAE attacks. This study evaluates the efficacy and safety of ecallantide for treatment of multiple HAE episodes in the DX-88/19 (continuation) study. Patients received 30 mg of subcutaneous ecallantide for acute HAE attack symptoms, with no limit on number of episodes treated. Primary end point was change in patient-reported mean symptom complex severity (MSCS) score at 4 hours. Additional end points included change in MSCS score at 24 hours, treatment outcome score (TOS) at 4 and 24 hours, and time to response. Safety parameters included adverse events. Statistical analyses were conducted on qualifying treatment episodes (those with ≥12 patients). One hundred forty-seven patients received treatment for 625 episodes; analyses were conducted through 13 treatment episodes. Across 13 episodes at 4 hours, mean change in MSCS score ranged from -1.04 to -1.36, and mean TOSs ranged from 56.2 to 79.8. Median time to onset of sustained improvement ranged from 59 to 113 minutes. There was no indication of reduced efficacy with repeated ecallantide use. No new safety signals were detected. Eight patients (5.4%) reported potential hypersensitivity reactions, six of whom met the definition of anaphylaxis based on National Institute of Allergy and Infectious Diseases criteria. Ecallantide is effective for acute recurrent HAE attacks and maintains its efficacy and safety during multiple treatment episodes in patients with HAE. Potential hypersensitivity reactions were consistent with prior reports.


Asunto(s)
Angioedemas Hereditarios/tratamiento farmacológico , Calicreínas/antagonistas & inhibidores , Péptidos/administración & dosificación , Enfermedad Aguda , Adolescente , Adulto , Anciano , Niño , Determinación de Punto Final , Femenino , Humanos , Inyecciones Subcutáneas , Masculino , Persona de Mediana Edad , Péptidos/efectos adversos , Péptidos/uso terapéutico , Resultado del Tratamiento , Adulto Joven
12.
J Allergy Clin Immunol Pract ; 11(11): 3391-3399.e3, 2023 11.
Artículo en Inglés | MEDLINE | ID: mdl-37544429

RESUMEN

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.


Asunto(s)
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/epidemiología
13.
medRxiv ; 2023 Jun 05.
Artículo en Inglés | MEDLINE | ID: mdl-37333367

RESUMEN

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.

14.
J Asthma ; 49(5): 450-5, 2012 Jun.
Artículo en Inglés | MEDLINE | ID: mdl-22540879

RESUMEN

OBJECTIVE: A possible association between long-acting beta-agonists (LABA) and severe asthma exacerbations including death remains controversial. We examined whether LABA in the setting of combination therapy with inhaled corticosteroids (ICS) increase the risk of near-fatal asthma in children using a case-control study design. METHODS: Medical records from admissions for asthma exacerbations in children 4-18 years of age during the 2005 calendar year at Children's Hospital of Pittsburgh of UPMC were reviewed. Cases and controls were determined by pediatric intensive care unit (PICU) and floor admission, respectively. Exposure was defined by LABA use in combination with ICS versus ICS alone. RESULTS: Records from 85 PICU and 96 pediatric floor admissions were reviewed. LABA use in combination with ICS did not increase the risk of PICU admission (odds ratio 1.07, 95% CI 0.46-2.52) compared to ICS only without LABA. After adjusting for demographics, asthma severity, history of PICU admissions, and concurrent infection, LABA/ICS use still did not increase the risk of PICU admission (adjusted odds ratio 0.84, 95% CI 0.26-2.76) compared to ICS alone. There were no deaths and five intubations within the study period. CONCLUSIONS: The combination of LABA and ICS did not appear to increase the risk of near-fatal asthma in children.


Asunto(s)
Corticoesteroides/uso terapéutico , Agonistas de Receptores Adrenérgicos beta 2/efectos adversos , Agonistas de Receptores Adrenérgicos beta 2/uso terapéutico , Albuterol/efectos adversos , Albuterol/uso terapéutico , Antiasmáticos/uso terapéutico , Asma/tratamiento farmacológico , Administración por Inhalación , Adolescente , Antiasmáticos/efectos adversos , Asma/fisiopatología , Estudios de Casos y Controles , Niño , Preescolar , Quimioterapia Combinada , Femenino , Humanos , Unidades de Cuidado Intensivo Pediátrico , Modelos Logísticos , Masculino , Estudios Retrospectivos , Factores Socioeconómicos
15.
Allergy Asthma Proc ; 33(2): 178-85, 2012.
Artículo en Inglés | MEDLINE | ID: mdl-22525395

RESUMEN

Hereditary angioedema (HAE) is characterized by episodic attacks of edema. HAE is caused by low levels of the protein C1 esterase inhibitor, which inhibits plasma kallikrein, the enzyme responsible for converting high-molecular-weight kininogen to bradykinin. Unregulated production of bradykinin leads to the characteristic clinical symptoms of swelling and pain. Ecallantide is a novel plasma kallikrein inhibitor effective for treatment of acute HAE attacks. This study was designed to analyze the efficacy of ecallantide for treating HAE attacks by attack location, attack severity, patient gender, and body mass index (BMI). An analysis of integrated data from two double-blind, placebo-controlled trials of ecallantide for treatment of acute HAE attacks was undertaken. For the purpose of analysis, symptoms were classified by anatomic location and, for each location, by the patient-assessed severity of the attack. Efficacy versus placebo was examined using two validated patient-reported outcomes: treatment outcome score and mean symptom complex severity score. One hundred forty-three attacks were analyzed (73 ecallantide and 70 placebo). Ecallantide was equally effective in both male and female subjects. Ecallantide had decreased efficacy for patients with BMI > 30 kg/m(2). Ecallantide showed efficacy for treatment of severe and moderate attacks, and was effective for abdominal, internal head and neck, external head and neck, and cutaneous locations. In summary, ecallantide is effective for treatment of acute HAE attacks of different symptom locations and severity; outcomes were similar for men and women. However, the standard dose was less effective for obese patients.


Asunto(s)
Angioedemas Hereditarios/tratamiento farmacológico , Angioedemas Hereditarios/fisiopatología , Inhibidores Enzimáticos/uso terapéutico , Calicreínas/antagonistas & inhibidores , Péptidos/uso terapéutico , Índice de Masa Corporal , Método Doble Ciego , Femenino , Humanos , Masculino , Ensayos Clínicos Controlados Aleatorios como Asunto , Índice de Severidad de la Enfermedad , Resultado del Tratamiento
16.
Acad Pediatr ; 22(1): 47-54, 2022.
Artículo en Inglés | MEDLINE | ID: mdl-34256177

RESUMEN

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.


Asunto(s)
Asma , Revisión de Utilización de Seguros , Asma/epidemiología , Niño , Estudios de Cohortes , Humanos , Lactante , Estudios Retrospectivos , Factores de Riesgo
17.
World Allergy Organ J ; 15(3): 100627, 2022 Mar.
Artículo en Inglés | MEDLINE | ID: mdl-35497649

RESUMEN

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.

18.
J Allergy Clin Immunol Pract ; 10(7): 1864-1873.e10, 2022 07.
Artículo en Inglés | MEDLINE | ID: mdl-34848381

RESUMEN

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.


Asunto(s)
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 farmacológico
19.
Pediatr Allergy Immunol ; 22(7): 671-5, 2011 Nov.
Artículo en Inglés | MEDLINE | ID: mdl-21449988

RESUMEN

OBJECTIVE: When to evaluate a child for possible immune deficiency is a challenge, as many children have frequent infections for which they are treated with antibiotics. We aimed to describe the clinical characteristics of children evaluated for possible primary immunodeficiency in a specialist clinic. We specifically aimed to evaluate widely promulgated 'warning signs of primary immunodeficiency' and to evaluate the relationship between primary immunodeficiency and atopy. METHODS: A retrospective analysis of 141 children who underwent testing for possible primary immunodeficiency was undertaken. RESULTS: Thirty-two (23%) children were diagnosed with an underlying primary immunodeficiency, and published warning signs were neither sensitive nor specific for primary immunodeficiency. Patients with allergy as determined by the presence of antigen-specific IgE were more likely to be diagnosed with immunodeficiency. CONCLUSIONS: Widely promulgated warning signs did not distinguish between patients with and without primary immunodeficiency. Likewise, primary immunodeficiency and allergy may coexist.


Asunto(s)
Síndromes de Inmunodeficiencia/diagnóstico , Síndromes de Inmunodeficiencia/fisiopatología , Agammaglobulinemia/diagnóstico , Agammaglobulinemia/epidemiología , Distribución por Edad , Niño , Preescolar , Inmunodeficiencia Variable Común/diagnóstico , Inmunodeficiencia Variable Común/epidemiología , Diagnóstico Diferencial , Femenino , Humanos , Hipersensibilidad Inmediata/diagnóstico , Hipersensibilidad Inmediata/epidemiología , Hipersensibilidad Inmediata/inmunología , Deficiencia de IgA/diagnóstico , Deficiencia de IgA/epidemiología , Inmunoglobulina E/sangre , Síndromes de Inmunodeficiencia/epidemiología , Masculino , Distribución por Sexo
20.
Allergy Asthma Proc ; 32(5): 372-6, 2011.
Artículo en Inglés | MEDLINE | ID: mdl-22195690

RESUMEN

Several studies have shown that sensitization to cockroach and mouse allergens is correlated with presence and severity of asthma, especially among children living in inner cities. This study evaluated the prevalence of positive skin testing to indoor allergens in the Pittsburgh area and the association with asthma and eczema. A retrospective analysis was performed of 540 children from the Pittsburgh area who underwent skin testing to indoor allergens. Presence of asthma and eczema were determined by parent and/or physician report. Asthma and eczema are not significantly more frequent among children who had positive skin testing to cockroaches or mice. However, asthma was more common among children who had positive skin testing to dogs (odds ratio [OR], 1.4; 95% CI, 1.23-1.65), cats (OR, 1.4; 95% CI, 1.21-1.58), and dust mites (OR, 1.2; 95% CI, 1.03-1.37). Eczema was more common in children who had positive skin testing to cats (OR, 1.5; 95% CI, 1.14-2.02). Both asthma (OR, 1.4; 95% CI, 1.18-1.58) and eczema (OR, 1.4; 95% CI, 1.07-1.92) were more prevalent among children with any positive skin test. We did not find that sensitization to cockroaches or mice was correlated with the diagnosis or asthma or eczema in the Pittsburgh area. However, sensitization to any allergen, and to cats and/or dogs specifically, was associated with diagnosis of both asthma and eczema. Our result suggests that allergic sensitization is associated with these diseases, but the implicated allergens may vary.


Asunto(s)
Alérgenos/inmunología , Asma/epidemiología , Eccema/epidemiología , Adolescente , Animales , Asma/inmunología , Gatos , Niño , Preescolar , Cucarachas/inmunología , Perros , Eccema/inmunología , Femenino , Humanos , Masculino , Ratones , Pennsylvania , Prevalencia , Pyroglyphidae/inmunología , Medición de Riesgo , Pruebas Cutáneas
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