Allergy & Asthma Proceedings最新文献

{"title":"Real-world outcomes in patients with hereditary angioedema prescribed lanadelumab versus other prophylaxis.","authors":"John Anderson,Daniel Soteres,Raffi Tachdjian,Jennifer Mellor,Lucy Earl,Hannah Connolly,Kieran Wynne-Cattanach,Kellyn Moran,Krystal Sing,Bob G Schultz,Salome Juethner","doi":"10.2500/aap.2024.45.240046","DOIUrl":"https://doi.org/10.2500/aap.2024.45.240046","url":null,"abstract":"BACKGROUNDHereditary angioedema (HAE) is a rare genetic disorder characterized by painful, debilitating, and potentiallyfatal swelling attacks. Lanadelumab is approved as long-term prophylaxis (LTP) in patients with HAE. However, realworld data on LTP use in patients with HAE are limited.OBJECTIVETo describe clinical characteristics, attack history, and quality of life (QoL) of patients with HAE type I/II whowere receiving lanadelumab or other LTPs.METHODSData were drawn from the Adelphi HAE Disease Specific Program, a cross-sectional survey of HAE physicians conducted in the United States from July to November 2021. Physician-reported disease characteristics, HAE attack frequency, and QoL were compared among patients receiving lanadelumab or other LTPs for at least 12 months.RESULTSPhysicians reported data for 144 patients, of whom 29 had received lanadelumab and 115 had received another prophylaxis for at least 12 months. The mean +/- standard deviation number of attacks in the previous 12 months was loweramong patients receiving lanadelumab than other LTPs (2.3 +/- 3.1 versus 3.4 +/- 2.8, respectively; p = 0.075). Although bothgroups had similar current disease activity and severity, more patients receiving lanadelumab versus other LTPs had high disease activity (51.7% versus 12.5%, respectively; p < 0.0001) and disease severity rated as severe (51.7% versus 16.1%, respectively; p = 0.0001) at diagnosis. Physicians reported that more patients who received lanadelumab had good or very good QoL(72.4%) than those receiving other LTPs (36.5%) (p = 0.003).CONCLUSIONAnalysis of these findings suggests lower attack frequency, lower symptomatic impact, and better QoL inpatients treated with lanadelumab than another prophylaxis in a real-world setting.","PeriodicalId":520163,"journal":{"name":"Allergy & Asthma Proceedings","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142269570","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Protective antibody concentrations in primary immunodeficiency following infusion with 5% or 10% intravenous immunoglobulin.","authors":"Martyn Paddick,Kim Clark,Eric Wolford,John More","doi":"10.2500/aap.2024.45.240033","DOIUrl":"https://doi.org/10.2500/aap.2024.45.240033","url":null,"abstract":"BACKGROUNDInadequate production of immunoglobulin G (IgG) antibodies renders patients with primary immunodeficiencysusceptible to infection by numerous pathogens, some of which can lead to severe asthma exacerbation and possible death. These patients who are immunocompromised are often reliant on intravenous immunoglobulin (IVIG) therapies, which provide passive antibodies against various respiratory pathogens, including measles virus and encapsulated bacteria.OBJECTIVEWe conducted a subanalysis of data from a multicenter, multinational, phase III, open-label bioequivalencestudy to compare protective concentrations of IgG antibodies provided by a 5% and a 10% IVIG product in patients with primaryimmunodeficiency.METHODSPatients on stable 21- or 28-day regimens of previous IVIG products were assigned to receive study treatment (adults: 5% IVIG and 10% IVIG; children: 10% IVIG) at doses of 300-800 mg/kg per infusion. Trough concentrations of total IgG, IgG subclasses, measles-neutralizing antibodies, and IgG against Haemophilus influenzae type b and Streptococcus pneumoniae serotypes were evaluated.RESULTSA total of 48 patients (33 adults ages 16-55 years; 15 children ages 2-15 years) were enrolled and received treatment.No statistically significant differences in trough concentrations of total IgG, IgG subclasses, measles neutralizing antibodies, or IgG directed at encapsulated bacteria were observed between the 5% and 10% formulations in analyses by age (adult or pediatric) or infusion schedule (every 21 or 28 days). All evaluated patients had trough IgG concentrations above accepted thresholds for protection against disease.CONCLUSIONThese findings support the conclusion that, at dose levels and infusion schedules prescribed in clinical practice,this 5% and 10% IVIG product provided consistent, predictable, and bioequivalent IgG concentrations for adult and pediatricpatients with primary immunodeficiency disease. Both formulations delivered trough antibody concentrations of total IgG, measles- neutralizing antibodies, and antibodies against encapsulated bacteria that are above thresholds accepted as protective.","PeriodicalId":520163,"journal":{"name":"Allergy & Asthma Proceedings","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142269580","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Diagnostic tests for primary immunodeficiency disorders: Classic and genetic testing.","authors":"Natchanun Klangkalya,Thomas A Fleisher,Sergio D Rosenzweig","doi":"10.2500/aap.2024.45.240051","DOIUrl":"https://doi.org/10.2500/aap.2024.45.240051","url":null,"abstract":"Primary immunodeficiency diseases encompass a variety of genetic conditions characterized by a compromised immune system and typically results in increased susceptibility to infection. In fact, they also manifest as autoimmunity, autoinflammation, atopic diseases, and malignancy. Currently, the number of recognized monogenic primary immunodeficiency disorders is set at ∼500 different entities, owing to the exponential use of unbiased genetic testing for disease discovery. In addition, the prevalence of secondary immunodeficiency has also been on the rise due to the increased use of immunosuppressive drugs to treat diseases based on immune dysregulation, an increase in the number of individuals undergoing hematopoietic stem cell transplantation, and other chronic medical conditions, including autoimmunity. Although the clinical symptoms of immunodeficiency disorders are broad, an early diagnosis and tailored management strategies are essential to mitigate the risk of infections and prevent disease-associated morbidity. Generally, the medical history and physical examination can provide useful information that can help delineate the possibility of immune defects. In turn, this makes it feasible to select focused laboratory tests that identify immunodeficiency disorders based on the specific immune cells and their functions or products that are affected. Laboratory evaluation involves quantitative and functional classic testing (e.g., leukocyte counts, serum immunoglobulin levels, specific antibody titers in response to vaccines, and enumeration of lymphocyte subsets) as well as genetic testing (e.g., individual gene evaluation via Sanger sequencing or unbiased evaluation based on next-generation sequencing). However, in many cases, a diagnosis also requires additional advanced research techniques to validate genetic or other findings. This article updates clinicians about available laboratory tests for evaluating the immune system in patients with primary immunodeficiency disorders. It also provides a comprehensive list of testing options, organized based on different components of host defense.","PeriodicalId":520163,"journal":{"name":"Allergy & Asthma Proceedings","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142269574","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Essentials of an immunodeficiency primer: A practical reference for the allergist/immunologist and the allergy-immunology fellow-in-training (FIT).","authors":"Joseph A Bellanti,Russell A Settipane","doi":"10.2500/aap.2024.45.240062","DOIUrl":"https://doi.org/10.2500/aap.2024.45.240062","url":null,"abstract":"","PeriodicalId":520163,"journal":{"name":"Allergy & Asthma Proceedings","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142269572","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Isotype deficiencies (IgG subclass and selective IgA, IgM, IgE deficiencies).","authors":"Kamran Imam,Jenny Huang,Andrew A White","doi":"10.2500/aap.2024.45.240055","DOIUrl":"https://doi.org/10.2500/aap.2024.45.240055","url":null,"abstract":"Background: Immunoglobulin G (IgG) subclass deficiencies and isolated IgA, IgM, IgE deficiencies have all been described in the literature with variable prevalence. Methods: These isotype deficiencies have a variable presentation from asymptomatic to recurrent infections resistant to prophylactic antibiotics. Results: Atopic disorders and autoimmune diseases are common comorbidities. IgE deficiency has been associated with impaired vaccine response and an increased risk of malignancy, particularly in patients with no allergic comorbidities and those with non-common variable immunodeficiency (CVID) humoral immunodeficiency, IgM deficiency, IgG2 deficiency, and CD4 lymphopenia. Conclusion: Close monitoring for malignancy should be strongly considered for these patients who are at risk. Treatment is variable and may include antimicrobial therapies for illnesses and prophylactic antibiotics in select patients, and immunoglobulin replacement can be considered for patients with refractory, recurrent infections.","PeriodicalId":520163,"journal":{"name":"Allergy & Asthma Proceedings","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142269577","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Immunodeficiency: Overview of primary immune regulatory disorders (PIRDs).","authors":"Cody S Nelson,Carolyn H Baloh","doi":"10.2500/aap.2024.45.240070","DOIUrl":"https://doi.org/10.2500/aap.2024.45.240070","url":null,"abstract":"Primary immune regulatory disorders (PIRD) comprise a heterogeneous group of monogenic disorders that impact homeostatic control of inflammation and immune tolerance. Patients with a PIRD typically present to medical care with symptoms of autoimmunity or hyperinflammation as the dominant clinical feature, symptoms that include fever, rash, lymphadenopathy, organomegaly, arthritis, and colitis are commonplace. Notably, PIRDs are a distinct entity from primary immune deficiency disorders (PIDD), which are primarily defined by a qualitative or quantitative defect in immunity, which manifests as a susceptibility to recurrent infections. PIDDs and PIRDs can be challenging to differentiate because the clinical presentations can be similar. Red flags for PIRDs include multiple autoimmune diagnoses in the same patient, chronic lymphadenopathy, hepatomegaly, and/or splenomegaly, chronic colitis, hemophagocytic lymphohistiocytosis (HLH), Epstein Barr virus (EBV) susceptibility, recurrent or persistent fever, vasculitis, and sterile inflammation. For simplicity in this brief review, we limit our discussion of PIRDs to the following categories multiple autoimmune diseases, immune dysregulation with colitis, disorders with HLH and/or EBV susceptibility, autoinflammatory syndromes, type 1 interferonopathies, and disorders of sterile inflammation. Diagnosing a PIRD requires a broad immune evaluation for both immune system deficiencies and inflammation, along with genetic testing. Given the complex nature of these diseases, treatment often requires a team of subspecialists. Treatment, depending on the specific diagnosis, may be somewhat empiric with nonspecific immune modulators, symptom-directed therapies, and, in severe cases, hematopoietic stem cell transplantation; however, with the increasing number of biologics available, we are often able to use targeted immune therapy or even gene therapy.","PeriodicalId":520163,"journal":{"name":"Allergy & Asthma Proceedings","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142269571","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Primary Immunodeficiency: Specific antibody deficiency with normal IgG.","authors":"Vishaka R Hatcher,Veronica C Alix,Tasha S Hellu,Meredith M Schuldt","doi":"10.2500/aap.2024.45.240057","DOIUrl":"https://doi.org/10.2500/aap.2024.45.240057","url":null,"abstract":"Specific antibody deficiency (SAD) is a common primary immunodeficiency disorder that should be considered in older children and adults with recurrent and/or severe sinopulmonary infections. The diagnosis is characterized by inadequate antibody response to polysaccharide vaccine, specifically, pneumococcal, with normal responses to protein antigens and normal levels of serum immunoglobulins as well as immunoglobulin G (IgG) subclasses. The underlying mechanism for SAD is not completely elucidated. It is understood that young children have limited polysaccharide responsiveness, which develops with increased age. Due to this phenomenon, the consensus is that there is adequate immune maturity after age 2 years, which is the earliest for the SAD diagnosis to be established. There remains a lack of consensus on thresholds for polysaccharide nonresponse, and there are several commercial laboratories that measure a range of serotypes, with the recommendation for patients to have their diagnostic evaluation with serotype testing both before vaccination and after vaccination to be conducted by the same laboratory. Once a diagnosis has been made, the management of SAD is based on the clinical severity. Clinicians may consider prophylactic antibiotics as well as immunoglobulin replacement. These patients should be closely followed up, with the possibility of discontinuation of IgG replacement after 12 to 24 months. Children are more likely to demonstrate resolution of SAD than are adolescents and adults. Patients with SAD may also progress to a more severe immunodeficiency; therefore, continued monitoring remains a crucial principle of practice in the care of patients with SAD.","PeriodicalId":520163,"journal":{"name":"Allergy & Asthma Proceedings","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142269579","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Genetically-determined defects of T cell development.","authors":"Luigi D Notarangelo","doi":"10.2500/aap.2024.45.240028","DOIUrl":"https://doi.org/10.2500/aap.2024.45.240028","url":null,"abstract":"Genetically determined defects of T-cell development comprise a heterogeneous group of conditions characterized by peripheral T-cell lymphopenia due to impaired intrathymic differentiation of T-cell progenitors. Collectively, these conditions are typically referred to as severe combined immune deficiency (SCID). In some cases (leaky SCID), residual function of the defective gene allows partial T-cell development. The vast majority of SCID disorders are due to genetic defects that affect the T-cell differentiation potential of hematopoietic stem cells, through a variety of mechanisms. However, some forms of SCID reflect impaired development or function of thymic stromal cells. A lack of peripheral T cells leads to increased susceptibility to severe infections since early in life. SCID is inevitably fatal unless immune reconstitution is achieved, most often through hematopoietic cell transplantation. Enzyme replacement therapy, gene therapy, and thymus implantation represent other forms of treatment in selected cases. The availability of newborn screening has greatly facilitated prompt recognition of SCID, which allows statistically significant improvement in survival after hematopoietic cell transplantation.","PeriodicalId":520163,"journal":{"name":"Allergy & Asthma Proceedings","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142269575","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Immunodeficiency: Quantitative and qualitative phagocytic cell defects.","authors":"David Mickey,Juanita Valdes Camacho,Adil Khan,David Kaufman","doi":"10.2500/aap.2024.45.240049","DOIUrl":"https://doi.org/10.2500/aap.2024.45.240049","url":null,"abstract":"The immune system is divided into two major branches: innate and adaptive. The innate immune system is the body's first line of defense and rapidly responds in a nonspecific manner to various microorganisms, foreign materials, or injuries. Phagocytes, which include macrophages, monocytes, and neutrophils, are innate immune cells that can surround and kill microorganisms, ingest foreign material, and remove dead cells. They also indirectly boost both innate and adaptive immune responses through various activation signals. Phagocytic defects characteristically lead to fungal and bacterial infections of the respiratory tract, lymph nodes, skin, and other organ systems, and they are commonly associated with inflammatory bowel disease. This primer will review high-yield innate defects of phagocytic cells, including defects of respiratory (oxidative) burst, defects of neutrophil migration, cyclic and severe congenital neutropenias and associated disorders, and other phagocyte defect disorders.","PeriodicalId":520163,"journal":{"name":"Allergy & Asthma Proceedings","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142269576","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Quality of life in primary immunodeficiency: Its contribution to shared decision-making and patient outcomes.","authors":"Mark Ballow","doi":"10.2500/aap.2024.45.240056","DOIUrl":"https://doi.org/10.2500/aap.2024.45.240056","url":null,"abstract":"Quality of life (QOL) measures have become increasingly important in the management of patients with complex diseases. There are a number of instruments to measure QOL that include broad areas or domains of physical, psychological, social, spiritual, and environmental issues. The number of potential domains plus the large number of items within each domain have led to the development of a large variety of QOL instruments and of different approaches by using both health-related and non-health-related factors. Health-related QOL (HRQOL) measures have been incorporated into clinical trials to assess changes in the patient's perspective on his or her disease and the effects of treatment. An important aspect of these HR-QOL instruments is that these questionnaires are patient reported and usually self-administered. The life-long therapy of intravenous immunoglobulin (IVIG) or subcutaneous immunoglobulin (SCIG) can be a challenge to the patient, his or her family and the physician. A number of patient-reported surveys have been published that examined treatment satisfaction and the comparison between IVIG and SCIG replacement therapy in patients with primary immunodeficiency. This review explores the use of HR-QOL surveys in patient preferences for mode and route of immunoglobulin therapy and site of care. Shared decision-making will be explored to emphasize the importance of this approach in optimizing patient care and compliance.","PeriodicalId":520163,"journal":{"name":"Allergy & Asthma Proceedings","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142269578","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}