Annals of Allergy Asthma & Immunology最新文献

{"title":"Gene therapy for inborn errors of immunity: current clinical progress.","authors":"Sathi Wijeyesinghe, Javier Chinen","doi":"10.1016/j.anai.2025.02.026","DOIUrl":"https://doi.org/10.1016/j.anai.2025.02.026","url":null,"abstract":"<p><p>Hematopoietic stem cell transplant has been the single curative treatment for inborn errors of immunity (IEI) and is recommended for the most severe IEI conditions, such as severe combined immunodeficiency (SCID). However, adverse outcomes primarily due to histocompatibility differences between the donor and the patient are still of concern. Progress in genetic and molecular mechanisms, as well as new technology to insert DNA sequences in cell genomes has allowed the development of strategies to treat genetic diseases by correcting the gene defect in patients' cells. This technology is named gene therapy. Gene therapy approaches being developed for IEI are mediated by gene insertion, using a retroviral vector, or by gene editing, using a combination of a nuclease and a DNA template. After the unexpected occurrence of oncogenesis associated with the initial retroviral vector designs, significant advances have led to successful gene therapy clinical trials for three forms of SCID, which demonstrated the safety and efficacy of this approach. Active preclinical and clinical studies are ongoing for diverse IEI, including chronic granulomatous disease, leukocyte adhesion deficiency, severe congenital neutropenia, Wiskott-Aldrich syndrome, X-linked agammaglobulinemia and familial forms of hemophagocytic lymphohistiocytosis.</p>","PeriodicalId":50773,"journal":{"name":"Annals of Allergy Asthma & Immunology","volume":" ","pages":""},"PeriodicalIF":5.8,"publicationDate":"2025-03-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143587933","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Dupilumab Efficacy in Relation to Changes in Club Cell Secretory Protein 16.","authors":"Yui Murai, Toshiyuki Koya, Hiroki Koda, Wakana Uji, Moe Tanaka, Masahiro Endo, Kyoichiro Ohshima, Takahiro Matsuda, Hiroshi Ueno, Ami Aoki, Kenjiro Shima, Yosuke Kimura, Toshiaki Kikuchi","doi":"10.1016/j.anai.2025.02.019","DOIUrl":"https://doi.org/10.1016/j.anai.2025.02.019","url":null,"abstract":"<p><strong>Background: </strong>Although the performance of dupilumab in severe asthma has been evaluated, the detailed mechanism underlying its effect remains unclear.</p><p><strong>Objective: </strong>We analyzed the effect of dupilumab on serum club cell secretory protein 16 (CC16).</p><p><strong>Methods: </strong>Twenty-five patients who were administered dupilumab and underwent computed tomography (CT) before and approximately 4 months after the introduction of dupilumab were included. Clinical and CT parameters before and after dupilumab administration, such as mucus plug score and wall area measurement, were compared along with serum CC16 levels. The correlation between the clinical background and treatment effects was also examined.</p><p><strong>Results: </strong>The number of mucus plugs and airway wall area decreased significantly after dupilumab introduction. The number of mucus plugs was positively correlated with both age and serum immunoglobulin E (IgE) levels. The number of mucus plugs and airway wall area was inversely correlated with percent of predicted forced expiratory volume in one second (FEV1) and maximal mid-expiratory flow (MMEF). Dupilumab treatment resulted in a significant increase in serum CC16 levels and led to a significant improvement in asthma symptoms, quality of life (QOL) scores, FEV1, and exacerbation frequency. Additionally, changes in CC16 were significantly correlated with changes in the fraction of exhaled nitric oxide, IgE, QOL score, FEV1, MMEF, and mucus plug score.</p><p><strong>Conclusion: </strong>These data suggest that dupilumab improves symptoms and respiratory functions by altering the airway epithelial environment.</p>","PeriodicalId":50773,"journal":{"name":"Annals of Allergy Asthma & Immunology","volume":" ","pages":""},"PeriodicalIF":5.8,"publicationDate":"2025-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143568686","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical burden, treatment, and disease control in patients with chronic spontaneous urticaria","authors":"Marc A. Riedl MD, MS ,&nbsp;Dhaval Patil MS ,&nbsp;Jonathan Rodrigues MD ,&nbsp;Merin Kuruvilla MD ,&nbsp;Tara Raftery PhD ,&nbsp;Irina Pivneva PhD ,&nbsp;Jason Doran MBA ,&nbsp;Arthur Voegel MA ,&nbsp;James Signorovitch PhD ,&nbsp;Gil Yosipovitch MD","doi":"10.1016/j.anai.2024.12.008","DOIUrl":"10.1016/j.anai.2024.12.008","url":null,"abstract":"<div><h3>Background</h3><div>Chronic spontaneous urticaria (CSU) is an unpredictable inflammatory skin condition with substantial clinical burden that affects 0.23% to 0.78% of the US population.</div></div><div><h3>Objective</h3><div>To describe the incidence and prevalence of patients with a record of CSU diagnosis, treatment patterns, disease control, and clinical and economic burden in a US cohort of patients with CSU.</div></div><div><h3>Methods</h3><div>Adults with a record of CSU diagnosis within the US HealthVerity claims database were eligible. Age- and gender-adjusted prevalence/incidence rates were calculated for January 2017 to December 2022. Clinical characteristics were described during the 1 year before CSU diagnosis (baseline) and the time after (follow-up). Proxy events representing uncontrolled CSU (any record of prescriptions for corticosteroids, biologics, or immunosuppressants [excluding all antihistamines and over-the-counter medication] or any CSU-related inpatient admissions or emergency department or urgent care visits) were used to identify patients with uncontrolled CSU. Health care resource utilization (HCRU) and health care costs were described.</div></div><div><h3>Results</h3><div>Overall, 200,298 patients were followed-up for a median of 2.3 years after diagnosis. Estimated cumulative prevalence of diagnosed CSU was 0.57% (women: 0.80%; men: 0.32%). The average annual incidence rate was 0.08%. Corticosteroids were the most prescribed treatment during follow-up among the 166,195 patients prescribed at least 1 treatment (94.3%). Proxy events were observed in 59.1% of the patients. HCRU and health care costs increased from baseline in patients with uncontrolled CSU during follow-up.</div></div><div><h3>Conclusion</h3><div>Of patients with CSU who were prescribed treatment, more than 50% experienced uncontrolled CSU, which was associated with increased HCRU and health care costs.</div></div>","PeriodicalId":50773,"journal":{"name":"Annals of Allergy Asthma & Immunology","volume":"134 3","pages":"Pages 324-332.e4"},"PeriodicalIF":5.8,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142856532","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Further evidence for the roles of computed tomography in managing severe asthma","authors":"Naoya Tanabe MD, PhD","doi":"10.1016/j.anai.2024.12.014","DOIUrl":"10.1016/j.anai.2024.12.014","url":null,"abstract":"","PeriodicalId":50773,"journal":{"name":"Annals of Allergy Asthma & Immunology","volume":"134 3","pages":"Pages 251-252"},"PeriodicalIF":5.8,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143529834","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mild asthma—What matters to patients and parents","authors":"Emma Greimann MD ,&nbsp;George E. Freigeh MD, MA ,&nbsp;Rachel P. Wettenstein BS ,&nbsp;Belinda Nelson MSW, PhD ,&nbsp;Laurie M. Carpenter MSW ,&nbsp;Arjun Mohan MBBS ,&nbsp;Alan Baptist MD, MPH","doi":"10.1016/j.anai.2024.11.017","DOIUrl":"10.1016/j.anai.2024.11.017","url":null,"abstract":"<div><h3>Background</h3><div>Mild asthma has received less attention despite accounting for most of patient with asthma. However, asthma complications including hospitalizations and progressive loss of lung function frequently occur in such patients. The priorities of patients with mild asthma are unknown, hindering the ability to advance care.</div></div><div><h3>Objective</h3><div>To identify patient and parent perspectives on the definition of mild asthma, treatment preferences, concerns and goals of care.</div></div><div><h3>Methods</h3><div>Participants with self-defined mild/intermittent asthma were recruited using emails distributed through the Allergy &amp; Asthma Network and Allergy Foundation of America. A demographic survey and measures of asthma control/quality of life were completed. Focus groups consisting of approximately 5 participants and a focus group leader were conducted.</div></div><div><h3>Results</h3><div>A total of 20 patients and 20 parents of children with mild asthma participated. Focus groups revealed significant variability in the definition and treatment preferences. Frequency of symptoms appears to be a key driver in treatment decisions for mild asthma, and those with infrequent symptoms were opposed to the addition of an inhaled corticosteroid to albuterol. Use of recommended asthma monitoring strategies such as asthma action plans or peak flow meters was low among adults. Participants desired more education from their providers regarding asthma remission and long-term complications associated with mild asthma.</div></div><div><h3>Conclusion</h3><div>There is significant heterogeneity in the definition and treatment preferences among patients and parents of those with mild asthma. Shared decision making between patients and providers is necessary to personalize medical decisions in those with mild asthma.</div></div>","PeriodicalId":50773,"journal":{"name":"Annals of Allergy Asthma & Immunology","volume":"134 3","pages":"Pages 290-295.e1"},"PeriodicalIF":5.8,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142752149","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Do no harm: An ethical approach to refractory anaphylaxis to chemotherapy","authors":"Timothy M. Buckey MD, MBE","doi":"10.1016/j.anai.2024.11.024","DOIUrl":"10.1016/j.anai.2024.11.024","url":null,"abstract":"","PeriodicalId":50773,"journal":{"name":"Annals of Allergy Asthma & Immunology","volume":"134 3","pages":"Pages 247-248"},"PeriodicalIF":5.8,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142774572","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"High prevalence of eosinophilic gastrointestinal disorders in patients with atopic disease","authors":"Alexandra A. Weir MS ,&nbsp;Onyinye I. Iweala MD, PhD ,&nbsp;Evan S. Dellon MD, MPH","doi":"10.1016/j.anai.2024.12.001","DOIUrl":"10.1016/j.anai.2024.12.001","url":null,"abstract":"","PeriodicalId":50773,"journal":{"name":"Annals of Allergy Asthma & Immunology","volume":"134 3","pages":"Pages 362-364"},"PeriodicalIF":5.8,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142803196","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Utility of using thoracic computed tomography to measure lung function decline and detect unsuspected emphysema in severe asthma","authors":"Arthur F. Gelb MD,&nbsp;Donald P. Tashkin MD","doi":"10.1016/j.anai.2024.11.007","DOIUrl":"10.1016/j.anai.2024.11.007","url":null,"abstract":"","PeriodicalId":50773,"journal":{"name":"Annals of Allergy Asthma & Immunology","volume":"134 3","pages":"Pages 253-254"},"PeriodicalIF":5.8,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143529422","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Performance of 5-domain rhinology-focused symptom score with dupilumab in chronic rhinosinusitis with nasal polyps","authors":"Kirsten Stewart MBChB ,&nbsp;Chris RuiWen Kuo MD ,&nbsp;Rory Chan PhD ,&nbsp;Brian Lipworth MD","doi":"10.1016/j.anai.2024.11.001","DOIUrl":"10.1016/j.anai.2024.11.001","url":null,"abstract":"","PeriodicalId":50773,"journal":{"name":"Annals of Allergy Asthma & Immunology","volume":"134 3","pages":"Pages 360-362"},"PeriodicalIF":5.8,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142632216","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Understanding advances in inborn errors of immunity for the practicing allergist-immunologist","authors":"Rebecca R. Saff MD, PhD ,&nbsp;Marcus S. Shaker MD, MS","doi":"10.1016/j.anai.2024.12.006","DOIUrl":"10.1016/j.anai.2024.12.006","url":null,"abstract":"","PeriodicalId":50773,"journal":{"name":"Annals of Allergy Asthma & Immunology","volume":"134 3","pages":"Pages 245-246"},"PeriodicalIF":5.8,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143529841","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}