Allergy Asthma and Clinical Immunology最新文献

{"title":"CTLA-4 haploinsufficiency presenting with chronic myeloid leukemia, bullous pemphigoid, and PLA2R-positive membranous nephropathy: a case report.","authors":"Nouraldeen Deeb, Salahaldeen Deeb, Fares Sayed Ahmed, Younis Malik Younis Amro, Abdallah Altell, Aliaa Khalili","doi":"10.1186/s13223-026-01011-7","DOIUrl":"10.1186/s13223-026-01011-7","url":null,"abstract":"<p><p>BACKGROUND: Cytotoxic T-lymphocyte–associated protein 4 (CTLA-4) haploinsufficiency is a primary immune-regulatory disorder characterized by T-cell overactivation and multisystem autoimmunity. Malignancies, particularly lymphomas and gastric cancer, have been reported in approximately 12–13% of individuals with CTLA-4 haploinsufficiency, but associations with chronic myeloid leukemia (CML) are very rarely described. We report a young adult with genetically and functionally confirmed CTLA-4 haploinsufficiency who developed a triad of BCR-ABL1–positive chronic myeloid leukemia (CML), bullous pemphigoid, and PLA2R-positive membranous glomerulonephritis (MGN), highlighting diagnostic and management lessons across immunology, hematology, dermatology, and nephrology. CASE PRESENTATION: A 21-year-old woman with Hashimoto thyroiditis and iron-deficiency anemia was found to have BCR-ABL1–positive CML. Within months she developed recurrent infections and paraneoplastic blistering disease. Whole-exome sequencing detected a heterozygous CTLA4 c.529_530insT (p.Tyr177LeufsTer2) frameshift; flow cytometry showed reduced CTLA-4 expression, establishing functional haploinsufficiency. Six months later she presented with edema and nephrotic-range proteinuria; serology revealed markedly elevated anti-PLA2R antibodies, and kidney biopsy confirmed immune-complex MGN. Management included a TKI (imatinib/nilotinib as indicated over the course), low-dose corticosteroids, monthly IVIG for infection/immune modulation, and rituximab for bullous disease and MGN. This resulted in molecular remission of CML, resolution of skin lesions, and sustained normalization of proteinuria over 12 months. CONCLUSIONS: The convergence of CML, bullous pemphigoid, and PLA2R-positive MGN in CTLA-4 haploinsufficiency broadens the clinical phenotype and underscores the importance of considering inborn errors of immunity in young adults with refractory, multisystem autoimmunity and hematologic abnormalities. Early genetic diagnosis can guide targeted immunomodulation and organ preservation, and multidisciplinary care is essential for optimal outcomes. </p>","PeriodicalId":51302,"journal":{"name":"Allergy Asthma and Clinical Immunology","volume":" ","pages":"5"},"PeriodicalIF":2.4,"publicationDate":"2026-01-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12879419/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146047289","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Attack rate reductions following berotralstat initiation among US patients with hereditary angioedema in the real-world.","authors":"Mark Davis-Lorton, Raffi Tachdjian, Lorena Lopez-Gonzalez, Sean D MacKnight, Ramya Ramasubramanian, François Laliberté, Patrick Gillard, Meri LiVecchi, Sandra Nestler-Parr, William R Lumry","doi":"10.1186/s13223-025-01005-x","DOIUrl":"10.1186/s13223-025-01005-x","url":null,"abstract":"<p><strong>Background: </strong>Hereditary angioedema (HAE) causes recurring swelling attacks, leading to substantial disease burden. This real-world, retrospective study aimed to evaluate HAE attack rates before and after berotralstat initiation stratified by patients' baseline attack frequency.</p><p><strong>Methods: </strong>Specialty Pharmacy data from Optime Care, Inc. (12/2020-01/2024), the sole berotralstat dispenser in the United States, were analyzed. Eligible patients had  ≥ 2 berotralstat dispensings (first =  index) and ≥ 1 self-assessment of attacks at baseline (90-days pre-index) and follow-up (first-to-last dispensing). Patients were classified by HAE type (based on laboratory measurements) and baseline attacks (≥ 5, 2-4, 1, and 0 attacks/month). Follow-up attack rates were compared with baseline using mean differences, confidence intervals, and P-values. Among those with 0 baseline attacks/month, proportions with 0 follow-up attacks/month were assessed.</p><p><strong>Results: </strong>Of 390 eligible patients with HAE with C1 esterase inhibitor (C1INH) deficiency (HAE-C1INH) and 311 with HAE with normal C1INH (HAE-nC1INH), most were female (64.1% and 77.5%) with mean ages of 39.3 and 48.1 years, respectively. Mean attack rates decreased from 2.50 to 0.79 attacks/month (HAE-C1INH) and from 4.59 to 1.68 attacks/month (HAE-nC1INH) at 12-months of berotralstat treatment (both P < 0 .001), with sustained reductions at 18-months. Patients with ≥ 1 baseline attack/month experienced significantly lower attack rates after berotralstat initiation. Among patients with 0 baseline attacks/month, most also maintained 0 attacks/month in each follow-up interval (HAE-C1INH: 70-85%; HAE-nC1INH: 61-81%).</p><p><strong>Conclusion: </strong>Berotralstat was associated with significant and sustained reductions in attack rates among patients with HAE, regardless of baseline attack rate. Patients with 0 baseline attacks/month maintained low attack rates following berotralstat initiation.</p>","PeriodicalId":51302,"journal":{"name":"Allergy Asthma and Clinical Immunology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-01-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13007376/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146047379","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-term effectiveness and safety of lanadelumab in Canadian patients with hereditary angioedema: a subanalysis of the EMPOWER study.","authors":"Stephen D Betschel, Hugo Chapdelaine, Remi Gagnon, M Dawn Goodyear, Paul K Keith, Ahmed El-Zoeiby, Natalie Khutoryansky, Daniel N Castaner","doi":"10.1186/s13223-025-01007-9","DOIUrl":"10.1186/s13223-025-01007-9","url":null,"abstract":"<p><strong>Background: </strong>The EMPOWER Study (NCT03845400) was a phase 4, observational, non-interventional, multicenter study evaluating the real-world effectiveness and safety of lanadelumab in patients with hereditary angioedema (HAE). This subanalysis focused on lanadelumab effectiveness and safety in patients from Canada.</p><p><strong>Methods: </strong>Enrollment included patients with HAE due to C1 inhibitor deficiency. Patients were categorized as \"newly treated\" or \"established on lanadelumab\" if they had received fewer than four or at least four doses before enrollment, respectively.</p><p><strong>Results: </strong>Thirteen patients from Canada were enrolled: seven newly treated and six established on lanadelumab; these patients received lanadelumab for a mean (standard deviation [SD]) duration of 315 (224) and 536 (366) days, respectively, during the study. In newly treated patients, the mean (SD) observed HAE attack rate decreased after lanadelumab initiation from 1.5 (2.9) to 0.3 (0.6) attacks per month. In patients established on lanadelumab, the mean (SD) observed HAE attack rate was 0.1 (0.1) attacks per month throughout the study. Most HAE attacks were mild or moderate in severity (newly treated, 94%; established on lanadelumab, 100%). No study discontinuations were attributed to treatment-emergent adverse events. All treatment-emergent adverse events were non-severe, non-serious, and unrelated to lanadelumab treatment.</p><p><strong>Conclusions: </strong>Lanadelumab lowered the HAE attack rate among newly treated Canadian patients and maintained a low attack rate among those previously established on lanadelumab, demonstrating robust effectiveness across both patient groups. Effectiveness, tolerability, and safety of lanadelumab were consistent with the overall population from the EMPOWER Study, supporting the use of lanadelumab as a first-line long-term prophylactic treatment for patients with HAE in Canada.</p><p><strong>Trial registration: </strong>NCT03845400, registered February 19, 2019.</p>","PeriodicalId":51302,"journal":{"name":"Allergy Asthma and Clinical Immunology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-01-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13011278/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146020434","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Abstracts of the 14th C1-inhibitor Deficiency and Angioedema Workshop.","authors":"","doi":"10.1186/s13223-025-00992-1","DOIUrl":"10.1186/s13223-025-00992-1","url":null,"abstract":"","PeriodicalId":51302,"journal":{"name":"Allergy Asthma and Clinical Immunology","volume":"21 Suppl 2","pages":"55"},"PeriodicalIF":2.4,"publicationDate":"2026-01-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12763819/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145896991","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"From guidelines to global impact: updates to address disparities in hereditary angioedema care.","authors":"Ankur Kumar Jindal, Philip H Li","doi":"10.1186/s13223-025-01008-8","DOIUrl":"10.1186/s13223-025-01008-8","url":null,"abstract":"<p><p>Hereditary angioedema (HAE) is a rare, potentially life-threatening genetic disorder characterized by recurrent subcutaneous and/or submucosal edema, most commonly caused by deficiency in C1-esterase inhibitor. There has been a paradigm shift in treatment goals—from merely managing acute attacks to improving or normalizing quality of life, thanks to the availability and accessibility of modern, HAE-specific medications. However, for a large majority of countries in the world, HAE-specific medications are still not available or accessible. The correspondence on ‘The International/Canadian Hereditary Angioedema Guideline’ tries to address global disparities in HAE care, highlight the key challenges in low- and middle-income countries and provide potentially viable and equitable solution to address disparities.</p>","PeriodicalId":51302,"journal":{"name":"Allergy Asthma and Clinical Immunology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-12-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13122898/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145769933","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Health-related quality of life in hereditary angioedema patients treated with subcutaneous C1 inhibitor (C1-INH) therapy in Canada.","authors":"Susan Waserman, Gina Lacuesta, Amin S Kanani, Gord L Sussman, Harold Kim, Hugo Chapdelaine, Hadi A Goubran Messiha, Chrystyna Kalicinsky, Adil Adatia, Belinda Yap, Marie-France Dansereau, Nataly Tanios, Rami El-Sayegh, Adriana Martin, Hachem Mahfouz, Maye Machnouk, Paul K Keith","doi":"10.1186/s13223-025-00991-2","DOIUrl":"10.1186/s13223-025-00991-2","url":null,"abstract":"<p><strong>Background: </strong>Hereditary angioedema (HAE) therapies have demonstrated improvements in clinical symptoms and health-related quality of life (HR-QoL) in patients with HAE. Specific causes for improvement in HR-QoL have not been sufficiently explored in Canada.</p><p><strong>Objective: </strong>To understand patient perspectives on the burden of HAE and their experiences taking subcutaneous plasma-derived C1 inhibitor concentrate (SC-pdC1INH), and to investigate changes in HR-QoL domains which include physical, mental, emotional, functional, social, and medical well-being.</p><p><strong>Methods: </strong>A qualitative research design was employed using telephone interviews from May to July 2023 and a quantitative self-administered Angioedema QoL questionnaire (AE-QoL), which has a recall period of 4 weeks. A conceptual map was generated from the interviews that illustrated HR-QoL domains most important to patients.</p><p><strong>Results: </strong>This study included 20 patients (50% female; mean age of 51 years; 85% HAE Type 1/2). Patients reported more controlled HAE since starting SC-pdC1INH, with fewer attacks (85%) and even no/nearly no attacks (60%) in the past year. Patients reported emotional benefits (reduced stress/anxiety regarding potential attacks [75%], and increased confidence in managing HAE [95%]). Positive impacts also included increased productivity/less missed days from work/school (40%) and improved physical ability/well-being (50%). The total mean AE-QoL Score was 34 (range 0-87; SD 24.9), indicating a small effect of HAE on HR-QoL in the 4-week pre-interview period.</p><p><strong>Conclusion: </strong>Overall, patients on long-term prophylaxis with SC-pdC1INH reported improvements in many HR-QoL domains, but most significantly relief of stress and anxiety associated with HAE, and improvement in social and physical well-being.</p>","PeriodicalId":51302,"journal":{"name":"Allergy Asthma and Clinical Immunology","volume":" ","pages":"53"},"PeriodicalIF":2.4,"publicationDate":"2025-12-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12717758/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145710263","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Oral corticosteroid overexposure: characterizing oral corticosteroid use in patients with chronic rhinosinusitis with nasal polypS in Canada (ACTIONS) results.","authors":"Yvonne Chan, Marie-Noëlle Corriveau, Jeffrey Beach, Jenna Reynolds, Koyo Usuba, Danya Thayaparan, Ali Tehrani, Juejing Ling, Huijuan Yang, Andrew Thamboo","doi":"10.1186/s13223-025-00989-w","DOIUrl":"10.1186/s13223-025-00989-w","url":null,"abstract":"<p><strong>Background: </strong>Chronic rhinosinusitis with nasal polyps (CRSwNP) is a type 2 inflammatory condition, often treated with oral corticosteroids (OCS). OCS overexposure can increase the risk of adverse effects, highlighting the need for better treatment strategies including timely biologic therapy use. This study explored OCS use patterns in patients with severe CRSwNP in Canada pre-biologic initiation.</p><p><strong>Methods: </strong>This retrospective, real-world study used IQVIA's Private Drug Plan (PDP) claims database to examine OCS use patterns 0-24 months pre-initiation of biologic therapy in patients with inferred CRSwNP in Canada (n = 747). Outcomes included OCS use patterns (proportions, claims per patient and by prescribing physician specialty, overexposure [≥ 1000 mg annually, prednisone-equivalent]), demographics, and stratified by comorbid asthma.</p><p><strong>Results: </strong>Of 747 patients indexed from the PDP, 81.0% had ≥ 1 OCS claim(s), with a median (interquartile range [IQR]) of 3.0 (4.0) claims per patient. Median (IQR) total OCS dose over 24 months was 1020 (1420) mg; 41.5% of patients experienced OCS overexposure. OCS was primarily prescribed by general practitioners (35.9%) and otolaryngologists (32.8%). Patients had a median (IQR) age of 51 (18) years at index, 52.1% were male, and most were from Ontario (48.7%). OCS use was highest in patients with comorbid severe asthma.</p><p><strong>Conclusions: </strong>This study highlights OCS overexposure in patients with CRSwNP pre-biologic initiation. Enhancing physician and patient awareness of appropriate OCS use and timely biologic initiation could reduce the risk of OCS-related adverse effects and improve CRSwNP management, benefiting long-term patient outcomes through shared decision-making. Trial registration GSK study number 221872.</p>","PeriodicalId":51302,"journal":{"name":"Allergy Asthma and Clinical Immunology","volume":" ","pages":"51"},"PeriodicalIF":2.4,"publicationDate":"2025-12-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12687512/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145679344","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical relevant Bruton's X-linked tyrosine kinase deficiency in a female with extreme X-chromosome inactivation.","authors":"Sundus M NoorSaeed, Abdulaziz S Alrafiaah, Manar Alghamdi, Adam J Shapiro, Christine McCusker","doi":"10.1186/s13223-025-01002-0","DOIUrl":"10.1186/s13223-025-01002-0","url":null,"abstract":"<p><strong>Background: </strong> X-linked agammaglobulinemia (XLA) is an inborn error of immunity resulting from mutations in the BTK gene. It is an X-linked inherited disease that almost exclusively affects males, while females are usually carriers of the disease. However, certain genetic conditions can lead to XLA disease expression in females. We report a 14-year-old girl who was diagnosed with XLA from a pathogenic BTK variant and skewed X chromosome inactivation (XCI).</p><p><strong>Case presentation: </strong>A 14-year-old female Ukrainian refugee was referred to the respirology clinic at the Montreal Children's Hospital with an abnormal chest radiograph found during her immigration medical screening process in Canada. She was reported to be previously well until the age of 11 years when she was diagnosed with pneumonia following SARS-CoV-2 infection. She subsequently developed recurrent pneumonias, persistent productive cough, and chronic sinusitis with polyposis. Laboratory investigations showed severely reduced serum immunoglobulin G, A, and M concentrations of 0.78 g/L, < 0.10 g/L, and 0.21 g/L, respectively. Flow cytometry for lymphocyte subsets showed a complete absence of circulating B cells in peripheral blood. Next-generation sequencing panel for inborn errors or immunity revealed a heterozygous pathogenic variant in BTK (c.862C > T, p.Arg288Trp). Analysis of XCI revealed markedly skewed inactivation (99:1), resulting in predominant expression of the mutant pathogenic BTK allele. These findings support the clinical diagnosis of X-linked agammaglobulinemia manifesting in a female patient due to skewed XCI.</p><p><strong>Conclusions: </strong>Female carriers of pathogenic mutations in BTK may develop clinically important disease as a result of extreme random X inactivation.</p>","PeriodicalId":51302,"journal":{"name":"Allergy Asthma and Clinical Immunology","volume":" ","pages":"4"},"PeriodicalIF":2.4,"publicationDate":"2025-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12777174/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145656098","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Isolated goat and sheep milk allergy in an adult in the absence of bovine sensitization.","authors":"Michael Aw, Gordon Sussman","doi":"10.1186/s13223-025-00996-x","DOIUrl":"10.1186/s13223-025-00996-x","url":null,"abstract":"<p><p>Cow's milk (CM) allergy is the most common food allergy in children, with cross-reactivity to goat and sheep milk (GSM) representing a common outcome due to high protein homology. However, isolated GSM allergy in CM-tolerant individuals is rare and poorly documented in the literature. We describe a 27-year-old male of Mediterranean origin who experienced two episodes of anaphylaxis after consuming sheep and goat cheese, despite regular CM consumption. Skin prick testing confirmed sensitization to GSM but not CM. This report contributes to the growing evidence of adult-onset isolated GSM allergy. Isolated GSM allergy typically presents later in life and may be underrecognized due to limited clinical awareness. We review the potential for species-specific mammalian milk protein allergy, likely mediated by structural differences in casein proteins. Clinicians should be cognisant to consider GSM sensitization in patients presenting with allergic reactions to dairy products, especially in multicultural populations with increasing GSM dietary exposure.</p>","PeriodicalId":51302,"journal":{"name":"Allergy Asthma and Clinical Immunology","volume":" ","pages":"3"},"PeriodicalIF":2.4,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12771896/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145656152","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"NLRP3 inflammasome expression in pediatric asthma: sputum-based insights, inflammatory mechanisms, and targeted therapeutic strategies.","authors":"Mohammed AbuBaha, Samia Aldwaik, Bara Abubaha, Anwar Zahran, Dana Sandouka, Kareem Istetieh, Husam Hamshary, Mohammad Abushehadeh, Sarah Saife, Nadeen Sandoqah","doi":"10.1186/s13223-025-01001-1","DOIUrl":"10.1186/s13223-025-01001-1","url":null,"abstract":"<p><p>Asthma is a common chronic inflammatory disease in children. Pediatric asthma has a wide range of immunologic phenotypes and different treatment responses. Recent data from various studies suggest that the NOD-like receptor family pyrin domain-containing 3 (NLRP3) inflammasome, a cytosolic multiprotein complex, has a central role in innate immunity, may be implicated in pediatric asthma pathogenesis, especially in the neutrophilic corticosteroid-resistant type. This review article investigates the mechanism of NLRP3 inflammasome activation as well as its role in airway inflammation and its expression in pediatric asthma based on sputum studies. The findings emphasize an association between elevated NLRP3 levels and poor asthma control. The need for novel treatments is important because Current using therapies such as corticosteroids and biologics demonstrate variable efficacy. According to data, their efficacy heavily depends on the underlying inflammatory phenotype. Biologics such as mepolizumab, benralizumab, and dupilumab are well known for their corticosteroid-sparing effects especially in cases of severe eosinophilic asthma. even though, their therapeutic benefits are limited when it comes to neutrophilic or steroid-resistant phenotypes. NLRP3 inhibitors are new, promising treatments which emerged recently and show potential capability in reducing airway inflammation in animal models. Furthermore, NLRP3-driven inflammation appears to play a role not only in asthma but also in inflammatory bowel disease and juvenile idiopathic arthritis, indicating a wider relevance for therapies that target the inflammasome pathway. Although promising data, application of this data in clinical practice is still challenging due to many causes, including diagnostic challenges, ethical considerations in trials involving children, and the lack of approved NLRP3 inhibitors for use in children. More research is required and essential to confirm that NLRP3 could be used as a biomarker or therapeutic target in pediatric asthma.</p>","PeriodicalId":51302,"journal":{"name":"Allergy Asthma and Clinical Immunology","volume":" ","pages":"2"},"PeriodicalIF":2.4,"publicationDate":"2025-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12771812/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145642432","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}