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Developing a patient journey map to improve care and experience in Chinese patients with hereditary angioedema 制定患者旅程图以改善中国遗传性血管性水肿患者的护理和经验
IF 4.3 2区 医学 Q2 ALLERGY Pub Date : 2026-02-01 DOI: 10.1016/j.waojou.2026.101333
Yin Wang MM , Yangxue Fu PhD , Hao Chen PhD , Jin Liu MD , Qingxiu Xu MD , Yaqi Yang MD , Si Zhang BS , Jing Cheng MD , Rongfei Zhu MD, PhD

Background

Hereditary angioedema (HAE) is a rare disorder that imposes a substantial burden on patient health and quality of life. Although international studies have highlighted specific aspects of the HAE care continuum, comprehensive evidence on patient experiences in China remains scarce.

Methods

We conducted a single-center, mixed-methods study to develop the first HAE Patient Journey Map (HAE-PJM) in China. Fifteen adult patients with HAE were recruited for structured interviews (July–November 2024), complemented by quantitative assessments using the Hospital Anxiety and Depression Scale (HADS) and the Angioedema Control Test (AECT). Stakeholder validation was obtained through expert–patient groups.

Results

Patients experienced a mean diagnostic delay of 16.3 years, with 80% reporting misdiagnosis and high rates of inappropriate interventions, underscoring systemic deficiencies in early recognition. Pre-diagnosis, activity limitations, and anxiety were common; genetic and economic anxieties persisted even after treatment initiation despite improvements in HADS and AECT scores. Prophylactic regimens achieved superior disease control compared with on-demand therapy, yet access barriers—including cost, reimbursement, and drug availability—remained prevalent. Expert-patient group validation confirmed the relevance of a phased patient journey map and emphasized multidisciplinary roles, including physicians, nurses, navigators, and societal support systems.

Conclusion

This study provides the first systematic HAE-PJM in China, integrating qualitative and quantitative evidence to delineate critical touchpoints and bottlenecks in HAE care. To address these identified challenges, we propose a “4T″ framework (Testing, Teaching, Therapeutic Monitoring, and Team working). These findings highlight urgent needs for standardized diagnostic pathways, integrated psychosocial and financial support, and access-oriented care models.
背景:遗传性血管性水肿(HAE)是一种罕见的疾病,对患者的健康和生活质量造成了严重的负担。尽管国际研究强调了HAE治疗连续性的具体方面,但关于中国患者经历的综合证据仍然很少。方法:我们开展了一项单中心、混合方法研究,在中国开发首个HAE患者旅程地图(HAE- pjm)。我们招募了15名患有HAE的成年患者进行结构化访谈(2024年7月至11月),并辅以使用医院焦虑和抑郁量表(HADS)和血管性水肿控制测试(AECT)进行定量评估。通过专家-患者分组获得利益相关者验证。结果患者平均诊断延迟16.3年,80%报告误诊,干预不当率高,突出了早期识别的系统性缺陷。诊断前、活动受限和焦虑是常见的;即使在治疗开始后,尽管HADS和AECT评分有所改善,遗传和经济焦虑仍然存在。与按需治疗相比,预防性治疗方案取得了更好的疾病控制效果,但获取障碍——包括成本、报销和药物可得性——仍然普遍存在。专家-患者组验证确认了分阶段患者旅程图的相关性,并强调了多学科作用,包括医生、护士、导航员和社会支持系统。本研究提供了中国第一个系统的HAE- pjm,整合了定性和定量证据来描述HAE护理的关键接触点和瓶颈。为了解决这些已确定的挑战,我们提出了“4T″框架(测试,教学,治疗监测和团队合作)”。这些发现突出表明,迫切需要标准化的诊断途径、综合的社会心理和经济支持以及面向可及性的护理模式。
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引用次数: 0
Delayed hypersensitivity reactions to taxanes: Clinical presentation and outcomes of post-medication and rapid drug desensitization 紫杉烷的迟发性超敏反应:临床表现和用药后及快速药物脱敏的结果
IF 4.3 2区 医学 Q2 ALLERGY Pub Date : 2026-02-01 DOI: 10.1016/j.waojou.2025.101157
Rosalaura Villarreal-González MD, PhD , Leslie Astrid De la Fuente MD , Alexia Moreno-Silvestre MD , María Guadalupe Durán-Hernández MD , Diego Armando Sánchez-Alvarado MD , David Hernández-Barajas MD , Oscar Vidal-Gutiérrez MD, PhD

Background

Taxanes are widely used in solid tumors but may induce delayed hypersensitivity reactions (DHRs), potentially compromising treatment. The aim was to characterize taxane-related DHRs and evaluate the role of post-medication and rapid drug desensitization (RDD) in treatment continuation.

Methods

A retrospective, observational study at University Hospital Dr. José Eleuterio González, in Monterrey, Mexico, from January 2020 to May 2025. Patients who developed delayed HSRs following taxane administration were included. After an intravenous challenge test, a post-medication and/or a 3-bag, 12-step rapid drug desensitization (RDD) protocol was applied, adjusted per patient tolerance.

Results

Among 4217 patients, 325 (7.7%) developed HSRs; 28 (8.8%) were delayed. Nine cases (32.1%) were attributed to taxanes: 66.6% paclitaxel and 33.4% docetaxel. Most patients were female (88.9%), with a mean age of 52.3 years. The predominant clinical presentation was maculopapular exanthema (77.8%), mainly involving the scalp. Mean time to HSR was 3 days post-infusion, during the second chemotherapy cycle. All patients received 3–7 days of post-medication; 7 of them tolerated the new administration of the drug, while the other 2 underwent a 3-bag, 12-step desensitization.

Conclusions

Delayed drug hypersensitivity reactions (DHRs) are a T cell-mediated immune response that occur more than 6 h after exposure, leading to treatment discontinuation or alternative therapies. Management remains poorly defined; however, our findings suggest that pre-medication and RDD may safely support continued chemotherapy. To our knowledge, this is the most extensive reported series of taxane-related DHRs managed with post-medication and RDD in Hispanic population phenotype.
紫杉烷广泛应用于实体瘤,但可能诱发迟发性超敏反应(DHRs),可能影响治疗。目的是表征紫杉烷相关的dhr,并评估用药后和快速药物脱敏(RDD)在治疗持续中的作用。方法2020年1月至2025年5月在墨西哥蒙特雷大学医院jos Eleuterio博士González进行回顾性观察研究。紫杉烷给药后发生迟发性HSRs的患者也包括在内。在静脉激发试验后,应用用药后和/或3袋、12步快速药物脱敏(RDD)方案,根据患者耐受性进行调整。结果4217例患者中,325例(7.7%)发生hsr;延误28例(8.8%)。紫杉醇类药物9例(32.1%),其中紫杉醇66.6%,多西紫杉醇33.4%。患者以女性为主(88.9%),平均年龄52.3岁。主要临床表现为黄斑丘疹(77.8%),主要累及头皮。在第二个化疗周期中,平均到HSR时间为输注后3天。所有患者均在给药后3-7 d接受治疗;其中7人对新药物耐受,而另外2人接受了3袋12步脱敏治疗。结论迟发性药物超敏反应(DHRs)是一种T细胞介导的免疫反应,发生在暴露后6小时以上,导致治疗中断或替代治疗。管理仍然定义不清;然而,我们的研究结果表明,药物前治疗和RDD可以安全地支持继续化疗。据我们所知,这是西班牙裔人群表型中紫杉烷相关dhr与用药后和RDD管理的最广泛报道系列。
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引用次数: 0
Clinical features of hereditary angioedema involving the gastrointestinal tract: A retrospective analysis 累及胃肠道的遗传性血管性水肿的临床特征:回顾性分析
IF 4.3 2区 医学 Q2 ALLERGY Pub Date : 2026-02-01 DOI: 10.1016/j.waojou.2026.101252
Haiyuan Ma MSC , Danping Zheng MD , Yangdi Wang MD , Chunyang Tian MSC , Zhoulin Huang MSC , Shanshan Xiong MD , Yangyang Ke MSC , Ren Mao MD , Yao He MD , Minhu Chen MD , Xuehua Li MD , Baili Chen MD

Background

Hereditary angioedema (HAE) is a rare, severe, disabling, and life-threatening disorder characterized by recurrent and unpredictable edema of skin and mucous membranes. Gastrointestinal edema, often presenting as abdominal pain, is common but frequently misdiagnosed, leading to unnecessary surgeries. This study aims to characterize gastrointestinal edema in HAE patients to improve early diagnosis and guide treatment.

Method

We analyzed 61 patients with HAE who were admitted in our hospital between April 1, 2023 and August 31, 2025. Data on demographic characteristics, clinical manifestations, laboratory findings, imaging results, and treatment outcomes were collected through questionnaires and hospital electronic records.

Result

Among the patients, 86.9% experienced gastrointestinal edema, mostly characterized by abdominal pain (94.3%), nausea (79.2%), and diarrhea (71.7%). Misdiagnosis occurred in 73.6% of cases, and 22.6% underwent unnecessary surgeries. The median age of gastrointestinal edema onset was 16.0 years, with the most severe episodes occurring at a median age of 25.0 years. The median annual frequency of attacks was 4.0/year, with a median severity score of 7.0. Acute attacks showed elevated D-dimer, white blood cells, neutrophils, and hemoglobin. CT imaging frequently revealed intestinal wall thickening and abdominal-pelvic effusion. Lanadelumab demonstrated superior efficacy over danazol in reducing both the frequency and severity of edema attacks, while icatibant significantly shortened the duration of edema episodes.

Conclusion

This study updates the clinical and imaging features of gastrointestinal edema in Chinese patients with HAE, identifies biomarkers of gastrointestinal edema attacks, and highlights the efficacy of lanadelumab and icatibant, aiding timely diagnosis and treatment.
背景:遗传性血管性水肿(HAE)是一种罕见的、严重的、致残的、危及生命的疾病,其特征是皮肤和粘膜复发性和不可预测的水肿。胃肠道水肿,通常表现为腹痛,是常见的,但经常误诊,导致不必要的手术。本研究旨在了解HAE患者胃肠道水肿的特征,以提高早期诊断和指导治疗。方法分析2023年4月1日至2025年8月31日在我院收治的61例HAE患者。通过问卷调查和医院电子记录收集人口统计学特征、临床表现、实验室检查结果、影像学结果和治疗结果的数据。结果86.9%的患者出现胃肠水肿,主要表现为腹痛(94.3%)、恶心(79.2%)和腹泻(71.7%)。误诊率为73.6%,不必要手术率为22.6%。胃肠道水肿发病的中位年龄为16.0岁,最严重发作的中位年龄为25.0岁。年发作频率中位数为4.0次/年,严重程度中位数为7.0分。急性发作表现为d -二聚体、白细胞、中性粒细胞和血红蛋白升高。CT影像常显示肠壁增厚及腹盆腔积液。Lanadelumab在降低水肿发作的频率和严重程度方面优于danazol,而icatibant显著缩短了水肿发作的持续时间。结论本研究更新了中国HAE患者胃肠道水肿的临床和影像学特征,确定了胃肠道水肿发作的生物标志物,突出了lanadelumab和icatibant的疗效,有助于及时诊断和治疗。
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引用次数: 0
Dupilumab in real-world pediatric asthma: Enhanced control and quality of life for children and caregivers Dupilumab在现实儿科哮喘中的应用:增强儿童和护理人员的控制和生活质量
IF 4.3 2区 医学 Q2 ALLERGY Pub Date : 2026-01-18 DOI: 10.1016/j.waojou.2026.101251
Ricardo Martínez-Tenopala MD , Jimena Prieto-Gomez MD , María Julia Rendón-Salazar MD , Tamara Hernández-Hernández MD , Javier Zermeño-Vallet MD , Diego Mauricio Gómez-González MD , Carlos Andrés Gómez-Nuñez MD , Luis Ángel Hernández-Zárate MD , Víctor Gonzalez-Uribe MD, MSc

Background

Dupilumab has demonstrated therapeutic benefits and improved quality of life (QoL) in children with uncontrolled asthma. This real-world study—the first in a Latin-American pediatric population—evaluated its impact on asthma control and QoL in children with moderate-to-severe uncontrolled type 2 asthma and their caregivers.

Methods

We conducted a retrospective observational study of patients aged 4–16 years with moderate-to-severe uncontrolled type 2 asthma (blood eosinophils ≥150 cells/μL or fractional exhaled nitric oxide ≥20 ppb). Eleven patients received dupilumab and 12 received conventional inhaled therapy, with 52-week follow-up. Asthma control was assessed using the 7-item Asthma Control Questionnaire interviewer-administered version (ACQ-7-IA). QoL was evaluated using the Pediatric Asthma Quality of Life Questionnaire interviewer-administered version (PAQLQ[S]-IA) and the Pediatric Asthma Caregiver's Quality of Life Questionnaire (PACQLQ). The primary endpoints were changes in ACQ-7-IA, PAQLQ(S)-IA, and PACQLQ scores, and the proportion achieving clinically significant improvement (≥0.5 points).

Results

Dupilumab significantly improved ACQ-7-IA scores from week 4, sustained through week 52 (least squares mean difference −0.44, 95% CI −0.59 to −0.30; p < 0.001). At week 52, 86% of dupilumab-treated patients achieved clinically significant improvement versus 75% with conventional therapy (p = 0.041). Well-controlled asthma (ACQ-7-IA ≤0.75) was achieved in 73% versus 36% (p < 0.003). Dupilumab also improved PAQLQ(S)-IA and PACQLQ scores from week 24, with 100% of patients and caregivers achieving clinically significant improvement at week 52.

Conclusions

In Latin-American children with moderate-to-severe type 2 asthma, dupilumab was associated with early and sustained improvements in asthma control and QoL for both patients and caregivers, supporting its role as an effective therapeutic option in real-world pediatric practice.
研究背景:dupilumab已经证明了治疗哮喘患儿的益处和改善生活质量(QoL)。这项真实世界的研究-首次在拉丁美洲儿科人群中进行-评估了其对中度至重度未控制的2型哮喘儿童及其护理人员的哮喘控制和生活质量的影响。方法对4-16岁中重度未控制的2型哮喘患者(血嗜酸性粒细胞≥150细胞/μL或呼气一氧化氮≥20 ppb)进行回顾性观察研究。11例患者接受dupilumab治疗,12例接受常规吸入治疗,随访52周。哮喘控制采用7项哮喘控制问卷(ACQ-7-IA)进行评估。生活质量采用儿童哮喘生活质量问卷(PAQLQ[S]-IA)和儿童哮喘护理人员生活质量问卷(PACQLQ)进行评估。主要终点为ACQ-7-IA、PAQLQ(S)-IA和PACQLQ评分的变化,以及达到临床显著改善(≥0.5分)的比例。结果dupilumab从第4周开始显著改善ACQ-7-IA评分,持续到第52周(最小二乘平均差为- 0.44,95% CI为- 0.59至- 0.30;p < 0.001)。在第52周,86%的dupilumab治疗患者获得临床显著改善,而75%的常规治疗患者(p = 0.041)。哮喘(ACQ-7-IA≤0.75)得到良好控制的比例分别为73%和36% (p < 0.003)。从第24周开始,Dupilumab也改善了PAQLQ(S)-IA和PACQLQ评分,100%的患者和护理人员在第52周达到临床显着改善。结论:在拉丁美洲患有中重度2型哮喘的儿童中,dupilumab与患者和护理人员的哮喘控制和生活质量的早期和持续改善相关,支持其作为现实世界儿科实践中有效的治疗选择的作用。
{"title":"Dupilumab in real-world pediatric asthma: Enhanced control and quality of life for children and caregivers","authors":"Ricardo Martínez-Tenopala MD ,&nbsp;Jimena Prieto-Gomez MD ,&nbsp;María Julia Rendón-Salazar MD ,&nbsp;Tamara Hernández-Hernández MD ,&nbsp;Javier Zermeño-Vallet MD ,&nbsp;Diego Mauricio Gómez-González MD ,&nbsp;Carlos Andrés Gómez-Nuñez MD ,&nbsp;Luis Ángel Hernández-Zárate MD ,&nbsp;Víctor Gonzalez-Uribe MD, MSc","doi":"10.1016/j.waojou.2026.101251","DOIUrl":"10.1016/j.waojou.2026.101251","url":null,"abstract":"<div><h3>Background</h3><div>Dupilumab has demonstrated therapeutic benefits and improved quality of life (QoL) in children with uncontrolled asthma. This real-world study—the first in a Latin-American pediatric population—evaluated its impact on asthma control and QoL in children with moderate-to-severe uncontrolled type 2 asthma and their caregivers.</div></div><div><h3>Methods</h3><div>We conducted a retrospective observational study of patients aged 4–16 years with moderate-to-severe uncontrolled type 2 asthma (blood eosinophils ≥150 cells/μL or fractional exhaled nitric oxide ≥20 ppb). Eleven patients received dupilumab and 12 received conventional inhaled therapy, with 52-week follow-up. Asthma control was assessed using the 7-item Asthma Control Questionnaire interviewer-administered version (ACQ-7-IA). QoL was evaluated using the Pediatric Asthma Quality of Life Questionnaire interviewer-administered version (PAQLQ[S]-IA) and the Pediatric Asthma Caregiver's Quality of Life Questionnaire (PACQLQ). The primary endpoints were changes in ACQ-7-IA, PAQLQ(S)-IA, and PACQLQ scores, and the proportion achieving clinically significant improvement (≥0.5 points).</div></div><div><h3>Results</h3><div>Dupilumab significantly improved ACQ-7-IA scores from week 4, sustained through week 52 (least squares mean difference −0.44, 95% CI −0.59 to −0.30; p &lt; 0.001). At week 52, 86% of dupilumab-treated patients achieved clinically significant improvement versus 75% with conventional therapy (p = 0.041). Well-controlled asthma (ACQ-7-IA ≤0.75) was achieved in 73% versus 36% (p &lt; 0.003). Dupilumab also improved PAQLQ(S)-IA and PACQLQ scores from week 24, with 100% of patients and caregivers achieving clinically significant improvement at week 52.</div></div><div><h3>Conclusions</h3><div>In Latin-American children with moderate-to-severe type 2 asthma, dupilumab was associated with early and sustained improvements in asthma control and QoL for both patients and caregivers, supporting its role as an effective therapeutic option in real-world pediatric practice.</div></div>","PeriodicalId":54295,"journal":{"name":"World Allergy Organization Journal","volume":"19 2","pages":"Article 101251"},"PeriodicalIF":4.3,"publicationDate":"2026-01-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146039141","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}
引用次数: 0
Clinical remission in patients with severe eosinophilic asthma treated with benralizumab over 24 months: Post hoc analysis of the ANANKE study benralizumab治疗严重嗜酸性哮喘患者超过24个月的临床缓解:ANANKE研究的事后分析
IF 4.3 2区 医学 Q2 ALLERGY Pub Date : 2026-01-14 DOI: 10.1016/j.waojou.2025.101159
Giorgio Walter Canonica MD , Gianenrico Senna MD , Luisa Brussino MD , Maria Aliani MD , Elena Altieri MD , Pietro Bracciale MD , Maria Filomena Caiaffa MD , Paolo Cameli MD , Marco Caminati MD , Cristiano Caruso MD , Stefano Centanni MD , Fausto De Michele MD , Stefano Del Giacco MD , Fabiano Di Marco MD , Laura Malerba MD , Francesco Menzella MD , Paola Rogliani MD , Micaela Romagnoli MD , Pietro Schino MD , Jan Walter Schroeder MD , Girolamo Pelaia MD

Background

Clinical remission is an emerging treatment goal in severe eosinophilic asthma (SEA). While benralizumab, an anti-IL-5Rα monoclonal antibody, has demonstrated efficacy in SEA, its ability to induce clinical remission in real-life settings over extended follow-up remains underexplored.

Methods

This post hoc analysis of the multicenter, retrospective ANANKE study evaluated clinical remission over 24 months in 167 Italian patients with SEA treated with benralizumab. Remission was defined according to the Severe Asthma Network Italy (SANI) criteria. Complete clinical remission (cCR) required the absence of oral corticosteroid (OCS) use and the presence of 3 criteria: no symptoms, no exacerbations, and stable lung function. Partial clinical remission (pCR) required the absence of OCS use and 2 of the 3 criteria. Outcomes were assessed at 3, 12, and 24 months.

Results

The proportion of patients achieving clinical remission increased over time: 87.2% at 3 months (40.4% pCR, 46.8% cCR), 95.0% at 12 months (17.5% pCR, 77.5% cCR), and 96.1% at 24 months (23.5% pCR, 72.6% cCR). No baseline demographic or clinical characteristics were found to significantly predict remission status. Blood eosinophil counts declined from a mean of 476.7 to 5.2 cells/μL at 24 months.

Conclusion

In this real-world Italian cohort, benralizumab was associated with rapid and sustained clinical remission in patients with SEA over 24 months. The high remission rates observed early and maintained throughout treatment support the role of benralizumab as a disease-modifying therapy and reinforce clinical remission as a meaningful therapeutic goal in SEA.
临床缓解是严重嗜酸性哮喘(SEA)的一个新兴治疗目标。虽然抗il - 5r α单克隆抗体benralizumab已证明对SEA有效,但其在长期随访中诱导临床缓解的能力仍未得到充分探索。该多中心回顾性ANANKE研究的事后分析评估了167例意大利SEA患者接受贝纳利珠单抗治疗后24个月的临床缓解。根据意大利严重哮喘网络(SANI)标准定义缓解。完全临床缓解(cCR)需要不使用口服皮质类固醇(OCS),并存在3个标准:无症状、无恶化和肺功能稳定。部分临床缓解(pCR)要求不使用OCS,满足3项标准中的2项。在3、12和24个月时评估结果。结果达到临床缓解的患者比例随时间增加:3个月时为87.2% (40.4% pCR, 46.8% cCR), 12个月时为95.0% (17.5% pCR, 77.5% cCR), 24个月时为96.1% (23.5% pCR, 72.6% cCR)。没有发现基线人口统计学或临床特征能显著预测缓解状态。24个月时,血嗜酸性粒细胞从平均476.7个细胞/μL下降到5.2个细胞/μL。在这个真实的意大利队列中,benralizumab与SEA患者在24个月以上的快速和持续的临床缓解相关。早期观察到的高缓解率和在整个治疗过程中保持的高缓解率支持benralizumab作为一种疾病改善疗法的作用,并加强临床缓解作为SEA有意义的治疗目标。
{"title":"Clinical remission in patients with severe eosinophilic asthma treated with benralizumab over 24 months: Post hoc analysis of the ANANKE study","authors":"Giorgio Walter Canonica MD ,&nbsp;Gianenrico Senna MD ,&nbsp;Luisa Brussino MD ,&nbsp;Maria Aliani MD ,&nbsp;Elena Altieri MD ,&nbsp;Pietro Bracciale MD ,&nbsp;Maria Filomena Caiaffa MD ,&nbsp;Paolo Cameli MD ,&nbsp;Marco Caminati MD ,&nbsp;Cristiano Caruso MD ,&nbsp;Stefano Centanni MD ,&nbsp;Fausto De Michele MD ,&nbsp;Stefano Del Giacco MD ,&nbsp;Fabiano Di Marco MD ,&nbsp;Laura Malerba MD ,&nbsp;Francesco Menzella MD ,&nbsp;Paola Rogliani MD ,&nbsp;Micaela Romagnoli MD ,&nbsp;Pietro Schino MD ,&nbsp;Jan Walter Schroeder MD ,&nbsp;Girolamo Pelaia MD","doi":"10.1016/j.waojou.2025.101159","DOIUrl":"10.1016/j.waojou.2025.101159","url":null,"abstract":"<div><h3>Background</h3><div>Clinical remission is an emerging treatment goal in severe eosinophilic asthma (SEA). While benralizumab, an anti-IL-5Rα monoclonal antibody, has demonstrated efficacy in SEA, its ability to induce clinical remission in real-life settings over extended follow-up remains underexplored.</div></div><div><h3>Methods</h3><div>This post hoc analysis of the multicenter, retrospective ANANKE study evaluated clinical remission over 24 months in 167 Italian patients with SEA treated with benralizumab. Remission was defined according to the Severe Asthma Network Italy (SANI) criteria. Complete clinical remission (cCR) required the absence of oral corticosteroid (OCS) use and the presence of 3 criteria: no symptoms, no exacerbations, and stable lung function. Partial clinical remission (pCR) required the absence of OCS use and 2 of the 3 criteria. Outcomes were assessed at 3, 12, and 24 months.</div></div><div><h3>Results</h3><div>The proportion of patients achieving clinical remission increased over time: 87.2% at 3 months (40.4% pCR, 46.8% cCR), 95.0% at 12 months (17.5% pCR, 77.5% cCR), and 96.1% at 24 months (23.5% pCR, 72.6% cCR). No baseline demographic or clinical characteristics were found to significantly predict remission status. Blood eosinophil counts declined from a mean of 476.7 to 5.2 cells/μL at 24 months.</div></div><div><h3>Conclusion</h3><div>In this real-world Italian cohort, benralizumab was associated with rapid and sustained clinical remission in patients with SEA over 24 months. The high remission rates observed early and maintained throughout treatment support the role of benralizumab as a disease-modifying therapy and reinforce clinical remission as a meaningful therapeutic goal in SEA.</div></div>","PeriodicalId":54295,"journal":{"name":"World Allergy Organization Journal","volume":"19 2","pages":"Article 101159"},"PeriodicalIF":4.3,"publicationDate":"2026-01-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145957886","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}
引用次数: 0
Prevalence and risk factors of papular urticaria among school children in Cartagena, Colombia 哥伦比亚卡塔赫纳学龄儿童流行性荨麻疹患病率及危险因素
IF 4.3 2区 医学 Q2 ALLERGY Pub Date : 2026-01-13 DOI: 10.1016/j.waojou.2025.101151
Josefina Zakzuk MD, PhD , Juliana Quintero MD, PhD , Devian Parra Eco , Victoria Marrugo MSc , Maria A. Forero Molina MD , Carlos Quiroz MD , Margarita Ochoa-Díaz MD, Esp., MSc, PhD , Doris Gómez MSc, PhD , Elizabeth García MD

Introduction

Papular urticaria (PU) is a chronic allergic reaction to arthropod bites and is a common cause of pediatric dermatologic consultations in tropical regions. This study aimed to assess the prevalence and associated factors of PU among children in Cartagena, Colombia, and to explore the relationship between insect presence and disease presentation.

Methods

Surveys were used to assess living conditions, and the diagnosis was confirmed by medical history and physical examination.

Results

A total of 725 children aged 1–6 years from 30 schools and 13 childcare centers were included, PU was diagnosed in 260 children, resulting in an age- and gender-adjusted prevalence rate of 35.9% (95% CI: 30.3–43.4). Lesions were primarily found on exposed areas, such as the lower limbs (97%) and arms (54%). Children aged 1–2 years (aOR = 3.01, 95% CI: 1.54–5.93, p < 0.001) and 3–4 years (aOR = 1.84, 95% CI: 1.11–3.08, p = 0.02) had a higher risk of PU than those aged 5–6 years. The antecedent of mosquito bites was reported in most PU cases (96.0%) and controls (93.0%), without significant differences between groups. The frequency of mosquito presence at home was similar between case and controls. Mosquito density was higher in the lower socioeconomic strata (p < 0.001). The most commonly identified species were Aedes aegypti (57.7%) and Culex quinquefasciatus (38.2%). Fleas were found in only 3% of the homes.

Conclusion

We identified a high prevalence of PU in the city. Despite the widespread presence of mosquitoes, no association between mosquito bites and PU was observed. Younger age emerged as a risk factor for PU, and socioeconomic disparities influenced higher mosquito density.
丘疹性荨麻疹(PU)是一种对节肢动物叮咬的慢性过敏反应,是热带地区儿童皮肤科会诊的常见原因。本研究旨在评估哥伦比亚卡塔赫纳地区儿童PU患病率及其相关因素,并探讨昆虫存在与疾病表现之间的关系。方法采用问卷调查的方式评估患者的生活状况,并结合病史和体格检查进行诊断。结果共纳入30所学校和13所托幼中心的1 ~ 6岁儿童725名,诊断为PU的儿童260名,经年龄和性别调整的患病率为35.9% (95% CI: 30.3 ~ 43.4)。病变主要发生在暴露部位,如下肢(97%)和手臂(54%)。1 ~ 2岁儿童(aOR = 3.01, 95% CI: 1.54 ~ 5.93, p < 0.001)和3 ~ 4岁儿童(aOR = 1.84, 95% CI: 1.11 ~ 3.08, p = 0.02)发生PU的风险高于5 ~ 6岁儿童。大多数PU病例(96.0%)和对照组(93.0%)报告有蚊虫叮咬史,组间差异无统计学意义。在病例和对照之间,蚊虫在家中出现的频率相似。低社会经济阶层的蚊密度较高(p < 0.001)。最常见的蚊种为埃及伊蚊(57.7%)和致倦库蚊(38.2%)。只有3%的家庭有跳蚤。结论我市PU患病率较高。尽管蚊子广泛存在,但没有观察到蚊子叮咬与PU之间的联系。年龄较小是PU的危险因素,社会经济差异影响了较高的蚊子密度。
{"title":"Prevalence and risk factors of papular urticaria among school children in Cartagena, Colombia","authors":"Josefina Zakzuk MD, PhD ,&nbsp;Juliana Quintero MD, PhD ,&nbsp;Devian Parra Eco ,&nbsp;Victoria Marrugo MSc ,&nbsp;Maria A. Forero Molina MD ,&nbsp;Carlos Quiroz MD ,&nbsp;Margarita Ochoa-Díaz MD, Esp., MSc, PhD ,&nbsp;Doris Gómez MSc, PhD ,&nbsp;Elizabeth García MD","doi":"10.1016/j.waojou.2025.101151","DOIUrl":"10.1016/j.waojou.2025.101151","url":null,"abstract":"<div><h3>Introduction</h3><div>Papular urticaria (PU) is a chronic allergic reaction to arthropod bites and is a common cause of pediatric dermatologic consultations in tropical regions. This study aimed to assess the prevalence and associated factors of PU among children in Cartagena, Colombia, and to explore the relationship between insect presence and disease presentation.</div></div><div><h3>Methods</h3><div>Surveys were used to assess living conditions, and the diagnosis was confirmed by medical history and physical examination.</div></div><div><h3>Results</h3><div>A total of 725 children aged 1–6 years from 30 schools and 13 childcare centers were included, PU was diagnosed in 260 children, resulting in an age- and gender-adjusted prevalence rate of 35.9% (95% CI: 30.3–43.4). Lesions were primarily found on exposed areas, such as the lower limbs (97%) and arms (54%). Children aged 1–2 years (aOR = 3.01, 95% CI: 1.54–5.93, p &lt; 0.001) and 3–4 years (aOR = 1.84, 95% CI: 1.11–3.08, p = 0.02) had a higher risk of PU than those aged 5–6 years. The antecedent of mosquito bites was reported in most PU cases (96.0%) and controls (93.0%), without significant differences between groups. The frequency of mosquito presence at home was similar between case and controls. Mosquito density was higher in the lower socioeconomic strata (p &lt; 0.001). The most commonly identified species were <em>Aedes aegypti</em> (57.7%) and <em>Culex quinquefasciatus</em> (38.2%). Fleas were found in only 3% of the homes.</div></div><div><h3>Conclusion</h3><div>We identified a high prevalence of PU in the city. Despite the widespread presence of mosquitoes, no association between mosquito bites and PU was observed. Younger age emerged as a risk factor for PU, and socioeconomic disparities influenced higher mosquito density.</div></div>","PeriodicalId":54295,"journal":{"name":"World Allergy Organization Journal","volume":"19 2","pages":"Article 101151"},"PeriodicalIF":4.3,"publicationDate":"2026-01-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145957885","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}
引用次数: 0
Increased glucose transporter 1 contributes to epithelial barrier dysfunction in allergic rhinitis 葡萄糖转运蛋白1升高与变应性鼻炎上皮屏障功能障碍有关
IF 4.3 2区 医学 Q2 ALLERGY Pub Date : 2026-01-01 DOI: 10.1016/j.waojou.2025.101158
Cui Xia PhD , Kang Zhu MD , Chao Yu MD , Jingguo Chen MD , Tianxi Gao MD , Yanni Zhang MD

Background

Epithelial barrier impairment is a characteristic pathological hallmark of allergic rhinitis (AR), yet the mechanisms contributing to this dysfunction remain incompletely understood. Our aim is to assess the impact of glucose transporter 1 (GLUT1) on epithelial barrier function in AR.

Methods

We performed proteomics analysis on nasal mucosa from AR patients and healthy controls (HCs) to identify differential proteins, which were validated using immunofluorescence, western blotting, and RT-qPCR. Nasal epithelial cells from HCs were isolated to evaluate house dust mite (HDM)-induced changes in GLUT1 expression and regulatory mechanisms. An AR mouse model was constructed to examine the effects of GLUT1 inhibition on nasal inflammation and barrier function.

Results

Tissue proteomics analysis revealed a distinct protein expression profile in AR patients compared with HCs, with GLUT1 identified as the most upregulated protein. Cohort validation demonstrated significantly elevated GLUT1 expression in the AR group, predominantly localized in epithelial cells. Moreover, GLUT1 mRNA levels showed a positive correlation with visual analogue scales and total nasal symptom scores, and a negative correlation with tissue ZO-1 and occludin expressions. In vitro studies indicated that HDM stimulation enhanced GLUT1 expression and reduced ZO-1 and occludin levels in nasal epithelial cells in a dose-dependent manner. Treatment with a GLUT1 inhibitor effectively restored ZO-1 and occludin expressions. Animal model experiments further confirmed that GLUT1 inhibition alleviated nasal inflammation and improved mucosal barrier function.

Conclusion

AR displays a distinct tissue-specific protein expression profile, with enhanced GLUT1 levels associated with disease severity and epithelial barrier dysfunction. Inhibition of GLUT1 has been shown to reduce nasal inflammation and improve epithelial barrier function in AR.
上皮屏障损伤是变应性鼻炎(AR)的一个特征性病理标志,但导致这种功能障碍的机制仍不完全清楚。我们的目的是评估葡萄糖转运蛋白1 (GLUT1)对AR上皮屏障功能的影响。方法我们对AR患者和健康对照(hc)的鼻黏膜进行蛋白质组学分析,以鉴定差异蛋白,并使用免疫荧光、western blotting和RT-qPCR验证。分离hc鼻上皮细胞,研究屋尘螨(HDM)诱导的GLUT1表达变化及其调控机制。建立AR小鼠模型,观察GLUT1抑制对鼻部炎症和屏障功能的影响。结果组织蛋白质组学分析显示,与hcc相比,AR患者的蛋白表达谱明显不同,其中GLUT1被确定为上调最多的蛋白。队列验证表明,在AR组中,GLUT1的表达显著升高,主要集中在上皮细胞中。GLUT1 mRNA水平与视觉模拟量表和鼻症状总分呈正相关,与组织ZO-1和occludin表达呈负相关。体外研究表明,HDM刺激增强了鼻上皮细胞中GLUT1的表达,降低了ZO-1和occludin的水平,并呈剂量依赖性。用GLUT1抑制剂治疗可有效恢复ZO-1和occludin的表达。动物模型实验进一步证实,抑制GLUT1可减轻鼻部炎症,改善粘膜屏障功能。结论ar表现出独特的组织特异性蛋白表达谱,GLUT1水平升高与疾病严重程度和上皮屏障功能障碍相关。抑制GLUT1已被证明可减少鼻炎并改善AR的上皮屏障功能。
{"title":"Increased glucose transporter 1 contributes to epithelial barrier dysfunction in allergic rhinitis","authors":"Cui Xia PhD ,&nbsp;Kang Zhu MD ,&nbsp;Chao Yu MD ,&nbsp;Jingguo Chen MD ,&nbsp;Tianxi Gao MD ,&nbsp;Yanni Zhang MD","doi":"10.1016/j.waojou.2025.101158","DOIUrl":"10.1016/j.waojou.2025.101158","url":null,"abstract":"<div><h3>Background</h3><div>Epithelial barrier impairment is a characteristic pathological hallmark of allergic rhinitis (AR), yet the mechanisms contributing to this dysfunction remain incompletely understood. Our aim is to assess the impact of glucose transporter 1 (GLUT1) on epithelial barrier function in AR.</div></div><div><h3>Methods</h3><div>We performed proteomics analysis on nasal mucosa from AR patients and healthy controls (HCs) to identify differential proteins, which were validated using immunofluorescence, western blotting, and RT-qPCR. Nasal epithelial cells from HCs were isolated to evaluate house dust mite (HDM)-induced changes in GLUT1 expression and regulatory mechanisms. An AR mouse model was constructed to examine the effects of GLUT1 inhibition on nasal inflammation and barrier function.</div></div><div><h3>Results</h3><div>Tissue proteomics analysis revealed a distinct protein expression profile in AR patients compared with HCs, with GLUT1 identified as the most upregulated protein. Cohort validation demonstrated significantly elevated GLUT1 expression in the AR group, predominantly localized in epithelial cells. Moreover, GLUT1 mRNA levels showed a positive correlation with visual analogue scales and total nasal symptom scores, and a negative correlation with tissue ZO-1 and occludin expressions. In vitro studies indicated that HDM stimulation enhanced GLUT1 expression and reduced ZO-1 and occludin levels in nasal epithelial cells in a dose-dependent manner. Treatment with a GLUT1 inhibitor effectively restored ZO-1 and occludin expressions. Animal model experiments further confirmed that GLUT1 inhibition alleviated nasal inflammation and improved mucosal barrier function.</div></div><div><h3>Conclusion</h3><div>AR displays a distinct tissue-specific protein expression profile, with enhanced GLUT1 levels associated with disease severity and epithelial barrier dysfunction. Inhibition of GLUT1 has been shown to reduce nasal inflammation and improve epithelial barrier function in AR.</div></div>","PeriodicalId":54295,"journal":{"name":"World Allergy Organization Journal","volume":"19 1","pages":"Article 101158"},"PeriodicalIF":4.3,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145926903","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}
引用次数: 0
Characterization of Pana g 1, an important cause of pollen-food allergy syndrome from Korean ginseng, Panax ginseng 人参花粉食物过敏综合征的重要致病因子Pana g1的鉴定
IF 4.3 2区 医学 Q2 ALLERGY Pub Date : 2026-01-01 DOI: 10.1016/j.waojou.2025.101164
Kyoung Yong Jeong PhD , Yoon Ji Shin BSc , Haeun Kim BSc , Yong Seok Lee PhD , Minkyu Sang PhD , Hyun Kyung Oh MD , Kyung Hee Park MD, PhD , Jae-Hyun Lee MD, PhD , Jung-Won Park MD, PhD

Background

Ginseng is a widely consumed herbal supplement. However, ginseng, especially raw ginseng, can cause allergic reactions, including pollen-food allergy syndrome (PFAS). This study aimed to identify the PFAS-causative allergen in Korean ginseng and to establish methods for its quantification.

Methods

Candidate allergens were screened using genomic and transcriptomic analyses. Proteomic profiling with patient sera was performed to identify the clinically relevant allergen. A recombinant protein was generated, and its allergenicity compared with the primary sensitizer, Que ac 1, by ELISA. A two-site ELISA was developed for the quantification of the ginseng allergen using monoclonal antibodies against recombinant protein. Multiple reaction monitoring (MRM) mass spectrometry was applied for validation.

Results

Genome and transcriptome analysis identified 4 candidate allergens: pathogenesis-related 10 (PR-10) protein, profilin, non-specific lipid transfer protein (nsLTP), and thaumatin-like protein. Among these, PR-10 (designated Pana g 1) was the sole allergen detected by proteomic analysis. Recombinant Pana g 1 was recognized by 4 of 5 patients. Inhibition ELISA showed stronger IgE reactivity to Que ac 1 than to Pana g 1, with marked cross-reactivity between the 2. Pana g 1 levels in ginseng extract were quantified as 4.26 μg/mg of protein by ELISA and 4.54 μg/mg by MRM in the ginseng extract.

Conclusion

Pana g 1 is the major PFAS-causative allergen in Korean ginseng. Recombinant Pana g 1 shows promise as a diagnostic tool for ginseng-induced PFAS. The quantification systems established here may also support standardization of ginseng extracts and allergen monitoring.
人参是一种被广泛食用的草药补充剂。然而,人参,尤其是生人参,会引起过敏反应,包括花粉食物过敏综合征(PFAS)。本研究旨在鉴定红参中诱发pfas的过敏原,并建立其定量方法。方法采用基因组学和转录组学分析筛选候选过敏原。对患者血清进行蛋白质组学分析,以确定临床相关的过敏原。制备了重组蛋白,并通过ELISA对其与原致敏剂Que ac 1的致敏性进行了比较。利用重组蛋白单克隆抗体,建立了人参过敏原的双位点ELISA定量方法。采用多反应监测(MRM)质谱法进行验证。结果基因组和转录组分析鉴定出4种候选过敏原:发病相关10蛋白(PR-10)、profilin、非特异性脂质转移蛋白(nsLTP)和thaumatin样蛋白。其中PR-10(命名为Pana g1)是唯一通过蛋白质组学分析检测到的过敏原。重组Pana g1在5例患者中被4例识别。抑制酶联免疫吸附试验显示,IgE对Que ac 1的反应性强于对Pana g1的反应性,且两者之间存在明显的交叉反应。ELISA法测定人参提取物中Pana g 1蛋白含量为4.26 μg/mg, MRM法测定人参提取物中Pana g 1蛋白含量为4.54 μg/mg。结论panag1是红参中主要的致pfas变应原。重组panag1有望作为人参诱导的PFAS的诊断工具。这里建立的定量系统也可以支持人参提取物和过敏原监测的标准化。
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引用次数: 0
Bronchiolitis and recurrent respiratory infections: The role of oxidative stress from early life inflammation to long-term outcomes – A narrative review 毛细支气管炎和复发性呼吸道感染:氧化应激从早期生活炎症到长期预后的作用-一篇叙述性综述
IF 4.3 2区 医学 Q2 ALLERGY Pub Date : 2026-01-01 DOI: 10.1016/j.waojou.2025.101162
Michele Piazza PhD , Alessandra Gori MD , Carlo Capristo MD , Attilio L. Boner MD
Bronchiolitis, primarily caused by respiratory syncytial virus (RSV), is a common respiratory infection in infants and a known precursor to recurrent wheezing and asthma. This review explores the role of oxidative stress and trace element deficiencies in the pathogenesis of bronchiolitis and its long-term sequelae.
Infants with reduced lung function due to prematurity or congenital airway anomalies exhibit heightened susceptibility to RSV infection. Growing evidence implicates oxidative stress and deficiencies in zinc, selenium, and magnesium as significant contributors to disease progression. Impaired antioxidant defenses exacerbate viral inflammatory responses, leading to prolonged symptoms and recurrent wheezing with potential developmental delays.
Studies consistently demonstrate that children with bronchiolitis exhibit elevated oxidative stress markers and reduced antioxidant capacity, with trace element deficiencies correlating with disease severity. Reduced defenses against oxidative stress may be associated with recurrent wheezing episodes, which are more frequent after rhinovirus bronchiolitis than after RSV bronchiolitis. Thus, RSV and rhinovirus (RV) bronchiolitis may unmask pre-existing vulnerabilities rather than directly causing long-term damage associated with later asthma.
Micronutrient supplementation, particularly zinc and selenium, has shown potential in reducing respiratory infection duration and severity. COVID-19 pandemic evidence further supports nutritional status as a key modulator of respiratory disease outcomes, with nutraceuticals like curcumin and flavonoids demonstrating anti-inflammatory benefits.
Given the safety and accessibility of micronutrient supplementation, early nutritional assessment and intervention in high-risk infants may offer a cost-effective strategy to improve long-term respiratory outcomes. Bronchiolitis should be viewed as a clinical signal warranting proactive, holistic pediatric care rather than merely an acute illness.
毛细支气管炎主要由呼吸道合胞病毒(RSV)引起,是一种常见的婴儿呼吸道感染,也是已知的复发性喘息和哮喘的前兆。本文综述了氧化应激和微量元素缺乏在毛细支气管炎发病机制及其长期后遗症中的作用。由于早产或先天性气道异常导致肺功能降低的婴儿对呼吸道合胞病毒感染的易感性增加。越来越多的证据表明,氧化应激和锌、硒、镁的缺乏是疾病进展的重要因素。抗氧化防御受损会加剧病毒炎症反应,导致症状延长和复发性喘息,并伴有潜在的发育迟缓。研究一致表明,毛细支气管炎儿童表现出氧化应激标志物升高和抗氧化能力降低,微量元素缺乏与疾病严重程度相关。抗氧化应激能力的降低可能与反复发作的喘息有关,鼻病毒细支气管炎比RSV细支气管炎更常见。因此,RSV和鼻病毒(RV)细支气管炎可能揭示了先前存在的脆弱性,而不是直接导致与后期哮喘相关的长期损害。补充微量营养素,特别是锌和硒,已显示出减少呼吸道感染持续时间和严重程度的潜力。COVID-19大流行的证据进一步支持营养状况是呼吸系统疾病结局的关键调节剂,姜黄素和类黄酮等营养保健品显示出抗炎作用。考虑到微量营养素补充的安全性和可及性,对高危婴儿进行早期营养评估和干预可能是改善长期呼吸预后的一种经济有效的策略。细支气管炎应被视为一个临床信号,保证积极,全面的儿科护理,而不仅仅是一种急性疾病。
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引用次数: 0
Efficacy and acceptability of lanadelumab for long-term prophylaxis in hereditary angioedema: A Chinese multicenter real-world analysis lanadelumab长期预防遗传性血管性水肿的疗效和可接受性:一项中国多中心现实世界分析
IF 4.3 2区 医学 Q2 ALLERGY Pub Date : 2026-01-01 DOI: 10.1016/j.waojou.2025.101165
Yijing Xu MD , Ruoyu Ji MD , Juan Liu MD , Xiangyi Cui MSc , Heng Zhang MD , Naiqing Cao MD , Yongmei Yu MD , Yingnan Li MD , He Lai MD , Yuxiang Zhi MD

Background

Hereditary angioedema (HAE) is a rare and potentially life-threatening genetic disorder. Lanadelumab, a monoclonal antibody inhibiting plasma kallikrein, has been approved for long-term prophylaxis (LTP) in HAE patients aged ≥12 years since 2020 in China, but real-world evidence in Chinese populations is insufficient. Here, we assessed the real-world effectiveness, safety, and treatment acceptability of lanadelumab for LTP in Chinese HAE patients.

Methods

This multicenter observational study enrolled type I/II HAE patients ≥12 years who initiated lanadelumab for LTP between June 2022 and December 2024 across 5 tertiary medical centers in China, with a minimum follow-up time of 6 months. Attack frequency, emergency interventions, patient-reported outcome measures (PROMs), dosing interval extension and acceptability were described and if feasible, compared pair-wise before and after lanadelumab treatment.

Results

Fifty HAE patients (36 prospectively enrolled and 14 retrospectively included) were included with a median follow-up of 17.5 months. Dosing intervals were successfully extended in 80% of patients. Attack-free rate (AFR) remarkably elevated from 0.0% to 65.6% (cumulative 1-year AFR) and 66.7% (observed 1-year AFR). The proportion of patients experiencing severe attacks significantly declined. Results of PROMs also substantially improved. Treatment-emergent adverse events were mostly mild. 78% of patients reported significant economic burden associated with lanadelumab use.

Conclusion

Lanadelumab demonstrates high efficacy in reducing HAE attacks and improving quality of life in Chinese patients in a safe manner. Successful symptom-guided dosing interval extension was achievable in most patients. Despite clinical benefits, its high cost imposes financial burdens on our patients, but this should be weighed against the cost savings achieved through improved disease control.
背景:遗传性血管性水肿(HAE)是一种罕见且可能危及生命的遗传性疾病。Lanadelumab是一种单克隆抗体抑制血浆激肽肽(kallikrein),自2020年以来已被批准用于中国≥12岁HAE患者的长期预防(LTP),但在中国人群中的实际证据不足。在这里,我们评估了lanadelumab治疗中国HAE患者LTP的实际有效性、安全性和治疗可接受性。该多中心观察性研究纳入了2022年6月至2024年12月期间在中国5个三级医疗中心开始使用lanadelumab治疗LTP的≥12岁的I/II型HAE患者,最小随访时间为6个月。描述了发作频率、紧急干预措施、患者报告的结果测量(PROMs)、给药间隔延长和可接受性,如果可行,对lanadelumab治疗前后进行两两比较。结果纳入50例HAE患者(36例前瞻性纳入,14例回顾性纳入),中位随访时间为17.5个月。80%的患者成功延长了给药间隔。无发作率(AFR)由0.0%显著提高到65.6%(累积1年AFR)和66.7%(观察1年AFR)。经历严重发作的患者比例显著下降。PROMs的结果也有了很大的改善。治疗后出现的不良事件大多是轻微的。78%的患者报告了与lanadelumab使用相关的显著经济负担。结论lanadelumab在减少HAE发作和改善中国患者生活质量方面具有较高的安全性。在大多数患者中,症状引导的给药间隔延长是可以实现的。尽管有临床效益,但它的高成本给我们的患者带来了经济负担,但这应该与通过改善疾病控制而节省的成本进行权衡。
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引用次数: 0
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World Allergy Organization Journal
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