过敏原免疫治疗的临床试验：当前内容和未来的需求
2018/05/03
摘要
过敏原免疫治疗（AIT）是一种安全，有效的治疗过敏性鼻结膜炎和过敏性哮喘的疗法。但是，AIT的临床效果仍然存在争议 - 主要是由于临床试验设计，研究人群，治疗制剂和疗效标准的异质性。在讨论了当前的概念和未满足的需求之后，国际专家小组提出了几项建议：（i）在AIT试验中探索和验证[临床]应答者的定义; （ii）在AIT试验中纳入具有相关疾病的受试者之前，使用记录完备的标准化激发试验; （iii）在AIT延伸试验中监测新发敏感和新发过敏，并排除多发过敏的受试者;（iv）验证基于自然暴露情况下的过敏原暴露; （v）在季节性过敏研究中，关注峰值暴露，但也考虑组织两个平行的，除地域外其他方面相同的试验; （vi）与监管机构讨论适应性试验设计; （vii）利用电子保健和移动健康技术获取更多个人接触过敏原的信息; （viii）开始研究安慰剂效应的潜在心理、生化、免疫、神经甚至基因组标记物;（ix）确定试验设计和主要终点，以便让过敏症儿童，更方便、更快地获得AIT制剂; （x）推广和应用标准化方法报告全身和局部不良事件。最新的技术和试验设计可能为AIT临床试验提供减少偏倚和异质性的新型的、伦理学方法。 医生，患者群体，公司和监管机构有可能改进AIT临床试验，并最终为患者提供更好的治疗。
Clinical trials in allergen immunotherapy: current concepts and future needs.
Oliver Pfaar, et al.
Abstract
Allergen immunotherapy (AIT) is a safe, effective treatment for allergic rhinoconjunctivitis and allergic asthma. However, AIT's clinical effect is still contested - primarily due to heterogeneity in clinical trial designs, study populations, therapeutic formulations and efficacy criteria. After discussing current concepts and unmet needs, an international panel of experts made several recommendations: (i) explore and validate definitions for [clinical] responders in AIT-trials; (ii) use of well-documented, standardized provocation tests prior to inclusion of subjects with relevant diseases in AIT trials; (iii) monitoring neosensitizations and occurrence of new allergy in extended AIT trials, and exclusion of polyallergic participants; (iv) validation of allergen exposure chambers with regard to natural exposure; (v) in studies of seasonal allergies, focus on peak exposure but also consider organising two parallel, geographically distinct but otherwise identical trials; (vi) discuss adaptive trial designs with the regulatory authorities; (vii) use e-health and m-health technologies to capture more information on individual exposure to allergens; (viii) initiate research on potential psychological, biochemical, immune, neural and even genomic markers of the placebo response; (ix) identify trial designs and primary endpoints that will give children with allergies easier, faster access to AIT formulations; and (x) promote and apply standardized methods for
reporting systemic and local adverse events. The latest technologies and trial designs may provide novel, ethical ways of reducing bias and heterogeneity in AIT clinical trials. There is scope for physicians, patient organizations, companies and regulators to improve clinical trials in AIT and, ultimately, to provide patients with better treatments.
过敏原免疫治疗（AIT）是一种安全，有效的治疗过敏性鼻结膜炎和过敏性哮喘的疗法。但是，AIT的临床效果仍然存在争议 - 主要是由于临床试验设计，研究人群，治疗制剂和疗效标准的异质性。在讨论了当前的概念和未满足的需求之后，国际专家小组提出了几项建议：（i）在AIT试验中探索和验证[临床]应答者的定义; （ii）在AIT试验中纳入具有相关疾病的受试者之前，使用记录完备的标准化激发试验; （iii）在AIT延伸试验中监测新发敏感和新发过敏，并排除多发过敏的受试者;（iv）验证基于自然暴露情况下的过敏原暴露; （v）在季节性过敏研究中，关注峰值暴露，但也考虑组织两个平行的，除地域外其他方面相同的试验; （vi）与监管机构讨论适应性试验设计; （vii）利用电子保健和移动健康技术获取更多个人接触过敏原的信息; （viii）开始研究安慰剂效应的潜在心理、生化、免疫、神经甚至基因组标记物;（ix）确定试验设计和主要终点，以便让过敏症儿童，更方便、更快地获得AIT制剂; （x）推广和应用标准化方法报告全身和局部不良事件。最新的技术和试验设计可能为AIT临床试验提供减少偏倚和异质性的新型的、伦理学方法。 医生，患者群体，公司和监管机构有可能改进AIT临床试验，并最终为患者提供更好的治疗。
（复旦大学附属中山医院 呼吸内科 罗锦龙 摘译 杨冬 审校）
（Allergy;2018,Feb 15）
（Allergy;2018,Feb 15）
Clinical trials in allergen immunotherapy: current concepts and future needs.
Oliver Pfaar, et al.
Abstract
Allergen immunotherapy (AIT) is a safe, effective treatment for allergic rhinoconjunctivitis and allergic asthma. However, AIT's clinical effect is still contested - primarily due to heterogeneity in clinical trial designs, study populations, therapeutic formulations and efficacy criteria. After discussing current concepts and unmet needs, an international panel of experts made several recommendations: (i) explore and validate definitions for [clinical] responders in AIT-trials; (ii) use of well-documented, standardized provocation tests prior to inclusion of subjects with relevant diseases in AIT trials; (iii) monitoring neosensitizations and occurrence of new allergy in extended AIT trials, and exclusion of polyallergic participants; (iv) validation of allergen exposure chambers with regard to natural exposure; (v) in studies of seasonal allergies, focus on peak exposure but also consider organising two parallel, geographically distinct but otherwise identical trials; (vi) discuss adaptive trial designs with the regulatory authorities; (vii) use e-health and m-health technologies to capture more information on individual exposure to allergens; (viii) initiate research on potential psychological, biochemical, immune, neural and even genomic markers of the placebo response; (ix) identify trial designs and primary endpoints that will give children with allergies easier, faster access to AIT formulations; and (x) promote and apply standardized methods for
reporting systemic and local adverse events. The latest technologies and trial designs may provide novel, ethical ways of reducing bias and heterogeneity in AIT clinical trials. There is scope for physicians, patient organizations, companies and regulators to improve clinical trials in AIT and, ultimately, to provide patients with better treatments.
上一篇：
稳定期寡粒细胞型哮喘患者的临床、功能和炎症特征：与痰液不同表型比较
下一篇：
室内细菌与成人哮喘：ECRHS II中一项多中心病例对照研究
