Al Said Abir, Cushen Breda, Costello Richard W
Department of Medicine, Royal College of Surgeons in Ireland, Beaumont Hospital, Dublin 9, Ireland.
Ther Adv Chronic Dis. 2017 Feb;8(2-3):31-45. doi: 10.1177/2040622317690494. Epub 2017 Feb 1.
The asthma syndrome has many manifestations, termed phenotypes, that arise by specific cellular and molecular mechanisms, termed endotypes. Understanding an individual's asthma phenotype helps clinicians make rational therapeutic decisions while the understanding of endotypes has led to the development of specific precision medications. Allergic asthma is an example of an asthma phenotype and omalizumab, a monoclonal antibody that neutralizes serum immunoglobulin (Ig)E, is a specific targeted treatment which was developed as a result of an understanding of the endotype of allergic asthma. Omalizumab has been widely used in clinical practice in Europe for over a decade as an add-on therapy to treat patients who have severe refractory allergic asthma. Over this period, many centres have reported their experience with omalizumab as an add-on therapy in patients with severe asthma. These 'real world' clinical effectiveness studies have confirmed the benefits, cost-effectiveness and clinical utility of this medication. Combining the outcomes of both sources of research has yielded important insights that may benefit patients with severe asthma, clinicians who treat them, as well as the funding agencies that reimburse the cost of this medication. The purpose of this review is to describe how to identify and evaluate a patient with asthma for whom treatment with omalizumab may be of clinical and cost-effective benefit. The assessment and investigations used to confirm allergic asthma, the objective assessment of adherence to asthma therapy and the expected benefits of add-on omalizumab treatment are described.
哮喘综合征有许多表现形式,称为表型,它们通过特定的细胞和分子机制产生,称为内型。了解个体的哮喘表型有助于临床医生做出合理的治疗决策,而对内型的了解则促成了特定精准药物的开发。过敏性哮喘是哮喘表型的一个例子,奥马珠单抗是一种中和血清免疫球蛋白(Ig)E的单克隆抗体,是基于对过敏性哮喘内型的了解而开发的一种特定靶向治疗药物。在欧洲,奥马珠单抗作为一种附加疗法用于治疗重度难治性过敏性哮喘患者,已在临床实践中广泛应用了十多年。在此期间,许多中心报告了他们使用奥马珠单抗作为重度哮喘患者附加疗法的经验。这些“真实世界”的临床疗效研究证实了这种药物的益处、成本效益和临床实用性。将这两种研究来源的结果相结合,产生了重要的见解,可能会使重度哮喘患者、治疗他们的临床医生以及报销这种药物费用的资助机构受益。本综述的目的是描述如何识别和评估可能从奥马珠单抗治疗中获得临床和成本效益益处的哮喘患者。描述了用于确诊过敏性哮喘的评估和检查、对哮喘治疗依从性的客观评估以及奥马珠单抗附加治疗的预期益处。