University of British Columbia, Vancouver, Canada.
St Paul's Hospital, Vancouver, Canada.
Med J Aust. 2019 May;210(9):424-428. doi: 10.5694/mja2.50138. Epub 2019 Apr 11.
Chronic obstructive pulmonary disease (COPD) is defined based on a reduced ratio of forced expiratory volume in one second (FEV ) to forced vital capacity (FVC) on spirometry. However, within this definition, there is significant heterogeneity of pathophysiological processes that lead to airflow obstruction and variation in phenotypic manifestations across patients. Current pharmacological treatments are based on large randomised clinical trials that apply to an "average" patient. Precision health enables tailoring of treatment for each individual patient by taking into account their unique characteristics. The number needed to treat (NNT) metric is often used to define implementation of precision health for specific interventions, with common endpoints requiring an NNT ≤ 5 to achieve precision therapy. Higher NNTs may be acceptable for rare but important endpoints such as mortality. Long-acting muscarinic antagonists and inhaled corticosteroids, which are commonly used in COPD, have 1-year treatment NNTs between 15 and 20 for exacerbation prevention in unselected patients with COPD. Subgroup identification using biomarkers or clinical traits may enable precision health. For example, NNT for inhaled corticosteroids is 9 in patients with a blood eosinophil count ≥ 300 cells/μL and 8 for long-acting muscarinic antagonists in patients with a body mass index ≤ 20 kg/m . Lung volume reduction surgery is associated with an NNT of 6 for survival over 5 years in patients with upper lobe-predominant disease and low exercise capacity (whereas the NNT is 245 when no bioimaging or exercise markers are used). Continuous domiciliary oxygen therapy (for at least 15 hours/day) has an NNT of 5 for survival over 5 years in patients with resting hypoxemia (PaO < 60 mmHg on room air). Emerging areas of precision health in COPD with potential for low NNTs in specific circumstances include anti-interleukin-5 therapy for eosinophilic COPD, and immunoglobulin replacement therapy for patients with severe immunoglobulin deficiency.
慢性阻塞性肺疾病(COPD)的定义基于肺活量测定中一秒用力呼气量(FEV )与用力肺活量(FVC)的比值降低。然而,在这个定义中,导致气流阻塞的病理生理过程存在显著的异质性,并且患者的表型表现也存在差异。目前的药物治疗基于大型随机临床试验,适用于“平均”患者。精准医疗通过考虑每个患者的独特特征,实现对每个患者的治疗进行个性化定制。需要治疗的人数(NNT)指标常用于定义特定干预措施的精准医疗实施,常见的终点需要 NNT≤5 才能实现精准治疗。对于死亡率等罕见但重要的终点,较高的 NNT 可能是可以接受的。长效抗胆碱能药物和吸入性皮质类固醇是 COPD 的常用药物,在未选择的 COPD 患者中,预防加重的 1 年治疗 NNT 分别为 15 至 20。使用生物标志物或临床特征进行亚组识别可能实现精准医疗。例如,在嗜酸粒细胞计数≥300 个/μL 的患者中,吸入性皮质类固醇的 NNT 为 9,在 BMI≤20kg/m 的患者中,长效抗胆碱能药物的 NNT 为 8。肺减容手术与上叶为主型疾病和低运动能力患者的 5 年生存率相关(NNT 为 6)(而没有生物成像或运动标志物时,NNT 为 245)。持续家庭氧疗(每天至少 15 小时)在静息低氧血症(空气状态下 PaO <60mmHg)患者中 5 年生存率的 NNT 为 5。在 COPD 中,精准医疗的新兴领域在特定情况下可能具有较低的 NNT,包括嗜酸性 COPD 的抗白细胞介素-5 治疗和严重免疫球蛋白缺乏症患者的免疫球蛋白替代治疗。