Ford Paul, Kreuter Michael, Brown Kevin K, Wuyts Wim A, Wijsenbeek Marlies, Israël-Biet Dominique, Hubbard Richard, Nathan Steven D, Nunes Hilario, Penninckx Bjorn, Prasad Niyati, Seghers Ineke, Spagnolo Paolo, Verbruggen Nadia, Hirani Nik, Behr Juergen, Kaner Robert J, Maher Toby M
Galapagos NV, Mechelen, Belgium.
Center for Pulmonary Medicine, Departments of Pneumology, Mainz University Medical Center and of Pulmonary, Critical Care and Sleep Medicine, Marienhaus Clinic Mainz, Mainz, Germany.
ERJ Open Res. 2024 Jan 29;10(1). doi: 10.1183/23120541.00636-2023. eCollection 2024 Jan.
There is no standard definition of respiratory-related hospitalisation, a common end-point in idiopathic pulmonary fibrosis (IPF) clinical trials. As diverse aetiologies and complicating comorbidities can present similarly, external adjudication is sometimes employed to achieve standardisation of these events.
An algorithm for respiratory-related hospitalisation was developed through a literature review of IPF clinical trials with respiratory-related hospitalisation as an end-point. Experts reviewed the algorithm until a consensus was reached. The algorithm was validated using data from the phase 3 ISABELA trials (clinicaltrials.gov identifiers NCT03711162 and NCT03733444), by assessing concordance between nonadjudicated, investigator-defined, respiratory-related hospitalisations and those defined by the adjudication committee using the algorithm.
The algorithm classifies respiratory-related hospitalisation according to cause: extraparenchymal (worsening respiratory symptoms due to left heart failure, volume overload, pulmonary embolism, pneumothorax or trauma); other (respiratory tract infection, right heart failure or exacerbation of COPD); "definite" acute exacerbation of IPF (AEIPF) (worsening respiratory symptoms within 1 month, with radiological or histological evidence of diffuse alveolar damage); or "suspected" AEIPF (as for "definite" AEIPF, but with no radiological or histological evidence of diffuse alveolar damage). Exacerbations ("definite" or "suspected") with identified triggers (infective, post-procedural or traumatic, drug toxicity- or aspiration-related) are classed as "known AEIPF"; "idiopathic AEIPF" refers to exacerbations with no identified trigger. In the ISABELA programme, there was 94% concordance between investigator- and adjudication committee-determined causes of respiratory-related hospitalisation.
The algorithm could help to ensure consistency in the reporting of respiratory-related hospitalisation in IPF trials, optimising its utility as an end-point.
在特发性肺纤维化(IPF)临床试验中,呼吸相关住院尚无标准定义,这是一个常见的终点指标。由于多种病因和并发合并症可能表现相似,有时采用外部判定来实现这些事件的标准化。
通过对以呼吸相关住院为终点的IPF临床试验进行文献综述,制定了呼吸相关住院的算法。专家对该算法进行评审,直至达成共识。使用3期ISABELA试验(clinicaltrials.gov标识符NCT03711162和NCT03733444)的数据对该算法进行验证,通过评估未经判定的、研究者定义的呼吸相关住院与判定委员会使用该算法定义的住院之间的一致性。
该算法根据病因对呼吸相关住院进行分类:肺实质外(因左心衰竭、容量超负荷、肺栓塞、气胸或创伤导致呼吸症状恶化);其他(呼吸道感染、右心衰竭或慢性阻塞性肺疾病加重);“明确的”IPF急性加重(AEIPF)(1个月内呼吸症状恶化,伴有弥漫性肺泡损伤的影像学或组织学证据);或“疑似”AEIPF(与“明确的”AEIPF相同,但无弥漫性肺泡损伤的影像学或组织学证据)。有明确触发因素(感染性、术后或创伤性、药物毒性或误吸相关)的加重(“明确的”或“疑似的”)归类为“已知AEIPF”;“特发性AEIPF”指无明确触发因素的加重。在ISABELA项目中,研究者和判定委员会确定的呼吸相关住院病因之间的一致性为94%。
该算法有助于确保IPF试验中呼吸相关住院报告的一致性,优化其作为终点指标的效用。