Popowicz Natalia, Wood Jamie, Tai Anna, Morey Sue, Mulrennan Siobhain
Pharmacy Department, Sir Charles Gairdner Hospital, Perth, Australia; Respiratory Medicine, Sir Charles Gairdner Hospital, Perth, Australia; School of Medicine & Pharmacology, University of Western Australia, Perth, Australia.
Respiratory Medicine, Sir Charles Gairdner Hospital, Perth, Australia; Physiotherapy Department, Sir Charles Gairdner Hospital, Perth, Australia.
J Cyst Fibros. 2017 May;16(3):392-394. doi: 10.1016/j.jcf.2017.02.009. Epub 2017 Mar 15.
Safety-data for lumacaftor/ivacaftor (LUM/IVA) combination therapy in patients with severe lung disease (percent predicted forced expiratory volume in 1s [ppFEV] <40) remain limited. We report immediate post-dose respiratory-related adverse events in 12 patients with severe cystic fibrosis (CF) lung disease (median [IQR] ppFEV: 34 [31-36]) prescribed LUM/IVA. All patients experienced a decline in ppFEV from baseline at 2-hours (median [IQR] relative change: -19 [-21 to -11]%, p<0.001) that persisted at 24-hours but recovered in most patients at 1-month. No pre- and post-differences in bronchodilator response were observed. Ten (83.3%) patients reported non-severe respiratory-related adverse events within 24-hours of LUM/IVA initiation. At 1-month, eight (67%) patients had persistent symptoms and six (50%) were treated for a pulmonary exacerbation. Our results highlight that LUM/IVA respiratory-related adverse events are common in patients with a ppFEV<40. We recommend close assessment of adverse events. Further studies are required to evaluate the efficacy of LUM/IVA in patients with severe lung disease.
针对严重肺部疾病（1秒用力呼气容积预测值百分比[ppFEV]<40）患者，鲁马卡托/依伐卡托（LUM/IVA）联合疗法的安全性数据仍然有限。我们报告了12例接受LUM/IVA治疗的严重囊性纤维化（CF）肺部疾病患者（ppFEV中位数[四分位间距]：34[31 - 36]）给药后立即出现的呼吸相关不良事件。所有患者在2小时时ppFEV均较基线下降（中位数[四分位间距]相对变化：-19[-21至-11]%，p<0.001），24小时时仍持续下降，但大多数患者在1个月时恢复。未观察到支气管扩张剂反应的前后差异。10例（83.3%）患者在开始LUM/IVA治疗后24小时内报告了非严重呼吸相关不良事件。在1个月时，8例（67%）患者有持续症状，6例（50%）因肺部加重接受治疗。我们的结果强调，ppFEV<40的患者中LUM/IVA呼吸相关不良事件很常见。我们建议密切评估不良事件。需要进一步研究来评估LUM/IVA在严重肺部疾病患者中的疗效。
