Gatt Dvir, Shaw Michelle, Waters Valerie, Kritzinger Fiona, Solomon Melinda, Dell Sharon, Ratjen Felix
Division of Respiratory Medicine.
Department of Translational Medicine, Research Institute, and.
Ann Am Thorac Soc. 2025 May;22(5):724-731. doi: 10.1513/AnnalsATS.202407-771OC.
Patients with primary ciliary dyskinesia (PCD) experience acute pulmonary exacerbations (PExs). In patients with cystic fibrosis (CF), PExs that were treated with oral antibiotics (oPExs) were found to be related to short- and long-term lung function deficits; however, the impact of oPExs on lung function in patients with PCD has not yet been assessed. We sought to assess the impact of oPExs on lung function recovery in PCD and determine the factors associated with poorer response. This was a retrospective study of pediatric patients with PCD who were followed from 2000 to 2022 at The Hospital for Sick Children (Toronto, Ontario, Canada). PExs were defined as an increase in baseline symptoms with a physician's decision to treat with systemic intravenous or oral antibiotics. Lung function recovery was defined as a forced expiratory volume in 1 second (FEV) measurement ⩾90% of a stable baseline within 12 months before the PEx. Univariate and multivariate analyses were completed to identify risk factors for nonresponse. A total of 337 PEx events in 85 patients were included in this analysis, of which 297 (88%) were treated with oral antibiotics. The mean follow up time for patients was 6.7 years (SD = 3.5), and the mean age of patients with an oPEx was 12.5 years (SD = 3.2). Patients with an oPEx had a significant drop from baseline in mean FEV values at the time of the PEx (85.1-69.5%), with absolute and relative changes of -10.4% and -12.9%, respectively. At follow up (3 mo post PEx) and up to 12 mo post PEx, the means for FEV were 79.6% and 84.1%, respectively. A total of 73.2% of the patients had lung function recovery at the follow up visit, which increased to 84.2% within 1 year postevent. We identified two risk factors for nonresponse: being a nonresponder on the last PEx and younger age at time of the oPEx. oPExs in patients with PCD show a similar pattern previously seen in patients with CF who showed a decrease in FEV during exacerbation and an improvement posttherapy. Most oPEx events recover to baseline FEV within the year postexacerbation, with younger age and being a nonresponder in the last PEx identified as risk factors for nonresponse.
原发性纤毛运动障碍（PCD）患者会经历急性肺部加重期（PExs）。在囊性纤维化（CF）患者中，口服抗生素治疗的PExs（oPExs）与短期和长期肺功能缺陷有关；然而，oPExs对PCD患者肺功能的影响尚未得到评估。我们试图评估oPExs对PCD患者肺功能恢复的影响，并确定与反应较差相关的因素。这是一项对2000年至2022年在加拿大多伦多病童医院随访的PCD儿科患者的回顾性研究。PExs被定义为基线症状加重且医生决定用全身静脉或口服抗生素治疗。肺功能恢复被定义为在PEx前12个月内1秒用力呼气量（FEV）测量值≥稳定基线的90%。进行单因素和多因素分析以确定无反应的危险因素。本分析纳入了85例患者的337次PEx事件，其中297次（88%）接受了口服抗生素治疗。患者的平均随访时间为6.7年（标准差=3.5），接受oPEx治疗的患者平均年龄为12.5岁（标准差=3.2）。接受oPEx治疗的患者在PEx时FEV平均值较基线有显著下降（85.1%-69.5%），绝对变化和相对变化分别为-10.4%和-12.9%。在随访时（PEx后3个月）以及PEx后长达12个月时，FEV平均值分别为79.6%和84.1%。共有73.2%的患者在随访时肺功能恢复，在事件发生后1年内这一比例增至84.2%。我们确定了两个无反应的危险因素：在上一次PEx时无反应以及接受oPEx治疗时年龄较小。PCD患者的oPExs表现出与CF患者先前相似的模式，CF患者在加重期FEV下降，治疗后改善。大多数oPEx事件在加重后一年内恢复到基线FEV，年龄较小以及在上一次PEx时无反应被确定为无反应的危险因素。
