Duman Ipek, Ünal Gokcen, Yilmaz Asli Imran, Güney Ahmet Yasin, Durduran Yasemin, Pekcan Sevgi
Department of Medical Pharmacology, Meram Medicine Faculty, Necmettin Erbakan University, Konya, Turkey.
Department of Pediatric Pulmonology, and Meram Medicine Faculty, Necmettin Erbakan University, Konya, Turkey.
Pediatr Allergy Immunol Pulmonol. 2022 Mar;35(1):19-26. doi: 10.1089/ped.2021.0127. Epub 2022 Mar 14.
Cystic fibrosis (CF) is a genetic disorder, in which defective clearance of airway secretions leads to progressive lung function loss. Inhaled mannitol is used to increase sputum and mucociliary clearance. There are little data from real-world studies on the effectiveness of mannitol in children. Our objective was to evaluate the spirometry and clinical results of mannitol in pediatric patients. We retrospectively reviewed the records of 30 children and adolescents with CF receiving inhaled mannitol who were already on recombinant human deoxyribonuclease (rhDNase) treatment. The change in forced expiratory volume in 1 second (FEV) from baseline at 2-4 months was the primary outcome. Secondary measures were other spirometry results, body mass index (BMI), hospital admissions, sputum characteristics, and positive bacterial colonization. Compared to baseline, we found significant improvement in percent predicted FEV at 2-4 months of treatment; 84.50 (58.00-99.00) vs. 96.00 (66.00-106.00) ( = 0.0007). The absolute change in FEV was +11.5% at 2-4 months, +6.5% at 5-7 months, and +4% at 8-12 months. Also, significant improvements in other spirometry results were observed. Adolescents had significantly lower FEV results, but the improvement in their lung function was sustained for a more extended period than children. Mannitol provided easier sputum removal, increased sputum volume, significant decline in hospitalizations, and significantly fewer patients with positive sputum cultures. A significant increase in BMI at 8-12 months was observed. Cough was the most frequent adverse effect. In a real-world setting, our results demonstrated that adding mannitol to rhDNase therapy is tolerable in pediatric patients with CF and may provide improved spirometry and clinical outcomes. In addition, our results showed that mannitol provided recovery in overall lung function at 2-4 months, which was sustained up to 12 months together with improved BMI, easier sputum removal, and a decline in bacterial colonization and hospital admissions. However, cough was the most frequent side effect.
囊性纤维化（CF）是一种遗传性疾病，其中气道分泌物清除缺陷会导致肺功能逐渐丧失。吸入甘露醇用于增加痰液和黏液纤毛清除率。关于甘露醇对儿童有效性的真实世界研究数据很少。我们的目的是评估甘露醇在儿科患者中的肺功能测定和临床结果。我们回顾性分析了30例接受吸入甘露醇治疗且已接受重组人脱氧核糖核酸酶（rhDNase）治疗的CF儿童和青少年的记录。治疗2 - 4个月时1秒用力呼气容积（FEV）相对于基线的变化是主要结局。次要指标包括其他肺功能测定结果、体重指数（BMI）、住院次数、痰液特征和阳性细菌定植情况。与基线相比，我们发现治疗2 - 4个月时预计FEV百分比有显著改善；84.50（58.00 - 99.00）对96.00（66.00 - 106.00）（P = 0.0007）。FEV的绝对变化在2 - 4个月时为 +11.5%，5 - 7个月时为 +6.5%，8 - 12个月时为 +4%。此外，其他肺功能测定结果也有显著改善。青少年的FEV结果显著较低，但他们的肺功能改善持续时间比儿童更长。甘露醇使痰液清除更容易，痰液量增加，住院次数显著减少，痰液培养阳性的患者显著减少。在8 - 12个月时观察到BMI显著增加。咳嗽是最常见的不良反应。在真实世界环境中，我们的结果表明，在CF儿科患者中，在rhDNase治疗基础上加用甘露醇是可耐受的，并且可能改善肺功能测定和临床结局。此外，我们的结果表明，甘露醇在2 - 4个月时可使整体肺功能恢复，这种恢复可持续至12个月，同时BMI改善、痰液清除更容易、细菌定植和住院次数减少。然而，咳嗽是最常见的副作用。
