There is emerging evidence for the role of posaconazole in the management of Aspergillus-related cystic fibrosis (CF) lung disease. The tolerability and efficacy of posaconazole in paediatric CF is not well established. We report a prospective study over a fifty-three month period evaluating the safety, tolerability, and efficacy of posaconazole in pediatric CF. Fourteen children (seven males, median age 13 years, range 3-17 years) received a total of twenty-three courses of posaconazole (13 oral suspension and 10 tablet formulation). Of these patient episodes, nine received posaconazole for emerging or active allergic bronchopulmonary aspergillosis (ABPA) and two required a combination of posaconazole and systemic corticosteroids for difficult-to-treat ABPA. A subgroup of patients (n = 12) with persistent isolates of Aspergillus fumigatus, in the absence of serological markers of ABPA, received posaconazole monotherapy for pulmonary exacerbations not responding to conventional broad-spectrum antibiotic treatment. Posaconazole levels, full blood count, electrolytes, and liver function were monitored on day 7 of treatment and then monthly. Posaconazole was well tolerated in all but three patients. Therapeutic plasma levels >1 mg/l were achieved in all receiving the tablet formulation in comparison to 60% on the liquid preparation. There was a modest but significant improvement in FEV1 (% predicted) demonstrated for the cohort as a whole (p = 0.015) following posaconazole therapy. Posaconazole is well tolerated in children as young as six years old, improvements in lung function are observed, and therapeutic plasma levels are readily achieved in patients taking the tablet formulation and in adherent patients taking the liquid formulation.

中文翻译：

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泊沙康唑治疗小儿囊性纤维化和曲霉菌相关的肺部疾病。

泊沙康唑在曲霉菌相关的囊性纤维化（CF）肺疾病的治疗中的作用的新兴证据。泊沙康唑在小儿CF中的耐受性和疗效尚不明确。我们报告了一项为期53个月的前瞻性研究，评估泊沙康唑在小儿CF中的安全性，耐受性和疗效。14名儿童（7名男性，中位年龄13岁，年龄3-17岁）总共接受了23疗程的泊沙康唑（13种口服混悬液和10片制剂）。在这些患者发作中，有9名因新出现的或活动性的过敏性支气管肺曲霉病（ABPA）接受了泊沙康唑治疗，另外2例因难治性ABPA需要联合使用泊沙康唑和全身性糖皮质激素治疗。一个亚组的患者（n = 12）持续存在烟曲霉菌，在没有ABPA血清学标志物的情况下，接受泊沙康唑单药治疗肺部加重，对常规广谱抗生素治疗无效。在治疗的第7天，然后每月一次监测泊沙康唑的水平，全血细胞计数，电解质和肝功能。除三名患者外，泊沙康唑的耐受性良好。与液体制剂的60％相比，所有接受片剂制剂的患者血浆血浆水平均> 1 mg / l。在泊沙康唑治疗后，整个队列的FEV1有适度但显着的改善（预测的百分比）（p = 0.015）。泊沙康唑在6岁以下的儿童中耐受良好，观察到肺功能有所改善，