Department of Pediatric Pneumology, Dr. von Haunersches Kinderspital, German Center for Lung Research, University of Munich, Munich, Germany.
Pediatric Pulmonology and Sleep Medicine, Cystic Fibrosis Center, Children's Hospital, University of Duisburg-Essen, Essen, Germany.
Pediatr Pulmonol. 2021 Aug;56(8):2627-2633. doi: 10.1002/ppul.25513. Epub 2021 May 28.
Hypersensitivity pneumonitis (HP) in children is a severe interstitial lung disease and potentially, a chronic condition, if not treated appropriately. No evidence-based guidelines are available; in particular, the role of systemic glucocorticoid therapy is unclear.
The aim of this randomized, double-blind, placebo-controlled, parallel-group, multi-center, phase II trial in pediatric HP was to assess the outcome of HP in children after 6 months of treatment and to compare 3 months of treatment with oral prednisolone or placebo.
After 1.5 years and the inclusion of only four children, we terminated the study prematurely. Two of the children randomized to prednisolone did not achieve the predefined response of FVC to normal. One child treated with placebo recovered to normal, similar to another child treated with prednisolone. All children treated with steroids developed drug-related side effects.
This uncompleted study illustrates the urgent medical need for evidence-based treatment protocols for this condition. We discuss the hurdles which were specific for completion of this trial in a rare condition. Among other options, we suggest the inclusion of children into an all-age study of HP, as in adults the same questions are unanswered.
儿童过敏性肺炎（HP）是一种严重的间质性肺病，如果治疗不当，可能会发展为慢性疾病。目前尚无基于证据的指南，特别是全身糖皮质激素治疗的作用尚不清楚。
本研究是一项针对儿科 HP 的随机、双盲、安慰剂对照、平行组、多中心、二期临床试验，旨在评估儿童 HP 治疗 6 个月后的结果，并比较口服泼尼松龙或安慰剂治疗 3 个月的效果。
1.5 年后，仅纳入了 4 名儿童，我们提前终止了研究。随机分配至泼尼松龙组的 2 名儿童的 FVC 未达到预设的正常反应。1 名接受安慰剂治疗的儿童恢复正常，与接受泼尼松龙治疗的另 1 名儿童相似。所有接受类固醇治疗的儿童均出现与药物相关的副作用。
这项未完成的研究说明了针对这种疾病制定基于证据的治疗方案的迫切医疗需求。我们讨论了在这种罕见情况下完成试验所面临的具体障碍。除了其他选择之外，我们还建议将儿童纳入成人 HP 的全年龄段研究中，因为成人中同样的问题也尚未得到解答。
