Systematic review of drug effects in humans and models with surfactant-processing disease

What is it about?

Patients with mutations in surfactant-processing genes develop progressive pulmonary fibrosis often resulting in fatal respiratory insufficiency. Although many mutations have been described, little is known about the optimal treatment strategy for lung fibrosis patients with surfactant-processing mutations. We performed a systematic literature review of studies that described a drug effect in patients, cell or mouse models with a surfactant-processing mutation. SP600125, a c-jun N-terminal kinase (JNK) inhibitor, hydroxychloroquine and 4-phenylbutyric acid were most frequently studied in disease models and lead to variable outcomes. This systematic review summarizes effect parameters for future studies on surfactant-processing disorders in disease models and provides directions for future trials in affected patients.

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