A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis
SCIENTIFIC REPORTS
Authors: Habib, Al-Rahim R.; Kajbafzadeh, Majid; Desai, Sameer; Yang, Connie L.; Skolnik, Kate; Quon, Bradley S.
Abstract
Several placebo-controlled trials have been recently published evaluating novel therapies targeting the defective CFTR protein. This systematic review examines the clinical efficacy and safety of CFTR modulators in individuals with cystic fibrosis (CF) with specific genetic mutations. Online sources were searched for placebo-controlled, parallel-design clinical trials investigating CFTR modulators from January 1, 2005 to March 31, 2018. The primary outcome of interest was FEV1% predicted (ppFEV(1)). Fourteen RCTs met our eligibility criteria. The largest improvement in ppFEV(1) favouring treatment was observed for ivacaftor (IVA) in G551D individuals (>= 6 years old). Both tezacaftor-ivacaftor (TEZ-IVA) and lumacaftor-ivacaftor (LUM-IVA) also improved ppFEV(1) in F508del homozygous individuals but there was increased reporting of respiratory adverse events with LUM-IVA compared to placebo. IVA also significantly improved ppFEV(1) in a sub-group of individuals >= 18 years old with an R117H mutation. No significant improvements in ppFEV(1) were observed for IVA, LUM, or TEZ in F508del homozygous individuals, LUM or LUM-IVA in F508del heterozygous individuals, or ataluren in individuals with a nonsense mutation. Significant improvements in ppFEV(1) and other clinical outcomes were observed for IVA in G551D individuals, TEV-IVA and LUM-IVA in F508del homozygous individuals, and IVA in adults with a R117H mutation.
Color-Tunable Circularly Polarized Luminescence with Helical Polyacetylenes as Fluorescence Converters
ADVANCED OPTICAL MATERIALS
Authors: Zhao, Biao; Gao, Xiaobin; Lu, Na; Deng, Jianping
Abstract
Circularly polarized luminescence (CPL) materials are currently drawing rapidly increasing interest. However, constructing color-tunable CPL materials is still a big challenge, primarily due to the lack of simple, powerful, and universal preparation strategies. In this contribution, color-tunable CPL emissions are easily realized by taking helical polyacetylenes as fluorescence converters. The investigated helical polyacetylenes are prepared by solution polymerization of a substituted acetylenic monomer with cholesteryl pendant. The prepared polymer exhibits dynamically reversible solvatochromism performance in different solvents, owing to the change of conjugated length of polymer main-chains. More excitingly, when circularly polarized light with blue emission passes through the as-prepared helical polyacetylene with different conjugated lengths, multicolor CPL emissions of cyan, green, and orange are obtained, with high dissymmetry factors (g(lum)) in the range of 10(-2)-10(-1). Also notably, neither any covalent nor noncovalent interaction is required during this CPL color-tuning process. The present study provides a new insight for developing multicolor CPL materials.