Symdeko (tezacaftor/ivacaftor) shows favourable results in Scottish real-world evidence study in cystic fibrosis

CONTEXT:  Publication of a real-world research study in cystic fibrosis in the European Journal of Hospital Pharmacy showed Vertex’s Symdeko (tezacaftor/ivacaftor) reduced hospital stays, number of days on antibiotics and favoured weight gain in 289 patients from a Scottish clinic.  However, the authors noted further work would be needed to assess the cost-effectiveness and impact on quality of life for patients receiving Symdeko.

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1. “Adults in this study had a baseline FEV1 of 46%, while those in the EVOLVE and EXPAND trials that showed significant lung function gains had baseline measures of 60% and 62%, respectively.” 

2. “Symdeko (tezacaftor/ivacaftor) favors weight gain, shortens hospital stays, and reduces the number of days on antibiotics in patients with cystic fibrosis (CF), a real-world study of its use at a Scottish clinic reported.” 

3. “To gather data from routine clinical practice, researchers in Scotland conducted a longitudinal study assessing the Symdeko’s effect in adults with CF.. They looked at the medical records of patients using Symdeko for at least four weeks — and followed for at least one year — at The West of Scotland Adult Cystic Fibrosis Unit.” 

4. “Symdeko’s use also shortened hospital stays by a median four days, and lowered by a median 21 days the number of days that patients’ used antibiotics to treat respiratory exacerbations.” 

5. “As such, it showed that “the use of tezacaftor-ivacaftor [Symdeko] in routine practice for people with cystic fibrosis was associated with improvements in weight, as well as reducing the number of days people needed to spend in hospital and receive antibiotics,” the investigators wrote.” 

Source URL: https://cysticfibrosisnewstoday.com/2021/06/29/symdeko-vertex-favors-weight-gain-limits-hospital-stays-real-life-study-scotland/