Real-world results are out for cystic fibrosis combo

Many more patients discontinued lumacaftor-ivacaftor than in pivotal clinical trials: researchers
Reuters Health
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Lumacaftor-ivacaftor treatment is associated with improved outcomes among adolescents and adults with cystic fibrosis who tolerate the therapy, a real-world study shows.

French researchers examined outcomes after the combination’s release in France in December 2015.

The analysed data on 292 adolescents and 553 adults who started lumacaftor-ivacaftor treatment in one of the 47 centres participating in the French Cystic Fibrosis Reference Network.

During the first year after treatment initiation, 18% of patients (24% of adults and 8% of adolescents) discontinued treatment, with nearly half of discontinuations due to respiratory adverse events.