Lumacaftor + Ivacaftor: A new treatment option for cystic fibrosis? |
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Lumacaftor + Ivacaftor: A new treatment option for cystic fibrosis?

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A combination of lumacaftor and ivacaftor has been found to improve lung function in cystic fibrosis patients and also reduced the need for intravenous (IV) antibiotics in a new study published online Oct. 11, 2019 in the American Journal of Respiratory and Critical Care Medicine.

The study conducted at 47 cystic fibrosis centers in France involved 845 patients, both adolescents and adults. Patients who took lumacaftor-ivacaftor continuously for 12 months experienced:

  • a 3.67% increase in their percent predicted FEV1.
  • A weight increase by approximately 2 to 3 kg (4.5-6.5 lbs.).
  • A 35% reduction in courses of intravenous antibiotics.

The combination therapy was also associated with a reduction in frequent (≥ 2) exacerbations per year.

  • Patients who discontinued treatment were more likely to have experienced an exacerbation after IV antibiotics in the year before taking the combination drug.
  • Adults were more likely than adolescents to discontinue treatment.
  • Adults, but not adolescents, who discontinued therapy were at high risk of rapid FEV1 decline, BMI decrease and multiple respiratory exacerbations.

About 18% of participants in the study dropped out. Nearly half the patients who discontinued treatment did so because of adverse respiratory events and more than a quarter discontinued the treatment due to adverse non-respiratory events, most often digestive problems.

Lumacaftor-ivacaftor combination has been approved by the US FDA and EMA in 2015 for treatment of cystic fibrosis patients who have inherited the same defective CFTR gene (Phe508del mutation)….(Excerpts from American Thoracic Society)


Dr KK Aggarwal

Padma Shri Awardee

President Confederation of Medical Associations in Asia and Oceania (CMAAO)

Group Editor-in-Chief IJCP Publications

President Heart Care Foundation of India

Past National President IMA

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