Predictive factors for lumacaftor/ivacaftor clinical response
Alexandra Masson, Elena K Schneider-Futschik, Nesrine Baatallah, Nguyen-Khoa Thao, Emmanuelle Girodon, Aurelie Hatton, Thomas Flament, Muriel Le Bourgeois, Frederique Chedevergne, Celine Bailly, Sylvia Kyrilli, Diane Achimastos, Alexandre Hinzpeter, Aleksander Edelman, Isabelle Sermet-Gaudelus
JOURNAL OF CYSTIC FIBROSIS | ELSEVIER SCIENCE BV | Published : 2019
BACKGROUND: Ivacaftor-lumacaftor combination therapy corrects the F508 del-CFTR mutated protein which causes Cystic Fibrosis. The clinical response of the patients treated with the combination therapy is highly variable. This study aimed to determine factors involved in the individual's response to lumacaftor-ivacaftor therapy. METHODS: Sweat test was assessed at baseline and after 6 months of ivacaftor-lumacaftor treatment in 41 homozygous F508del children and young adults. β-adrenergic peak sweat secretion, nasal potential difference (NPD) and intestinal current measurements (ICM) were performed in patients accepting these tests. Seric level of lumacaftor and ivacaftor were determined and ..View full abstract
Awarded by Vaincre La Mucoviscidose Grant
This work was supported by AARM (Association pour l'Aide a la Recherche contre la Mucoviscidose), ABCF Protein Association and Vaincre La Mucoviscidose Grant RC20160501612/1/3/8.