Breast development in a 7 year old girl with CF treated with ivacaftor: An indication for personalized dosing?
Drug plasma concentration
Ivacaftor
Side effect
Journal
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
ISSN: 1873-5010
Titre abrégé: J Cyst Fibros
Pays: Netherlands
ID NLM: 101128966
Informations de publication
Date de publication:
09 2021
09 2021
Historique:
received:
13
10
2020
revised:
27
05
2021
accepted:
01
06
2021
pubmed:
28
6
2021
medline:
8
2
2022
entrez:
27
6
2021
Statut:
ppublish
Résumé
Substantial progress has been made in the treatment of Cystic fibrosis due to introduction of CFTR modulators. However, little is known about the long term side effects of treatment with these drugs. We here present a 7 year old girl with CF who presented with breast development as a rare dose dependent side effect of treatment with ivacaftor and we report data on the correlation between drug plasma concentration and clinical effect, bodyweight, and BSA in 16 patients. Higher plasma concentrations did not correlate with clinical effect, as change in FEV1 and sweat chloride concentration. Patients with low bodyweight or BSA tended to have higher plasma concentrations. This might indicate that the current recommended dose of ivacaftor is at the top of the dose-response curve and that some patients can be treated with lower doses of ivacaftor with similar clinical effect.
Identifiants
pubmed: 34175243
pii: S1569-1993(21)01285-6
doi: 10.1016/j.jcf.2021.06.001
pii:
doi:
Substances chimiques
Aminophenols
0
Chloride Channel Agonists
0
Quinolones
0
ivacaftor
1Y740ILL1Z
Types de publication
Case Reports
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
e63-e66Informations de copyright
Copyright © 2021. Published by Elsevier B.V.
Déclaration de conflit d'intérêts
Declaration of Competing Interest Dr. Jeyaratnam, Dr. van der Meer, Dr. Berkers and Dr. Heijerman have nothing to disclose. Dr. Beekman reports grants from proteostasis, personal fees from proteostasis, outside the submitted work; In addition, Dr. Beekman is inventor on a patent related to Intestinal Organoid swelling and received royalties paid from Hubrecht Organoid Technology. Dr. van der Ent reports grants from GSK, grants from Nutricia, grants from TEVA, grants from Gilead, grants from Vertex, grants from ProQR, grants from Proteostasis, grants from Galapagos NV, grants from Eloxx, outside the submitted work; In addition, Dr. van der Ent has a patent 10,006,904 with royalties paid.