TY - JOUR
T1 - Breast development in a 7 year old girl with CF treated with ivacaftor
T2 - An indication for personalized dosing?
AU - Jeyaratnam, Joshena
AU - van der Meer, Renske
AU - Berkers, Gitte
AU - Heijerman, Harry Gm
AU - Beekman, Jeffrey M
AU - van der Ent, Cornelis K
N1 - Funding Information:
This work was supported by grants of the Dutch Cystic Fibrosis Foundation (NCFS) as part of the HIT-CF program and the Dutch Health Organization ZonMw, Netherlands.
Publisher Copyright:
© 2021
PY - 2021/9
Y1 - 2021/9
N2 - 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.
AB - 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.
KW - Drug plasma concentration
KW - Ivacaftor
KW - Side effect
UR - https://www.scopus.com/pages/publications/85108508716
U2 - 10.1016/j.jcf.2021.06.001
DO - 10.1016/j.jcf.2021.06.001
M3 - Article
C2 - 34175243
SN - 1569-1993
VL - 20
SP - e63-e66
JO - Journal of Cystic Fibrosis
JF - Journal of Cystic Fibrosis
IS - 5
ER -