TY - JOUR
T1 - Maternal and fetal outcomes following elexacaftor-tezacaftor-ivacaftor use during pregnancy and lactation
AU - Taylor-Cousar, Jennifer L.
AU - Jain, Raksha
N1 - Funding Information:
Supported by the Asher family fund. We would like to acknowledge the vital contributions of women with CF, their children and the caregivers that contributed data for this study.
Publisher Copyright:
© 2021
PY - 2021/5
Y1 - 2021/5
N2 - Background: With the improved health afforded by cystic fibrosis transmembrane conductance regulator (CFTR) modulators, pregnancy rates are increasing in women with CF. In animal reproductive models, the three components of elexacaftor-tezacaftor-ivacaftor (ETI) did not cause teratogenicity at normal human doses. Although the limited human data available in the literature for previously approved modulators did not suggest cause for concern, there is currently no data in the literature regarding use of ETI in pregnant women. Thus, the decision to continue therapy during pregnancy (with the associated unknown fetal impact) versus discontinuing therapy (with the known risk of maternal health decline) is challenging. Methods: CF Center staff completed an anonymous questionnaire regarding pregnancy and infant outcomes for women who used ETI during pregnancy and/or lactation. Results: Of 45 ETI-exposed pregnancies reported to date, complications in 2 mothers and in 3 infants (2 born to mothers with poorly controlled diabetes) were rated by clinicians as unknown (possible) or suspected relatedness to ETI use. Two women terminated unplanned pregnancies. Miscarriage rates were consistent with that known in the general U.S. population. Five of the six women who discontinued ETI out of concern for unknown fetal risk restarted because of clinical deterioration. No infant cataracts were reported though only two infants were formally evaluated. Conclusions: In the context of the known increased rate of complications in women with CF and their infants, data from this retrospective survey is reassuring for women who choose to continue ETI during pregnancy. However, a large, multi-center prospective study is needed to assess impact of use of ETI in pregnancy.
AB - Background: With the improved health afforded by cystic fibrosis transmembrane conductance regulator (CFTR) modulators, pregnancy rates are increasing in women with CF. In animal reproductive models, the three components of elexacaftor-tezacaftor-ivacaftor (ETI) did not cause teratogenicity at normal human doses. Although the limited human data available in the literature for previously approved modulators did not suggest cause for concern, there is currently no data in the literature regarding use of ETI in pregnant women. Thus, the decision to continue therapy during pregnancy (with the associated unknown fetal impact) versus discontinuing therapy (with the known risk of maternal health decline) is challenging. Methods: CF Center staff completed an anonymous questionnaire regarding pregnancy and infant outcomes for women who used ETI during pregnancy and/or lactation. Results: Of 45 ETI-exposed pregnancies reported to date, complications in 2 mothers and in 3 infants (2 born to mothers with poorly controlled diabetes) were rated by clinicians as unknown (possible) or suspected relatedness to ETI use. Two women terminated unplanned pregnancies. Miscarriage rates were consistent with that known in the general U.S. population. Five of the six women who discontinued ETI out of concern for unknown fetal risk restarted because of clinical deterioration. No infant cataracts were reported though only two infants were formally evaluated. Conclusions: In the context of the known increased rate of complications in women with CF and their infants, data from this retrospective survey is reassuring for women who choose to continue ETI during pregnancy. However, a large, multi-center prospective study is needed to assess impact of use of ETI in pregnancy.
KW - CFTR
KW - cystic fibrosis
KW - infant
KW - lactation
KW - modulators
KW - pregnancy
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U2 - 10.1016/j.jcf.2021.03.006
DO - 10.1016/j.jcf.2021.03.006
M3 - Article
C2 - 33762125
AN - SCOPUS:85103053174
SN - 1569-1993
VL - 20
SP - 402
EP - 406
JO - Journal of Cystic Fibrosis
JF - Journal of Cystic Fibrosis
IS - 3
ER -