Outcomes of pregnancy in women with cystic fibrosis (CF) taking CFTR modulators – an international survey

Nash, Edward F.; Middleton, Peter G.; Taylor‐Cousar, Jennifer L.

doi:10.1016/j.jcf.2020.02.018

Cited by 64 publications

(61 citation statements)

References 20 publications

(31 reference statements)

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“…One of the main aims of CF registers in the future will be to follow the pregnancy outcomes of women receiving CFTR modulators. [21] In conclusion, analysis of the ICFR data relating to this large cohort of mother-child dyads showed that CF women can successfully undergo pregnancy, and that this often leads to the birth a full-term newborn of adequate weight. The very high percentage of caesarean sections transversally related to various factors is often an a priori choice rather than being determined by the clinical condition of the mother, and deserves further investigation.…”

Section: Discussionmentioning

confidence: 80%

Perinatal Outcomes in Cystic Fibrosis Women: Data From the Italian Cystic Fibrosis Registry.

Padoan¹,

Quattrucci²,

Amato³

et al. 2020

Preprint

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Data from the Italian Cystic Fibrosis Registry (ICFR) concerning pregnancies in the period 2010-2015 were used to investigate the association between the pre-conception clinical status and perinatal outcomes of women with CF. The assessed clinical variables were genotype, pre-conception age, the body mass index (BMI), and the percentage of predicted forced expiratory volume in one second (ppFEV1); the analysed outcomes were gestational age, birth weight (BW), and the frequency of caesarean deliveries. Complete information concerning mother and child was available for 56 pregnanciesthat came to term. The median pre-conception age was 30.8 years (18.7-42.3); median BMI was 21.5 kg/m2(16.5-26.8); and median ppFEV1was 73.9% (30-128%).Twenty-four women (42.8%) carried class I-III mutations, and 25 (44.8%) at least one mutation conferring residual CFTR function or having variable clinical consequences. The median duration of pregnancy was 37 weeks (31-41), and the frequency of prematurity was 28.30%. Median BW was 2,910 g (1,300-3,650). There was a positive correlation between BMI and ppFEV1 in comparison with BW (r=0.29 andr=0.31 by p=0.048 and p=0.032, respectively), but no correlation between age and BW. The overall frequency of caesarean sections was 63.64%, but it was 77.27% among the women with severe genotypes. A lower ppFEV1 corresponded to a lower BW (p=0.047). Respiratory function had no effect on the percentage of caesarean deliveries. The nutritional status and respiratory function of CF mothers correlate with their newborn’s BW. Caesarean sections are very frequent, even in young women with normal lung function.

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Section: Discussionmentioning

confidence: 80%

Perinatal Outcomes in Cystic Fibrosis Women: Data From the Italian Cystic Fibrosis Registry.

Padoan¹,

Quattrucci²,

Amato³

et al. 2020

Preprint

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“…In addition to the case reports, which provided reassurance for normal infant outcomes following modulator exposure during pregnancy, Nash et al recently reported the results of an international survey of CF center physicians regarding modulator use in women with CF during pregnancy [ 47 ]. Results from a total of 64 pregnancies in 61 women were summarized (31 pregnancies with ivacaftor exposure, 26 pregnancies with lumacaftor/ivacaftor exposure, and 7 pregnancies with tezacaftor/ivacaftor exposure).…”

Section: Impact Of Cftr Modulators On the Sexual And Reproductive mentioning

confidence: 99%

“…No modulator-related complications were reported in infants exposed in utero. The miscarriage rate for women with CF on modulator therapy was 4.7% [ 47 ], which was lower than the reported 10–15% miscarriage rate expected in the general population [ 48 ].…”

Section: Impact Of Cftr Modulators On the Sexual And Reproductive mentioning

confidence: 99%

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CFTR Modulators: Impact on Fertility, Pregnancy, and Lactation in Women with Cystic Fibrosis

Taylor‐Cousar

2020

JCM

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Cystic fibrosis (CF) is a life-shortening genetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. These mutations lead to abnormal ion transport in mucous membranes throughout the body, including in the respiratory and gastrointestinal and reproductive tracts. Improvements in care and therapy have led to substantial increases in the quantity and quality of life for those with CF. Consequently, women with CF are increasingly interested in having families. Although pregnancy was once discouraged for women with CF, at this point, even women with moderately severe lung disease can successfully navigate pregnancy. With the recent approval of a triple combination CFTR modulator therapy that improves lung function, nutritional status, and quality of life for people with a single copy of the most common CFTR mutation, it is expected that the number of women with CF who choose to become pregnant will continue to increase. Although animal reproduction models show no alarming signals for use during pregnancy at normal human doses, there is a paucity of human safety data in pregnancy and lactation. This review summarizes what is currently known about the impact of use of CFTR modulators on fertility, pregnancy, and lactation in women with CF.

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“…Earlier this year, Nash and his colleagues published an international survey looking at the safety of cystic fibrosis drugs taken during pregnancy 10 . All the cases they looked at involved women with cystic fibrosis who were taking the drugs for themselves, not their unaffected fetuses, so the survey could not gauge the efficacy of in utero drug treatment.…”

Section: The Leap To Humansmentioning

confidence: 99%

In utero intervention to stem the damage of cystic fibrosis

Dolgin¹

2020

Nature

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hen ivacaftor hit the market in 2012, becoming the first medicine to treat the underlying cause of cystic fibrosis (see page S2), it was initially available only for people aged six years and older. Trial data from younger children helped to lower the age limit to two. Then one. And in 2019, regulatory authorities approved the drug for infants as young as six months old who carry certain mutations in cystic fibrosis trans membrane conductance regulator (CFTR), the gene responsible for the disease. Doctors are already evaluating the safety and efficacy of giving ivacaftor to newborn babies diagnosed with cystic fibrosis. And it's conceivable that they could begin treatment even earlier, perhaps months before birth-thanks in part to the advent of a simple blood test for pregnant women that can diagnose cystic fibrosis in utero. Many babies born with cystic fibrosis already have damage to their organs that can lead to diabetes, gastrointestinal issues and male infertility. "The reality is that there's a lot A scientist prepares samples for the UNITY blood test, which can screen for cystic fibrosis. BILLIONTOONE, INC.

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Outcomes of pregnancy in women with cystic fibrosis (CF) taking CFTR modulators – an international survey

Cited by 64 publications

References 20 publications

Perinatal Outcomes in Cystic Fibrosis Women: Data From the Italian Cystic Fibrosis Registry.

Perinatal Outcomes in Cystic Fibrosis Women: Data From the Italian Cystic Fibrosis Registry.

CFTR Modulators: Impact on Fertility, Pregnancy, and Lactation in Women with Cystic Fibrosis

In utero intervention to stem the damage of cystic fibrosis

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