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

J Cyst Fibros. 2020 Jul;19(4):521-526. doi: 10.1016/j.jcf.2020.02.018. Epub 2020 Mar 6.

Abstract

Background: As their long-term prognosis improves, women with CF are increasingly choosing to have children, but the safety of CFTR modulators in pregnancy and breastfeeding is currently unknown.

Methods: A survey was sent to lead clinicians of adult CF centres in Europe, the United Kingdom (UK), United States of America (USA), Australia and Israel requesting anonymised data on pregnancy outcomes in women using CFTR modulators before and during pregnancy and lactation.

Results: We identified 64 pregnancies in 61 women taking IVA (n = 31), LUM/IVA (n = 26) or TEZ/IVA (n = 7), resulting in 60 live births. In 44 pregnancies, CFTR modulators were either continued throughout pregnancy or temporarily stopped and then restarted. Two maternal complications were deemed related to CFTR modulator therapy; cessation of modulator therapy resulted in clinical decline in 9 women prompting resumption of therapy during pregnancy. No modulator-related complications were reported in infants exposed in utero and/or during breastfeeding.

Conclusions: CFTR modulators were reported to be generally well tolerated in pregnancy and breastfeeding, with only 2 maternal complications that were deemed related to CFTR modulator therapy. Women stopping CFTR modulators in pregnancy may experience a decline in clinical status and in the cases identified in this survey, restarting therapy led to a clinical improvement. Current experience remains limited and longer-term prospective follow-up is required to exclude delayed adverse effects.

Keywords: Breastfeeding; CFTR; Cystic fibrosis; Infant; Modulators; Pregnancy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Breast Feeding* / methods
  • Breast Feeding* / statistics & numerical data
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics
  • Cystic Fibrosis* / diagnosis
  • Cystic Fibrosis* / drug therapy
  • Cystic Fibrosis* / epidemiology
  • Cystic Fibrosis* / genetics
  • Drug Monitoring / methods
  • Drug-Related Side Effects and Adverse Reactions* / diagnosis
  • Drug-Related Side Effects and Adverse Reactions* / epidemiology
  • Drug-Related Side Effects and Adverse Reactions* / etiology
  • Female
  • Humans
  • Infant, Newborn
  • International Cooperation
  • Medication Therapy Management / standards
  • Medication Therapy Management / statistics & numerical data
  • Membrane Transport Modulators* / administration & dosage
  • Membrane Transport Modulators* / adverse effects
  • Membrane Transport Modulators* / classification
  • Needs Assessment
  • Pregnancy
  • Pregnancy Complications* / diagnosis
  • Pregnancy Complications* / drug therapy
  • Pregnancy Complications* / epidemiology
  • Pregnancy Complications* / genetics
  • Pregnancy Outcome
  • Surveys and Questionnaires

Substances

  • Membrane Transport Modulators
  • Cystic Fibrosis Transmembrane Conductance Regulator