ECFS 2020 - Optimizing pharmaceutical care in cystic fibrosis

302    www.ecfs.eu OPTIMIZING PHARMACEUTICAL CARE IN CYSTIC FIBROSIS CFTR MODULATORS AND PREGNANCY, A CASE STUDY CHAPTER 18 ivacaftor and one used tezacaftor/ivacaftor (Symkevi). In five of the 12 pregnancies CFTR modulating therapy was continued throughout the entire pregnancy. Of the seven patients who discontinued CFTR modulators, the medication had to be restarted due to pulmonary deterioration. Five out of 12 patients experienced compli- cations during pregnancy, including three who developed gestational diabetes/ CFRD [3]. Rebecca, who also had CFRD, also had an increase in serum glucose during her pregnancy. The two cases described here illustrate that, in the absence of direct evidence for safety or harm, treatment with CFTR modu- lating therapy during pregnancy is a tailored decision. For both cases, the patient and consultant are content with the decision made and would do the same in an iden- tical situation. Both patients state they were informed and counselled well by their consultant and that the advice was very important. They trusted and relied heavily on their doctor’s opinion about whether or not to use lumacaftor/ivacaftor during pregnancy, as well as the assessment of the potential risks of use or non-use. This places a heavy responsibility on the shoul- ders of the consultant, who has to explain clearly and carefully all considerations. Patients should be informed that risks cannot be excluded and birth defects are possible – whether or not related to the use of medication. To assist clinicians, as much data as possible on CFTR-modulating ther- apies during pregnancy should be gathered and made accessible. until after birth. Starting lumacaftor/ivacaftor during pregnancy would still be possible if the clinical condition of the mother started to decline. Several times during the course of the pregnancy, the need for starting lumacaftor/ivacaftor was evaluated but rejected every time. Based on the mother’s health, the use of lumacaftor/ivacaftor was not warranted at any stage during the preg- nancy. Deborah too had an uncomplicated pregnancy and delivered a healthy girl. 3 Discussion In the case of Rebecca, both the patient and the pulmonologist deemed the risk of a decline in health too high to discontinue lumacaftor/ivacaftor. This was motivated by the major benefit of lumacaftor/ivacaftor, that turned Rebecca’s unstable disease into a much improved and stable condition. The patient did have some worries concerning the effects on the unborn child from her medications and lumacaftor/ivacaftor specifically. But when the detailed ultra- sound scan at 20 weeks revealed no abnor- malities, her concerns were eased. The second patient, Deborah, had a FEV 1 similar to that of Rebecca before she started lumacaftor/ivacaftor. But because she had a relatively stable disease, it was chosen not to start lumacaftor/ivacaftor due to the unknown effects on the unborn child. Several case reports mention a successful pregnancy with the use of lumacaftor/ ivacaftor and in a case series, all 12 preg- nancies resulted in a live birth. Of these 12 pregnancies, 11 women used lumacaftor/