Effect in Transplant
PwCF who have had a transplant, were not included in the original trials
for modulators, however many have started to explore the outcomes. A
case series of 9 PwCF who had a lung transplant and started on ETI
demonstrated stability or improvement in their graft function,
improvements in BMI, and no one required changes in immunosuppression
dosing45.
PwCF who had liver transplants, the use of ETI has been shared in
multiple case reports, showing increased/stable FEV1pp and
BMI46-48. Based on the known side effects to the liver
with CFTR modulators, the starting dose of ETI ranged from full dose, AM
dose only, and half of the AM dose46-48. The ultimate
dose remained at full dosing regimen, only the AM dose, and some
discontinued ETI46-48. For those with a reduced final
dose, it was for several reasons, including clinicians never increasing,
elevation in liver function tests (LFTs), and one patient with
tacrolimus toxicity and acute kidney disease. Laboratory monitoring
varied with each patient but included LFTs and immunosuppressive
medication levels. Changes in dosing based on the interaction between
immunosuppressive medications and the CFTR modulator also varied, no
change in dose was seen in one PwCF on mycophenolate, while those on
tacrolimus/sirolimus required no changes, reductions and in only one
case an increase in dosing. Overall, the patients showed improvements
while being monitored closely for changes in immunosuppressive dosing
and LFTs. Therefore, these cases highlight additional benefits in
patients who have had transplants, with the importance of careful
monitoring.