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.