Do you often see clinical improvement that is significant enough to de-list patients with CF for transplant after the initiation of CFTR modulator therapy?
2 Answers
Mednet Member
Pulmonology · Hospital of the University of Pennsylvania
Yes, this is actually very common and has led to a dramatic decrease in lung transplants and referrals for persons with CF.
Here is a paper from France that shows this more systematically Burgel et al., PMID 33600738.
There is also a follow-up paper, which I cannot find right now.
Denis Hadjiliaids
Mednet Member
Pulmonology · Cleveland Clinic
https://www.cff.org/medical-professionals/patient-registry
You can download the CF Patient Registry Annual Data Report with this link. On page 71 of the report, the number of individuals receiving a transplant by year is provided. There was a steadily increasing trend (with 2016-19 averaging about 2...