Thank you to the @CF_Foundation Teen Advocates from Alabama for stoping by to meet with my staff and share your stories!
Proud to see our youth using their voices to advocate on these important issues.
Thank you to Dr. Steven Rowe and Dr. Susan Walley who took time to address our Middle School students yesterday and our parents tonight about the health effects and increase in usage of… https://t.co/vqeDuQqmHO
@respandsleepjc @JenTaylorCousar @torontoadultcf Studies being planned to figure this out - what could be stopped, when, in whom? all key questions. #rsjc
@respandsleepjc @JenTaylorCousar @ElizabethTulli1 No one knows, but there has to be at some point. Where will it be? GOAL results suggest starts to slow > FEV1 95 baseline. https://t.co/ghyNHbQW6P. Need more experience to be sure. Ceiling for short term vs. long term changes likely to be different. #rsjc
@drstanbrook@respandsleepjc Yes, ongoing. FEV1 is primary. See: https://t.co/rsTZDksKnp. Early results announced in press release look promising: https://t.co/lWXyeS4KHX #rsjc
@drstanbrook@respandsleepjc Sweat CL, a measure of CFTR activity, dropped 51 mEQ at the 400mg dose. This brings levels to ~diagnostic threshold for CF (~60), and very similar to effects of IVA in G551D pwCF. This is not normal (~25), but in range of CF related d/o, c/w a better long term prognosis #rsjc
@EmadSaa05065769 The study was too short to pick up change in exacerbations. You need 6 months of on drug monitoring minimum, and 250 subjects per arm or more to pick up meaningful effects on PEx. PEx only examined for safety issues here. #rsjc
@lbudd3 @JenTaylorCousar @respandsleepjc Yes. In the cases where we recommended using to a txp patient, we suggested waiting ~6mos to be sure rejection issues and prograf levels stable. Prograf was one of the interactions, so obviously key to watch closely.
@lbudd3 One argument is that the txp lung does not have CF, thus no modulator is needed. But the systemic manifestations remain, thus there could be a role. We have used in selected patients where non respiratory disease is dominant, but be mindful of DDIs including txp meds.
@drstanbrook@respandsleepjc Their efficacy and mechanism are very similar, thus their discriminating factors haven't yet evolved enough to say if one is better than the other. The ongoing Phase 3 programs may be able to deliver that.
@respandsleepjc the in vitro correlates to the clinical response is one of the more remarkable aspects of the CFTR modulator development programs. This has enabled major development acceleration, since long, expensive and variable studies in animal models could be circumvented. #rsjc
@respandsleepjc It certainly is, and we don't yet know the cost of this treatment given its developmental status. Fortunately, in the US access to drugs in #CF has not so far been limited, to my knowledge, for the approved indications, but we will need to remain vigilant as a community. #rsjc