Abstract
Objectives: In patient-derived rectal organoids (PDRO), CFTR modulator efficacy can be evaluated in the forskolin-induced swelling (FIS) assay. In this assay, PDRO swelling in response to acute stimulation with forskolin (Fsk) and potentiator(s) is quantified as a measure of restoration of CFTR function after incubation with corrector(s). However, with high residual CFTR function and/or modulator efficacy (eg. potentiator effect of elexacaftor), PDRO may swell already during the incubation step, before any FIS measurements. The magnitude of modulator efficacy is underestimated in these cases. Comparison with non-CF organoids is therefore also not informative. Rectal organoid morphology analysis (ROMA) indexes have been shown previously (Cuyx Thorax 2021) to discriminate between PDRO from subjects with and without CF.
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