Preservation of β-cell Function in Pancreatic Insufficient Cystic Fibrosis With Highly Effective CFTR Modulator Therapy
Authors' conclusion
ETI preserves β-cell function in CF through effects on glucose-dependent insulin secretion, proinsulin processing, and meal-related insulin secretion. Further work should determine whether early intervention with ETI can prevent deterioration of glucose tolerance in PI-CF.
Study context
- Population
- Individuals with pancreatic insufficient cystic fibrosis (PI-CF); Elexacaftor/tezacaftor/ivacaftor (ETI) mean ± SD age 25 ± 5 yearsmatched controls
- Participants
- 9 vs 8
- Comparison
- ETI vs controls not yet treated with CFTR modulator therapy
- ETIn = 9
- Controlsn = 8reference
- Outcome
- Changes in β-cell function and secretory capacity measures over 2 visits
Methodological detailsmatched controls
- Matched controls
Main finding
As reported in the abstract
β-cell function (glucose-potentiated arginine)
- Glucose-potentiated insulin and C-peptide responses deteriorated in controls but not in ETI
- Basal proinsulin secretory ratio deteriorated in controls but improved in ETI, as did maximal arginine-induced proinsulin secretory ratio (P < .05 for all comparisons)
- ETI vs Controls: P < 0.05
Observational data: associations, not effects.
Further findings
Follow-up duration
P < 0.01
- Follow-up duration — ETI initiation before follow-up (median)
- 1 year
Mixed-meal tolerance test
- Early insulin secretion improved, with more rapid insulin secretory rate kinetics
Original publication
Preservation of β-cell Function in Pancreatic Insufficient Cystic Fibrosis With Highly Effective CFTR Modulator Therapy. J Clin Endocrinol Metab. Published 28 July 2023. doi:10.
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