ESPE Abstracts (2022) 95 P1-233

ESPE2022 Poster Category 1 Diabetes and Insulin (86 abstracts)

Effects of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)-Modulating Therapy on Glucose Metabolism in Cystic Fibrosis Patients

Pamela Yesquen , Ariadna Campos , Eduard Mogas , Diego Yeste , Silvia Gartner & María Clemente

Vall d'Hebron Hospital, Barcelona, Spain

Introduction: CFTR-modulating therapy aims to restore CFTR function. It improves lung function and quality of life in CF patients however, its effects on glucose metabolism are not yet well defined.

Methodology: Retrospective and longitudinal study.

Inclusion criteria: patients with CF genetic diagnosis ≥10 years old on modulator therapy (ivacaftor + lumacaftor or ivacaftor + tezacaftor or triple therapy: elexacaftor + tezacaftor + ivacaftor) and who had OGTT ± Continuous-glucose-monitoring (CGM) performed before and after therapy onset. Tests were performed without underlying disease exacerbations or corticosteroid at the time of test or 4 weeks prior. OGTT classifies patients into normal tolerance (NGT), impaired tolerance (AGT) or diabetes (CFRD). CGM (IproTM2-Medtronic) performed for 6 days with regular exercise and diet; patients were classified as NGT: <4.5% of monitoring time >140mg/dl, AGT: ≥4.5% of monitoring time >140mg/dl or one glucose peak ≥200mg/dl and CFRD: ≥2glucose peaks ≥200mg/dl on different days.


Patients and glucose metabolism
Therapy F/M Age (years) Pre-therapy Time to control study (months) Control
1. A F 10,25 AGT (INDET) -- 17 NGT --
2. B M 14,58 NGT AGT 13 NGT NTG
3. M 15,50 NGT NGT 7 NGT --
4. M 15,60 NGT NGT 17 NGT NTG
5. M 17,25 NGT NGT 13 NGT --
6. F 17,33 NGT NGT 18 NGT CFRD
7. F 20,50 AGT AGT 19 NGT NGT
8. C M 17,50 CFRD -- 21 AGT --
A:lumacaftor and ivacaftor. B: tezacafor and ivacaftor. C: ivacaftor, tezacaftor and elexacaftor

HbA1c was <6% before and at therapy control, except for patient Nº6 who started with HbA1c7.9%. Five patients (62.5%) were homozygous and 3 heterozygous for F508del mutation. Patients with pre-therapy glucose abnormalities improved and one patient without glucose abnormalities worsened. Patient nº8 required nasogastric tube feeding, insulin (CFRD) and was a lung transplant candidate before beginning triple therapy; nowadays he doesn`t need nasogastric tube feeding, stopped insulin after 16 months on therapy and he is no longer a transplant candidate. Patient nº6 had 11 peaks of postprandial glucose >200mg/dl on control CGM while OGTT showed normal glucose values (maximum 138mg/dl).

Conclusions: • CFTR-modulating therapy appears to improve glucose metabolism in patients who had glucose abnormalities.

• The effects of CFTR-modulating therapy on glucose metabolism seem to take longer than the rapid improvement observed in lung function and nutritional status.

• CGM is more sensible than OGTT to changes of glucose metabolism in CF patients.

Volume 95

60th Annual ESPE (ESPE 2022)

Rome, Italy
15 Sep 2022 - 17 Sep 2022

European Society for Paediatric Endocrinology 

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