ESPE Abstracts

Background: There is limited knowledge about the natural history of growth in patients with congenital hyperinsulinism (CHI). The disease itself, as well as its treatment methods with common long-term sequalae in terms of pancreatic endocrine and exocrine dysfunction have the potential to affect growth. We investigated longitudinal height growth of CHI patients in a large Finnish cohort.

Materials and methods: In this cross-sectional study, comprehensive auxological and clinical data of 93 eligible patients from the nationwide cohort of 106 patients with persistent CHI (born 1972-2015) were collected from medical records and examined from birth until 6.5 years of age. Growth parameters and outcome measures of height were statistically compared to the national growth references, parental heights, and between different treatment subgroups. Growth failure was defined as end-height below 10th percentile (<-1.3 SD).

Results: The median follow-up time of the 93 patients (53% male, 66% with identified genetic etiology, 33% surgically treated, 77% in clinical remission without medication for hyperinsulinism) was 5.0 (IQR 2.4 – 6.1) years. The patients reached mean end-height of -0.22 SD (95% CI -0.48 – 0.04), corresponding to mean of -0.19 SD (95% CI -0.36 – -0.02) compared to their target height SD. Growth failure was found in 43% of the patients treated with octreotide, 26% of those with near-total pancreatectomy (nt-Px), 13% of patients treated with diazoxide, and in 9% of patients who had lesionectomy for focal CHI. Patients treated with octreotide (n=7) showed markedly lower meandian end-heights of -1.27 SD (95% CI -2.35 – -0.19) compared to standard references, corresponding -0.72 SD (95% CI -1.51 – 0.08) difference compared to their target height. The patients with nt-Px reached lower meandian end-height of -0.26 SD (95% CI -0.99 – 0.46, n=19) compared to their target height, with a statistical difference compared to 0.88 SD (95% CI -0.41 – 2.18, n=12) of the patients with pancreatic lesionectomy (P=0.04) and a non-significant tendency of higher proportion of clinical exocrine pancreatic insufficiency in nt-Px group (25% vs 58%, P=0.092).

Conclusions: The overall prognosis of childhood growth is favorable in CHI but varies between patients treated with different treatment methods. Especially the patients with diazoxide-unresponsive CHI form treated with octreotide are at significant risk of childhood growth failure.