ESPE2019 Poster Category 1 Growth and Syndromes (to include Turner Syndrome) (13 abstracts)
Clinical Outcomes in Primary Empty Sella (ES) Syndrome in Childhood-Onset Growth Hormone Deficiency: Data from KIGS (Pfizer International Growth Database)
Mohamad Maghnie 1 , Martin Carlsson 2 , Ferah Aydin 3 & Cecilia Camacho-Hübner 2
1Department of Pediatrics, IRCCS Giannina Gaslini, University of Genova, Genova, Italy. 2Endocrine Care, Pfizer Inc, New York, NY, USA. 3Endocrine Care, Pfizer Health AB, Sollentuna, Sweden
Background: The incidence of ES in children varies greatly depending on the population surveyed, ranging from 1.2%-9% to 68% (children without and with known endocrinopathies, respectively). MRI is the main diagnostic tool for screening pituitary and in a previous KIGS study, 3.0% of patients with GHD were identified with ES and 7.8% with pituitary hypoplasia (Maghnie et al, EJE (2013).
Aim: To evaluate the clinical outcomes to GH treatment in patients with ES and compare the clinical response in patients with pituitary hypoplasia (PH), hypoplastic anterior pituitary, missing stalk and ectopic posterior pituitary (HME) and other central malformation (OCM).
Patients and Methods: All patients diagnosed with GHD and neuroimaging findings of ES, PH, HME and OCM in KIGS were included in this study. Descriptive statistics for the ES cohort compared growth response to GH treatment at yr 1, 5 and near adult height (NAH). Delta height SDS (ΔHt SDS) among the other cohorts were compared at yr1. Wilcoxon signed rank and Kruskal–Wallis tests were applied. Significance level=5%.
Results: Clinical characteristics and outcomes in patients with ES.
| At visit | Baseline | Yr 1 | Yr 5 | NAH | P-value** |
| Variable | Mean (SD) | Mean SD) | Mean SD) | Mean(SD) | |
| N (boys %) | 702 (69) | 702 (69) | 370 (69) | 89 (61) | |
| Age at diagnosis (yr) | 7.7 (5.0) | ||||
| Chronological age | 8.0 (4.9) | 9.0 (4.9) | 11.3 (4.1) | 17.6 (1.4) | <.001 |
| Mid-parental Ht SDS Prader | -0.8 (1.2) | ||||
| Height (SDS) Prader | -3.5 (1.6) | -2.5 (1.5) | -1.1 (1.4) | -0.8 (1.3) | <.001 |
| ΔHt SDS Prader | 1.0 (0.8) | 2.5 (1.4) | 3.1 (1.5) | <.001 | |
| Ht - MPH (SDS) Prader | -2.6 (1.6) | -1.6 (1.4) | -0.3 (1.4) | 0.1 (1.1) | <.001 |
| Weight (SDS) | -2.4 (2.0) | -1.7 (1.7) | -0.4 (1.6) | -0.3 (1.9) | <.001 |
| In puberty | 11% | 20% | 37% | 100% | |
| Bone Age (yr) | 6.2 (4.1) | 7.3 (4.3) | 10.0 (3.6) | 15.8 (1.4)* | 0.031 |
| Max GH peak (µg/L) | 4.6 (5.7) | ||||
| Dose (mg/kg/week) | 0.22 (0.08) | 0.21 (0.06) | 0.20 (0.06) | 0.15 (0.09) | <.001 |
| Years on GH treatment | 10.6 (3.6) | ||||
| *N=13 at NAH; **Comparing NAH vs baseline | |||||
Summary results for other cohorts included height SDS at baseline and year 1 ΔHt SDS: PH (n=180; -3.4(1.6); 0.9(0.7), HME (n=485; -3.3(1.7);1.1 (1.0) and OCM (n=121; -3.5(1.6); 1.0(1.0). P-values=0.09, 0.134. No new safety signals were reported.
Conclusion: A significant clinical response to GH treatment in ES patients was observed at all treatment time points. Patients with other diagnosis also demonstrated a positive response to treatment at year 1.
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