ESPE2014 Poster Presentations Growth (2) (13 abstracts)
Safety and Effectiveness of Increlex® Therapy in Children Enrolled in the Increlex® Growth Forum Database in Europe: 4 Years Interim Results
Michel Polak a , Joachim Woelfle b , Peter Bang c , Aude Sicsic d & Bruno Fiorentino d
aHôpital Necker Enfants Malades, AP-HP, Université Paris Descartes, Paris, France; bChildren’s Hospital, University of Bonn, Bonn, Germany; cFaculty of Health Sciences, Linköping University, Linköping, Sweden; dIPSEN Pharma, Boulogne Billancourt, France
Background: The post-authorization registry, European Increlex® (Mecasermin (rDNA Origin) injection) growth forum database (EU-IGFD) was initiated in Dec 2008 to collect data on children with growth failure treated with Increlex®.
Objective: To report 4-year safety and effectiveness data.
Methods: Multicenter, open-label observational study, eCRF data collection.
Results: As of 30/09/13, 195 patients were enrolled in nine countries: 34% female, 85% severe primary IGF1 deficiency, 81% pre-pubertal, and 67% growth treatment-naïve. Median age at first injection was 10.6 years. Mean (95% CI) treatment duration was 832 (762; 902) days. Median dose was 40 μg/kg BID at treatment initiation, 116 at year 1 and 120 from years 2 to 4. Effectiveness data are summarized by mean (S.D.) of height SDS and Δheight SDS.
Targeted adverse events were reported for 74 pts (39%), the most frequent (≥5%) being hypoglycaemia (18%), lipohypertrophy (11%), tonsillar hypertrophy (7%), headaches (6%), and injection site reactions (6%). Hypoglycaemia tended to be related to age at start of treatment (OR [95% CI]=0.92 (0.83; 1.01) by 1 year increment) and to whether the patient has Laron syndrome (OR [95% CI]=4.3 (1.8; 10.6)).
| All pts | na | Height SDS | na | ΔHeight SDS |
| Baseline | 176 | −3.50 (1.34) | ||
| Year 1 | 151 | −3.18 (1.37) | 144 | 0.34 (0.42) |
| Year 2 | 104 | −3.14 (1.58) | 98 | 0.59 (0.64) |
| Year 3 | 66 | −2.76 (1.78) | 62 | 0.73 (0.74) |
| Year 4 | 25 | −2.87 (1.75) | 23 | 0.92 (0.80) |
| Treatment-naive pre-pubertal pts | ||||
| Baseline | 102 | −3.35 (1.34) | ||
| Year 1 | 71 | −2.92 (1.33) | 67 | 0.47 (0.38) |
| Year 2 | 41 | −3.06 (1.59) | 39 | 0.78 (0.64) |
| Year 3 | 22 | −2.70 (2.00) | 21 | 0.90 (0.79) |
| aData available at the timepoint. | ||||
Conclusions: The overall increase in height SDS was confirmed on a larger cohort, with better results in naïve pre-pubertal pts. The safety profile remains consistent with the product labeling.
Article tools
My recent searches
My recently viewed abstracts
ESPE Abstracts
© Bioscientifica 2024 |
Privacy policy |
Cookie settings
BiosciAbstracts
Bioscientifica Abstracts is the gateway to a series of products that provide a permanent, citable record of abstracts for biomedical and life science conferences.
Find out more