ESPE Abstracts (2014) 82 P-D-1-3-161

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)).

Table 1.
All ptsnaHeight SDSnaΔHeight SDS
Baseline176−3.50 (1.34)
Year 1151−3.18 (1.37)1440.34 (0.42)
Year 2104−3.14 (1.58)980.59 (0.64)
Year 366−2.76 (1.78)620.73 (0.74)
Year 425−2.87 (1.75)230.92 (0.80)
Treatment-naive pre-pubertal pts
Baseline102−3.35 (1.34)
Year 171−2.92 (1.33)670.47 (0.38)
Year 241−3.06 (1.59)390.78 (0.64)
Year 322−2.70 (2.00) 210.90 (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

No recent searches.