ESPE Abstracts (2015) 84 FC7.1

ESPE2015 Free Communications Growth-promoting therapies (6 abstracts)

Safety and Effectiveness of Increlex® Therapy in Children with Laron Syndrome and Enrolled in the European Increlex® Growth Forum Database in Europe

Peter Bang a , Michel Polak b , Joachim Woelfle c , Aude Houchard d & Caroline Sert d

aFaculty of Health Sciences, Linköping University, Linköping, Sweden; bHôpital Universitaire Necker Enfants Malades, Université Paris Descartes, Paris, France; cChildren’s Hospital, University of Bonn, Bonn, Germany; dIPSEN Pharma, Boulogne Billancourt, France

Background: The post-authorisation registry, European Increlex® Growth Forum Database (EU-IGFD), initiated in December 2008, collects data in children receiving Increlex (mecasermin (rDNA origin) injection) for growth failure, including Laron syndrome (LS) (severe primary IGF1 deficiency with confirmed GH-receptor mutation).

Objective and hypotheses: Report safety and effectiveness data in children with LS.

Method: Multicentre, open-label observational study, eCRF data collection.

Results: As of 2nd October 2014, nine countries enrolled 205 patients (115 naïve-prepubertal), including 29 LS patients (13 naïve-prepubertal). In LS patients (vs non-LS), 45% were females (vs 34%), 48% treatment-naïve (vs 69%). Mean (95% CI) treatment duration (days): 1413 (1190; 1636) (vs 916 (837; 995)); median dose (μg/kg BID): 40 at treatment initiation (both subgroups), 105 (vs 120) at year 1, 120 at year 2. Naïve-prepubertal LS patients had significantly higher Δ height SDS at year 1, vs naïve-prepubertal non-LS (linear regression, P=0.006).

Targeted adverse events (TAEs) were reported in 69% LS patients (vs 36% non-LS); most frequently hypoglycaemia (41% vs 13%), lipohypertrophy (21% vs 9%), tonsillar hypertrophy (17% vs 5%), and otitis media (14% vs 3%). LS was a predictive factor for hypoglycaemia (OR (CI 95%): 0.21 (0.09;0.50); P<0.001).

naHeight SDSnaΔ Height SDS
LS patients
Baseline26−5.22 (1.63)
Year 123−4.46 (1.88)220.50 (0.61)
Year 222−4.51 (1.72)200.77 (0.79)
Baseline12−5.80 (1.60)
Year 110−4.76 (2.09)90.77 (0.54)
Non-LS patients
Baseline163−3.50 (1.04)
Year 1139−3.19 (1.10)1340.32 (0.44)
Year 2102−2.99 (1.24)990.57 (0.62)
Baseline97−3.42 (0.95)
Year 184−3.03 (0.95)810.36 (0.40)
aAvailable at timepoint.

Conclusion: Height SDS increase was higher in LS patients vs non-LS and this was significant at year 1 in the naïve-prepubertal subgroup; however, they remained shorter. TAEs were more frequent in LS patients; LS was identified as predictive factor for hypoglycaemia.

Declaration of interest: A Houchard and C Sert: employees of Ipsen. M Polak and J Woelfle: advisory boards for Ipsen and Novo Nordisk.

Funding: This work and the study on which it is based were supported by Ipsen.

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