ESPE Abstracts (2015) 84 FC7.1

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

Peter Banga, Michel Polakb, Joachim Woelflec, Aude Houchardd & Caroline Sertd

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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