hrp0084p2-419 | GH & IGF | ESPE2015

Safety Evaluation of Long-Term Recombinant GH Treatment in Childhood: Interim Analysis of the NordiNet® International Outcome Study (IOS)

Savendahl Lars , Pournara Effie , Rohrer Tilman R , Pedersen Birgitte Tonnes , Saha Marja-Terttu , Blankenstein Oliver

Background: Long-term safety data are reported for paediatric patients treated with recombinant GH (GH; Norditropin®, Novo Nordisk A/S) at the treating physician’s discretion and enrolled in the observational NordiNet® International Outcome Study (IOS) (NCT00960128).Objective and hypotheses: To evaluate incidence rates (IR) (events/1 000 patient-years) of adverse drug reactions (ADR), serious adverse events (SAE), and...

hrp0094fc9.3 | Growth Hormone and IGFs | ESPE2021

Once-weekly somapacitan versus daily growth hormone in growth hormone deficiency: 3 year efficacy and safety results from REAL 3, a randomised controlled phase 2 trial

Savendahl Lars , Battelino Tadej , Rasmussen Michael Hojby , Saenger Paul , Horikawa Reiko ,

Growth hormone (GH) replacement therapy currently requires daily subcutaneous injections. Somapacitan is a long-acting GH-derivative in development for once-weekly use in children with GH deficiency (GHD). A phase 2, multinational, randomised, open-label, controlled trial (NCT02616562) investigated the efficacy and safety of somapacitan compared with daily GH (Norditropin®). GH-treatment-naïve prepubertal children with GHD received 0.04 (n = 16), 0.08 (n...

hrp0084fc7.4 | Growth-promoting therapies | ESPE2015

A Novel Reversible Albumin-Binding GH Derivative Possesses a Promising Once-Weekly Treatment Profile in Children with GH Deficiency

Zuckerman-Levin Nehama , Gucev Zoran , de Schepper Jean , Rasmussen Michael Hojby , Battelino Tadej , Olsen Minna Braendholt , Savendahl Lars

Background: GH administration restores normal growth in children with GH deficiency (GHD). However, current daily s.c. injection treatment regimens may be inconvenient leading to impaired adherence and subsequently suboptimal treatment outcomes. NNC0195-0092 is a novel, reversible albumin-binding GH developed for once-weekly administration.Objective and hypotheses: This was a randomised, open-label, active-controlled, dose-escalation trial (NCT01973244) ...

hrp0095p1-498 | GH and IGFs | ESPE2022

A Long-Acting Growth Hormone Module For Paediatric Growth Hormone Deficiency In The Global Registry For Novel Therapies In Rare Bone & Endocrine Conditions – The GloBE-Reg LAGH Study

Miller Bradley , Savendahl Lars , Hickman Krystina , Smythe Christopher , Chen Ching , Choi Jin-Ho , Dou Xinyu , Gong Chunxiu , Hamza Rasha , Horikawa Reiko , Jorge Alexander , Faisal Ahmed S.

Introduction: Post-marketing surveillance registries provided extensive information about the safety and efficacy of daily growth hormone (GH) therapy during treatment. With the availability of novel long-acting GH (LAGH) therapies, it is important to determine whether the novel molecules or the different pattern of GH exposure lead to changes in the efficacy and safety profile. Therefore, new surveillance registries of LAGH are warranted.<p class="abstext...

hrp0086fc8.6 | Growth: Clinical | ESPE2016

Birth Characteristics Explain One Third of Expected Deaths in rhGH-treated Patients Diagnosed with IGHD, ISS & SGA

Albertsson-Wikland Kerstin , Martensson Anton , Savendahl Lars , Niklasson Aimon , Bang Peter , Dahlgren Jovanna , Gustafsson Jan , Kristrom Berit , Norgren Svante , Pehrsson Nils-Gunnar , Oden Anders

Background: That mortality is not increased in rhGH-treated patients when adjusting for birth characteristics was recently published (1). When applying a developed mortality model of the general population, the observed and expected deaths in rhGH-treated IGHD, ISS and SGA patients (n=3847) where 21 and 21.99, respectively. The model includes gender, age, calendar year, gestational age (GA), birth lengthSDS (BLSDS), birth weightSDS (BW...

hrp0086rfc14.2 | Growth : Mechanisms | ESPE2016

Contribution of GHR and IGFALS Mutations to Growth Hormone Resistance – Identification of New Variants and Impact on the Inheritance Pattern

Legendre Marie , Dastot Florence , Collot Nathalie , Duquesnoy Philippe , Cohen Enzo , Sobrier Marie-Laure , Adiceam Paola , Anderson Donald , Baron Sabine , Cabrol Sylvie , Callewaert Bert , Cartigny Maryse , Craen Margarita , Crock Patricia , Ladjouze Asmahane , Lazea Cecilia , Polak Michel , Savendahl Lars , Touzani Asmae , Amselem Serge

Background: Bi-allelic GHR mutations are classically responsible for Laron syndrome, a severe growth hormone (GH) resistance syndrome. A few GHR missense mutations have also been implicated in mild GH resistance or idiopathic short stature. IGFALS mutations are responsible for recessive or semi-dominant short stature with normal GH provocative test contrasting with extremely low IGF-I levels.Objective and hypotheses: To assess the contribution of GHR and...

hrp0095p1-108 | Growth and Syndromes | ESPE2022

Efficacy, Observer-Reported Outcomes, and Safety of Once-Weekly Somapacitan in Children with Growth Hormone Deficiency (GHD): 4-Year Results from the REAL 3 Trial

Sävendahl Lars , Battelino Tadej , Højby Rasmussen Michael , Brod Meryl , Wai Lee Kai , Saenger Paul , Horikawa Reiko

Children with GHD are currently treated with daily subcutaneous growth hormone (GH) injections, which can be burdensome. Somapacitan is a long-acting GH derivative in development for once-weekly use in children with GHD. REAL 3 (NCT02616562) is a phase 2, multinational, randomised, open label, controlled trial assessing efficacy and safety of somapacitan vs daily GH (Norditropin®). Prepubertal, GH-naïve children with GHD received 0.04 (n=16), 0.08 (n</e...

hrp0089fc10.2 | Late Breaking | ESPE2018

Efficacy and Safety of Once-Weekly Somapacitan in Childhood Growth Hormone Deficiency: Results of a Randomised Open-Label, Controlled Phase 2 Trial

Savendal Lars , Rasmussen Michael , Horikawa Reiko , Khadilkar Vaman , Battelino Tadej , Saenger Paul

Background: Growth hormone deficiency (GHD) requires long-term daily injections with GH replacement therapy and is associated with considerable treatment burden by patients and caregivers. Somapacitan is a long-acting GH derivative that is being developed for once-weekly dosing in adults and children with GHD. A well-established protraction method, which is successfully in use to extend the half-life of insulin and glucagon-like peptide (GLP)-1, has been applied in developing ...

hrp0086rfc6.4 | Syndromes: Mechanisms and Management | ESPE2016

Growth Hormone (GH) Treatment in Skeletal Dysplasias – Short-term Results in Prepubertal Children Reported in KIGS

Hagenas Lars , Lindberg Anders , Camacho-Hubner Cecilia , Rooman Raoul

Background: A total of 83,803 patients who received rhGH therapy were enrolled in KIGS (Pfizer International Growth Database) including 748 patients diagnosed with a specified or unspecified skeletal dysplasia. The most prevalent diagnoses were hypochondroplasia (n=238: Female=111, Male=127), achondroplasia (n=113: F=51, M=62) and Leri-Weill dyschondrosteosis, LWD (n=88: F=59, M=29).Objective: To analyse the first year response...

hrp0084p2-474 | Growth | ESPE2015

Growth Curves for Height, Weight, BMI and Head Circumference in Children with Achondroplasia

Merker Andrea , Hagenas Lars , Hertel Thomas , Neumeyer Lo

Background: Close monitoring of growth is vital when following children with achondroplasia yet existing growth curves suffer from a simple chart format and their clinical use is therefore limited. Also, references for body proportions; i.e. sitting height, relative sitting height and arm span, are lacking.Objective and hypotheses: The aim of this study was to construct age-specific growth curves for height, weight, body mass index, head circumference an...