hrp0082p2-d3-439 | Growth Hormone (2) | ESPE2014

GH Dosing Patterns in Short Children Born Small for Gestational Age

Petit Isabelle Oliver , Blankenstein Oliver , Zdravkovic Dragan , Tonnes Pedersen Birgitte , Pournara Effie

Introduction: In Europe, GH is approved for short (height SDS <−2.5) children born small for gestational age (SGA) who fail to show catch-up growth by 4 years of age.Methods: This report analysed the patterns of GH dose used in everyday clinical practice based on short children born SGA enrolled in NordiNet® IOS, an observational study evaluating the long-term effectiveness and safety of Norditropin®. Average GH ...

hrp0094p2-31 | Adrenals and HPA Axis | ESPE2021

Investigation of usage and acceptance of hydrocortisone granules in capsules for opening in children with adrenal insufficiency

Hilger Julia , Blankenstein Oliver , Neumann Uta ,

Context: Off-label and unlicensed use is widespread in medical therapy of children. Since 2007 there is an EU regulation, which includes the need for a pediatric investigation plan (PIP) in the drug approval process. In 2018, hydrocortisone granules in capsules for opening (Alkindi®) became the first medicine to be approved for the treatment of adrenal insufficiency in children from birth.Methods: An anonymised onli...

hrp0082p2-d1-589 | Thyroid | ESPE2014

No Difference in Cognitive Development of Young Adults and Adolescents Affected by Congenital Hypothyroidism Compared to Their Sibling Controls Despite High Dose L-Thyroxin Treatment

Aleksander Paulina , Blankenstein Oliver , Gruters Annette , Krude Heiko

Background: An early diagnosis and treatment based on neonatal screening offers a normal cognitive development in patients affected with congenital hypothyroidism (CH). However, several studies within cohorts of young adults have shown a still existing difference compared to control groups of up to eight IQ points. Moreover it has been claimed recently that a high L-T4 dose with subsequent episodes of overtreatment results in less favourable IQ outcom...

hrp0084p3-940 | GH &amp; IGF | ESPE2015

GH Dosing Patterns in Children with Isolated GH Deficiency and Multiple Pituitary Hormone Deficiency Enrolled in the NordiNet® International Outcome Study

Snajderova Marta , Pournara Effie , Pedersen Birgitte Tonnes , Blankenstein Oliver

Background: Long-term monitoring of GH treatment in children is very important.Objective and hypotheses: To describe patterns of GH dosing in clinical practice in children with isolated GH deficiency (IGHD) and multiple pituitary hormone deficiency (MPHD).Method: We analysed 7 years of GH treatment data from NordiNet® International Outcome Study (IOS) (NCT00960128), an observational study evaluating the long-term effectiveness...

hrp0084p3-1069 | Hypo | ESPE2015

Experience Based on 193 18F-DOPA PET CTs in Patients with Congenital Hyperinsulinism: Pearls and Pitfalls in Imaging Diagnostics in Patients with CHI

Kuhnen Peter , Prasad Vikas , Brenner Winfried , Mohnike Wolfgang , Blankenstein Oliver

Background: In congenital hyperinsulinism (CHI) 18F-DOPA PET CT plays an essential role in differentiating between focal and diffuse CHI forms and in the analysis of the localization of a potential focus before surgery.Objective and hypotheses: The aim of this retrospective analysis was the evaluation of the 18F-DOPA PET CT efficacy in a large cohort of CHI patients.Method: In the last few years 193 18</s...

hrp0086p1-p15 | Adrenal P1 | ESPE2016

Individualized Optimization with 17OHP-Saliva Profiles Leads to Changes in Hydrocortisone Dosing Pattern in Children with Congenital Adrenal Hyperplasia

Neumann Uta , Lankes Erwin , Bathelt-Tok Franziska , Krude Heiko , Blankenstein Oliver

Background: Treatment of CAH in children is compromised by the pharmacokinetic of available hydrocortisone (HC) preparations resulting in un-physiological early morning rise of ACTH followed by elevated androgens. HC substitution usually follows a fixed dosing scheme (50%–25%–25%) monitored by blood sampling.Objective and hypotheses: We describe the individualized optimization of HC treatment by 17-OHP saliva profiles and the effects of the res...

hrp0086p2-p643 | Growth P2 | ESPE2016

An Analysis of the Safety of Childhood Growth Hormone (GH) Therapy: Data from the NordiNet® International Outcome Study (IOS)

Savendahl Lars , Rohrer Tilman R , Pournara Effie , Pedersen Birgitte Tonnes , Blankenstein Oliver

Background: NordiNet® IOS (NCT00960128), a non-interventional study, collects long-term effectiveness and safety data of GH (Norditropin®, Novo Nordisk) treatment in everyday clinical practice.Objective and hypotheses: Identify paediatric patients more likely to experience a second adverse event (AE).Method: Based on diagnosis at GH treatment start and associated risk for mortality, patients were cla...

hrp0084p2-419 | GH &amp; 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...

hrp0094p2-411 | Sex differentiation, gonads and gynaecology or sex endocrinology | ESPE2021

Rationale for a reduced dexamethasone dose in prenatal congenital adrenal hyperplasia therapy based on pharmacokinetic modelling

Neumann Uta , Stachanow Viktoria , Blankenstein Oliver , Fuhr Uwe , Huisinga Wilhelm , Reisch Nicole , Kloft Charlotte

Context: Prenatal dexamethasone (Dex) therapy is used in female foetuses with congenital adrenal hyperplasia (CAH) to suppress adrenal androgen excess and prevent virilisation of the external genitalia. The prenatal dexamethasone dose of 20 µg/kg/d has been used for decades in prenatal CAH. Despite the risks for the treated mother and potentially for the unborn child, no clinical study or evaluation had been conducted in order to determine a Dex dose with a scientific rat...

hrp0092p1-156 | Adrenals and HPA Axis (1) | ESPE2019

Prospective, Open-Label, Long-Term Follow-Up of Neonates and Young Children with Adrenal Insufficiency Treated with Hydrocortisone Granules

Neumann Uta , Braune Katarina , Whitaker Martin , Wiegand Susanna , Krude Heiko , Porter John , Digweed Dena , Voet Bernard , Ross Richard , Blankenstein Oliver

Introduction: Children with congenital adrenal hyperplasia (CAH) and adrenal insufficiency (AI) rely on lifelong hormone replacement with hydrocortisone (HC). Alkindi® is the first HC licensed for children from birth to 18 years with AI, available in small doses of 0.5, 1, 2 and 5mg required for the needs of neonates, infants and children.Objectives: Primary: long-term safety of Alkindi®; Secondary: long-term d...