hrp0089rfc15.6 | Growth and syndromes | ESPE2018

Latest Results from PATRO Children, a Multi-Centre, Non-Interventional Study of the Long-Term Safety and Efficacy of Omnitrope® in Children Requiring Growth Hormone Treatment

Kanumakala Shankar , Pfaffle Roland , Hoybye Charlotte , Kristrom Berit , Battelino Tadej , Zabransky Markus , Zouater Hichem

Objectives: PATRO Children is a non-interventional, international, longitudinal study of the long-term safety of a recombinant human growth hormone (rhGH; Omnitrope®, Sandoz). In particular, the study will assess the diabetogenic potential of rhGH and the risk of malignancies. The long-term efficacy is a secondary objective of the study.Methods: The study population includes infants, children and adolescents receiving Omnitrope®...

hrp0089p1-p153 | GH & IGFs P1 | ESPE2018

Testing the Performance of a Preexisting Growth Prediction Model in a Cohort of Prepubertal Patients Born Small for Gestational Age (SGA) Receiving GH Treatment in PATRO Children

Land Christof , Pfaffle Roland , Schwab Karl Otfried , Sommer Heide , Partsch Carl Joachim

Aim and background: Growth hormone (GH) treatment of short children born SGA and its effects on growth varies greatly between treated individuals. In the present study we tested the performance of a preexisting growth prediction model (Ranke et al.; JCE&M 2003 pp. 125–131) to estimate 1st-year height velocity (HV) in a german subcohort of prepubertal children born SGA treated with GH (Omnitrope).Methods: 190 treatment-naïve prepu...

hrp0089p2-p398 | Thyroid P2 | ESPE2018

Diagnosis and Clinical Course of Amiodarone Induced Hyperthyroidism in Three Adolescent Patients

Gesing Julia , Hoppmann Julia , Gebauer Roman , Pfaffle Roland , Bertsche Astrid , Kiess Wieland

Amiodarone induced hyperthyroidism is a known side effect of amiodarone treatment. In the pediatric population long-term amiodarone treatment is rarely indicated because of its severe side effects including thyroid function impairment and therefore treatment is restricted to therapy resistant arrhythmias. In the literature scarce data is available on management and therapy of amiodarone induced thyroid dysfunction at a young age. We present three adolescent patients developin...

hrp0086p1-p634 | Growth P1 | ESPE2016

Four-Year Results from PATRO Children, a Multi-Centre, Non-Interventional Study of the Long-Term Safety and Efficacy of Omnitrope® in Children Requiring Growth Hormone Treatment

Pfaffle Roland , Kanumakala Shankar , Charlotte Hoybye , Berit Kristrom , Markus Zabransky , Tadej Battelino , Michel Colle

Background: PATRO Children is an international, open, longitudinal, non-interventional study of the long-term safety and efficacy of Omnitrope®, a biosimilar recombinant human GH (rhGH).Objective and hypotheses: The primary objective of PATRO Children is to assess long-term safety of Omnitrope® (particularly the diabetogenic potential of GH in short children born small for gestational age, the risk of malignancies, and oth...

hrp0086p2-p857 | Syndromes: Mechanisms and Management P2 | ESPE2016

A Case of Familial Silver-Russell Syndrome

Hoppmann Julia , Netchine Irene , Eggermann Thomas , Jamra Rami Abou , Kiess Wieland , Pfaffle Roland

Background: Silver-Russell syndrome (SRS) is a heterogeneous condition characterized by intrauterine growth restriction, relative macrocephaly at birth, postnatal growth retardation, body asymmetry, feeding difficulties/ low body mass index and dysmorphic craniofacial features. SRS is caused by DNA hypomethylation at the H19/IGF2-imprinting control region (ICR1) on chromosome 11p15 or maternal uniparental disomy of chromosome 7 (mUPD7) in approximately 50% and 10%, respectivel...

hrp0082p2-d3-442 | Growth Hormone (2) | ESPE2014

Two-Year Results from Patro Children, a Multi-Centre, Non-Interventional Study of the Long-Term Efficacy and Safety of Omnitrope® in Children Requiring GH Treatment

Pfaffle Roland , Kanumakala Shankar , Hoybye Charlotte , Kristrom Berit , Schuck Ellen , Zabransky Markus , Battelino Tadej , Colle Michel

Background: PATRO children is an international, open, longitudinal, noninterventional study of the long-term safety and efficacy of Omnitrope®, a biosimilar recombinant human GH (rhGH).Objective and hypotheses: The primary objective is to assess the long-term safety of Omnitrope®, particularly the diabetogenic potential of GH in short children born small for gestational age, the risk of malignancies, and potential risks of...

hrp0082p3-d1-813 | Growth | ESPE2014

BMI, IGF1–SDS, and rhGH Treatment

Vasiliakis Ioannis Anargyros , Gausche Ruth , Beger Christoph , Kratzsch Jurgen , Kiesz Wieland , Pfaffle Roland

Background: CrescNet is a large data base used to document children’s growth and weight development in more than 300 primary care practices and in eight specialized paediatric endocrinological centres in Germany.Aims and method: We investigated 3281 patients with IGF1 measurements during their consultations and subsequent checkups. We analysed 2269 children without an indication for rhGH treatment and 1012 who were subsequently treated with rhGH. A ...

hrp0084p2-413 | GH & IGF | ESPE2015

Results up to January 2015 from PATRO Children, a Multi-Centre, Non-Interventional Study of the Long-Term Safety and Efficacy of Omnitrope® in Children Requiring GH Treatment

Pfaffle Roland , Kanumakala Shankar , Hoybye Charlotte , Kristrom Berit , Schuck Ellen , Zabransky Markus , Battelino Tadej , Colle Michel

Background: PATRO Children is an international, open, longitudinal, non-interventional study designed to evaluate the long-term safety and efficacy of Omnitrope®, a biosimilar recombinant human GH (rhGH).Objective and hypotheses: Long-term safety of Omnitrope® is the primary objective of PATRO Children (particularly the diabetogenic potential of GH in short children born small for gestational age, the risk of malignancies,...

hrp0089p2-p213 | GH & IGFs P2 | ESPE2018

Different Genetic Causes of Short Stature in a Family

Tulun Alev , Pfaffle Roland , Rockstroh Denise , Jamra Rami Abou , Schmidt Julia , Gillessen-Kaesbach Gabriele , Hoppmann Julia , Hiort Olaf

Background: The most common endocrine cause of growth disorders in childhood is growth hormone deficiency (GHD). The rare monogenic forms of GHD are inherited as autosomal dominant or recessive traits and manifest as isolated deficiency or in combination with other hormone deficiencies. Here, we report on a three-year-old girl with a severe growth retardation (height 77 cm, – 5.6 S.D.S.). She is the only child of non-consanguineous parents from northern Ira...

hrp0082p1-d1-140 | Growth | ESPE2014

Severe Short Stature and GH Insensitivity Due to a De Novo Heterozygous STAT5B Missense Mutation

Klammt Jurgen , Neumann David , Andrew Shayne F , Drahosova Marcela , Stobbe Heike , Buckham Kyle , Rosenfeld Ron G , Pfaffle Roland , Hwa Vivian

Background: GH insensitivity is caused by disturbances of GH receptor function or inability to transduce the hormone signal. Affected children are severely growth retarded and may also present immune complications when the transducer STAT5B is defective. Only autosomal-recessive STAT5B mutations have been described to date.Clinical case: Two male 14.5 year monozygotic twins presented with heights of 131.5 cm (−5.3 SDS). Bone age of the ind...