hrp0089rfc14.6 | Multisystem Endocrine Disorders | ESPE2018

Identification of Epithelial Sodium Channel (ENaC) in Endometrial Pipelle Biopsy Samples

Boggula Vijay , Hanukoglu Israel , Sagiv Ron , Enuka Yehoshua , Hanukoglu Aaron

Background: The fluid milieu along the female reproductive tract has a major role in a complex series of events that follow oocyte ovulation. These include oocyte transport in the fallopian tube, the transport and capacitation of sperm, fertilization, transport of the blastocyst and implantation of the embryo in the uterus. These processes are regulated by the activity of ion channels located on the surface of endometrial epithelia. In our previous studies we showed that epith...

hrp0089con1.1 | Should growth hormone be used in ISS? | ESPE2018

PRO: Should GH Be Used in ISS?

Rosenfeld Ron

There are few issues in pediatric endocrinology which have generated as much controversy as that of GH treatment of ISS. The genesis of this debate is totally understandable, given that ISS is not a ‘disease,’ but, rather, a heterogeneous collection of conditions that are defined auxologically as a height below −2 S.D., without evidence of an underlying systemic, nutritional, endocrine or chromosomal disorder. One can quite rightfully state that ...

hrp0092fc1.6 | Diabetes and Insulin Session 1 | ESPE2019

A Novel Biochemical Marker, Fatty Acid-Binding Protein 4, in Diabetic Ketoacidosis in Children

Gruber Noah , Ron Idit , Sheinvald Sharon , Tirosh Amir , Pinhas-Hamiel Orit

Introduction: Diabetic ketoacidosis (DKA) is the most common cause of hospitalization, cerebral edema, and death among children with type 1 diabetes (T1D). Fatty acid-binding protein 4 (FABP4 or aP2) is one of the most abundant proteins in adipocytes and has been shown to be actively secreted from adipocytes. Circulating FABP4 is regulated by fasting- and lipolysis-related signals and contributes to hyperglycemia by promoting hepatic gluconeogenesis and interf...

hrp0092rfc12.1 | Growth and Syndromes (to include Turner syndrome) | ESPE2019

Karyotyping of oocytes, granulosa cells and stromal cells in the ovarian tissue from patients with Turner syndrome: a pilot study

Peek Ron , Schleedoorn Myra , Beerendonk Catharina , van der Velden Janielle , Braat Didi , Smeets Dominique , Fleischer Kathrin

Summary Answer: Most primordial/primary oocytes were found to have a 46,XX karyotype. Chromosome patterns of the ovarian cells were different from that observed in other tissues.Background: TS is a chromosomal condition associated with partial or complete absence of one of the two X-chromosomes. Females with TS have a limited reproductive lifespan due to an accelerated loss of germ cells. It has been hypothesized that vi...

hrp0092p1-365 | GH and IGFs (2) | ESPE2019

De Novo Formation of Neutralizing IGF-I Antibodies During rhIGF-1 Treatment in a Girl with IGFALS Deficiency as Distinct Adverse Event Interfering with Growth Promotion

Mittnacht Janna , Breil Thomas , Choukair Daniela , Duffert Christin , Hwa Vivian , Rosenfeld Ron , Bettendorf Markus

Background: IGFALS deficiency is a rare cause of GH insensitivity (GHI). We report a German girl with short stature who was born as 2nd child at 40 weeks of gestation. Her Caucasian parents were unrelated and healthy (target height 168 cm, SDS 0.2). She was born appropriately sized for gestational age (49 cm, SDS -1.2; 2950 g, SDS-1.2). Height (104.8 cm; SDS -2.94) and height velocity (5.1 cm / year; SDS -1.67) were reduced at the age of 6 years.</p...

hrp0082fc1.6 | Adrenal | ESPE2014

Molecular Characterization of Testicular Adrenal Rest Tumours in Congenital Adrenal Hyperplasia; Lesions with both Adrenocortical and Leydig Cell Features

Smeets Evelien , Span Paul , van Herwaarden Antonius , Wevers Ron , Sweep Fred , Claahsen-van der Grinten Hedi

Background: Characterization of testicular adrenal rest tumours (TART) are one of the major long-term complications in congenital adrenal hyperplasia (CAH) patients. Although several adrenal-like properties have been assigned to these benign lesions, the exact etiology has not been established yet.Objective and Hypotheses: The aim of this study was to analyze several (steroidogenic) characteristics of TART tissue which may be classified as adrenal cortex...

hrp0092p1-361 | GH and IGFs (2) | ESPE2019

Long-Term Safety of a Once-Weekly Somatrogon (hGH-CTP): 4-Year Results of a Phase 2 Extension Study in Children with Growth Hormone Deficiency

Zielinska Nataliya , Skorodok Yulia , Malievsky Oleg , Iotova Violeta , Rosenfeld Ron G. , Zadik Zvi , Vander Shelly , Pastrak Aleksandra

Background: Once-daily growth hormone (GH) therapy is an effective treatment for children with growth hormone deficiency (GHD), but compliance wanes with ongoing treatment. A once -weekly GH, somatrogon (hGH-CTP), is being developed to reduce the treatment burden of daily dosing for children and caregivers and potentially improve compliance and long-term efficacy. The impact of once-weekly somatrogon on long-term safety, local tolerability and immunogenicity w...

hrp0086rfc8.7 | Growth: Clinical | ESPE2016

Safety and Tolerability of Once-Weekly Administration of CTP-Modified Human Growth Hormone (MOD-4023): 24-month Complete Dataset Results of a Phase 2 Study in Children with Growth Hormone Deficiency

Zelinska Nataliya , Skorodok Julia , Malievsky Oleg , Rosenfeld Ron G. , Zadik Zvi , Koren Ronit , Vander Shelly , Hart Gili , Radziuk Klaudziya

Background: Daily injections are currently required for growth hormone (GH) replacement therapy, which may cause poor compliance, inconvenience and distress for patients. MOD-4023 is a CTP-modified human GH (hGH) developed for once-weekly administration in growth hormone deficient (GHD) adults and children.Objective and hypotheses: In the present Phase 2 study, the safety and tolerability of once-weekly subcutaneous (SC) administration of MOD-4023 were a...

hrp0086rfc8.8 | Growth: Clinical | ESPE2016

Efficacy of Once-Weekly Administration of CTP-Modified Human Growth Hormone (MOD-4023): 24-month Complete Database Results of a Phase 2 Study in Children with Growth Hormone Deficiency

Zelinska Nataliya , Skorodok Julia , Malievsky Oleg , Rosenfeld Ron G. , Zadik Zvi , Koren Ronit , Vander Shelly , Hart Gili , Raduk Dmitri

Background: Growth hormone (GH) replacement therapy currently requires daily injections, which may cause poor compliance, inconvenience and distress for patients. CTP-modified hGH (MOD-4023) has been developed by OPKO Biologics for once-weekly administration in growth hormone-deficient (GHD) adults and children.Objective and hypotheses: The 24-month efficacy of once-weekly subcutaneous (SC) administration of MOD-4023 was evaluated in a Phase 2 study in G...

hrp0086p1-p628 | Growth P1 | ESPE2016

Height Gain and Safety Outcomes in Growth Hormone (GH)-Treated Girls and Boys with Idiopathic Short Stature (ISS): Experience from the Prospective GeNeSIS Observational Study

Child Christopher , Quigley Charmian , Zimmermann Alan , Deal Cheri , Ross Judith , Rosenfeld Ron , Cutler Jr Gordon , Blum Werner

Background: GH treatment for ISS received first approval in the USA in 2003 based on data from two controlled clinical trials. Eligibility is restricted to those with baseline (BL) height standard deviation score (HtSDS) ≤−2.25; other approvals followed, but not in Europe.Objective and hypotheses: To assess outcomes of GH therapy in a large cohort of patients (pts) treated in routine clinical practice.Methods: Short-ter...