Webinar 6 - Novel Therapeutic Options for CAH Management

Webinar 6 - Novel Therapeutic Options for CAH Management

Date: Wednesday 13 July 2022

Time: 16:00-17:30 (CEST) /15:00-16:30 (BST)

Programme: Novel Therapeutic Options for CAH Management

Welcome and introduction - Dr Senthil Senniappan (Liverpool, UK) - ESPE Connect Webinar Convenor

  • Chronotherapy: Modified Release Hydrocortisone to treat CAH – Professor Richard Ross (Sheffield, UK)
  • CRF1 Receptor antagonists for CAH - Professor Kyriakie Sarafoglou (Minnesota, USA)
  • Continuous subcutaneous hydrocortisone infusion in the treatment of CAH- Professor Katerina Nella (Texas, USA)
  • Panel Discussion
  • Webinar Close - Dr Senthil Senniappan

Our Speakers

Professor Richard Ross

Richard Ross trained in medicine at The Royal London Hospital (1974-1979) and in Endocrinology at St Bartholomew’s Hospital, London (1983-1988). He was appointed to Sheffield University in 1995 and was Head of Section and the Academic Unit of Diabetes, Endocrinology and Metabolism and is currently Theme Lead for Diabetes and Endocrinology. He retired from clinical practice in June 2020.

Richard’s research and clinical interests are in pituitary and adrenal disease, transition endocrinology and the late effects of cancer. Richard has a particular interest in commercial research and founded Sheffield Healthcare Gateway. He is a founding Director of two university spin-out companies; Asterion Ltd developing long-acting biologicals and Diurnal Ltd developing circadian endocrine therapies. Richard has served on the editorial boards of: Clinical Endocrinology (1996-2000), Growth & Growth Factors (1986-2006), Hormones (2004-2018), and J Clin Endocrinol Metab (2010-2014). Council member for the Society of Endocrinology (1999-2002), Editor Endocrinologist (2001-2004), Chair of CaHASE (2002-2015), member of the Bioscientifica Board (2006-2010), Society for Endocrinology Public Engagement Committee (2008-2011) and Nominations Committee (2010-2012). Executive Committee of the European Society of Endocrinology (2011-2015), Treasurer of the European Society of Endocrinology (2013-2015), The Growth Hormone Research Society Council (2011-2018), The Pituitary Society Board of Directors (2019-). Currently Chief Scientific Officer and Director on the Board of Diurnal Group Plc.

Professor Kyriakie Sarafoglou

Kiki Sarafoglou is a tenured Associate Professor in the Department of Pediatrics (Divisions of Pediatric Endocrinology and Genetics & Metabolism) and in the Department of Experimental and Clinical Pharmacology at the University of Minnesota. Dr. Sarafoglou specializes in the clinical research and care of rare endocrine and metabolic disorders with a primary focus on congenital adrenal hyperplasia (CAH). Her research examines cortisol pharmacokinetics and pharmacodynamic response in developing more targeted therapies and improving monitoring.

Currently Dr. Sarafoglou is conducting an FDA-funded R01 examining pulsatile delivery of hydrocortisone to replicate circadian and ultradian rhythms of endogenous cortisol production; the first clinical trial of gene therapy (Adrenas) in patients with CAH; and multiple clinical trials examining CRF receptor antagonists. She sees both pediatric and adult patients in her multidisciplinary CAH clinics. Dr. Sarafoglou is also the editor of the textbook Pediatric Endocrinology and Inborn Errors of Metabolism, 2nd Edition, McGraw Hill, 2018.

Learning Objectives:

  • Understanding current treatment modalities and their relationship to outcomes
  • Describing challenges in monitoring of adrenal androgens and disease control
  • Recognizing how different approaches and new treatments for CAH may impact outcomes

Professor Katerina Nella

Aikaterini (Katerina) Nella, MD, FAAP is Assistant Professor of Pediatrics and Pediatric Endocrinologist at Baylor College of Medicine (BCM) and Texas Children’s Hospital (TCH) in Houston, Texas. She is also Associate Program Director for the BCM Pediatric Endocrinology fellowship program.

Dr. Nella received her medical degree from the University Of Thessaloniki School of Medicine in Greece. She completed a pediatric residency at the University of Nebraska-Creighton University and a pediatric endocrinology fellowship at the National Institutes of Health (NIH). Her clinical and research interests include novel approaches in the treatment of congenital adrenal hyperplasia and mitochondrial dysfunction in endocrine disease. She was the lead associate investigator of the NIH open label phase I-II study examining the use of continuous subcutaneous hydrocortisone infusion in congenital adrenal hyperplasia. At BCM, Dr. Nella is also studying mitochondrial dysfunction and its role in the pathogenesis of pediatric type 2 diabetes.

A summary of Dr. Nella’s talk

Conventional cortisol replacement with oral glucocorticoids is suboptimal and many patients suffer from adverse outcomes due to hyperandrogenism, hypercortisolism or a combination of these two states. Continuous subcutaneous hydrocortisone infusion (CSHI) is a novel, safe and well-tolerated modality of cortisol replacement that effectively approximates physiologic cortisol secretion in patients with classic CAH poorly controlled on conventional therapy. Studies with CSHI in adults with difficult-to-treat classic CAH have shown improved outcomes, including better adrenal steroid control, improved comorbidities and quality of life, making CSHI an attractive therapy for a select set of patients.

Learning objectives from Dr. Nella’s talk

  • To describe the tolerability and safety profile of the continuous subcutaneous hydrocortisone infusion (CSHI) in patients with congenital adrenal hyperplasia (CAH)
  • To understand the role of CSHI in the management of patients with difficult-to-treat classic CAH