Philippe F Backeljauw MD

PubMed

• International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with achondroplasia.
  Ravi Savarirayan, Julie Hoover-Fong, Keiichi Ozono, Philippe Backeljauw, Valérie Cormier-Daire
  Nature Reviews. Endocrinology. 2025-05-01
• 1 citations
  Somapacitan in Children Born SGA: 52-Week Efficacy, Safety, and IGF-I Response Results From the Phase 2 REAL5 Study.
  Anders Juul, Philippe Backeljauw, Michael Højby, Jan Frystyk, Masanobu Kawai
  The Journal of Clinical Endocrinology and Metabolism. 2025-03-17
• Growth Response to Weekly Somapacitan Therapy in Children With GH Deficiency Is Related to GH Thresholds in GH Stimulation Testing.
  Philippe F Backeljauw, Claudia Boettcher, Michael Højby, Nicky Kelepouris, Mathias Lerbech Jeppesen
  Journal of the Endocrine Society. 2025-03-03

Journal Articles

• Response to Letter to the Editor: Low incidence of pathology detection and high cost of screening in the evaluation of asymptomatic children with short stature  
  Sisley S, Backeljauw PF, J Pediatr, 11/1/2013
• The recommended dose for IGF-I therapy does not compromise long-term growth (Letter to the editor)  
  Backeljauw PF, Kuntze J, Frane J, Calicoglu A, Chernausek SD, J Clin Endocrinol Metab, 7/18/2013
• Low incidence of pathology detection and high cost of screening in the evaluation of asymptomatic children with short stature  
  Sisley S, Vargas-Trujillo M, Khoury J, Backeljauw PF, J Pediatr, 1/1/2013
• Adult height in patients with severe primary IGF-I deficiency  
  Backeljauw PF, Chernausek SD, Calicoglu A, Kuntze J, Frane J, Horm Res Paediatr, 1/1/2013
• The impact of aortic arch geometry on flow characteristics  
  Prahl Wittberg L, van Wyk S, Mihaescu M, Fuchs, L, Gutmark E, Backeljauw P, Gutmark-Little I, American Institute of Aeronautics and Astronautics, 51st AIAA Aerospace Sciences Meeting Proceedings, 1/1/2013
• Cardiac magnetic resonance imaging in Turner syndrome  
  Gutmark-Little I, Backeljauw P, Clinical Endocrinology, 1/1/2013
• Partial anomalous pulmonary venous return is common in Turner syndrome  
  Gutmark-Little I, Hor K, Gottliebson W, Cnota J, Gottliebson WM, Backeljauw PF, J Pediatr Endocr and Met, 1/1/2012
• Identification and management of poor response to growth-promoting therapy in children with short stature  
  Bang P, Ahmed F, Argente J, Backeljauw P, Bettendorf M, Bona G, Coutant R, Rosenfeld RG, Walenkamp MJ, Savage MO, Clinical Endocrinology, 1/1/2012
• The insulin-like growth factors and growth disorders of childhood  
  Backeljauw PF, Chernausek SD, Endocrinol Metab Clin N Am, 1/1/2012
• Cardiovascular Anomalies in Turner Syndrome: Spectrum, Prevalence, and Cardiac MRI Findings in a Pediatric and Young Adult Population  
  Hee Kyung Kim, William Gottliebson, Kan Hor, Philippe Backeljauw, Iris Gutmark-Little, Shelia R. Salisbury, Judy M. Racadio, Kathy Helton-Skally, and Robert Fleck, Am. J. Roentgenol, 1/1/2011
• Superior mesenteric artery syndrome as a presentation of Graves' disease in adolescence  
  Howell JC, Cafasso M, Backeljauw PF, Endotrends, 1/1/2011
• Non-classic congenital adrenal hyperplasia Clinical experience versus clinical guidelines  
  Shah AS, Backeljauw PF, Endocrinology Studies, 1/1/2011
• Recombinant insulin-like growth factor (IGF)-I treatment in short children with low IGF-I levels: first-year results from a randomized clinical trial  
  Midyett LK, Rogol AD, Van Meter QL, Frane J, Bright GM; MS301 Study Group. Abdullatif H, Allen H, Backeljauw P, Banks R, Bloch C, et al, J Clin Endocrinol Metab, 2/1/2010
• Endocrine sequelae of traumatic brain injury in children  
  Kaulfers AM, Backeljauw PF, Reifschneider K, Blum S, Michaud L, Weiss M, Rose SR, J Pediatr, 1/1/2010
• Insulin-like growth factor-I (IGF-I) in growth and metabolism  
  Backeljauw P, Bang P, Dunger DB, Juul A, Le Bouc Y, Rosenfeld R, J Ped Endocrinol Metab, 1/1/2010
• Dental development in children with growth hormone insensitivity syndrome: Demirjian analysis of serial panoramic radiographs  
  Campbell R, Weinshel R, Backeljauw P, Wilson S, Bean J, Shao M, Cleft Palate-Craniofacial Journal, 1/1/2009
• Allergic reaction to mecasermin, recombinant human insulin-like growth factor-I  
  Allred Metz K, Assa'ad A, Lierl MB, Backeljauw P, Ann Allergy, Asthma & Clin Immunol, 1/1/2009
• Prevalence of adrenal insufficiency following systemic glucocorticoid therapy in infants with hemangiomas  
  Lomenick JP, Reifschneider KL, Lucky AW, Woo JG, Backeljauw PF, Archives in Dermatology, 1/1/2009
• Treatment of severe IGF-I deficiency with recombinant human IGF-I (mecasermin)  
  Backeljauw PF, Chernausek SD, Growth-Growth hormone, IGF-1, and Metabolism, 1/1/2009
• Puberty in Turner syndrome  
  Backeljauw P, Highlights, 1/1/2009
• Does growth hormone therapy before 4 years of age enhance the linear growth in girls with Turner's syndrome?  
  Philippe F. Backeljauw, Nat Clin Pract Endocrinol Metab, 1/1/2008
• Algorithms for the evaluation and management of short stature Part III of: treatment perspectives in idiopathic short stature with a focus on IGF-1 deficiency  
  Rosenthal S, Cohen P, Clayton P, Backeljauw P, Bang P, Ten S, Rosenfeld R, Pediatr Endocrinol Rev, 1/1/2007
• Defining and managing growth hormone treatment failure Part II of: treatment perspectives in idiopathic short stature with a focus on IGF-1 deficiency  
  Rosenthal S, Cohen P, Clayton P, Backeljauw P, Bang P, Ten S, Rosenfeld R, Pediatr Endocrinol Rev, 1/1/2007
• Clinical Diagnosis of IGF-I deficiency Part I of: treatment perspectives in idiopathic short stature with a focus on IGF-1 deficiency  
  Rosenthal S, Cohen P,Clayton P, Backeljauw P, Bang P, Ten S, Rosenfeld R, Pediatr Endocrinol Rev, 1/1/2007
• Treatment of insulin-like growth factor deficiency: studies in humans  
  Philippe F. Backeljauw, Steven D. Chernausek, Horm Res, 1/1/2006
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Books/Book Chapters

• Pediatric Endocrinology 
  Chapter: Disorders of growth hormone/insulin-like growth factor secretion and action 
  Page 292 - 404Backkeljauw PF, Dattani MT, Cohen P, Rosenfeld RGEditors: Sperling MA.Elsevier-Saunders, PhiliadelphiaEdition 4,2014
• Pediatric Endocrinology: A Practical Clinical Guide 
  Chapter: Turner syndrome. 
  Page 109 - 136Davenport M, Ross J, Backeljauw PEditors: Sally Radovick, Margaret H. MacGillivray.Humana Press, New YorkEdition 2nd,2013
• The 5 Minute OB/GYN Consult 
  Chapter: Turner syndrome. 
  Page 186 - 187Philippe F. BackeljauwEditors: Adams Hillard, PJ.Lippincott Williams & Wilkins, PhiladelphiaEdition 1st,2008
• Educational booklet of The Human Growth Foundation 
  Chapter: Insulin-like growth factor I deficiency. 
  Philippe BackeljauwThe Human Growth Foundation, Inc.2008
• A Current Review of Pediatric Endocrinology 
  Chapter: Hypercalcemia. 
  Page 155 - 162Philippe BackeljauwBiosymposia, Inc.2007
• Central and Peripheral Mechanisms in Pituitary Disease 
  Chapter: Growth hormone resistance as a model for understanding IGF-I actions. 
  Page 43 - 52L Underwood, P Backeljauw and the GHIS Collaborative GroupEditors: Kleinberg DL and Clemmons DR.BioScientifica Ltd, Bristol2002
• The 5 Minute Pediatric Consult 
  Chapter: Short stature. 
  Page 48 - 49Philippe F. BackeljauwEditors: Schwartz MW.Williams & Wilkins, BaltimoreEdition 1st,1996
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Abstracts/Posters

• SNP Microarray analysis reveals whole gene deletion of the mineralocorticoid receptor in a patient with features of pseudohypoaldosteronism type 1.
  Dyer L, Wasserman H, Backeljauw P, MD, Smolarek T, Journal of Molecular Diagnostics; Annual Meeting of the Association for Molecular Pathology, Phoenix, AZ, 11/1/2013
• Recombinant human insulin-like growth factor-I (IGF-I) in Duchenne Muscular Dystrophy (DMD).
  Rutter MM, Collins J, Wong B, Horn P, Taylor MD, Hu S, Blum S, Morehart P, Sawnani H, Backeljauw PF, 18th International WMS Congress, Asilomar, CA, 10/1/2013
• Height predictions using ulna length are inaccurate in glucocorticoid-treated boys with Duchenne Muscular Dystrophy (DMD).
  Meilan M Rutter, James Collins, Hemant Sawnani, Paul Horn, Philippe F Backeljauw, Samantha Blum, Shengyong Hu, Paula Morehart, Michael D Taylor, Brenda Wong, 18th International WMS Congress, Asilomar, CA, 10/1/2013
• Aortic blood flow characteristics in Turner syndrome.
  Gutmark-Little I, Prahl Wittberg L, van Wyk S, Backeljauw PF, 95th Annual Meeting of the Endocrine Society, San Francisco, CA, 6/1/2013
• Safety results from a phase II, randomized, open-label, active-controlled trial of rhGH/rhIGF-1 combination therapy in children with short Stature, low IGF-1 and GH su...
  Miller B, Backeljauw P, Dutailly P, Sicsic A, Lawson E, Hale D, Reiner B, Sperling M, 95th Annual Meeting of the Endocrine Society, San Francisco, CA, 6/1/2013
• Liver function test abnormalities begin early in life in Turner syndrome.
  Jimenez-Vega JM, Backeljauw P, Khoury J, Lawson S, Gutmark-Little I, Horm Res; 9th Joint Meeting of Pediatric Endocrinology, Milan, Italy, September 2013, 1/1/2013
• Recombinant human insulin-like growth factor-I (IGF-I) in Duchenne Muscular Dystrophy (DMD).
  Rutter MM, Collins J, Wong B, Horn P, Taylor MD, Hu S, Blum S, Morehart P, Sawnani H, Backeljauw PF, Horm Res; 9th Joint Meeting of Pediatric Endocrinology, Milan, Italy, September 2013, 1/1/2013
• Efficacy of rhGH/rhIGF-1 co-administration therapy in children with short stature, low IGF-1 and GH sufficiency-results from a phase II, randomized, open-label, active...
  Backeljauw P, Miller B, Dutailly P, Sicsic A, Lawson E, Hale D, Reiner B, Sperling M, Horm Res; 9th Joint Meeting of Pediatric Endocrinology, Milan, Italy, September 2013, 1/1/2013
• Characteristics of the flow in the aortic arch.
  Prahl-Wittberg L, van Wyk S, Mihaescu M, Fuchs L, Gutmark EJ, Backeljauw P, Gutmark-Little I, 51st AIAA Aerospace Sciences Meeting, Grapevine, TX, 1/1/2013
• Oophorectomy versus salpingo-oophorectomy in Turner syndrome patients with Y-chromosome material: current practice patterns assessment.
  Shankar RK, Inge T, Backeljauw PF, The 94th Annual Meeting of the Endocrine Society, Houston, TX, 6/1/2012
• Efficacy and cost-effectiveness of laboratory screening in short stature.
  Sisley S, Vargas M, Backeljauw PF, The 94th Annual Meeting of the Endocrine Society, Houston, TX, 6/1/2012
• Vasculopathy in the young Turner syndrome population.
  Lawson S, Urbina E, Khoury P, Gutmark-Little I, Backeljauw P, Meeting on Strategies To Improve Care Outcome In Turner syndrome, Winthrop University Hospital, 9/1/2011
• Computational airway modeling: a novel method to analyze airway anomalies in Turner syndrome (TS).
  Gutmark-Little I, Mylavarapu G, Mihaescu M, Gutmark E, Backeljauw P, Annual PAS/PES/SPR Joint Meeting, Denver, CO, 5/1/2011
• Vasculopathy in the young Turner syndrome population.
  Lawson S, Urbina E, Khoury P, Sucharew H, Gao Z, Backeljauw B, Gutmark-Little I, Backeljauw P, Annual PAS/PES/SPR Joint Meeting, Denver, CO, 5/1/2011
• Long-term treatment with recombinant human IGF-I: Results in patients with primary IGF-I deficiency.
  Backeljauw P, International Symposium on GH, IGF-I, Ghrelin/GHS: Climbing The Scientific Growth Ladder, Orlando, FL, 3/1/2011
• Danazol therapy-induced central precocious puberty in a patient with hereditary angioedema.
  Auble B, Backeljauw P, 93rd Annual Meeting of the Endocrine Society, Boston, MA, 1/1/2011
• Craniofacial growth with insulin-like growth factor-I therapy in children with severe primary IGF-I deficiency.
  Backeljauw P, Campbell R, Annual PAS/LWPES/SPR Joint Meeting, Vancouver, 5/1/2010
• Turner syndrome aortopathy demonstrated by cardiac MRI-determined pulse wave velocity (PWV).
  Hagenbuch S, Hor K, Mazur W, Fleck R, Backeljauw P, Benson DW, Gotttliebson W, Wansapura J, Annual Meeting of the American College of Cardiology, Atlanta, GA, 3/1/2010
• Twenty years of efficacy and safety information with recombinant human IGF-I therapy: how will our clinical experience alter the approach to the treatment of short sta...
  Backeljauw P, GH & IGF-I Research, 1/1/2010
• Efficacy and safety of intranasal desmopressin acetate administered orally for the management of infants with neurogenic diabetes insipidus (DI).
  Gutmark-Little I, Repaske D, Backeljauw P, 92nd Annual Meeting of the Endocrine Society, San Diego, CA, 1/1/2010
• Final and near-final adult height in patients with severe primary insulin-like growth factor-I deficiency (IGFD) after long-term treatment with recombinant human insul...
  Philippe F. Backeljauw, Ali S Calicoglu, Joyce Kuntze, James W Frane, Steven Chernausek, Horm Res, 1/1/2009
• Partial anomalous pulmonary venous return (PAPVR) in adolescent and young adult Turner syndrome (TS) patients: prevalence and hemodynamic significance as determined by...
  Iris Gutmark-Little, William Gottliebson, Kan Hor, James Cnota, Philippe Backeljauw, Horm Res, 1/1/2009
• Therapy with rhIGF-I for severe primary IGF-I deficiency-data analysis from the US IGFD Registry.
  Backeljauw P, Abstract Book for the ESPE Satellite Symposium, 9/1/2008
• Endocrine sequelae of traumatic brain injury in children.
  Kaulfers A, Blum S, Backeljauw P, Rose S, Annual PAS/LWPES/SPR Joint Meeting; EPAS, 5/1/2008
• Under-recognized prevalence of elevated blood pressure in girls with Turner syndrome.
  Gutmark-Little I, Woo JG, Backeljauw PF, Annual PAS/LWPES/SPR Joint Meeting, 5/1/2008
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Lectures

• Pediatric Transgender Medicine, Growth Disorders, and Puberty 
  ENDO 2019 - New Orleans, LA - 3/24/2019
• Reproductive Issues and Fertility Preservation in Individuals with Turner Syndrome 
  ENDO 2018: 100th Endocrine Society Annual Meeting - Chicago, Illinios - 03/18/2018
• Clinical guidelines for Turner syndrome care-revisited. 
  Jacksonville, FL - 7/14/2014
• Co-chairman and moderator: Turner Syndrome Health and Wellness in the 21st Century: the Crossroads of Health Care Research and Health Care Delivery. 
  Jacksonville, FL - 7/13/2014
• The development of a Turner syndrome resource center-viewpoint from the health care provider. 
  Jacksonville, FL - 7/13/2014
• Liver disease in Turner syndrome. 
  Jacksonville, FL - 7/12/2014
• An update on novel growth promoting therapies. 
  Chicago, IL - 3/22/2014
• Growth Hormone plus Insulin-Like Growth Factor-1 Co-Administration Therapy in Short Children with Low IGF-I and GH Sufficiency: Results from a Randomized, Multi-Center... 
  Birmingham, AL - 10/1/2013
• Co-chairman: Growth Disorders 1-guided poster tour. 
  Milan, Italy - 9/1/2013
• Chair: The Gland-Brain Connection: Hormones, Cognition, and Behavior. 
  Washington, DC - 5/1/2013
• Moderator: "Clinical cases seminar on IGF deficiency" 
  Berlin, Germany - 2/1/2013
• Co-chairman: "Basic Growth Genetics for Clinicians" 
  Berlin, Germany - 2/1/2013
• Co-chairman: "Online Genetic Resources for Clinicians" 
  Berlin, Germany - 2/1/2013
• Characteristics of the flow in the aortic arch. 
  Grapevine, TX - 1/1/2013
• Turner syndrome-update on cardiovascular research. 
  Children's Hospital of Fudan University, Shanghai, China - 11/1/2012
• When to stop GH: pathophysiological and management considerations for ending GH treatment in GH-deficient and Turner syndrome patients. 
  Leipzig, Germany - 9/1/2012
• The incidence of pathology and cost of screening in the evaluation of children with short stature. 
  Houston, TX - 6/1/2012
• Long-term results of IGF-I treatment in severe primary IGF-I deficiency-Adult and near-adult efficacy and safety data. 
  Houston, TX - 6/1/2012
• The approach to the growth hormone deficient patient during the period of transitional care: current data, recommendations and guidelines. 
  Philadelphia, PA - 5/1/2012
• The diagnosis of short stature-A practical approach. 
  Philadelphia, PA - 5/1/2012
• Adult and near-adult efficacy and safety data of long-term IGF-I therapy in children with IGF deficiency. 
  Chicago, IL - 3/1/2012
• Response to IGF-1 therapy in severe primary IGF-1 deficiency. 
  Stockholm, Sweden - 1/1/2012
• Grand Rounds: "An update on cardiovascular disease in Turner syndrome." 
  Rush University Medical Center, Chicago, IL - 10/1/2011
• Long-term results of IGF-1 treatment in severe primary IGF-1 deficiency. 
  Glasgow, Scotland - 9/1/2011
• An update on vasculopathy research in Turner syndrome. 
  Cincinnati, OH - 5/1/2011
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Other

• Growth as a Clinical Barometer of Health in the Pediatric Patient. 
  Philippe Backeljauw, PV Updates, Peer Direct Publishing (Sponsored by Novo Nordisk, Inc.)
  1/1/2012
• PREP Endocrinology. 
  Philippe Backeljauw, Pediatrics Review and Education Program, American Academy of Pediatrics
  1/1/2012
• Growth Hormone Deficiency and Other Causes of Short Stature: Trends in Diagnosis, Evaluation, and Treatment in Children and Adults. 
  Mark A. Sperling, MD; David B. Allen, MD; Philippe F. Backeljauw, MD; Robert Rapaport, MD, Endocrine Today Education Lab
  http://www.Haelio.com
  1/1/2012
• Treating the IGF-1-Deficient Patient: Challenges and Solutions. (CME-approved educational activity.) 
  Philippe Backeljauw, Medscape, LLC
  1/1/2011
• Exploring first-line therapy options for growth failure. (CME-approved educational slide kit) 
  Philippe Backeljauw (chair), Ciccarelli C, Cohen J, Estrada E, Geffner M, Luetjen T, Miller K, Savage M, Medical Education Resources, Inc and Consensus Medical Communications
  1/1/2009
• IGF-I therapy of primary IGF-I deficiency: efficacy and safety considerations. 
  Philippe F Backeljauw
  http://www.IGFDForum.com
  1/1/2009
• Clinical debates on the treatment and management of patients with IGFD: How do we manage primary and secondary IGFD? 
  Philippe F. Backeljauw
  http://www.IGFDForum.com
  1/1/2008
• New areas for growth: diagnosis and treatment of IGF-I deficiency. 
  Philippe F. Backeljauw
  http://www.IGFDForum.com
  1/1/2006
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