R Teissier1, I Flechtner1, A Colmenares1, K Lambot-Juhan1, G Baujat1, C Pauwels1, D Samara-Boustani1, J Beltrand1, A Simon1, C Thalassinos1, H Crosnier1, H Latrech1, G Pinto1, M Le Merrer1, V Cormier-Daire2, J C Souberbielle1, M Polak3. 1. Pediatric EndocrinologyDiabetology and Gynecology Unit, Centre des Maladies Endocriniennes Rares de la CroissancePediatric Radiology UnitDepartment of Medical GeneticsHôpital Necker Enfants-Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), 149 Rue de Sèvres, 75743 Paris Cedex 15, FrancePediatric UnitCentre Hospitalier Intercommunal de Poissy-Saint-Germain-en-Laye, Saint-Germain-en-Laye, FranceOujda University HospitalOujda, MoroccoINSERM U871Université Paris Descartes, Sorbonne Paris Cité, Paris, FranceHormonal Biochemistry UnitHôpital Necker Enfants-Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, FranceINSERM U845Université Paris Descartes, Sorbonne Paris Cité, Paris, France. 2. Pediatric EndocrinologyDiabetology and Gynecology Unit, Centre des Maladies Endocriniennes Rares de la CroissancePediatric Radiology UnitDepartment of Medical GeneticsHôpital Necker Enfants-Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), 149 Rue de Sèvres, 75743 Paris Cedex 15, FrancePediatric UnitCentre Hospitalier Intercommunal de Poissy-Saint-Germain-en-Laye, Saint-Germain-en-Laye, FranceOujda University HospitalOujda, MoroccoINSERM U871Université Paris Descartes, Sorbonne Paris Cité, Paris, FranceHormonal Biochemistry UnitHôpital Necker Enfants-Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, FranceINSERM U845Université Paris Descartes, Sorbonne Paris Cité, Paris, FrancePediatric EndocrinologyDiabetology and Gynecology Unit, Centre des Maladies Endocriniennes Rares de la CroissancePediatric Radiology UnitDepartment of Medical GeneticsHôpital Necker Enfants-Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), 149 Rue de Sèvres, 75743 Paris Cedex 15, FrancePediatric UnitCentre Hospitalier Intercommunal de Poissy-Saint-Germain-en-Laye, Saint-Germain-en-Laye, FranceOujda University HospitalOujda, MoroccoINSERM U871Université Paris Descartes, Sorbonne Paris Cité, Paris, FranceHormonal Biochemistry UnitHôpital Necker Enfants-Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, FranceINSERM U845Université Paris Descartes, Sorbonne Paris Cité, Paris, France. 3. Pediatric EndocrinologyDiabetology and Gynecology Unit, Centre des Maladies Endocriniennes Rares de la CroissancePediatric Radiology UnitDepartment of Medical GeneticsHôpital Necker Enfants-Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), 149 Rue de Sèvres, 75743 Paris Cedex 15, FrancePediatric UnitCentre Hospitalier Intercommunal de Poissy-Saint-Germain-en-Laye, Saint-Germain-en-Laye, FranceOujda University HospitalOujda, MoroccoINSERM U871Université Paris Descartes, Sorbonne Paris Cité, Paris, FranceHormonal Biochemistry UnitHôpital Necker Enfants-Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, FranceINSERM U845Université Paris Descartes, Sorbonne Paris Cité, Paris, FrancePediatric EndocrinologyDiabetology and Gynecology Unit, Centre des Maladies Endocriniennes Rares de la CroissancePediatric Radiology UnitDepartment of Medical GeneticsHôpital Necker Enfants-Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), 149 Rue de Sèvres, 75743 Paris Cedex 15, FrancePediatric UnitCentre Hospitalier Intercommunal de Poissy-Saint-Germain-en-Laye, Saint-Germain-en-Laye, FranceOujda University HospitalOujda, MoroccoINSERM U871Université Paris Descartes, Sorbonne Paris Cité, Paris, FranceHormonal Biochemistry UnitHôpital Necker Enfants-Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, FranceINSERM U845Université Paris Descartes, Sorbonne Paris Cité, Paris, France michel.polak@nck.aphp.fr.
Abstract
OBJECTIVE: The prevalence of severe primary IGF1 deficiency (IGFD) is unclear. IGFD must be identified promptly as treatment with recombinant human IGF1 (rhIGF1) is now available. Our objective was to characterize and assess the prevalence of severe primary IGFD in a large cohort of patients evaluated for short stature at a pediatric endocrinology unit in France. DESIGN: Observational study in a prospective cohort. METHODS: Consecutive patients referred to our unit between 2004 and 2009 for suspected slow statural growth were included. Patients were classified into eight etiological categories. IGFD was defined by height ≤-3 SDS, serum IGF1 levels <2.5th percentile, GH sufficiency, and absence of causes of secondary IGFD. RESULTS: Out of 2546 patients included, 337 (13.5%) were born small for gestational age and 424 (16.9%) had idiopathic short stature. In these two categories, we identified 30 patients who met our criterion for IGFD (30/2546, 1.2%). In these 30 patients, we assessed the response to IGF1 generation test, time course of IGF1 levels, and efficiency of GH replacement therapy. The results indicated that only four of the 30 children were definite or possible candidates for rhIGF1 replacement therapy. CONCLUSION: The prevalence of severe primary IGFD defined using the standard criterion for rhIGF1 treatment was 1.2%, and only 0.2% of patients were eligible for rhIGF1 therapy.
OBJECTIVE: The prevalence of severe primary IGF1 deficiency (IGFD) is unclear. IGFD must be identified promptly as treatment with recombinant humanIGF1 (rhIGF1) is now available. Our objective was to characterize and assess the prevalence of severe primary IGFD in a large cohort of patients evaluated for short stature at a pediatric endocrinology unit in France. DESIGN: Observational study in a prospective cohort. METHODS: Consecutive patients referred to our unit between 2004 and 2009 for suspected slow statural growth were included. Patients were classified into eight etiological categories. IGFD was defined by height ≤-3 SDS, serum IGF1 levels <2.5th percentile, GH sufficiency, and absence of causes of secondary IGFD. RESULTS: Out of 2546 patients included, 337 (13.5%) were born small for gestational age and 424 (16.9%) had idiopathic short stature. In these two categories, we identified 30 patients who met our criterion for IGFD (30/2546, 1.2%). In these 30 patients, we assessed the response to IGF1 generation test, time course of IGF1 levels, and efficiency of GH replacement therapy. The results indicated that only four of the 30 children were definite or possible candidates for rhIGF1 replacement therapy. CONCLUSION: The prevalence of severe primary IGFD defined using the standard criterion for rhIGF1 treatment was 1.2%, and only 0.2% of patients were eligible for rhIGF1 therapy.
Authors: Peter Bang; Michel Polak; Valérie Perrot; Caroline Sert; Haris Shaikh; Joachim Woelfle Journal: Front Endocrinol (Lausanne) Date: 2022-02-18 Impact factor: 5.555
Authors: S Y Wang; Y Y Cheng; S C Liu; Y X Xu; Y Gao; C L Wang; Z G Wang; T Q Feng; G H Lu; J Song; P J Xia; L L Hao Journal: Mol Ther Nucleic Acids Date: 2021-08-19 Impact factor: 8.886