scholarly journals Characterization and prevalence of severe primary IGF1 deficiency in a large cohort of French children with short stature

2014 ◽  
Vol 170 (6) ◽  
pp. 847-854 ◽  
Author(s):  
R Teissier ◽  
I Flechtner ◽  
A Colmenares ◽  
K Lambot-Juhan ◽  
G Baujat ◽  
...  
Keyword(s):  
Replacement Therapy ◽  
Short Stature ◽  
Time Course ◽  
Large Cohort ◽  
Test Time ◽  
Pediatric Endocrinology ◽  
Gh Replacement ◽  
Standard Criterion ◽  
Igf1 Deficiency ◽  
Generation Test

ObjectiveThe 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.DesignObservational study in a prospective cohort.MethodsConsecutive 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.ResultsOut 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.ConclusionThe 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.

scholarly journals Prevalence of IGF1 deficiency in prepubertal children with isolated short stature

2009 ◽  
Vol 161 (1) ◽  
pp. 43-50 ◽  
Author(s):  
T Edouard ◽  
S Grünenwald ◽  
I Gennero ◽  
J P Salles ◽  
M Tauber
Keyword(s):  
Short Stature ◽  
Gestational Age ◽  
Bone Age ◽  
Pediatric Endocrinology ◽  
Exclusion Criteria ◽  
Prepubertal Children ◽  
Gh Secretion ◽  
Appropriate For Gestational Age ◽  
Low Levels ◽  
Igf1 Deficiency

Background/aims‘Primary IGF1 deficiency (IGFD)’ is defined by low levels of IGF1 without a concomitant impairment in GH secretion in the absence of secondary cause. The aims of this study were to evaluate the prevalence of non-GH deficient IGFD in prepubertal children with isolated short stature (SS) and to describe this population.MethodsThis retrospective study included all children with isolated SS seen in our Pediatric Endocrinology Unit from January 2005 to December 2007. Children were included based on the following criteria: i) SS with current height SDS ≤ −2.5, ii) age≥2 years, and iii) prepubertal status. Exclusion criteria were: i) identified cause of SS and ii) current or past therapy with rhGH. IGF1-deficient children were defined as children without GH deficiency and with IGF1 levels below or equal to −2 SDS.ResultsAmong 65 children with isolated SS, 13 (20%) had low IGF1 levels, consistent with a diagnosis of primary IGFD, four of which were born small for gestational age and nine were born appropriate for gestational age. When compared with non-IGFD children, IGFD children had higher birth weight (−0.7 vs −1 SDS, P=0.02) and birth height (−1.7 vs −2 SDS, P=0.04) and more delayed bone age (2.6 vs 1.7 years, P=0.03).ConclusionThe prevalence of primary IGFD was 20% in children with isolated SS. Concerning the pathophysiology, our study emphasizes that IGFD in some children may be secondary to nutritional deficiency or to maturational delay.

scholarly journals Metabolic Co-Morbidities Revealed in Patients with Childhood-Onset Adult GH Deficiency after Cessation of GH Replacement Therapy for Short Stature

2008 ◽  
Vol 55 (6) ◽  
pp. 977-984 ◽  
Author(s):  
Izumi FUKUDA ◽  
Naomi HIZUKA ◽  
Kumiko YASUMOTO ◽  
Junko MORITA ◽  
Makiko KURIMOTO ◽  
...  
Keyword(s):  
Replacement Therapy ◽  
Short Stature ◽  
Gh Deficiency ◽  
Childhood Onset ◽  
Gh Replacement ◽  
Adult Gh Deficiency

scholarly journals Long-term effects of growth hormone (GH) replacement therapy on hematopoiesis in a large cohort of children with GH deficiency

Endocrine ◽  
2015 ◽  
Vol 53 (1) ◽  
pp. 192-198 ◽  
Author(s):  
Andrea Esposito ◽  
Donatella Capalbo ◽  
Lucia De Martino ◽  
Martina Rezzuto ◽  
Raffaella Di Mase ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Replacement Therapy ◽  
Gh Deficiency ◽  
Large Cohort ◽  
Long Term Effects ◽  
Gh Replacement

scholarly journals Pregnancy outcomes in women receiving growth hormone replacement therapy enrolled in the NordiNet® International Outcome Study (IOS) and the American Norditropin® Studies: Web-Enabled Research (ANSWER) Program

Pituitary ◽  
2021 ◽  
Author(s):  
Beverly M. K. Biller ◽  
Charlotte Höybye ◽  
Paul Carroll ◽  
Murray B. Gordon ◽  
Anna Camilla Birkegård ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Replacement Therapy ◽  
Hormone Replacement ◽  
Outcome Study ◽  
Real World Data ◽  
Multicenter Studies ◽  
Clinical Trial Registration ◽  
Normal Delivery ◽  
Growth Hormone Replacement Therapy ◽  
Gh Replacement

Abstract Purpose Data on the safety of growth hormone (GH) replacement therapy during pregnancy are limited. We report a combined analysis of data from pregnant women treated with GH while enrolled in two non-interventional, multicenter studies: NordiNet® International Outcome Study (IOS) and the American Norditropin® Studies: Web-Enabled Research (ANSWER) Program. Methods Pregnancy data were pooled from NordiNet® IOS and the ANSWER Program. Data were collected during routine clinic visits by participating physicians using a web-based system. Patients exposed to GH replacement therapy during pregnancy were included in the analysis. Results The study population included 40 female patients with typical causes of adult GH deficiency (GHD). Overall, there were 54 pregnancies. Of these, 47 were exposed to GH between conception and delivery. In 48.9% of pregnancies exposed to GH, the dose was > 0.6 mg/day. GH was continued past conception and then stopped during the first, second, and third trimester, in 27.7%, 17.0%, and 2.1% of pregnancies, respectively. In 29.8%, GH was continued throughout pregnancy, with an unchanged dose in most cases. Of the 47 GH-exposed pregnancies, 37 (78.7%) progressed to normal delivery. There were three adverse events reported in two pregnancies. Conclusion These real-world data suggest that there were no new safety signals related to GH exposure in women with GHD during pregnancy. These results are consistent with findings from previous studies reporting data in pregnancies exposed to GH at conception or throughout pregnancy. This observational study in additional pregnancies provides further evidence that GH exposure does not adversely affect pregnancy outcome. Clinical trial registration: ClinicalTrials.gov NCT00960128 (date of registration: August 13, 2009) and NCT01009905 (date of registration: November 5, 2009).

scholarly journals Frequency of myelin oligodendrocyte glycoprotein antibodies in a large cohort of neurological patients

2021 ◽  
Vol 7 (2) ◽  
pp. 205521732110227
Author(s):  
Friederike Held ◽  
Sudhakar Reddy Kalluri ◽  
Achim Berthele ◽  
Ana-Katharina Klein ◽  
Markus Reindl ◽  
...  
Keyword(s):  
Time Course ◽  
Neurological Diseases ◽  
External Validation ◽  
Diagnostic Value ◽  
Myelin Oligodendrocyte Glycoprotein ◽  
Large Cohort ◽  
Wide Range ◽  
Neurological Patients ◽  
A Cell ◽  
Nosological Entity

Background Myelin oligodendrocyte glycoprotein (MOG) antibody disease (MOG-AD) is recognized as a distinct nosological entity. IgG antibodies against MOG (MOG-Ab) overlap with neuromyelitis optica spectrum disorders (NMOSD) phenotype in adults. However, an increasing number of clinical phenotypes have been reported to be associated with MOG-Ab. Objective To investigate the seroprevalence of MOG-Ab under consideration of demographics, disease entities and time course in a large cohort of unselected neurological patients. Methods Blood samples of 2.107 consecutive adult neurologic patients admitted to our department between 2016-2017 were tested for MOG-Ab using a cell-based assay. MOG-Ab persistence was analyzed in follow-up samples. External validation was performed in two independent laboratories. Results We found MOG-Ab in 25 of 2.107 (1.2%) patients. High antibody ratios were mostly associated with NMOSD and MOG-AD phenotype (5/25). Low ratios occurred in a wide range of neurological diseases, predominantly in other demyelinating CNS diseases (5/25) and stroke (6/25). MOG-Ab persistence over time was not confined to NMOSD and MOG-AD phenotype. Conclusion The present study demonstrates the occurrence of MOG-Ab in a wide range of neurological diseases. Only high MOG-Ab ratios were associated with a defined clinical phenotype, but low MOG-Ab ratios were not. The diagnostic value of low MOG-Ab is thus highly limited.

Changes In Prothrombin And Activated Partial Thromboplastin Time During Replacement Therapy With Human Recombinant Growth Hormone In Growth Hormone Deficient Adults

2020 ◽  
Vol 154 (Supplement_1) ◽  
pp. S102-S103
Author(s):  
Y S Kamel
Keyword(s):  
Growth Hormone ◽  
Replacement Therapy ◽  
Healthy Subjects ◽  
Recombinant Growth Hormone ◽  
Gh Replacement ◽  
Coagulation Tests ◽  
Before And After ◽  
Human Recombinant Growth Hormone ◽  
Male Patients ◽  
One Year

Abstract Introduction/Objective The aim of this study was to investigate the effects of GH administration on basic coagulation parameters: PT, aPTT and fibrinogen concentrations in adult GHD patients before and during one year of GH replacement. Methods Twenty-one adult patients with severe GHD (mean age +/- SE: 38.6 +/- 2.8 years) were included in this hospital based, prospective, interventional study. All patients were treated with rhGH for 12 months (GH dose: 0.4 mg/day for male and 0.6 mg/day for female patients). IGF-1 concentrations were determined using RIA-INEP kits. Basic coagulation tests, i.e. aPTT and fibrinogen concentrations, were measured before and after 3, 6 and 12 months of treatment with rhGH. Control values were obtained from fourteen “healthy” subjects matched by age, sex and body mass index (BMI). Results At baseline, we observed no significant differences in PT, aPTT and fibrinogen values between GHD and healthy subjects. IGF-1 concentrations increased significantly within 3 months of GH therapy (8.2 +/- 1.5 vs. 24.2 +/- 2.9 nmol/l, p &lt;0.05) and remained stable thereafter. A significant increase in PT values, which was more pronounced in female subjects, was noted after 6 and 12 months of treatment with GH. aPTT values increased significantly after 12 months of treatment only in male patients (28.8 +/- 4.6 vs. 39.7 +/- 2.1 s.; p &lt;0.05). No significant changes in fibrinogen concentrations were found during the study. Conclusion Twelve months of GH replacement therapy led to a significant increase in PT and aPTT values in adult GHD patients, while fibrinogen concentrations did not change. Changes in PT were more pronounced in female GHD patients, while an increase in aPTT values was observed only in male patients with GHD. The clinical significance of these changes needs further evaluation.

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